Verastem, Inc. (VSTM)
SIC breadcrumb: Manufacturing > Chemicals And Allied Products > SIC 2834 Pharmaceutical Preparations
SEC company page: https://www.sec.gov/edgar/browse/?CIK=1526119. Latest filing source: 0001104659-26-023444.
Informational only - descriptive public-record data, not investment advice.
Business
Read VSTM's verbatim Item 1 Business section from its latest 10-K: Business.
Risk Factors
Read VSTM's verbatim Item 1A Risk Factors from its latest 10-K: Risk Factors.
Selected Fundamentals
| Metric | Value | Unit | FY | Filed |
|---|---|---|---|---|
| Revenue | 30,914,000 | USD | 2025 | 2026-03-04 |
| Net income | -209,471,000 | USD | 2025 | 2026-03-04 |
| Assets | 246,442,000 | USD | 2025 | 2026-03-04 |
Financials
Annual standardized facts from SEC companyfacts as of latest extracted filing date 2026-03-04. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001526119.json. Derived margins, ratios, and free cash flow are computed from the extracted annual SEC facts.
| Metric | 2012 | 2013 | 2014 | 2015 | 2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Revenue | 2,053,000 | 2,596,000 | 10,000,000 | 30,914,000 | ||||||||||
| Net income | -36,440,000 | -67,802,000 | -72,434,000 | -149,209,000 | -67,726,000 | -71,200,000 | -73,812,000 | -87,367,000 | -130,637,000 | -209,471,000 | ||||
| Operating income | -37,002,000 | -67,804,000 | -94,783,000 | -132,341,000 | -49,360,000 | -61,409,000 | -72,937,000 | -92,084,000 | -114,956,000 | -170,129,000 | ||||
| Diluted EPS | -0.99 | -1.76 | -1.37 | -2.00 | -0.44 | -4.90 | -4.57 | -3.96 | -3.66 | -3.02 | ||||
| Operating cash flow | -29,484,000 | -57,310,000 | -74,515,000 | -138,518,000 | -33,506,000 | -53,502,000 | -63,673,000 | -86,460,000 | -104,771,000 | -137,509,000 | ||||
| Capital expenditures | 310,000 | 56,000 | 2,429,000 | 211,000 | 39,000 | 1,507,000 | 7,000 | 33,000 | 196,000 | 28,000 | ||||
| Assets | 83,629,000 | 89,791,000 | 277,236,000 | 145,046,000 | 154,349,000 | 108,662,000 | 95,050,000 | 149,718,000 | 101,538,000 | 246,442,000 | ||||
| Liabilities | 11,332,000 | 32,107,000 | 152,937,000 | 137,872,000 | 39,075,000 | 21,103,000 | 47,659,000 | 71,185,000 | 130,431,000 | 189,245,000 | ||||
| Stockholders' equity | 72,297,000 | 57,684,000 | 124,299,000 | 7,174,000 | 115,274,000 | 87,559,000 | 47,391,000 | 57,374,000 | -28,893,000 | 57,197,000 | ||||
| Cash and cash equivalents | 32,349,000 | 82,176,000 | 129,867,000 | 43,514,000 | 67,782,000 | 21,252,000 | 74,933,000 | 77,909,000 | 88,818,000 | 204,990,000 | ||||
| Free cash flow | -29,523,000 | -76,022,000 | -138,525,000 | -33,539,000 | -53,698,000 | -104,799,000 |
Ratios
| Metric | 2012 | 2013 | 2014 | 2015 | 2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Return on equity | -50.40% | -117.54% | -58.27% | -58.75% | -81.32% | -155.75% | -152.28% | -366.23% | ||||||
| Return on assets | -43.57% | -75.51% | -26.13% | -102.87% | -43.88% | -65.52% | -77.66% | -58.35% | -128.66% | -85.00% | ||||
| Liabilities / equity | 0.16 | 0.56 | 1.23 | 19.22 | 0.34 | 0.24 | 1.01 | 1.24 | 3.31 | |||||
| Current ratio | 7.40 | 5.13 | 6.83 | 2.84 | 8.48 | 5.69 | 4.29 | 5.45 | 3.07 | 3.09 |
Industry Peer Context
ROE peer context
ROA peer context
Financial Bridges
Free cash flow = operating cash flow - capital expenditures
Figure provenance: SEC companyfacts FY 2024. Operating cash flow: accession 0001104659-26-023444; concept NetCashProvidedByUsedInOperatingActivities; source concepts us-gaap:NetCashProvidedByUsedInOperatingActivities | Capital expenditures: accession 0001104659-26-023444; concept PaymentsToAcquirePropertyPlantAndEquipment; source concepts us-gaap:PaymentsToAcquirePropertyPlantAndEquipment | Free cash flow: accession 0001104659-26-023444; concept NetCashProvidedByUsedInOperatingActivities - PaymentsToAcquirePropertyPlantAndEquipment; source concepts us-gaap:NetCashProvidedByUsedInOperatingActivities; us-gaap:PaymentsToAcquirePropertyPlantAndEquipment
Financial Charts
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: Revenues. Source concepts: us-gaap:Revenues.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: NetIncomeLoss. Source concepts: us-gaap:NetIncomeLoss.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: OperatingIncomeLoss. Source concepts: us-gaap:OperatingIncomeLoss.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: EarningsPerShareDiluted. Source concepts: us-gaap:EarningsPerShareDiluted.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: NetCashProvidedByUsedInOperatingActivities. Source concepts: us-gaap:NetCashProvidedByUsedInOperatingActivities.
Figure provenance: SEC companyfacts. Latest point: FY 2024 ended 2024-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: PaymentsToAcquirePropertyPlantAndEquipment. Source concepts: us-gaap:PaymentsToAcquirePropertyPlantAndEquipment.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: Assets. Source concepts: us-gaap:Assets.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: Liabilities. Source concepts: us-gaap:Liabilities.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: StockholdersEquity. Source concepts: us-gaap:StockholdersEquity.
Figure provenance: SEC companyfacts. Latest point: FY 2025 ended 2025-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: CashAndCashEquivalentsAtCarryingValue. Source concepts: us-gaap:CashAndCashEquivalentsAtCarryingValue.
Figure provenance: SEC companyfacts. Latest point: FY 2024 ended 2024-12-31; accession 0001104659-26-023444; filed 2026-03-04. Concept: NetCashProvidedByUsedInOperatingActivities - PaymentsToAcquirePropertyPlantAndEquipment. Source concepts: us-gaap:NetCashProvidedByUsedInOperatingActivities; us-gaap:PaymentsToAcquirePropertyPlantAndEquipment.
Quarterly
Quarterly standardized facts from SEC companyfacts as of latest extracted filing date 2026-05-07. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001526119.json.
| Quarter | End Date | Revenue | Net Income | Diluted EPS | Method |
|---|---|---|---|---|---|
| 2018-Q2 | 2018-06-30 | 10,000,000 | reported discrete quarter | ||
| 2021-Q3 | 2021-09-30 | -0.13 | reported discrete quarter | ||
| 2022-Q1 | 2022-03-31 | -0.09 | reported discrete quarter | ||
| 2022-Q2 | 2022-06-30 | -0.12 | reported discrete quarter | ||
| 2022-Q3 | 2022-09-30 | -0.09 | reported discrete quarter | ||
| 2023-Q1 | 2023-03-31 | -15,714,000 | -0.08 | reported discrete quarter | |
| 2023-Q2 | 2023-03-31 | -15,714,000 | -0.08 | reported discrete quarter | |
| 2023-Q3 | 2023-06-30 | -24,281,000 | reported discrete quarter | ||
| 2023-Q4 | 2023-12-31 | -27,368,000 | derived Q4 = FY annual - nine-month YTD | ||
| 2024-Q1 | 2024-03-31 | -33,863,000 | reported discrete quarter | ||
| 2024-Q1 | 2024-06-30 | 10,000,000 | -0.31 | reported discrete quarter | |
| 2024-Q3 | 2024-06-30 | -8,256,000 | reported discrete quarter | ||
| 2024-Q3 | 2024-09-30 | -0.60 | reported discrete quarter | ||
| 2024-Q4 | 2024-12-31 | 0.00 | -64,551,000 | derived Q4 = FY annual - nine-month YTD | |
| 2025-Q1 | 2025-03-31 | -52,103,000 | -0.96 | reported discrete quarter | |
| 2025-Q2 | 2025-03-31 | -52,103,000 | reported discrete quarter | ||
| 2025-Q2 | 2025-06-30 | 2,137,000 | -0.62 | reported discrete quarter | |
| 2025-Q3 | 2025-06-30 | -25,934,000 | reported discrete quarter | ||
| 2025-Q3 | 2025-09-30 | 11,242,000 | -1.35 | reported discrete quarter | |
| 2025-Q4 | 2025-12-31 | 17,535,000 | -32,916,000 | derived Q4 = FY annual - nine-month YTD | |
| 2026-Q1 | 2026-03-31 | 18,671,000 | -36,591,000 | -0.46 | reported discrete quarter |
Quarterly Charts
Figure provenance: SEC companyfacts. Latest point: FY 2026 ended 2026-03-31; accession 0001104659-26-057114; filed 2026-05-07. Concept: Revenues. Source concepts: us-gaap:Revenues.
Figure provenance: SEC companyfacts. Latest point: FY 2026 ended 2026-03-31; accession 0001104659-26-057114; filed 2026-05-07. Concept: NetIncomeLoss. Source concepts: us-gaap:NetIncomeLoss.
Figure provenance: SEC companyfacts. Latest point: FY 2026 ended 2026-03-31; accession 0001104659-26-057114; filed 2026-05-07. Concept: EarningsPerShareDiluted. Source concepts: us-gaap:EarningsPerShareDiluted.
Macro Cross-References
- CPIAUCSL - Consumer Price Index for All Urban Consumers: All Items in U.S. City Average
- UNRATE - Unemployment Rate
- FEDFUNDS - Federal Funds Effective Rate
- CES0500000003 - Average Hourly Earnings of All Employees, Total Private
- DFEDTARU - Federal Funds Target Range - Upper Limit
- DFEDTARL - Federal Funds Target Range - Lower Limit
- DGS3MO - Market Yield on U.S. Treasury Securities at 3-Month Constant Maturity
- DGS2 - Market Yield on U.S. Treasury Securities at 2-Year Constant Maturity
- DGS10 - Market Yield on U.S. Treasury Securities at 10-Year Constant Maturity
- DGS30 - Market Yield on U.S. Treasury Securities at 30-Year Constant Maturity
- T10Y2Y - 10-Year Treasury Constant Maturity Minus 2-Year Treasury Constant Maturity
- CPILFESL - Consumer Price Index for All Urban Consumers: All Items Less Food and Energy
- CPIUFDSL - Consumer Price Index for All Urban Consumers: Food
- CPIENGSL - Consumer Price Index for All Urban Consumers: Energy
- CUSR0000SAH1 - Consumer Price Index for All Urban Consumers: Shelter
- PCEPI - Personal Consumption Expenditures: Chain-type Price Index
- PCEPILFE - Personal Consumption Expenditures Excluding Food and Energy: Chain-type Price Index
- PPIACO - Producer Price Index by Commodity: All Commodities
- T10YIE - 10-Year Breakeven Inflation Rate
- U6RATE - Total Unemployed, Plus All Marginally Attached Workers Plus Total Employed Part Time for Economic Reasons
- PAYEMS - All Employees, Total Nonfarm
- CIVPART - Labor Force Participation Rate
- EMRATIO - Employment-Population Ratio
- UNEMPLOY - Unemployed
- CE16OV - Employment Level
- ICSA - Initial Claims
- JTSJOL - Job Openings: Total Nonfarm
- JTSQUR - Quits: Total Nonfarm
- GDPC1 - Real Gross Domestic Product
- A191RL1Q225SBEA - Real Gross Domestic Product: Percent Change from Preceding Period
- INDPRO - Industrial Production: Total Index
- TCU - Capacity Utilization: Total Index
- HOUST - New Privately-Owned Housing Units Started: Total Units
- PERMIT - New Privately-Owned Housing Units Authorized in Permit-Issuing Places: Total Units
- RSAFS - Advance Retail Sales: Retail Trade
- PCE - Personal Consumption Expenditures
- DSPIC96 - Real Disposable Personal Income
- PSAVERT - Personal Saving Rate
- M2SL - M2
- BOPGSTB - U.S. International Trade in Goods and Services: Balance
- MSPUS - Median Sales Price of Houses Sold for the United States
- HSN1F - New One Family Houses Sold: United States
- RHORUSQ156N - Homeownership Rate in the United States
- TTLCONS - Total Construction Spending: Total Construction in the United States
- RRVRUSQ156N - Rental Vacancy Rate in the United States
- TOTALSL - Total Consumer Credit Owned and Securitized
- REVOLSL - Revolving Consumer Credit Owned and Securitized
- DRCCLACBS - Delinquency Rate on Credit Card Loans, All Commercial Banks
- GDP - Gross Domestic Product
- GPDI - Gross Private Domestic Investment
- GCE - Government Consumption Expenditures and Gross Investment
- PCEC - Personal Consumption Expenditures
- NETEXP - Net Exports of Goods and Services
- GFDEBTN - Federal Debt: Total Public Debt
- GFDEGDQ188S - Federal Debt: Total Public Debt as Percent of Gross Domestic Product
- FYFSD - Federal Surplus or Deficit
- FGRECPT - Federal Government Current Receipts
- FGEXPND - Federal Government: Current Expenditures
- MANEMP - All Employees, Manufacturing
- USCONS - All Employees, Construction
- USTRADE - All Employees, Retail Trade
- USFIRE - All Employees, Financial Activities
- USGOVT - All Employees, Government
- AWHAETP - Average Weekly Hours of All Employees, Total Private
- DGORDER - Manufacturers' New Orders: Durable Goods
- NEWORDER - Manufacturers' New Orders: Nondefense Capital Goods Excluding Aircraft
- BUSINV - Total Business Inventories
- EXPGS - Exports of Goods and Services
- IMPGS - Imports of Goods and Services
- IR - Import Price Index (End Use): All Commodities
- PPIFIS - Producer Price Index by Commodity: Final Demand
Latest quarter (10-Q)
Latest 10-Q source: 0001104659-26-057114.
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and elsewhere in this Quarterly Report on Form 10-Q and in our Annual Report on Form 10-K for our fiscal year ended December 31, 2025. Please also refer to the sections under headings “Forward-Looking Statements” and “Risk Factors” in this Quarterly Report on Form 10-Q and in our Annual Report on Form 10-K for our fiscal year ended December 31, 2025.
OVERVIEW
We are a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with challenging RAS/MAPK pathway-driven cancers. We market AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the United States (“U.S.”), the first treatment specifically FDA-approved for adults with KRAS-mutated recurrent LGSOC who have received prior systemic therapy.
Our pipeline includes clinical-stage programs, preclinical research programs and externally partnered early-stage programs. Our focus is on novel small molecule drugs developed both as monotherapy and in combination, which inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including targeting RAS directly with KRAS G12D inhibition, targeting the pathway downstream with RAF/MEK inhibition, and targeting the parallel pathway that drives resistance with FAK inhibition. Our focus is to expeditiously develop and deliver transformative therapies that truly change outcomes for people living with RAS/MAPK pathway-driven cancers.
COMMERCIAL PRODUCTS
AVMAPKI FAKZYNJA CO-PACK
AVMAPKI FAKZYNJA CO-PACK is the first treatment specifically approved by the FDA for adults with KRAS-mutated recurrent LGSOC who have received prior systemic therapy. AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.
The combination is being evaluated in an ongoing international Phase 3 trial, RAMP 301, in recurrent LGSOC with or without a KRAS mutation. The trial was fully enrolled, as of December 2025, and will serve as a confirmatory study for the initial indication and has the potential to expand the indication regardless of KRAS mutation status. The results will also be leveraged for potential geographic expansion.
| Column 1 | Column 2 |
|---|---|
| Ø | In April 2026, we announced new two-year median follow up data from the Phase 2 RAMP 201 trial that demonstrated durable benefit of avutometinib plus defactinib across both KRAS-mutant and KRAS wild-type recurrent LGSOC patients, with discontinuation rates consistent with the primary analysis, presented at the Society of Gynecologic Oncology 2026 annual meeting on women’s cancers. A new exposure-response analysis further demonstrated that the approved dose and schedule of avutometinib plus defactinib achieve the optimal therapeutic effect. |
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Table of Contents
CLINICAL PIPELINE
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor
VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 has a differentiated profile compared to other RAS inhibitors. Based on preclinical data, VS-7375 offers dual, potent inhibition of both ON and OFF states of KRAS G12D. We believe this correlates with better in vivo efficacy and durability versus ON-only RAS inhibitors. VS-7375 has demonstrated a high affinity for KRAS G12D with long residence time (18-24 hours) in preclinical models. We believe this correlates with a more rapid and durable suppression of pERK signaling (which controls growth and cell survival) when compared to other ON-only KRAS G12D inhibitors in tumor cell lines. The selective inhibition of VS-7375 to KRAS G12D has shown, in preclinical models, to spare T cell proliferation to maintain a normal healthy immune response, versus a RAS-multi-inhibitor which impairs T cell proliferation at increasing concentrations of drug. The once daily oral dosing of VS-7375 achieves exposures corresponding to maximal tumor regressions across preclinical models for pancreatic, lung and colorectal cancers. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for (VS-7375-101) was cleared, and initiated a Phase 1/2 clinical trial in June 2025 in patients with advanced KRAS G12D mutant solid tumors, including pancreatic ductal adenocarcinoma (“PDAC”), non-small cell lung cancer (“NSCLC”) and colorectal cancer (“CRC”).
In the Phase 1/2 study the Company cleared multiple monotherapy dose levels with no dose-limiting toxicities (DLTs) and no major toxicities and continues to dose-escalate. The Company also cleared multiple dose levels in combination with cetuximab with no DLTs and higher doses are being evaluated. The Company continues to enroll patients across various cohorts in the Phase 1/2 study. Following recent feedback from the FDA, the Company is amending its Phase 1/2 trial protocol to separate out disease-specific Phase 2 registration-directed trials for KRAS G12D mutated 2L PDAC, 2L/3L NSCLC and 2L+ CRC.
| Column 1 | Column 2 |
|---|---|
| Ø | In April 2026 we branded the trials as the VS-7375 TARGET-D Clinical Trial Program. In the ongoing TARGET-D 101 (VS-7375-101) Phase 1/2 dose escalation, dose expansion and combination-evaluation trial, we are now evaluating the 1200 mg daily dose (“QD”). We are also evaluating VS-7375 at 900 mg QD in combination with cetuximab. We expect to finish enrollment across the various expansion cohorts and combination cohorts with chemotherapies in the near term. In addition, we shared updated pharmacokinetic (“PK”) data that showed the 900 mg QD dose achieves target plasma levels of VS-7375 and provides clear separation from the 600 mg QD dose. |
| Column 1 | Column 2 |
|---|---|
| Ø | We have initiated three Phase 2 registration-directed trials, including: |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| 1. | TARGET-D 201 to evaluate VS-7375 at the 900 mg QD both as monotherapy and in combination with cetuximab in patients with second-line PDAC. The study is also evaluating VS-7375 and cetuximab in the first-line PDAC setting. |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| 2. | TARGET-D 202 to evaluate VS-7375 at the 900 mg QD in patients with advanced NSCLC who have received one-to-two prior lines of therapy. A final dose will be selected to evaluate VS-7375 in more patients, based on additional data from the TARGET-D 101 trial, which is currently evaluating VS-7375 at 600 mg QD in NSCLC. The study is also evaluating VS-7375 in patients with asymptomatic untreated NSCLC with brain metastasis. |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| 3. | TARGET-D 203 to evaluate VS-7375 at 900 mg QD as both monotherapy and in combination with EGFR inhibitors, including cetuximab or panitumumab, and chemotherapy in patients with metastatic CRC. |
| Column 1 | Column 2 |
|---|---|
| Ø | In March 2026, we announced that several late-breaking and regular abstracts were selected for presentation at the American Associate for Cancer Research (“AACR”) Annual Meeting. The abstracts included preclinical data demonstrating tumor regressions with VS-7375 in combination with PRMT5 inhibition in MTAP-deleted KRAS G12D-mutant pancreatic cancer and when compared to ON-only KRAS G12D inhibitors. |
| Column 1 | Column 2 |
|---|---|
| Ø | In March 2026, we reported an update on our VS-7375 clinical program, highlighting progress with our dose-escalation and dose-expansion trial, TARGET-D 101, a PK analysis, and a safety update, which demonstrated VS- |
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Table of Contents
| Column 1 | Column 2 |
|---|---|
| 7375 was generally well-tolerated across all monotherapy dose levels evaluated to date. Patients (n=23) receiving VS-7375 at either 400 mg QD, 600 mg QD or 900 mg QD with a mean duration of therapy of 1.6 months (0.7-5.6), reported no drug related liver function test abnormalities. As of the January 30, 2026 data cutoff, there was no drug-related neutropenia greater than Grade 2 and rates of nausea, vomiting and diarrhea remained lower than those reported by our partner in China. We also reported that the FDA requested the Company develop separate Phase 2 trial protocols for their trials in PDAC, NSCLC and CRC, where we are seeking marketing authorization. |
GENFLEET THERAPEUTICS (SHANGHAI), Inc
We shared multiple updates from GenFleet Therapeutics, our partner developing VS-7375, known as GFH375, in China.
| Column 1 | Column 2 |
|---|---|
| Ø | In April 2026, GenFleet announced that GFH375 was granted Breakthrough Therapy Designation (“BTD”) in China for patients with KRAS G12D-mutated metastatic pancreatic cancer who have received at least one prior systemic therapy. |
| Column 1 | Column 2 |
|---|---|
| Ø | In March 2026, GenFleet announced that GFH375 was granted BTD in China for patients with KRAS G12D-mutated NSCLC who have received prior systemic therapy. |
FINANCIAL OPERATIONS OVERVIEW
As of March 31, 2026, we had an accumulated deficit of $1,201.6 million. Our net loss was $36.6 million and $52.1 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had cash, cash equivalents, and investments of $181.7 million. In accordance with applicable accounting standards, we are required to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within twelve months after the date of the issuance of these condensed consolidated financial statements.
In our Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 4, 2026, we disclosed that our existing cash resources, including proceeds from exercise of warrants in January 2026, along with revenue we expected to generate from sales of AVMAPKI FAKZYNJA CO-PACK, was expected to be sufficient to fund our planned operations through twelve months from the date of issuance of those consolidated financial statements. As of March 4, 2026, our liquidity analysis excluded projected expenses for the Phase 2 studies entitled TARGET-D 201, TARGET-D 202, and TARGET-D 203, as we had not yet formally committed to the spend for those studies. Subsequent to the filing of our Annual Report on Form 10-K for the year ended December 31, 2025, we initiated TARGET-D-201, TARGET-D 202, and TARGET-D 203, resulting in a significant increase in projected research and development expenses. Consequently, the significant increase in costs we now expect to incur raises substantial doubt about our ability to continue as a going concern within the twelve months after the date of the issuance of these consolidated financial statements.
We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura AP
[Excerpt truncated for page length; source filing is linked above.]
Latest 10-K MD&A
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and related notes appearing elsewhere in this Annual Report on Form 10-K. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and as set forth under “Risk Factors.” Please also refer to the section under the heading “Forward-Looking Statements.”
OVERVIEW
We are a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with challenging RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the U.S., the first treatment specifically FDA-approved for adults with KRAS mutated recurrent LGSOC who have received prior systemic therapy. AVMAPKI FAKZYNJA CO-PACK received accelerated approval in the U.S. on May 8, 2025. We are also conducting RAMP 301, a Phase 3 trial designed to evaluate avutometinib plus defactinib versus Investigator’s Choice of Treatment (“ICT”) in patients with recurrent LGSOC with and without a KRAS mutation. This trial will serve as a confirmatory study for the initial U.S. indication and has the potential to expand the indication regardless of KRAS mutation status. Results of the RAMP 301 trial may also support future regulatory filings in Europe and Japan.
Our pipeline includes clinical-stage programs, preclinical research programs and externally partnered early-stage programs. Our focus is on novel small molecule drugs developed both as monotherapy and in combination, which inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including targeting RAS directly with KRAS G12D inhibition, targeting the pathway downstream with RAF/MEK inhibition, and targeting the parallel pathway that drives resistance with FAK inhibition. Our focus is to expeditiously develop and deliver transformative therapies that truly change outcomes for people living with RAS/MAPK pathway-driven cancers.
Our operations to date have been focused on organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020 and in anticipation of and following the approval of AVMAPKI FAKZYNJA CO-PACK in May 2025.
We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our Note Purchase Agreement, former loan agreements, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, we have also financed a portion of our operations through product revenue, including from AVMAPKI FAKZYNJA CO-PACK, beginning with our U.S. commercial launch in May 2025 and from COPIKTRA, from its U.S. commercial launch in September 2018 through our sale of the COPIKTRA license in September 2020.
As of December 31, 2025, we had an accumulated deficit of $1,165.0 million. Our net loss was $209.5 million, $130.6 million, and $87.4 million, for the years ended December 31, 2025, 2024, and 2023, respectively. We anticipate to incur significant expenses and operating losses may continue for the foreseeable future as we continue to incur operating costs to execute our strategic plan, including costs related to research and development of our product candidates and commercial activities. As of December 31, 2025, we had cash, and cash equivalents of $205.0 million and received $29.4 million in January 2026 from exercises of warrants. We expect our existing cash resources including proceeds from exercise of warrants in January 2026, along with revenue we expect to generate from sales of AVMAPKI FAKZYNJA CO-PACK, will be sufficient to fund our planned operations through 12 months from the date of issuance of these consolidated financial statements.
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We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura APA, through the Note Purchase Agreement, through future product revenues or through other strategic financing opportunities that could include, but are not limited to collaboration agreements, offerings of our equity, or the incurrence of debt. If we fail to obtain additional capital or generate sufficient revenue from our commercialization activities in the future, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities.
FINANCIAL OPERATIONS OVERVIEW
Revenue
Product Revenue, net
Product revenue, net, is recognized when earned on gross sales of AVMAPKI FAKZYNJA CO-PACK in the U.S. less provisions for all variable consideration. These provisions include trade allowances, rebates, chargebacks and discounts, product returns and other incentives. We sell AVMAPKI FAKZYNJA CO-PACK to a limited number of specialty pharmacies and specialty distributors. Although we expect net product revenues to increase over time, the provisions for product sales allowances may fluctuate based on the mix of sales to either specialty pharmacy or specialty distributor customers. See “Critical Accounting Policies and Significant Judgements and Estimates” below for more information on the components of net U.S. product sales of AVMAPKI FAKZYNJA CO-PACK.
Sales of Intellectual Property
Sales of intellectual property represents revenue generated from the sale of our COPIKTRA license and related assets to Secura. The sale included intellectual property related to duvelisib in oncology indications, certain existing duvelisib inventory, certain manufacturing equipment and, claims and rights under certain contracts pertaining to duvelisib, including net contract prepaid balances.
Costs of Sales - Product
Cost of sales - product consists of costs of AVMAPKI FAKZYNJA CO-PACK on which product revenue was recognized, royalties owed on such sales, and certain period costs including inventory write downs. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the product costs of AVMAPKI FAKZYNJA CO-PACK units sold during the year ended December 31, 2025, were expensed prior to obtaining regulatory approval and, therefore, are not included in cost of sales - product during this period. We expect cost of sales – product to increase in relation to product revenues as we deplete these inventories. There was no cost of sales – product recognized during the year ended December 31, 2024.
Cost of Sales - Intangible Amortization
Cost of sales – intangible amortization represents amortization expense recognized on finite-lived AVMAPKI FAKZYNJA CO-PACK-related intangible assets, which we began amortizing during the second quarter of 2025. There was no cost of sales – intangible amortization recognized during the year ended December 31, 2024.
Research and Development Expenses
Research and development expenses consist of costs associated with our research activities, including the development of our product candidates. Research and development expenses include product/ product candidate and/or project-specific costs, as well as unallocated costs. We allocate external research and development services, expenses incurred by third parties such as CROs, clinical sites, manufacturing organizations and consultants, by project and/or product candidate. We use our employee and infrastructure resources in a cross-functional manner
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across multiple research and development projects. Our project costing methodology does not allocate personnel, infrastructure and other indirect costs to specific clinical programs or projects.
Product/ product candidate/ project specific costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct third-party costs, which include expenses incurred under agreements with CROs, the cost of consultants who assist with the development of our product candidates on a program-specific basis, clinical site costs, and any other third-party expenses directly attributable to the development of the product candidates; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct costs related to avutometinib or defactinib that are not specific to a clinical trial such as the costs relating to contract manufacturing operations including manufacturing costs in connection with producing avutometinib and defactinib are included within “Avutometinib and defactinib manufacturing and non-clinical trial specific” as the cost to manufacture avutometinib and defactinib is not allocated to specific clinical trials; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | license fees. |
Unallocated costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | research and development employee-related expenses, including salaries, benefits, travel, and stock-based compensation expense; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | cost of consultants, including our scientific advisory board, who assist with our research and development but are not allocated to a specific program; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | facilities, depreciation, and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, and laboratory supplies. |
The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the years ended December 31, 2025, 2024, and 2023:
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2025 | 2024 | 2023 | ||||||
| | | (in thousands) | | (in thousands) | | (in thousands) | ||||
| Product/ product candidate / project specific costs | | | | | | | | | | |
| Avutometinib + defactinib - LGSOC | | $ | 35,778 | | $ | 25,079 | | $ | 13,360 | |
| Avutometinib + defactinib - NSCLC | | | 5,434 | | | 7,271 | | | 8,487 | |
| Avutometinib + defactinib - pancreatic cancer | | | 4,854 | | | 2,966 | | | — | |
| Avutometinib + defactinib - other indications | | | 1,431 | | | 1,282 | | | 1,225 | |
| Avutometinib and defactinib manufacturing and non-clinical trial specific | | 10,273 | | 15,020 | | 16,462 | | |||
| GenFleet / VS-7375 | | | 25,490 | | | 3,939 | | | 2,177 | |
| COPIKTRA | | — | | — | | 93 | | |||
| Unallocated costs | | | | | | | | | | |
| Personnel costs, excluding stock-based compensation | | | 18,799 | | | 14,657 | | | 12,299 | |
| Stock-based compensation expense | | | 2,527 | | | 2,134 | | | 1,987 | |
| Other unallocated expenses | | | 10,013 | | | 8,986 | | | 5,266 | |
| Total research and development expense | | $ | 114,599 | | $ | 81,334 | | $ | 61,356 | |
Costs for certain development activities, such as clinical trial expenses, are recognized based on an evaluation of the progress to completion of specific tasks using patient enrollment, clinical site activations, and other information based on actual costs incurred or level of effort expended provided by our vendors. Payments for these activities are based on the terms of individual arrangements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid expenses and other current assets or accrued expenses.
Our research and development expenses may increase significantly in future periods as we undertake costlier development activities for our existing and future product candidates, including larger and later-stage clinical trials.
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The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete development of our product candidates or the period, if any, in which material net cash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | clinical trial results; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, rate of progress, and expense of our research and development activities, including preclinical research and clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the potential benefits of our product candidates over other therapies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to market, commercialize, and achieve market acceptance of any of our product candidates for which we receive regulatory approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the terms and timing of regulatory approvals; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the expense of filing, prosecuting, defending, and enforcing patent claims and other intellectual property rights; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | changes in government regulation. |
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us to conduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or if we experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.
Selling, General, and Administrative Expenses
Selling, general, and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, in our executive, finance, legal, information technology, commercial, communication, human resources, and business development functions. Other selling, general, and administrative expenses include allocated facility costs, commercial costs, professional fees for legal, patent, investor and public relations, consulting, insurance premiums, audit, tax, and other public company costs.
Other Income, Other Expense, Interest Income and Interest Expense
Other expense for each of the years ended December 31, 2025, 2024 and 2023 primarily represent transaction losses recognized due to changes in foreign currency exchange rates.
Interest income reflects interest earned on our cash, cash equivalents and available-for-sale securities.
Interest expense reflects interest expense due on our Loan Agreement with Oxford and our convertible notes, interest expense related to vendor financing arrangements, as well as non-cash interest related to the amortization of debt discount and issuance costs.
Loss on Debt Extinguishment
The loss on debt extinguishment for the year ended December 31, 2025, represents the loss recognized on early extinguishment of our Loan Agreement with Oxford. On January 13, 2025, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Loan Agreement in an aggregate amount. The amount of repayment, excluding accrued interest, which exceeded the carrying of the loan was recorded as loss on debt extinguishment. There was no loss on debt extinguishment for the year ended December 31, 2024 and 2023.
Change in Fair Value of Preferred Stock Tranche Liability
The change in fair value of preferred stock tranche liability for the years ended December 31, 2024 and December 31, 2023, represents the mark-to-market adjustment of the second tranche right issued as part of the Securities
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Purchase Agreement (the “Series B Convertible Preferred Stock Securities Purchase Agreement”), dated January 24, 2023 with certain purchasers pursuant to which we agreed to sell and issue to the purchasers in a private placement up to 2,144,160 shares of its Series B Convertible Preferred Stock in two tranches. The preferred stock tranche liability expired in July 2024 and is no longer outstanding. There was no preferred stock tranche liability outstanding during the year ended December 31, 2025.
Change in Fair Value of Common Stock Warrant Liability
The change in fair value of warrant liability for the years ended December 31, 2025, and 2024 represents the mark-to-market adjustment of the liability classified warrants issued as part of the July 2024 Offering (defined herein). There were no warrants outstanding during the year ended December 31, 2023.
Change in Fair Value of Notes
We elected the fair value option to account for the Notes (defined herein) and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk, are recorded as change in fair value of Notes in the consolidated statements of operations and comprehensive loss. The Notes were not outstanding during the years ended December 31, 2024, and 2023.
CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with United States generally accepted accounting principles (“U.S. GAAP”). The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued and prepaid research and development expenses, stock-based compensation, fair value of Notes and revenue recognition described in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding and evaluating our financial condition and results of operations.
Revenue Recognition
We recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration that we expect to receive in exchange for those goods or services in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 606 Revenue from Contracts with Customers (“ASC 606”).
Product Revenue, Net
We sell AVMAPKI FAKZYNJA CO-PACK to a limited number of specialty pharmacies and specialty distributors in the U.S. The specialty pharmacies dispense AVMAPKI FAKZYNJA CO-PACK directly to patients while the specialty distributors resell AVMAPKI FAKZYNJA CO-PACK to healthcare entities who then resell AVMAPKI FAKZYNJA CO-PACK to patients. In addition to distribution agreements with specialty distributors, we also enter into arrangements with (1) certain government agencies and various private organizations (“Third-Party Purchasers”), which may provide for chargebacks or discounts with respect to the purchase of AVMAPKI FAKZYNJA CO-PACK, and (2) Medicare and Medicaid, which may provide for certain rebates with respect to their reimbursement of AVMAPKI FAKZYNJA CO-PACK.
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We recognize revenue on sales of AVMAPKI FAKZYNJA CO-PACK when a customer obtains control of the product, which occurs at a specific point in time (typically upon delivery). Product revenues are recorded at the wholesale acquisition costs, net of applicable reserves for variable consideration. Components of variable consideration include trade discounts and allowances, Third-Party Payer chargebacks and discounts, government rebates, product returns, other patient focused allowances, such as voluntary co-pay assistance, benefits verification, and other patient support programs that are offered within contracts between us and customers, payors, and other indirect customers relating to our sale of AVMAPKI FAKZYNJA CO-PACK. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable or a current liability. These estimates take into consideration a range of possible outcomes based upon relevant factors such as, customer contract terms, information received from third parties regarding the anticipated payor mix for AVMAPKI FAKZYNJA CO-PACK, known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled with respect to sales made.
The amount of variable consideration included within a transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under contracts will not occur in a future period. Our analyses contemplate the application of the constraint in accordance with FASB ASC 606. For the year ended December 31, 2025, we determined a material reversal of revenue would not occur in a future period for the estimates detailed below and, therefore, transaction prices would not be reduced further. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.
Trade Discounts and Allowances: We generally provide customers with invoice discounts on sales of AVMAPKI FAKZYNJA CO-PACK for prompt payment and other discounts, which are explicitly stated in our contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we compensate the specialty pharmacy and specialty distributor customers for sales order management, data, distribution, and certain other services. We have determined such services are not distinct from our sale of AVMAPKI FAKZYNJA CO-PACK to the specialty pharmacy and specialty distributor customers and, therefore, these payments have also been recorded as a reduction of revenue within the consolidated statements of operations and comprehensive loss.
Third-Party Payer Chargebacks, Discounts and Fees: We execute contracts with Third-Party Purchasers that allow for eligible purchases of AVMAPKI FAKZYNJA CO-PACK at prices lower than the wholesale acquisition cost. In some cases, customers will charge us for the difference between what they pay for AVMAPKI FAKZYNJA CO-PACK and the ultimate selling price to the Third-Party Purchasers to whom they sell the product. Reserves will generally be established in the same period that the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable, net. Chargeback amounts will generally be determined at the time of resale to the qualified Third-Party Purchasers by customers, and we generally will issue credits for such amounts within a few weeks of the customer’s notification of the resale. The reserves for chargebacks are expected to consist of credits that we expect to issue for units that remain in customer inventories at the end of each reporting period that we expect will be sold to Third-Party Purchasers, and chargebacks that customers have claimed, but for which we have not yet issued a credit.
Government Rebates: We are subject to discount and rebate payment obligations under various government programs including Federal and state Medicaid programs, Medicare, and others. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability, which is included as a component of accrued expenses on the consolidated balance sheets. Our liability for these rebates consist of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in customer inventories at the end of each reporting period.
Other Patient Support Initiatives: Other patient support initiatives that we offer include voluntary co-pay assistance programs, which are intended to provide financial assistance to qualified commercially-insured patients with prescription drug co-payments required by payors. The calculation of the accrual for co-pay assistance is based on
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an estimate of claims and the cost per claim that we expect to receive for product that has been recognized as revenue but remains in the distribution channel inventories at the end of each reporting period. The adjustments are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included as a component of accrued expenses on the consolidated balance sheets.
Product Returns: Consistent with industry practice, we generally offer customers a limited right of return for product that has been purchased from us either directly or through one of its distribution channels. We estimate the amount of our product sales that may be returned by our customers and records this estimate as a reduction of revenue in the period the related product revenue is recognized. We estimate product return liabilities using available industry data and our own sales information, including our visibility into the inventory remaining in the distribution channel.
Our return policy generally allows for eligible returns of AVMAPKI FAKZYNJA CO-PACK for credit under the following circumstances:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Receipt of damaged product; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Shipment errors that were a result of an error by us; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Expired product that is returned during the period beginning three months prior to the product’s expiration and ending six months after the expiration date; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Quantities of product received by a customer in excess of quantity ordered; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Product subject to a recall; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Product that we at our sole discretion, has specified can be returned for credit. |
As of December 31, 2025, we have not received any product returns.
Sales of Intellectual Property
Upon the sale of licenses or intellectual property that incorporate sale-based royalties, including milestone payments based on a level of sales, we evaluate whether the royalties and sales-based milestones are considered probable of being achieved and estimate the amount of royalties to include over the contractual term using the expected value method and estimate the sales-based milestones using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated royalty and milestone value is included in the transaction price. Royalties and sales-based milestones for territories for which there is not regulatory approval are not considered probable until such regulatory approval is achieved. We evaluate factors such as whether consideration is outside of our control, timeline for when the uncertainty will be resolved and historical sales of COPIKTRA if applicable. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, we reevaluate the probability of achievement of all milestones subject to constraint and amount of royalty revenue to be received and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. At December 31, 2025, we determined no future potential royalties pursuant to the Secura APA were constrained.
Refer to Note 2. Significant accounting policies and Note 16. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of revenue.
Accrued and Prepaid Research and Development Expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued expenses. This process involves reviewing contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly in arrears for
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services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to CROs in connection with research and development activities for which we have not yet been invoiced.
We base our research and development expenses on estimates of services received and efforts expended pursuant to quotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract, and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided and result in a prepayment of research and development expenses. When recognizing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. To date, there have been no material differences between our estimates of such expenses and the amounts actually incurred.
Refer to Note 2. Significant accounting policies, and Note 7. Accrued expenses to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of accrued research and development expenses.
Stock-Based Compensation
We recognize stock-based compensation expense for service-based awards, such as stock options and restricted stock units (“RSUs”), issued to employees, directors and consultants based on the grant date fair value of the awards on a straight-line basis over the requisite service period. In addition, we issue shares to employees under our employee stock purchase plan (“ESPP”). The fair value of our stock options and ESPP grants is estimated at the date of grant using the Black-Scholes option pricing model. We are precluded from utilizing the simplified method as described in SEC SAB Topic 14.D.2 to calculate the expected term as a key assumption in the Black-Scholes pricing model when determining fair value of stock options not in the money because of a modification. Therefore, when valuing stock options that are not at the money, we utilize a binomial lattice model to calculate the fair value of the stock option.
We have also granted performance-based RSUs and stock options with terms that allow the recipients to vest in a certain number of shares based upon the achievement of performance-based milestones as specified in the grants. Stock-based compensation expense associated with these performance-based RSUs and stock options is recognized if the performance condition is considered probable of achievement using management’s best estimates of the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from our estimates, stock-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current and prior periods of a change in estimate for performance-based RSUs and stock options will be recognized as compensation cost in the period of the revision and recorded as a change in estimate.
While the assumptions used to calculate and account for stock-based compensation awards represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, our stock-based compensation expense could vary significantly from period to period.
During the year ended December 31, 2025, we recorded $9.4 million of stock-based compensation expense. As of December 31, 2025, there was approximately $5.4 million of unrecognized stock-based compensation related to stock options, which is expected to be recognized over a weighted-average period of 2.2 years. As of December 31, 2025, there was approximately $4.6 million of unrecognized stock-based compensation related to RSUs, which is expected to be recognized over a weighted-average period of 1.9 years. See Note 2. Significant accounting policies
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and Note 11. Stock-based compensation to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of stock-based compensation.
Fair Value of Notes
The fair value of the Notes pursuant to the Note Purchase Agreement represents the present value of estimated future payments, including interest, principal, Repayment Amount, and Revenue Participation Payments (each as defined in the Note Purchase Agreement). The fair value measurement is based on significant Level 3 unobservable inputs such as the probability and timing of Revenue Participation Payments, Repayment Amount, and the discount rate. We determined the fair value of the Notes utilizing a discounted cash flow model of estimated future payments including interest, principal, Repayment Amount and Revenue Participation Payments utilizing a discount rate calculated as the term matched risk-free rate plus credit spread. At January 13, 2025, we utilized a discount rate between 11.9%-12.4% and at December 31, 2025, we utilized a discount rate between 12.6%-13.0%. The fair value of the Notes at December 31, 2025 was determined to be $76.3 million which differed from the contractual principal amount of $75.0 million by $1.3 million. Significant increases or decreases in any of these inputs in isolation could result in a significantly lower or higher fair value measurement.
While the assumptions used to calculate and account for the fair value of Notes represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, the fair value of Notes and consequently the change in fair value of Notes could vary significantly from period to period.
Refer to Note 2. Significant accounting policies, and Note 8. Long-term debt to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of fair value of Notes.
RESULTS OF OPERATIONS
All financial information presented has been consolidated and includes the accounts of our wholly-owned subsidiaries, Verastem Securities Company and Verastem Europe GmbH. All intercompany balances and transactions have been eliminated in consolidation.
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year Ended December 31, | |||||||
| | | | | | | | | | |
| | 2025 | | 2024 | | 2023 | ||||
| Revenue: | | | | | | | | | |
| Product revenue, net | | $ | 30,914 | | $ | — | | $ | — |
| Sale of COPIKTRA license and related assets | | | — | | | 10,000 | | | — |
| Total revenue | | 30,914 | | 10,000 | | — | |||
| Operating expenses: | | | | | | | | | |
| Cost of sales - product | | | 4,600 | | | — | | | — |
| Cost of sales - intangible amortization | | | 698 | | | — | | | — |
| Research and development | | | 114,599 | | | 81,334 | | | 61,356 |
| Selling, general and administrative | | 81,146 | | 43,622 | | 30,728 | |||
| Total operating expenses | | 201,043 | | 124,956 | | 92,084 | |||
| Loss from operations | | (170,129) | | (114,956) | | (92,084) | |||
| Other expense | | | (203) | | | (123) | | | (109) |
| Interest income | | 4,068 | | 4,149 | | 6,214 | |||
| Interest expense | | (1,138) | | (4,562) | | (4,139) | |||
| Loss on debt extinguishment | | | (1,826) | | | — | | | — |
| Change in fair value of preferred stock tranche liability | | | — | | | 4,189 | | | 2,751 |
| Change in fair value of warrant liability | | | (27,492) | | | (19,149) | | | — |
| Change in fair value of Notes | | | (12,751) | | | — | | | — |
| Net loss before taxes | | | (209,471) | | | (130,452) | | | (87,367) |
| Income tax expense | | | — | | | (185) | | | — |
| Net loss | | $ | (209,471) | | $ | (130,452) | | $ | (87,367) |
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Comparison of the Year Ended December 31, 2025 to the Year Ended December 31, 2024
Product Revenue, Net. We initiated commercial sales of AVMAPKI FAKZYNJA CO-PACK in the U.S. in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the year ended December 31, 2025 (the “2025 Period”) we recognized approximately $30.9 million of net product revenue. We had no product revenue during the year ended December 31, 2024 (the “2024 Period”).
Sale of COPIKTRA license and related assets revenue. Sale of COPIKTRA license and related assets revenue for the 2025 Period was $0.0 million compared to $10.0 million for the 2024 Period. Sale of COPIKTRA license and related assets revenue for the 2024 Period was comprised of one sales milestone of $10.0 million due upon Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Period. The $10.0 million milestone payment was received by us in July 2024. There was no milestone achieved during the 2025 Period. Refer to Note 16. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the Secura APA.
Costs of Sales – Product. Costs of sales – product of $4.6 million for the 2025 Period consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed on such sales, and certain period costs including inventory write downs. We began capitalizing inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2025 Period, or approximately $0.2 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales - product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We had no cost of sales – product during the 2024 Period.
Research and Development Expense. Research and development expense was $114.6 million for the 2025 Period compared to $81.3 million for the 2024 Period. The increase of $33.3 million from the 2024 Period to the 2025 Period was primarily a result of the following incremental expenses incurred: $9.3 million in CRO costs, $7.1 million in investigator fees, $6.8 million in drug substance and drug product costs, $5.3 million in clinical supply costs, $4.6 million in personnel related costs, including non-cash stock-based compensation, $0.7 million in preclinical costs, and $0.6 million in other costs The increase was also attributable to a $6.0 million payment made to exercise the GenFleet Option with respect to VS-7375. These increases were partially offset by a decrease of $4.1 million in consulting costs and the non-recurrence of a $3.0 million milestone payment made to GenFleet in the 2024 Period. The increase in Research and Development costs was primarily driven by the RAMP 301 study, which began in the fourth quarter of 2023, and the VS-7375-101 study, which began in the second quarter of 2025.
We anticipate an increase in research and development expenses in future periods as we continue development of VS-7375 for the treatment of patients with PDAC, NSCLC, CRC and other KRAS G12 cancers, complete the RAMP 301 clinical trial for the treatment of patients with recurrent LGSOC with and without KRAS mutation, and address post-marketing commitments for AVMAPKI FAKZYNJA CO-PACK as agreed upon with the FDA. This increase is expected to be partially offset by a reduction in costs incurred as a result of the discontinuation of the RAMP 203 clinical trial.
Selling, General and Administrative Expense. Selling, general and administrative expense was $81.1 million for the 2025 Period compared to $43.6 million for the 2024 Period. The increase of $37.5 million from the 2024 Period to the 2025 Period was primarily a result of the following incremental expenses incurred: $28.2 million in consulting and professional fees related to the launch of AVMAPKI FAKZYNJA CO-PACK for the treatment of KRAS mutant recurrent LGSOC, $8.2 million in personnel costs including non-cash stock-based compensation, $1.1 million in commercial operations expense, and $2.3 million in travel related and other costs. These increases were partially offset by a decrease of $2.2 million in financing fees.
Other Expense. Other expense was $0.2 million for the 2025 Period compared to $0.1 million for the 2024 Period. Other expense was comprised of transaction losses due to changes in foreign currency exchange rates in both periods.
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Interest Income. Interest income for the 2025 Period and the 2024 Period was $4.1 million.
Interest Expense. Interest expense for the 2025 Period was $1.1 million compared to $4.6 million for the 2024 Period. The decrease of $3.5 million from the 2024 Period to the 2025 Period was primarily driven by the termination of the Loan Agreement with Oxford on January 13, 2025 resulting in a reduction of interest expense recorded in the 2025 Period. We have elected to present interest expense associated to the Notes within change in fair value of Notes within the consolidated statements of operations and comprehensive loss.
Loss on Debt Extinguishment. The loss on debt extinguishment for the 2025 Period of $1.8 million represents the loss recognized on early extinguishment of our Loan Agreement with Oxford. On January 13, 2025, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Loan Agreement in an aggregate amount of $42.7 million (the “Payoff Amount”). The Payoff Amount, excluding accrued interest, exceeded the carrying amount of the Term Loans on January 13, 2025 by $1.8 million which was recorded as a loss on debt extinguishment. There was no loss on debt extinguishment in the 2024 Period.
Change in Fair Value of Preferred Stock Tranche Liability. The change in fair value of the preferred stock tranche liability was $4.2 million income for the 2024 Period. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement. The preferred stock tranche liability expired in July 2024 and therefore was not outstanding during the 2025 Period. The fair value of the preferred stock tranche liability decreased from $4.2 million at the beginning of the 2024 Period to $0.0 million at the end of the 2024 Period resulting in $4.2 million income in the 2024 Period.
Change in Fair Value of Warrant Liability. The change in fair value of the warrant liability was $27.5 million in expense for the 2025 Period compared to $19.1 million in expense for the 2024 Period. The change in fair value of warrant liability represents the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The $27.5 million expense recognized in the 2025 Period was driven by an increase in fair value per Warrant from December 31, 2024 to the exercise date for 9.7 million Warrants exercised during the 2025 Period and an increase in fair value per Warrant for 8.4 million Warrants that remained outstanding at December 31, 2025 primarily driven by an increase in our stock price. The $19.1 million expense recognized in the 2024 Period was primarily driven an increase in fair value per Warrant from July 23, 2024, to the exercise date for 0.3 million Warrants exercised during the 2024 Period and an increase in fair value per Warrant for the 18.1 million Warrants that remained outstanding at December 31, 2024 primarily driven by an increase in our stock price.
Change in Fair Value of Notes. We elected the fair value option to account for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the consolidated statements of operations and comprehensive loss. The change in fair value of $12.8 million for the 2025 Period was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Period. There were no Notes outstanding in the 2024 Period.
Income Tax Expense. No income tax expense was recognized during the 2025 Period. Income tax expense of $0.2 million for the 2024 Period was comprised of interest under IRC section 453A related to the $10.0 million milestone payment from Secura because it was an installment sale for tax purposes.
LIQUIDITY AND CAPITAL RESOURCES
Sources of Liquidity
We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our Note Purchase Agreement, former loan agreements, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, we have financed a portion of our operations through product revenue, including from AVMAPKI FAKZYNJA CO-PACK, beginning with our U.S. commercial launch in May 2025, and from COPIKTRA, from its
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U.S. commercial launch in September 2018 through our sale of the COPIKTRA license in September 2020. We expect to finance a portion of our business through future potential milestones and royalties received pursuant to the Secura APA.
As of December 31, 2025, we had $205.0 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
Risks and uncertainties include those identified under Item 1A. Risk Factors, in this Annual Report on Form 10-K.
Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below (in thousands):
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2025 | | 2024 | | 2023 | ||||
| Net cash (used in) provided by: | | | | | | | | | | |
| Operating activities | | $ | (137,509) | | $ | (104,771) | | $ | (86,460) | |
| Investing activities | | (9,624) | | 59,972 | | (44,447) | | |||
| Financing activities | | 263,305 | | 54,782 | | 134,194 | | |||
| Increase in cash, cash equivalents and restricted cash | | $ | 116,172 | | $ | 9,983 | | $ | 3,287 | |
Operating activities. Cash used in operating activities was $137.5 million and $104.8 million for the 2025 Period and the 2024 Period, respectively. The use of cash in operating activities in the 2025 Period and 2024 Period resulted primarily from our net losses adjusted for non-cash adjustments and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash adjustments was $163.5 million and $108.5 million for the 2025 Period and 2024 Period, respectively. Non-cash charges and adjustments for the 2025 Period were primarily related to the change in fair value of warrant liability, non-cash changes in fair value of the Notes, loss on debt extinguishment and stock-based compensation expense. Non-cash charges and adjustments for the 2024 Period were primarily related to the changes in fair value of the preferred stock tranche liability, the change in fair value of warrant liability and stock-based compensation expense. Our cash inflow from operating activities due to changes in operating assets and liabilities was $26.0 million for the 2025 Period primarily driven by an increase of $30.8 million in accrued expenses and other liabilities and an increase of $8.4 million in accounts payable, partially offset by an increase of $8.8 million in accounts receivable, an increase of $2.6 million in prepaid expenses, other current assets and other assets, and an increase of $1.8 million in inventory. Our cash inflow from operating activities due to changes in operating assets and liabilities for the 2024 Period was $3.7 million primarily driven by an increase of $8.0 million in accrued expenses and other liabilities, partially offset by a decrease of $3.2 million in accounts payable, an increase of $0.6 million in prepaid expenses, other current assets and other assets, a decrease of $0.3 million in deferred liabilities and an increase of $0.2 million in grant receivable.
Investing activities. Cash used in investing activities for the 2025 Period primarily represents milestone payments recorded as intangible assets in the amounts of $7.5 million under the Pfizer Agreement and $2.1 million under the License Agreement. Cash provided by investing activities for the 2024 Period primarily relates to net maturities of investments of $60.0 million.
Financing activities. Cash provided by financing activities for the 2025 Period includes $96.9 million of net proceeds from issuance of common stock and pre-funded warrants as part of the November 2025 Public Offering, $75.0 million of proceeds received pursuant to the Note Purchase Agreement, $69.9 million of net proceeds from issuance of common stock and pre-funded warrants as part of the 2025 Private Placement, $33.8 million of proceeds from the exercise of Warrants, $22.7 million of net proceeds received from issuance of common stock under the ATM Programs, $7.4 million of net proceeds received from issuance of common stock under the Stock Purchase Agreement, $1.2 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received from exercise of stock options and employee stock purchase program, partially offset by the $42.6 million repayment of our Loan Agreement and $1.2 million of payments for insurance premium financing. Cash provided by financing activities for the 2024 Period includes $53.8 million of net proceeds received from the
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issuance of shares of common stock, pre-funded warrants, and warrants as part of the July 2024 Offering, $1.3 million of proceeds received from insurance premium financing, $0.9 million of proceeds received from exercise of Warrants and $0.2 million of proceeds received from exercise of stock options and our employee stock purchase plan, partially offset by $1.3 million of payments on insurance premium financing and $0.2 million of fees paid to the Lenders to amend our Loan Agreement with Oxford.
Refer to Note 10. Capital Stock to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the November 2025 Public Offering, 2025 Private Placement, ATM Programs, the Stock Purchase Agreement, the July 2024 Offering and Warrants; Note 8 Long-Term Debt to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the Note Purchase Agreement and Loan Agreement; and Note 17. Notes Payable to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the finance agreements related to insurance premium financing and the monthly payments of principal and interest related thereto.
Funding requirements
We expect to continue to incur significant expenses and may continue to incur operating losses. Refer to risk factor titled We have incurred significant losses since our inception. We may incur losses for the foreseeable future and may never achieve or maintain profitability within section Item 1A. Risk Factors for detailed activities which may drive our continued operating losses and expenses in future periods.
Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs and timing of activities of commercialization for AVMAPKI FAKZYNJA CO-PACK and product candidates for which we expect to receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, progress, and results of our ongoing and potential future clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the extent to which we acquire or in-license other product candidates and technologies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs, timing, and outcome of regulatory review of our product candidates (including our efforts to seek approval and fund the preparation and filing of regulatory submissions); |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | revenue received from commercial sales of AVMAPKI FAKZYNJA CO-PACK and our product candidates, should any of our product candidates also receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property rights, and defending intellectual property related claims; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to establish collaborations or partnerships on favorable terms, if at all. |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | receipt of milestone payments and royalties pursuant to the Secura APA including timing of such receipt. |
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and through future potential milestones and royalties received pursuant to the Secura APA. To the extent that we raise additional capital through the sale of equity, warrants or securities convertible into common stock, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. To the extent that we enter into certain licensing arrangements, the ownership interest of our existing stockholders may be diluted if we elect to make certain payments in shares of our common stock. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings
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when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
CONTRACTUAL OBLIGATIONS AND COMMITMENTS
On April 15, 2014, we entered into a lease agreement for approximately 15,197 square feet of office and laboratory space in Needham, Massachusetts. The lease term commenced on April 15, 2014 and was scheduled to expire on September 30, 2019. Effective February 15, 2018, we amended our lease agreement to relocate within the facility to another location consisting of 27,810 square feet of office space (the “February 2018 Amended Lease Agreement”). The February 2018 Amended Lease Agreement extended the expiration date of the lease from September 2019 through June 2025. Pursuant to the February 2018 Amended Lease Agreement, the initial annual base rent amount was approximately $0.7 million, which increased during the lease term to $1.1 million for the last 12-month period. Effective November 1, 2024, we amended the February 2018 Amended Lease Agreement to extend the expiration date from June 2025 to June 2026 (the “November 2024 Amended Lease Agreement”). The payment terms of the November 2024 Amended Lease Agreement are $1.1 million per annum through the expiration date in June 2026. As of December 31, 2025, the total future lease payments under the agreement are $0.5 million through June 2026.
In the fourth quarter of 2024, we entered into a master services agreement with IQVIA (“IQVIA Master Services Agreement”) to leverage IQVIA’s infrastructure and established commercialization solutions to complement our AVMAPKI FAKZYNJA CO-PACK launch strategy. In signing the IQVIA Master Service Agreement we committed to spend $60.0 million, of which $48.1 million remains unpaid at December 31, 2025 and is expected to be spent in the next two to three years. As of December 31, 2025, approximately $13.7 million of this commitment is included within vendor financing arrangements, accrued expenses, and accounts payable on the consolidated balance sheets.
As discussed in Note 16. License, collaboration and commercial agreements to the consolidated financial statements located in this Annual Report on Form 10-K, we are party to several agreements to license intellectual property. The license agreements may require us to pay upfront license fees, ongoing annual license maintenance fees, milestone payments, minimum royalty payments, as well as reimbursement of certain patent costs incurred by the licensors, as applicable. As of December 31, 2025, we do not have any minimum contractual obligations in relation to these agreements because: there were no upfront license fees payable in future periods; no annual license maintenance fees; we cannot estimate if milestone and/or royalty payments will occur in future periods; and patent cost reimbursement costs are perpetual and the agreements are cancelable by us at any time upon prior written notice to the licensor.
TAX LOSS CARRYFORWARDS
As of December 31, 2025, we had federal and state NOL carryforwards of $460.5 million and $99.5 million, respectively, which are available to reduce future taxable income. We also had federal and state tax credits of $12.3 million and $0.7 million, respectively, which may be used to offset future tax liabilities. The NOL and tax credit carryforwards will expire at various dates through 2044, except for $423.3 million of federal NOL carryforwards which may be carried forward indefinitely. NOL and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the IRC, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. At December 31, 2025 we recorded a 100% valuation allowance against our NOL and tax credit carryforwards, as we believe it is more likely than not that the tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our tax carryforwards will be realized, net income would increase in the period of determination.
Based on our analysis under Section 382 of the IRC and similar provisions under state law, we believe that our federal NOL carryforwards, our state NOL carryforwards, our research and development (“R&D”) credits and our
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Orphan Drug (“OD”) credits will be limited as of December 31, 2025. The portion of federal NOL, state NOL, R&D credits and OD credits that were determined to be limited by Section 382 have been written off as of December 31, 2025. The remaining unused carryforwards remain available for future periods. During 2024, we believe we triggered ownership changes under Section 382 of the IRC and similar provisions under state law. We have approximately $346.4 million of federal NOLs generated prior to such ownership changes inclusive of $309.3 million of federal NOLs which may be carried forward indefinitely. Since the $309.3 million of federal NOLs may be carried forward indefinitely these have not been written off as of December 31, 2025, but due to the limitations under Section 382, generally we can only use $1.6 million per year against taxable income in the future. Due to our full valuation allowance the write off of NOLs, R&D credits, and OD credits did not have any impact to the statements of operation and comprehensive loss for the 2025 Period and 2024 Period.
RECENTLY ADOPTED ACCOUNTING STANDARDS
Refer to Note 2. Significant accounting policies to our consolidated financial statements located in this Annual Report on Form 10-K for recently adopted accounting standards.
MD&A history
Prior-year 10-K MD&A spans are extracted from SEC filings with the same bounded parser used for the latest filing. The latest 10-K appears above; prior years are below.
FY 2024 10-K MD&A
SEC filing source: 0001558370-25-003372.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and related notes appearing elsewhere in this Annual Report on Form 10-K. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and as set forth under “Risk Factors.” Please also refer to the section under the heading “Forward-Looking Statements.”
OVERVIEW
We are a late-stage development biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with RAS/ MAPK pathway-driven cancers. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition and KRAS G12D inhibition.
Our most advanced product candidates, avutometinib and defactinib, are being investigated in both preclinical and clinical studies for the treatment of various solid tumors, including, but not limited to LGSOC, NSCLC and pancreatic cancer. We believe that avutometinib may be beneficial as a therapeutic, as a single agent or when used together in combination with defactinib, other agents, other pathway inhibitors, or other current and emerging standard of care treatments in cancers that do not adequately respond to currently available therapies.
Our operations to date have been organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020. We have financed our operations to date primarily through public and private offerings of our common stock, warrants and pre-funded warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement, the Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue.
As of December 31, 2024, we had an accumulated deficit of $955.5 million. Our net loss was $130.6 million, $87.4 million, and $73.8 million, for the years ended December 31, 2024, 2023, and 2022, respectively. As of December 31, 2024, we had cash, cash equivalents, and investments of $88.8 million. In accordance with applicable accounting standards, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within 12 months after the date of the issuance of the consolidated financial statements. We anticipate operating losses may continue for the foreseeable future since we do not yet have regulatory approval to sell any of our product candidates, and we continue to incur operating costs to execute our strategic plan, including costs related to research and development of our product candidates and commercial readiness activities. As a result of the assessment in accordance with the applicable accounting standards, these conditions raise substantial doubt about our ability to continue as a going concern for 12 months after the date the consolidated financial statements are issued.
We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura APA, through the Note Purchase Agreement or through other strategic financing opportunities that could include, but are not limited to collaboration agreements, future offerings of our equity, or the incurrence of debt. However, given the risks associated with these potential strategic or financing opportunities, they are not deemed probable for purposes of the going concern assessment. If we fail to obtain additional future capital, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities. Therefore, there is substantial doubt about our ability to continue as a going concern.
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FINANCIAL OPERATIONS OVERVIEW
Revenue
Sale of licenses and related assets revenue to date have been generated through our sale of our duvelisib license and related assets to Secura. The sale included intellectual property related to duvelisib in oncology indications, certain existing duvelisib inventory, certain manufacturing equipment and, claims and rights under certain contracts pertaining to duvelisib including net contract prepaid balances.
Research and development expenses
Research and development expenses consist of costs associated with our research activities, including the development of our product candidates. Research and development expenses include product/ product candidate and/or project-specific costs, as well as unallocated costs. We allocate the expenses related to external research and development services, such as CROs, clinical sites, manufacturing organizations and consultants, by project and/or product candidate. We use our employee and infrastructure resources in a cross-functional manner across multiple research and development projects. Our project costing methodology does not allocate personnel, infrastructure and other indirect costs to specific clinical programs or projects.
Product/ product candidate/ project specific costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct third-party costs, which include expenses incurred under agreements with CROs, the cost of consultants who assist with the development of our product candidates on a program-specific basis, clinical site costs, and any other third-party expenses directly attributable to the development of the product candidates; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct costs related to avutometinib or defactinib that are not specific to a clinical trial such as the costs relating to contract manufacturing operations including manufacturing costs in connection with producing avutometinib and defactinib are included within “Avutometinib and defactinib manufacturing and non-clinical trial specific” as the cost to manufacture avutometinib and defactinib is not allocated to specific clinical trials; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | license fees. |
Unallocated costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | research and development employee-related expenses, including salaries, benefits, travel, and stock-based compensation expense; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | cost of consultants, including our scientific advisory board, who assist with our research and development but are not allocated to a specific program; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | facilities, depreciation, and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, and laboratory supplies. |
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The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the years ended December 31, 2024, 2023, and 2022:
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2024 | 2023 | 2022 | ||||||
| | | (in thousands) | | (in thousands) | | (in thousands) | ||||
| Product/ product candidate / project specific costs | | | | | | | | | | |
| Avutometinib + defactinib - LGSOC | | $ | 25,079 | | $ | 13,360 | | $ | 9,000 | |
| Avutometinib + defactinib - NSCLC | | | 7,271 | | | 8,487 | | | 7,666 | |
| Avutometinib + defactinib - pancreatic cancer | | | 2,966 | | | — | | | — | |
| Avutometinib + defactinib - other indications | | | 1,282 | | | 1,225 | | | 70 | |
| Avutometinib and defactinib manufacturing and non-clinical trial specific | | 15,020 | | 16,462 | | 16,215 | | |||
| GenFleet | | | 3,939 | | | 2,177 | | | — | |
| COPIKTRA | | — | | 93 | | 183 | | |||
| Unallocated costs | | | | | | | | | | |
| Personnel costs, excluding stock-based compensation | | | 14,657 | | | 12,299 | | | 10,848 | |
| Stock-based compensation expense | | | 2,134 | | | 1,987 | | | 1,766 | |
| Other unallocated expenses | | | 8,986 | | | 5,266 | | | 4,810 | |
| Total research and development expense | | $ | 81,334 | | $ | 61,356 | | $ | 50,558 | |
Costs for certain development activities, such as clinical trial expenses, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, and information provided us by our vendors on their actual costs incurred or level of effort expended. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid expenses and other current assets or accrued expenses.
Our research and development expenses may increase significantly in future periods as we undertake costlier development activities for our existing and future product candidates, including larger and later-stage clinical trials.
The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete development of our product candidates or the period, if any, in which material net cash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | clinical trial results; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, rate of progress, and expense of our research and development activities, including preclinical research and clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the potential benefits of our product candidates over other therapies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to market, commercialize, and achieve market acceptance for any of our product candidates that we receive regulatory approval for; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the terms and timing of regulatory approvals; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the expense of filing, prosecuting, defending, and enforcing patent claims and other intellectual property rights. |
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us to conduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or if we experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.
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Selling, general, and administrative expenses
Selling, general, and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, in our executive, finance, legal, information technology, commercial, communication, human resources, and business development functions. Other selling, general, and administrative expenses include allocated facility costs, commercial costs, professional fees for legal, patent, investor and public relations, consulting, insurance premiums, audit, tax, and other public company costs.
Other income, other expense, interest income and interest expense
Other expense for each of the years ended December 31, 2024 and 2023 was comprised of transaction losses due to changes in foreign currency exchange rates. Other income for the year ended December 31, 2022 was comprised of a gain on the sale of fixed assets and transaction gains due to changes in foreign currency exchange rates.
Interest income reflects interest earned on our cash, cash equivalents and available-for-sale securities.
Interest expense reflects interest expense due on our Loan Agreement with Oxford and our convertible notes, as well as non-cash interest related to the amortization of debt discount and issuance costs.
Change in fair value of preferred stock tranche liability
The change in fair value of preferred stock tranche liability for the years ended December 31, 2024 and December 31, 2023, was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement (the “Series B Convertible Preferred Stock Securities Purchase Agreement”), dated January 24, 2023 with certain purchasers pursuant to which the Company agreed to sell and issue to the purchasers in a private placement up to 2,144,160 shares of its Series B Convertible Preferred Stock in two tranches. The preferred stock tranche liability expired in July 2024 and is no longer outstanding. There was no preferred stock tranche liability outstanding during the year ended December 31, 2022.
Change in fair value of common stock warrant liability
The change in fair value of warrant liability for the year ended December 31, 2024 was comprised of the mark-to-market adjustment related to liability classified warrants issued as part of the July 2024 Offering (defined herein). There were no warrants outstanding during the years ended December 31, 2023, or December 31, 2022.
CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with United States generally accepted accounting principles (“U.S. GAAP”). The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued and prepaid research and development expenses, stock-based compensation, revenue recognition, and collaborative agreements, described in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding and evaluating our financial condition and results of operations.
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Revenue recognition
We recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 606 Revenue from Contracts with Customers (“ASC 606”).
Sales of intellectual property
For sales of license and intellectual property, that include sale-based royalties, including milestone payments based on a level of sales, we evaluate whether the royalties and sales-based milestones are considered probable of being achieved and estimate the amount of royalties to include over the contractual term using the expected value method and estimate the sales-based milestones using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated royalty and milestone value is included in the transaction price. Royalties and sales-based milestones for territories for which there is not regulatory approval are not considered probable until such regulatory approval is achieved. We evaluate factors such as whether consideration is outside of our control, timeline for when the uncertainty will be resolved and historical sales of COPIKTRA if applicable. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, we reevaluate the probability of achievement of all milestones subject to constraint and amount of royalty revenue to be received and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. At December 31, 2024, we determined no future potential royalties pursuant to the Secura APA were not constrained.
Refer to Note 2. Significant Accounting Policies and Note 13. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of revenue.
Collaborative Arrangements
Collaborative Arrangements: Contracts are considered to be collaborative arrangements pursuant to U.S. GAAP when they satisfy the following criteria defined in ASC Topic 808, Collaborative Arrangements: (i) the parties to the contract must actively participate in the joint operating activity and (ii) the joint operating activity must expose the parties to the possibility of significant risk and rewards, based on whether or not the activity is successful. Payments received from or made to a partner that are the result of a collaborative relationship with a partner, instead of a customer relationship, such as co-development activities, are recorded as a reduction or increase to research and development expense, respectively.
Accrued and prepaid research and development expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued expenses. This process involves reviewing contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly in arrears for services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to CROs in connection with research and development activities for which we have not yet been invoiced.
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We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant to quotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided and result in a prepayment of the research and development expense. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. To date, there have been no material differences between our estimates of such expenses and the amounts actually incurred.
Refer to Note 2. Significant Accounting Policies, and Note 4. Accrued expenses to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of accrued research and development expenses.
Stock-based compensation
For service-based awards, we recognize stock-based compensation expense for stock options, and restricted stock units (“RSUs”) issued to employees, directors and consultants based on the grant date fair value of the awards on a straight-line basis over the requisite service period. In addition, we issue shares under our employee stock purchase plan (“ESPP”) to employees. The fair value of our stock options and ESPP grants is estimated at the date of grant using the Black-Scholes option pricing model. For determining fair value of stock options when the stock options are not at the money because of a modification, we are precluded from utilizing the simplified method as described in SEC SAB Topic 14.D.2 to calculate the expected term as a key assumption in the Black-Scholes pricing model. Therefore, when valuing stock options that are not at the money, we utilize a binomial lattice model to calculate the fair value of the stock option.
We have also granted performance-based RSUs and stock options with terms that allow the recipients to vest in a specific number of shares based upon the achievement of performance-based milestones as specified in the grants. Stock-based compensation expense associated with these performance-based RSUs and stock options is recognized if the performance condition is considered probable of achievement using management’s best estimates of the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from our estimates, stock-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current and prior periods of a change in estimate for performance-based RSUs and stock options will be recognized as compensation cost in the period of the revision, and recorded as a change in estimate.
While the assumptions used to calculate and account for stock-based compensation awards represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, our stock-based compensation expense could vary significantly from period to period.
During the year ended December 31, 2024, we recorded $7.3 million of stock-based compensation expense. As of December 31, 2024, there was approximately $6.0 million of unrecognized stock-based compensation related to stock options, which are expected to be recognized over a weighted-average period of 2.1 years. As of December 31, 2024, there was approximately $4.0 million of unrecognized stock-based compensation related to RSUs, which are expected to be recognized over a weighted-average period of 2.3 years. See Note 2. Significant accounting policies and Note 8. Stock-based compensation to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of stock-based compensation.
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RESULTS OF OPERATIONS
All financial information presented has been consolidated and includes the accounts of our wholly-owned subsidiaries, Verastem Securities Company and Verastem Europe GmbH. All intercompany balances and transactions have been eliminated in consolidation.
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year Ended December 31, | |||||||
| | | | | | | | | | |
| | 2024 | 2023 | 2022 | ||||||
| Revenue: | | | | | | | | | |
| Sale of COPIKTRA license and related assets | | | 10,000 | | | — | | | 2,596 |
| Total revenue | | 10,000 | | — | | 2,596 | |||
| Operating expenses: | | | | | | | | | |
| Research and development | | | 81,334 | | | 61,356 | | | 50,558 |
| Selling, general and administrative | | 43,622 | | 30,728 | | 24,975 | |||
| Total operating expenses | | 124,956 | | 92,084 | | 75,533 | |||
| Loss from operations | | (114,956) | | (92,084) | | (72,937) | |||
| Other expense | | | (123) | | | (109) | | | 47 |
| Interest income | | 4,149 | | 6,214 | | 1,215 | |||
| Interest expense | | (4,562) | | (4,139) | | (2,137) | |||
| Change in fair value of preferred stock tranche liability | | | 4,189 | | | 2,751 | | | — |
| Change in fair value of warrant liability | | | (19,149) | | | — | | | — |
| Net loss before taxes | | | (130,452) | | | (87,367) | | | (73,812) |
| Income tax expense | | | (185) | | | — | | | — |
| Net Loss | | | (130,637) | | | (87,367) | | | (73,812) |
| | | | | | | | | | |
Comparison of the Year Ended December 31, 2024 to the Year Ended December 31, 2023
Sale of COPIKTRA license and related assets revenue. Sale of COPIKTRA license and related assets revenue for the year ended December 31, 2024 (the “2024 Period”) was $10.0 million compared to $0.0 million for the year ended December 31, 2023 (the “2023 Period”). Sale of COPIKTRA license and related assets revenue for the 2024 Period was comprised of one sales milestone of $10.0 million due upon Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Period. The $10.0 million milestone payment was received by us in July 2024.
Research and development expense. Research and development expense for the 2024 Period was $81.3 million compared to $61.4 million for the 2023 Period. The $19.9 million increase from the 2023 Period to the 2024 Period was primarily related to an increase of $6.4 million in CRO costs, an increase of $4.7 million in investigator fees, an increase of $4.5 million in consulting costs, an increase of $2.5 million in personnel related costs, including non-cash stock-based compensation, and an increase of $1.8 million in clinically supply costs. The increase in CRO costs and investigator fees was primarily driven by increased costs related to the RAMP 301 study which we commenced in the last quarter of 2023. The increase in consulting costs was primarily driven by additional consultants in 2024 to support RAMP 301 startup and enrollment and additional consulting costs related to completing the NDA submission for avutometinib and defactinib for treatment of patients with recurrent LGSOC.
Selling, general and administrative expense. Selling, general and administrative expense for the 2024 Period was $43.6 million compared to $30.7 million for the 2023 Period. The increase of $12.9 million from the 2023 Period to the 2024 Period primarily resulted from an increase of $4.8 million in personnel related costs, including non-cash stock-based compensation, an increase of $4.7 million of costs in anticipation of the potential launch of avutometinib and defactinib in LGSOC, an increase of $3.0 million in July 2024 Offering financing fees in the 2024 Period, and an increase of $1.0 million in consulting and professional fee, partially offset by $0.6 million in financing fees for the offering of our Series B Convertible Preferred Stock in the 2023 Period.
Other expense. Other expense of $0.1 million for the 2024 Period and the 2023 Period was comprised of transaction losses due to changes in foreign currency exchange rates.
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Interest income. Interest income for the 2024 Period was $4.1 million compared to $6.2 million for the 2023 Period. The decrease of $2.1 million in interest income was primarily driven by a decrease in interest rates and investment balances on short-term investments and cash equivalents.
Interest expense. Interest expense for the 2024 Period was $4.6 million compared to $4.1 million for the 2023 Period. The increase of $0.5 million from the 2023 Period to the 2024 Period was primarily driven by additional interest expense in the 2024 Period on the Loan Agreement as a result of the additional $15.0 million debt drawdown on March 22, 2023.
Change in fair value of preferred stock tranche liability. The change in fair value of the preferred stock tranche liability was $4.2 million income for the 2024 Period compared to $2.8 million income for the 2023 Period. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Series B Convertible Preferred Stock Securities Purchase Agreement. The fair value of the preferred stock tranche liability decreased from $4.2 million at the beginning of the 2024 Period and expired in July 2024 resulting in $4.2 million income in the 2024 Period. The fair value of the preferred stock tranche liability decreased from $6.9 million upon issuance on January 24, 2023, to $4.1 million at the end of the 2023 Period resulting in $2.8 million income in the 2023 Period.
Change in fair value of warrant liability. The change in fair value of the warrant liability of $19.1 million expense for the 2024 Period was comprised of the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The liability classified warrants increased in value from July 23, 2024, to December 31, 2024 primarily driven by an increase in our stock price. There was no warrant liability outstanding during the 2023 Period.
Income tax expense. Income tax expense of $0.2 million for the 2024 Period was comprised of interest under IRC section 453A related to the $10.0 million milestone payment from Secura because it was an installment sale for tax purposes. There was no income tax expense in the 2023 Period.
LIQUIDITY AND CAPITAL RESOURCES
Sources of liquidity
We have financed our operations to date primarily through public and private offerings of our common stock, warrants and pre-funded warrants, offerings of convertible notes, convertible preferred stock sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement, the Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, and the upfront payment and milestone payments received under the Secura APA. Additionally, from our commercial launch of COPIKTRA in the United States on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue. As of September 30, 2020, we have sold our COPIKTRA license and no longer sell COPIKTRA in the United States. We expect to finance a portion of our business through future potential milestones and royalties received pursuant to the Secura APA.
As of December 31, 2024, we had $88.8 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
Risks and uncertainties include those identified under Item 1A. Risk Factors, in this Annual Report on Form 10-K.
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Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below (in thousands):
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2024 | | 2023 | | 2022 | ||||
| Net cash (used in) provided by: | | | | | | | ||||
| Operating activities | | $ | (104,771) | | $ | (86,460) | | $ | (63,673) | |
| Investing activities | | 59,972 | | (44,447) | | | 66,185 | | ||
| Financing activities | | 54,782 | | 134,194 | | | 51,784 | | ||
| Increase in cash, cash equivalents and restricted cash | | $ | 9,983 | | $ | 3,287 | | | 54,296 | |
Operating activities. The use of cash in operating activities in the 2024 Period and 2023 Period resulted primarily from our net losses adjusted for non-cash adjustments and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash adjustments was $108.5 million and $85.5 million for the 2024 Period and 2023 Period, respectively. Non-cash adjustments for the 2024 Period were primarily related to stock-based compensation expense, change in the fair value of warrant liability and change in the fair value of preferred stock tranche liability. Non-cash adjustments for the 2023 Period were primarily related to stock-based compensation expense, non-cash interest, net and change in the fair value of preferred stock tranche liability. Our cash inflow for the 2024 Period from operating activities due to changes in operating assets and liabilities was $3.7 million primarily driven by an increase of $8.0 million in accrued expenses and other liabilities, partially offset by a decrease of $3.2 million in accounts payable, an increase of $0.6 million in prepaid expenses, other current assets and other assets, a decrease of $0.3 million in deferred liabilities and an increase of $0.2 million in grant receivable. Our cash outflow for the 2023 Period from operating activities due to changes in operating assets and liabilities was $1.0 million for the 2023 Period primarily driven by an increase of $5.8 million in prepaid expenses, other current assets and other assets, and a decrease of $0.4 million in deferred liabilities, partially offset by an increase of $2.9 million in accrued expenses and other liabilities, and an increase of $2.3 million in accounts payable. The increases in both periods in prepaid expenses, other current assets, and other asserts is exclusive of cash received from PanCAN and used on the RAMP 205 study. Cash used in operating activities was $104.8 million and $86.5 million for the 2024 Period and the 2023 Period, respectively.
Investing activities. The cash provided by investing activities for the 2024 Period primarily relates to the net maturities of investments of $60.0 million. The cash used in investing activities for the 2023 Period primarily relates to the net purchases of investments of $44.4 million.
Financing activities. The cash provided by financing activities for the 2024 Period represents $53.8 million of net proceeds received from the issuance of shares of common stock, pre-funded warrants, and warrants as part of the July 2024 Offering, $1.3 million of proceeds received from insurance premium financing, $0.9 million of proceeds received from exercise of warrants and $0.2 million of proceeds received from exercise of stock options and our employee stock purchase plan, partially offset by $1.3 million of payments on insurance premium financing, and $0.2 million of fees paid to the Lenders to amend our Loan Agreement with Oxford. The cash provided by financing activities for the 2023 Period primarily represents $91.4 million of proceeds from our public offering in June 2023 of common stock and pre-funded warrants to purchase shares of our common stock, net of issuance costs, $28.1 million of proceeds received from issuance of Series B Convertible Preferred Stock, net of issuance costs, $14.9 million of proceeds received pursuant to the Loan Agreement, $1.4 million of proceeds received from insurance premium financing and $0.1 million of proceeds received related to our employee stock purchase plan, partially offset by $1.4 million of payments on insurance premium financing, and $0.3 million of repayment of our 5.00% Convertible Senior Notes due 2048.
Refer to Note 7. Capital Stock to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the July 2024 Offering of common stock, pre-funded warrants, and warrants, the June 2023 Offering of common stock and pre-funded warrants, and the January 2023 offering of our Series B Convertible Preferred Stock; Note 5. Debt to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the Loan Agreement; and Note 14. Notes Payable to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the finance agreement with
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AFCO Premium Credit LLC related to insurance premium financing and the monthly payments of principal and interest related thereto.
Funding requirements
We expect to continue to incur significant expenses and may continue to incur operating losses. Refer to risk factor titled We have incurred significant losses since our inception. We may incur losses for the foreseeable future and may never achieve or maintain profitability within section Item 1A. Risk Factors for detailed activities which may drive our continued operating losses and expenses in future periods.
Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs and timing of activities in anticipation of potential commercialization for avutometinib and defactinib and product candidates for which we expect to receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, progress, and results of our ongoing and potential future clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the extent to which we acquire or in-license other product candidates and technologies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs, timing, and outcome of regulatory review of our product candidates (including our efforts to seek approval and fund the preparation and filing of regulatory submissions); |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | revenue, if any, received from commercial sales of our product candidates, including avutometinib and defactinib, should any of our product candidates receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property rights, and defending intellectual property related claims; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to establish collaborations or partnerships on favorable terms, if at all; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | receipt of milestone payments and royalties pursuant to the Secura APA including timing of such receipt. |
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and through future potential milestones and royalties received pursuant to the Secura APA. To the extent that we raise additional capital through the sale of equity, warrants or convertible debt securities, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Without additional funding we believe that we may not have sufficient funds to meet our obligations within the next 12 months from the date of issuance of these consolidated financial statements. While we believe that we may have sufficient funds to meet our obligations within the next 12 months from the date of the issuance of the consolidated financial statements for the year ended December 31, 2024, in performing this analysis under the applicable accounting standards management excluded certain elements of our operating plan that cannot be considered probable of occurring. Accordingly, management has concluded that substantial doubt exists about our ability to continue as a going concern for 12 months from the date the consolidated financial statements are issued.
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CONTRACTUAL OBLIGATIONS AND COMMITMENTS
On April 15, 2014, we entered into a lease agreement for approximately 15,197 square feet of office and laboratory space in Needham, Massachusetts. The lease term commenced on April 15, 2014 and it was scheduled to expire on September 30, 2019. Effective February 15, 2018, we amended our lease agreement to relocate within the facility to another location consisting of 27,810 square feet of office space (the “February 2018 Amended Lease Agreement”). The February 2018 Amended Lease Agreement extended the expiration date of the lease from September 2019 through June 2025. Pursuant to the February 2018 Amended Lease Agreement, the initial annual base rent amount was approximately $0.7 million, which increased during the lease term to $1.1 million for the last 12-month period. Effective November 1, 2024, we amended the February 2018 Amended Lease Agreement to extend the expiration date from June 2025 to June 2026 (the “November 2024 Amended Lease Agreement”). The payment terms of the November 2024 Amended Lease Agreement are $1.1 million per annum through the expiration date in June 2026. As of December 31, 2024, the total future lease payments under the agreement are $1.6 million through June 2026.
In 2024 we entered into a master services agreement with IQVIA (“IQVIA Master Services Agreement”) for our strategic collaboration with IQVIA to leverage IQVIA’s infrastructure and established commercialization solutions to complement our launch strategy for the potential launch of avutometinib and defactinib in patients with KRAS mt LGSOC planned for middle of 2025. As of December 31, 2024, we have committed to spend approximately $60.0 million under the IQVIA Master Services Agreement which we expect to spend in the next three to four years.
As discussed in Note 13. License, collaboration and commercial agreements to the consolidated financial statements located in this Annual Report on Form 10-K, we are party to several agreements to license intellectual property. The license agreements may require us to pay upfront license fees, ongoing annual license maintenance fees, milestone payments, minimum royalty payments, as well as reimbursement of certain patent costs incurred by the licensors, as applicable. As of December 31, 2024, we do not have any minimum contractual obligations in relation to these agreements because: there were no upfront license fees payable in future periods; no annual license maintenance fees; we cannot estimate if milestone and/or royalty payments will occur in future periods; and patent cost reimbursement costs are perpetual and the agreements are cancelable by us at any time upon prior written notice to the licensor.
TAX LOSS CARRYFORWARDS
As of December 31, 2024, we had federal and state NOL carryforwards of $370.6 million and $56.7 million, respectively, which are available to reduce future taxable income. We also had federal and state tax credits of $2.6 million and $0.2 million, respectively, which may be used to offset future tax liabilities. The NOL and tax credit carryforwards will expire at various dates through 2044, except for $333.4 million of federal NOL carryforwards which may be carried forward indefinitely. NOL and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the IRC, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. At December 31, 2024, we recorded a 100% valuation allowance against our NOL and tax credit carryforwards, as we believe it is more likely than not that the tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our tax carryforwards will be realized, net income would increase in the period of determination.
Based on our analysis under Section 382 of the IRC and similar provisions under state law, we believe that our federal NOL carryforwards, our state NOL carryforwards, our research and development (“R&D”) credits and our Orphan Drug (“OD”) credits will be limited as of December 31, 2024. The portion of federal NOL, state NOL, R&D credits and OD credits that were determined to be limited by Section 382 have been written off as of December 31, 2024. The remaining unused carryforwards remain available for future periods. During 2024, we believe we triggered ownership changes under Section 382 of the IRC and similar provisions under state law. We
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have approximately $346.4 million of federal NOLs generated prior to such ownership changes inclusive of $309.3 million of federal NOLs which may be carried forward indefinitely. Since the $309.3 million of federal NOLs may be carried forward indefinitely these have not been written off as of December 31, 2024, but due to the limitations under Section 382, generally we can only use $1.6 million per year against taxable income in the future. Due to our full valuation allowance the write off of NOLs, R&D credits, and OD credits did not have any impact to the statements of operation and comprehensive loss for the 2024 Period and 2023 Period.
RECENTLY ADOPTED ACCOUNTING STANDARDS
Refer to Note 2. Significant Accounting Policies to our consolidated financial statements located in this Annual Report on Form 10-K for recently adopted accounting standards.
FY 2023 10-K MD&A
SEC filing source: 0001558370-24-003250.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and related notes appearing elsewhere in this Annual Report on Form 10-K. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and as set forth under “Risk Factors.” Please also refer to the section under the heading “Forward-Looking Statements.”
OVERVIEW
We are a late-stage development biopharmaceutical company, with an ongoing registration directed trial, committed to advancing new medicines for patients battling cancer. Our pipeline is focused on novel anticancer agents that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, particularly RAF/MEK inhibition and FAK inhibition.
Our most advanced product candidates, avutometinib and defactinib, are being investigated in both preclinical and clinical studies for the treatment of various solid tumors, including, but not limited to LGSOC, NSCLC, pancreatic cancer, CRC, and melanoma. We believe that avutometinib may be beneficial as a therapeutic as a single agent or when used together in combination with defactinib, other agents, other pathway inhibitors, or other current and emerging standard of care treatments in cancers that do not adequately respond to currently available therapies.
On August 10, 2020, we and Secura signed the Secura APA and on September 30, 2020, the transaction closed. Pursuant to the Secura APA, we sold to Secura our exclusive worldwide license for the research, development, commercialization, and manufacture in oncology indications of products containing duvelisib. Refer to Note 13. License, collaboration and commercial agreements in our consolidated financial statements located in this Annual Report on Form 10-K for a detailed description of the terms and conditions of the Secura APA. With the transition of the duvelisib program to Secura, we are focusing our efforts on our lead product candidates, avutometinib and defactinib.
Our operations to date have been organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020. We have financed our operations to date primarily through public offerings of our common stock and pre-funded warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, the proceeds in connection with the private investment in public equity (the “PIPE”), and our loan and security agreement executed with Oxford in March 2022, sales of Series B Convertible Preferred Stock. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue.
As of December 31, 2023, we had an accumulated deficit of $824.9 million. Our net loss was $87.4 million, $73.8 million, and $71.2 million, for the years ended December 31, 2023, 2022, and 2021, respectively. As of December 31, 2023, we had cash, cash equivalents, and investments of $137.1 million. In accordance with applicable accounting standards, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within 12 months after the date of the issuance of the consolidated financial statements. We anticipate operating losses may continue for the foreseeable future since we do not yet have regulatory approval to sell any of our product candidates, and we continue to incur operating costs to execute our strategic plan, including costs related to research and development of our product candidates and commercial readiness activities. These conditions raise substantial doubt about our ability to continue as a going concern for 12 months after the date the consolidated financial statements are issued.
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We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura APA, through the loan and security agreement with Oxford, or through other strategic financing opportunities that could include, but are not limited to collaboration agreements, future offerings of our equity, or the incurrence of debt. However, given the risk associated with these potential strategic or financing opportunities, they are not deemed probable for purposes of the going concern assessment. If we fail to obtain additional future capital, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities. Therefore, there is substantial doubt about our ability to continue as a going concern.
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FINANCIAL OPERATIONS OVERVIEW
Revenue
Sale of licenses and related assets revenue to date have been generated through our sale of our duvelisib license and related assets to Secura. The sale included intellectual property related to duvelisib in oncology indications, certain existing duvelisib inventory, certain manufacturing equipment and, claims and rights under certain contracts pertaining to duvelisib including net contract prepaid balances.
Research and development expenses
Research and development expenses consist of costs associated with our research activities, including the development of our product candidates. Research and development expenses include product/ product candidate and/or project-specific costs, as well as unallocated costs. We allocate the expenses related to external research and development services, such as contract research organizations (“CROs”), clinical sites, manufacturing organizations and consultants, by project and/or product candidate. We use our employee and infrastructure resources in a cross-functional manner across multiple research and development projects. Our project costing methodology does not allocate personnel, infrastructure and other indirect costs to specific clinical programs or projects.
Product/ product candidate/ project specific costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct third-party costs, which include expenses incurred under agreements with CROs, the cost of consultants who assist with the development of our product candidates on a program-specific basis, clinical site costs, and any other third-party expenses directly attributable to the development of the product candidates; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | costs related to contract manufacturing operations including manufacturing costs in connection with producing product candidates for use in conducting preclinical and clinical studies. Costs associated with manufacturing avutometinib are included in “Avutometinib manufacturing and non-clinical trial specific” category below as these costs relate to both the “Avutometinib + defactinib” and “Avutometinib + other combinations” categories and are not specifically allocated to any particular project. Costs to produce defactinib are included in “Avutometinib + defactinib” below; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | license fees. |
Unallocated costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | research and development employee-related expenses, including salaries, benefits, travel, and stock-based compensation expense; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | cost of consultants, including our scientific advisory board, who assist with our research and development but are not allocated to a specific program; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | facilities, depreciation, and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, and laboratory supplies. |
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The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the years ended December 31, 2023, 2022, and 2021:
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2023 | 2022 | 2021 | ||||||
| | | (in thousands) | | (in thousands) | | (in thousands) | ||||
| Product/ product candidate / project specific costs | | | | | | | | | | |
| Avutometinib + defactinib | | $ | 28,683 | | $ | 22,374 | | $ | 17,025 | |
| Avutometinib + other combinations | | | 5,323 | | | 2,373 | | | 416 | |
| Avutometinib manufacturing and non-clinical trial specific | | 5,528 | | 8,204 | | 5,441 | | |||
| GenFleet | | | 2,177 | | | — | | | — | |
| COPIKTRA | | 93 | | 183 | | 1,194 | | |||
| Unallocated costs | | | | | | | | | | |
| Personnel costs, excluding stock-based compensation | | | 12,299 | | | 10,848 | | | 9,953 | |
| Stock-based compensation expense | | | 1,987 | | | 1,766 | | | 2,099 | |
| Other unallocated expenses | | | 5,266 | | | 4,810 | | | 3,219 | |
| Total research and development expense | | $ | 61,356 | | $ | 50,558 | | $ | 39,347 | |
Costs for certain development activities, such as clinical trial expenses, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, and information provided us by our vendors on their actual costs incurred or level of effort expended. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid expenses and other current assets or accrued expenses.
Our research and development expenses may increase significantly in future periods as we undertake costlier development activities for our existing and future product candidates, including larger and later-stage clinical trials.
The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete development of our product candidates or the period, if any, in which material net cash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | clinical trial results; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, rate of progress, and expense of our research and development activities, including preclinical research and clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the potential benefits of our product candidates over other therapies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to market, commercialize, and achieve market acceptance for any of our product candidates that we receive regulatory approval for; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the terms and timing of regulatory approvals; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the expense of filing, prosecuting, defending, and enforcing patent claims and other intellectual property rights. |
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us to conduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or if we experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.
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Selling, general, and administrative expenses
Selling, general, and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, in our executive, finance, legal, information technology, commercial, communication, human resources, and business development functions. Other selling, general, and administrative expenses include allocated facility costs, commercial costs, professional fees for legal, patent, investor and public relations, consulting, insurance premiums, audit, tax, and other public company costs.
Other income, other expense, interest income and interest expense
Other expense for the year ended December 31, 2023 was comprised of transaction losses due to changes in foreign currency exchange rates. Other income for the year ended December 31, 2022 was comprised of a gain on the sale of fixed assets and transaction gains due to changes in foreign currency exchange rates. There was no other income or other expense for the year ended December 31, 2021.
Interest income reflects interest earned on our cash, cash equivalents and available-for-sale securities.
Interest expense reflects interest expense due on our Loan Agreement with Oxford and our convertible notes, as well as non-cash interest related to the amortization of debt discount and issuance costs.
Change in fair value of preferred stock tranche liability
The change in fair value of preferred stock tranche liability for the year ended December 31, 2023, was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement (the “Series B Preferred Stock Securities Purchase Agreement”), dated January 24, 2023 with certain purchasers pursuant to which the Company agreed to sell and issue to the purchasers in a private placement up to 2,144,160 shares of its Series B Convertible Preferred Stock in two tranches. There was no preferred stock tranche liability outstanding during the years ended December 31, 2022 and December 31, 2021.
CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles (“GAAP”). The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued and prepaid research and development expenses, stock-based compensation, revenue recognition, and collaborative agreements, described in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding and evaluating our financial condition and results of operations.
Revenue recognition
We recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services in accordance with Accounting Standards Codification (“ASC”) Topic 606 Revenue from Contracts with Customers.
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Sales of intellectual property
For sales of license and intellectual property, that include sale-based royalties, including milestone payments based on a level of sales, we evaluate whether the royalties and sales-based milestones are considered probable of being achieved and estimate the amount of royalties to include over the contractual term using the expected value method and estimate the sales-based milestones using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated royalty and milestone value is included in the transaction price. Royalties and sales-based milestones for territories for which there is not regulatory approval are not considered probable until such regulatory approval is achieved. We evaluate factors such as whether consideration is outside of our control, timeline for when the uncertainty will be resolved and historical sales of COPIKTRA if applicable. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, we reevaluate the probability of achievement of all milestones subject to constraint and amount of royalty revenue to be received and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. At December 31, 2023, we determined less than $0.1 million of future potential royalties, we expect to receive pursuant to the Secura APA, were not constrained and have been recorded in the transaction price. As the consideration for future royalties is conditional, we recorded a corresponding contract asset for the expected future royalties. Portions of the contract asset are reclassified to accounts receivable when the right to consideration becomes unconditional.
Refer to Note 2. Significant Accounting Policies and Note 13. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of revenue.
Collaborative Arrangements
Collaborative Arrangements: Contracts are considered to be collaborative arrangements pursuant to GAAP when they satisfy the following criteria defined in ASC Topic 808, Collaborative Arrangements: (i) the parties to the contract must actively participate in the joint operating activity and (ii) the joint operating activity must expose the parties to the possibility of significant risk and rewards, based on whether or not the activity is successful. Payments received from or made to a partner that are the result of a collaborative relationship with a partner, instead of a customer relationship, such as co-development activities, are recorded as a reduction or increase to research and development expense, respectively.
Accrued and prepaid research and development expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued expenses. This process involves reviewing contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly in arrears for services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to CROs in connection with research and development activities for which we have not yet been invoiced.
We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant to quotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided and result in a prepayment of the research and development expense. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular
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period. To date, there have been no material differences between our estimates of such expenses and the amounts actually incurred.
Refer to Note 2. Significant Accounting Policies, and Note 4. Accrued expenses to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of accrued research and development expenses.
Stock-based compensation
For service-based awards, we recognize stock-based compensation expense for stock options, and restricted stock units (“RSUs”) issued to employees, directors and consultants based on the grant date fair value of the awards on a straight-line basis over the requisite service period. In addition, we issue shares under our employee stock purchase plan (“ESPP”) to employees. The fair value of our stock options and ESPP grants is estimated at the date of grant using the Black-Scholes option pricing model.
We have also granted performance-based RSUs and stock options with terms that allow the recipients to vest in a specific number of shares based upon the achievement of performance-based milestones as specified in the grants. Stock-based compensation expense associated with these performance-based RSUs and stock options is recognized if the performance condition is considered probable of achievement using management’s best estimates of the time to vesting for the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from our estimates, stock-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current and prior periods of a change in the estimated time to vesting for performance-based RSUs and stock options will be recognized as compensation cost in the period of the revision, and recorded as a change in estimate.
While the assumptions used to calculate and account for stock-based compensation awards represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, our stock-based compensation expense could vary significantly from period to period.
During the year ended December 31, 2023, we recorded $5.9 million of stock-based compensation expense. As of December 31, 2023, there was approximately $9.2 million of unrecognized stock-based compensation related to stock options, which are expected to be recognized over a weighted-average period of 2.6 years. As of December 31, 2023, there was approximately $3.1 million of unrecognized stock-based compensation related to RSUs, which are expected to be recognized over a weighted-average period of 2.1 years. See Note 2. Significant accounting policies and Note 8. Stock-based compensation to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of stock-based compensation.
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RESULTS OF OPERATIONS
All financial information presented has been consolidated and includes the accounts of our wholly-owned subsidiaries, Verastem Securities Company and Verastem Europe GmbH. All intercompany balances and transactions have been eliminated in consolidation.
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year Ended December 31, | |||||||
| | | | | | | | | | |
| | 2023 | 2022 | 2021 | ||||||
| Revenue: | | | | | | | | | |
| Sale of COPIKTRA license and related assets | | | — | | | 2,596 | | | 1,447 |
| Transition services revenue | | | — | | | — | | | 606 |
| Total revenue | | — | | 2,596 | | 2,053 | |||
| Operating expenses: | | | | | | | | | |
| Research and development | | | 61,356 | | | 50,558 | | | 39,347 |
| Selling, general and administrative | | 30,728 | | 24,975 | | 24,115 | |||
| Total operating expenses | | 92,084 | | 75,533 | | 63,462 | |||
| Loss from operations | | (92,084) | | (72,937) | | (61,409) | |||
| Other income (expense) | | | (109) | | | 47 | | | — |
| Interest income | | 6,214 | | 1,215 | | 181 | |||
| Interest expense | | (4,139) | | (2,137) | | (9,972) | |||
| Change in fair value of preferred stock tranche liability | | | 2,751 | | | — | | | — |
| Net loss | | (87,367) | | (73,812) | | (71,200) |
Comparison of the Year Ended December 31, 2023 to the Year Ended December 31, 2022
Sale of COPIKTRA license and related assets revenue. Sale of COPIKTRA license and related assets revenue for the year ended December 31, 2023 (the “2023 Period”) was $0.0 million compared to $2.6 million for the year ended December 31, 2022 (the “2022 Period”). Sale of COPIKTRA license and related assets revenue for the 2022 Period was comprised of one regulatory milestone for $2.5 million achieved by Secura’s sublicensee, CSPC, and $0.1 million related to royalties on COPIKTRA sales in the 2022 Period and future royalties expected to be received pursuant to the Secura APA that are not constrained.
Research and development expense. Research and development expense for the 2023 Period was $61.4 million compared to $50.6 million for the 2022 Period. The $10.8 million increase from the 2022 Period to the 2023 Period was primarily related to an increase of $6.1 million in CRO costs, a $2.0 million upfront payment made in September 2023 pursuant to the GenFleet Agreement, an increase of $1.8 million in personnel related costs, including non-cash stock-based compensation, an increase of $1.5 million in IST costs, an increase of $0.9 million in consulting costs, and an increase of $0.7 million in investigator fees. The increase is partially offset by a decrease of $2.2 million in drug substance and drug manufacturing costs.
Selling, general and administrative expense. Selling, general and administrative expense for the 2023 Period was $30.7 million compared to $25.0 million for the 2022 Period. The increase of $5.7 million from the 2022 Period to the 2023 Period primarily resulted from an increase of $1.7 million in personnel related costs, including non-cash stock-based compensation, an increase of $1.4 million of costs in anticipation of the potential launch of avutometinib and defactinib in LGSOC, an increase of $1.2 million in consulting and professional fees, an increase of $0.6 million in costs associated with financing activities, and an increase of $0.8 million in travel and other costs.
Other Income (expense). Other expense of $0.1 million for the 2023 Period was comprised of transaction losses due to changes in foreign currency exchange rates compared to other income of less than $0.1 million for the 2022 Period comprised of a gain on the sale of fixed assets and transaction gains due to changes in foreign currency exchange rates.
Interest income. Interest income for the 2023 Period was $6.2 million compared to $1.2 million for the 2022 Period. The increase of $5.0 million in interest income was primarily driven by an increase in interest rates and investment balances on short-term investments and cash equivalents.
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Interest expense. Interest expense for the 2023 Period was $4.1 million compared to $2.1 million for the 2022 Period. The increase of $2.0 million from the 2022 Period to the 2023 Period was primarily driven by increased expense on the Loan Agreement as a result of the additional $15.0 million debt drawdown on March 22, 2023.
Change in fair value of preferred stock tranche liability. The change in fair value of the preferred stock tranche liability of $2.8 million for the 2023 Period was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Series B Convertible Preferred Stock Securities Purchase Agreement. There was no preferred stock tranche liability outstanding during the 2022 Period.
LIQUIDITY AND CAPITAL RESOURCES
Sources of liquidity
We have financed our operations to date primarily through public and private offerings of our common stock and pre-funded warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the upfront payments under our license and collaboration agreements with Sanofi, Yakult, and CSPC, the upfront payment under the Secura APA, the proceeds in connection with the PIPE, the Loan Agreement with Oxford, and the issuance of Series B Convertible Preferred Stock. With the commercial launch of COPIKTRA in the United States in September 2018 through our ownership period ending in September 2020, we financed a portion of our operations through product revenue. As of September 30, 2020, we have sold our COPIKTRA license and no longer sell COPIKTRA in the United States. We expect to finance a portion of our business through future potential milestones and royalties received pursuant to the Secura APA.
As of December 31, 2023, we had $137.1 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
Risks and uncertainties include those identified under Item 1A. Risk Factors, in this Annual Report on Form 10-K.
Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below (in thousands):
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2023 | | 2022 | | 2021 | ||||
| Net cash (used in) provided by: | | | | | | | ||||
| Operating activities | | $ | (86,460) | | $ | (63,673) | | $ | (53,502) | |
| Investing activities | | (44,447) | | 66,185 | | | 87 | | ||
| Financing activities | | 134,194 | | 51,784 | | | 6,885 | | ||
| Increase (decrease) in cash, cash equivalents and restricted cash | | $ | 3,287 | | $ | 54,296 | | | (46,530) | |
Operating activities. The use of cash in operating activities in the 2023 Period and 2022 Period resulted primarily from our net losses adjusted for non-cash adjustments and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash adjustments was $85.5 million and $67.6 million for the 2023 Period and 2022 Period, respectively. Non-cash adjustments were primarily related to stock-based compensation expense, non-cash interest, net, and change in fair value on preferred stock tranche liability in the 2023 Period and stock-based compensation expense in the 2022 Period. Our cash outflow for the 2023 Period from operating activities due to changes in operating assets and liabilities was $1.0 million for the 2023 Period primarily driven by an increase of $5.8 million in prepaid expenses, other current assets and other assets, and a decrease of $0.4 million in deferred liabilities, partially offset by an increase of $2.9 million in accrued expenses and other liabilities, and an increase of $2.3 million in accounts payable. Our cash inflow for the 2022 Period from operating activities due to changes in operating assets and liabilities was $3.9 million primarily driven by an increase of $2.6
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million in accounts payable, an increase of $0.7 million in deferred liabilities, a decrease of $0.7 million of prepaid expenses, other current assets and other assets, and a decrease of $0.5 million in accounts receivable, partially offset by a $0.6 million decrease in accrued expenses and other liabilities. The changes in prepaid expenses, other current assets, and other assets in both periods are exclusive of cash received from PanCAN and used in the RAMP 205 study. Cash used in operating activities was $86.5 million and $63.7 million for the 2023 Period and the 2022 Period, respectively.
Investing activities. The cash used in investing activities for the 2023 Period primarily relates to the net purchases of investments of $44.4 million. The cash provided by investing activities for the 2022 Period primarily relates to the net maturities of investments of $66.2 million.
Financing activities. The cash provided by financing activities for the 2023 Period primarily represents $91.4 million of proceeds from our previously disclosed public offering in June 2023 of common stock and pre-funded warrants to purchase shares of our common stock, net of issuance costs, $28.1 million of proceeds received from issuance of Series B Convertible Preferred Stock, net of issuance costs, $14.9 million of proceeds received pursuant to the loan and security agreement with Oxford, $1.4 million of proceeds received from insurance premium financing and $0.1 million of proceeds received related to our employee stock purchase plan, partially offset by $1.4 million of payments on insurance premium financing, and $0.3 million of repayment of our 5.00% Convertible Senior Notes due 2048 (the “2018 Notes”). The cash provided by financing activities for the 2022 Period primarily represents $27.4 million of net proceeds received under our at-the market equity offering program, $24.1 million of net proceeds received from the loan and security agreement with Oxford, and $0.3 million of proceeds received related to exercise of stock options and our employee stock purchase plan.
Refer to Note 11. Capital Stock to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the January 2023 offering of our Series B Convertible Preferred Stock, the June 2023 offering of our common stock and pre-funded warrants to purchase shares of our common stock, and our at-the-market equity offering program; Note 5. Debt to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the Loan Agreement; Note 16. Notes Payable to our consolidated financial statements located in this Annual Report on Form 10-K for additional details on the finance agreement with AFCO Premium Credit LLC related to insurance premium financing and the monthly payments of principal and interest related thereto; and Note 10. Convertible Senior Notes to our consolidated financial statements located in this Annual Report on Form 10-K for details on the 2018 Notes.
Funding requirements
We expect to continue to incur significant expenses and may continue to incur operating losses. Refer to risk factor titled We have incurred significant losses since our inception. We may incur losses for the foreseeable future and may never achieve or maintain profitability within section Item 1A. Risk Factors for detailed activities which may drive our continued operating losses and expenses in future periods.
Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs and timing of commercialization activities for our product candidates for which we expect to receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, progress, and results of our ongoing and potential future clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the extent to which we acquire or in-license other product candidates and technologies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs, timing, and outcome of regulatory review of our product candidates (including our efforts to seek approval and fund the preparation and filing of regulatory submissions); |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | revenue received from commercial sales our product candidates, should any of our other product candidates receive marketing approval; |
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| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property rights, and defending intellectual property related claims; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to establish collaborations or partnerships on favorable terms, if at all; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | receipt of milestone payments and royalties pursuant to the Secura APA including timing of such receipt. |
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and through future potential milestones and royalties received pursuant to the Secura APA. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Without additional funding we do not believe that we have sufficient funds to meet our obligations within the next 12 months from the date of issuance of these consolidated financial statements. While we believe that we may have sufficient funds to meet our obligations within the next 12 months from the date of the issuance of the consolidated financial statements for the year ended December 31, 2023, in performing this analysis under the applicable accounting standards management excluded certain elements of our operating plan that cannot be considered probable of occurring. Accordingly, management has concluded that substantial doubt exists about our ability to continue as a going concern for 12 months from the date the consolidated financial statements are issued.
CONTRACTUAL OBLIGATIONS AND COMMITMENTS
On April 15, 2014, we entered into a lease agreement for approximately 15,197 square feet of office and laboratory space in Needham, Massachusetts. The lease term commenced on April 15, 2014, and it was scheduled to expire on September 30, 2019. Effective February 15, 2018, we amended the lease agreement to relocate within the facility to another location consisting of 27,810 square feet of office space (the “Amended Lease Agreement”). The Amended Lease Agreement extends the expiration date of the lease from September 2019 through June 2025. Pursuant to the Amended Lease Agreement, the initial annual base rent amount is approximately $0.7 million, which increases during the lease term to $1.1 million for the last 12-month period. As of December 31, 2023, the total future minimum lease payments under the agreement are $1.6 million through June 2025.
As discussed in Note 13. License, collaboration and commercial agreements to the consolidated financial statements located in this Annual Report on Form 10-K, we are party to several agreements to license intellectual property. The license agreements may require us to pay upfront license fees, ongoing annual license maintenance fees, milestone payments, minimum royalty payments, as well as reimbursement of certain patent costs incurred by the licensors, as applicable. As of December 31, 2023, we do not have any minimum contractual obligations in relation to these agreements because: there were no upfront license fees payable in future periods; no annual license maintenance fees; we cannot estimate if milestone and/or royalty payments will occur in future periods; and patent cost reimbursement costs are perpetual and the agreements are cancelable by us at any time upon prior written notice to the licensor.
TAX LOSS CARRYFORWARDS
As of December 31, 2023, we had federal and state net operating loss (“NOL”) carryforwards of $473.6 million and $189.0 million, respectively, which are available to reduce future taxable income. We also had federal and state tax credits of $9.5 million and $2.2 million, respectively, which may be used to offset future tax liabilities.
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The net operating loss and tax credit carryforwards will expire at various dates through 2043, except for $277.9 million of federal net operating loss carryforwards which may be carried forward indefinitely. Net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. At December 31, 2023, we recorded a 100% valuation allowance against our NOL and tax credit carryforwards, as we believe it is more likely than not that the tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our tax carryforwards will be realized, net income would increase in the period of determination.
Based on our analysis under Section 382 of the Internal Revenue Code and similar provisions under state law, we believe that our federal net operating loss carryforwards, our state net operating loss carryforwards, our research and development (“R&D”) credits and our Orphan Drug (“OD”) credits will be limited as of December 31, 2023. The portion of federal NOL, state NOL, R&D credits and OD credits that were determined to be limited by Section 382 have been written off as of December 31, 2023. The remaining unused carryforwards remain available for future periods. Due to our full valuation allowance the write off of NOLs, R&D credits, and OD credits did not have any impact to the statements of operation and comprehensive loss for the 2023 Period and 2022 Period.
RECENTLY ADOPTED ACCOUNTING STANDARDS
Refer to Note 2. Significant Accounting Policies to our consolidated financial statements located in this Annual Report on Form 10-K for recently adopted accounting standards.
FY 2022 10-K MD&A
SEC filing source: 0001558370-23-003738.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and related notes appearing elsewhere in this Annual Report on Form 10-K. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and as set forth under “Risk Factors.” Please also refer to the section under the heading “Forward-Looking Statements.”
OVERVIEW
We are a late-stage development biopharmaceutical company, with an ongoing registration directed trial, committed to advancing new medicines for patients battling cancer. Our pipeline is focused on novel anticancer agents that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, particularly RAF/MEK inhibition and FAK inhibition.
Our most advanced product candidates, avutometinib (VS-6766) and defactinib, are being investigated in both preclinical and clinical studies for the treatment of various solid tumors, including, but not limited to low-grade serous ovarian cancer (“LGSOC”), non-small cell lung cancer (“NSCLC”), colorectal cancer (“CRC”), pancreatic cancer, and melanoma. We believe that avutometinib may be beneficial as a therapeutic as a single agent or when used together in combination with defactinib, other agents, other pathway inhibitors or other current and emerging standard of care treatments in cancers that do not adequately respond to currently available therapies.
On August 10, 2020, we and Secura Bio, Inc. (“Secura”) signed an Asset Purchase Agreement (“Secura APA”) and on September 30, 2020, the transaction closed. Pursuant to the Secura APA, we sold our exclusive worldwide license for the research, development, commercialization, and manufacture in oncology indications of products containing duvelisib. Refer to Note 13. License, collaboration and commercial agreements in our consolidated financial statements located in this Annual Report on Form 10-K for a detailed description of the terms and conditions of the Secura APA. With the transition of the duvelisib program to Secura, we are focusing our efforts on our lead product candidates, avutometinib and defactinib.
Our operations to date have been organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020. We have financed our operations to date primarily through public offerings of our common stock, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules Capital, Inc. (“Hercules”) in March 2017, as amended, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC Pharmaceutical Group Limited (“CSPC”), and Yakult Honsha Co., Ltd. (“Yakult”), the upfront payment and milestone payments received under the Secura APA, the issuance of the 2018 Notes (defined herein) in October 2018, the proceeds in connection with the private investment in public equity (the “PIPE”), and our loan and security agreement executed with Oxford Finance LLC (“Oxford”) in March 2022. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue.
As of December 31, 2022, we had an accumulated deficit of $737.5 million. Our net loss was $73.8 million, $71.2 million, and $67.7 million, for the years ended December 31, 2022, 2021, and 2020, respectively. We expect to incur significant expenses and may continue to incur operating losses for the foreseeable future as a result of the continued research and development of avutometinib and defactinib. As of December 31, 2022, we had cash, cash equivalents, and investments of $87.9 million. We expect our existing cash resources, $30.0 million gross proceeds from our Series B Preferred Stock issuance in January 2023, and expected $15.0 million debt drawdown through our loan and security agreement with Oxford expected in March 2023 will be sufficient to fund our planned operations through at least 12 months from the date of issuance of these consolidated financial statements. See Note
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16. Subsequent events located in this Annual Report on Form 10-K for further discussion of the Series B Preferred Stock issuance.
We expect to finance the future development costs of our clinical product portfolio with our existing cash, cash equivalents and investments, through future milestones and royalties received pursuant to the Secura APA, through our loan and security agreement with Oxford, or through other strategic financing opportunities that could include, but are not limited to, collaboration agreements, future offerings of our equity, or the incurrence of debt. However, there is no guarantee that any of these strategic or financing opportunities will be executed or executed on favorable terms, and some could be dilutive to existing stockholders. If we fail to obtain additional future capital, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities.
COVID-19 pandemic
The current COVID-19 pandemic has presented a substantial public health and economic challenge around the world and is affecting our employees, patients, communities and business operations, as well as the U.S. economy and financial markets. We have been carefully monitoring the COVID-19 pandemic and its impact on our operations. Our corporate headquarters remains open and we have adopted a hybrid work program allowing our employees the option to primarily work from home. Shortages in personnel in clinics and hospitals have caused some United States sites to institute limits on new clinical trials which could impact our ability to open new sites for our clinical trials. In addition, clinics in Europe and the United States continue to have delays in startup activities due to the ongoing pandemic and the increase in COVID-19 variant infections. The full extent to which the COVID-19 pandemic will directly or indirectly impact our business, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted, including new information that may emerge concerning COVID-19, new variants, the actions taken to contain it or treat its impact and the economic impact on local, regional, national, and international markets.
For additional information on the various risks posed by the COVID-19 pandemic, please read Item 1A. Risk Factors included within.
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FINANCIAL OPERATIONS OVERVIEW
Revenue
From our U.S. commercial launch of COPIKTRA on September 24, 2018 through our ownership period ending in September 2020 product revenue, net represented the gross sales of COPIKTRA in the United States less provisions for product sales allowances and accruals. These provisions included trade allowances, rebates, chargebacks and discounts, product returns and other incentives. We sold COPIKTRA to a limited number of specialty pharmacies and specialty distributors. The provisions for product sales and allowances fluctuated based on the mix of sales to either specialty pharmacy or specialty distributor customers. See “Critical Accounting Policies and Significant Judgements and Estimates” below and Note 2. Significant Accounting Policies located within the notes to the financial statements included within for more information on the components of net U.S. product sales of COPIKTRA.
License and collaboration revenue to date has been generated through our license and collaboration agreements for the development and commercialization of duvelisib with Sanofi in the Sanofi Territory, CSPC in China, and Yakult in Japan. The terms of these agreements contain multiple deliverables which may include (i) licenses, (ii) research and development activities, and (iii) the manufacture of finished drug product, active pharmaceutical ingredient (“API”), or development materials for a partner, which are reimbursed at a contractually determined rate. Payments to us may include (i) up‑front license fees, (ii) payments for research and development activities, (iii) payments for the manufacture of finished drug product, API or development materials, (iv) payments based upon the achievement of certain milestones, and (v) royalties on product sales.
Sale of licenses and related assets revenue to date have been generated through our sale of our duvelisib license and related assets to Secura. The sale included intellectual property related to duvelisib in oncology indications, certain existing duvelisib inventory, certain manufacturing equipment and, claims and rights under certain contracts pertaining to duvelisib including net contract prepaid balances.
Costs of sales - product
Cost of sales - product consisted of costs of COPIKTRA on which product revenue was recognized, royalties owed to Healthcare Royalty Partners (“HCR”) and Infinity we incurred as a result of such sales of COPIKTRA, and certain period costs. Certain of the costs of COPIKTRA units recognized as revenue during 2020 were expensed prior to the September 2018 FDA marketing approval and, therefore, are not included in cost of sales during 2020. There were no cost of sales – product in 2022 and 2021.
Costs of sales - sale of COPIKTRA license and related assets
Cost of sales - sale of COPIKTRA license and related assets represent assets delivered to Secura as part of the sale pursuant to the Secura APA. This included our intangible assets, certain duvelisib inventory, net duvelisib contract prepaid balances, and manufacturing equipment. There were no cost of sales –sale of COPIKTRA license and related assets in 2022 and 2021.
Research and development expenses
Research and development expenses consist of costs associated with our research activities, including the development of our product candidates. Research and development expenses include product/ product candidate and/or project-specific costs, as well as unallocated costs. We allocate the expenses related to external research and development services, such as contract research organizations (“CROs”), clinical sites, manufacturing organizations and consultants, by project and/or product candidate. We use our employee and infrastructure resources in a cross-functional manner across multiple research and development projects. Our project costing methodology does not allocate personnel, infrastructure and other indirect costs to specific clinical programs or projects.
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Product/ product candidate/ project specific costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | direct third-party costs, which include expenses incurred under agreements with CROs, the cost of consultants who assist with the development of our product candidates on a program-specific basis, clinical site costs, and any other third-party expenses directly attributable to the development of the product candidates; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | costs related to contract manufacturing operations including manufacturing costs in connection with producing product candidates for use in conducting preclinical and clinical studies. Costs associated with manufacturing avutometinib are included in “Avutometinib manufacturing and non-clinical trial specific” category below as these costs relate to both the “Avutometinib + defactinib” and “Avutometinib + other combinations” categories and are not specifically allocated to any particular project. Costs to produce defactinib are included in “Avutometinib + defactinib” below; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | license fees. |
Unallocated costs include:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | research and development employee-related expenses, including salaries, benefits, travel, and stock-based compensation expense; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | cost of consultants, including our scientific advisory board, who assist with our research and development but are not allocated to a specific program; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | facilities, depreciation, and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, and laboratory supplies. |
The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the years ended December 31, 2022, 2021, and 2020:
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2022 | 2021 | 2020 | ||||||
| | | (in thousands) | | (in thousands) | | (in thousands) | ||||
| Product/ product candidate / project specific costs | | | | | | | | | | |
| Avutometinib + defactinib | | $ | 22,374 | | $ | 17,025 | | $ | 6,199 | |
| Avutometinib + other combinations | | | 2,373 | | | 416 | | | — | |
| Avutometinib manufacturing and non-clinical trial specific | | 8,204 | | 5,441 | | 5,874 | | |||
| COPIKTRA | | 183 | | 1,194 | | 13,454 | | |||
| Unallocated costs | | | | | | | | | | |
| Personnel costs, excluding stock-based compensation | | | 10,848 | | | 9,953 | | | 8,937 | |
| Stock-based compensation expense | | | 1,766 | | | 2,099 | | | 1,935 | |
| Other unallocated expenses | | | 4,810 | | | 3,219 | | | 4,977 | |
| Total research and development expense | | $ | 50,558 | | $ | 39,347 | | $ | 41,376 | |
Costs for certain development activities, such as clinical trial expenses, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, and information provided us by our vendors on their actual costs incurred or level of effort expended. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid expenses and other current assets or accrued expenses.
Our research and development expenses may increase significantly in future periods as we undertake costlier development activities for our existing and future product candidates, including larger and later-stage clinical trials.
The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete
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development of our product candidates or the period, if any, in which material net cash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | clinical trial results; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, rate of progress, and expense of our research and development activities, including preclinical research and clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the potential benefits of our product candidates over other therapies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to market, commercialize, and achieve market acceptance for any of our product candidates that we receive regulatory approval for; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the terms and timing of regulatory approvals; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the expense of filing, prosecuting, defending, and enforcing patent claims and other intellectual property rights. |
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us to conduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or if we experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.
Selling, general, and administrative expenses
Selling, general, and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, in our executive, finance, legal, information technology, commercial, communication, human resources, and business development functions. Other selling, general, and administrative expenses include allocated facility costs, commercial costs, professional fees for legal, patent, investor and public relations, consulting, insurance premiums, audit, tax, and other public company costs.
Other income, other expense, interest income and interest expense
Other income for the year ended December 31, 2022 was comprised of a gain on the sale of fixed assets and changes in foreign currency exchange rates. There was no other income or other expense for the year ended December 31, 2021. Other expense for the year ended December 31, 2020, consists entirely of the mark-to-market adjustment of the bifurcated make-whole interest provision derivative liability related to the 2019 Notes.
Interest income reflects interest earned on our cash, cash equivalents and available-for-sale securities.
Interest expense reflects interest expense due on our Loan Agreement with Oxford, our term loan facility executed with Hercules and the Notes, as well as non-cash interest related to the amortization of debt discount and issuance costs.
CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles (“GAAP”). The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued and prepaid research and development expenses, leases, stock-based compensation, revenue recognition, and collaborative agreements, described in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
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Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding and evaluating our financial condition and results of operations.
Revenue recognition
We recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services in accordance with Accounting Standards Codification (“ASC”) Topic 606 Revenue from Contracts with Customers (ASC 606).
Product revenue, net
Product Revenue, Net – From September 24, 2018 (the date of our U.S. commercial launch of COPIKTRA) through September 30, 2020 (the date we sold COPIKTRA to Secura), we sold COPIKTRA to a limited number of specialty pharmacies and specialty distributors in the United States. We recognized revenue on sales of COPIKTRA when a customer obtains control of the product, which occurs at a point in time (typically upon delivery). Product revenues are recorded at the wholesale acquisition costs, net of applicable reserves for variable consideration. Components of variable consideration include trade discounts and allowances, Third-Party Payer chargebacks and discounts, government rebates, other incentives, such as voluntary co-pay assistance, product returns, and other allowances that are offered within contracts between us and customers, payors, and other indirect customers relating to our sale of COPIKTRA. These reserves, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable or a current liability. These estimates take into consideration a range of possible outcomes based upon relevant factors such as customer contract terms, information received from third parties regarding the anticipated payor mix for COPIKTRA, known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which it is entitled with respect to sales made.
The amount of variable consideration which is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under contracts will not occur in a future period. Our analyses contemplate the application of the constraint in accordance with ASC 606. For the year ended December 31, 2020, we determined a material reversal of revenue would not occur in a future period for variable consideration, and the transaction price was not reduced further. There was not any product revenue, net recorded for the years ended December 31, 2022 and 2021. Actual amounts of consideration ultimately received could differ from our estimates. However, as of December 31, 2022, we do not have any reserve balances associated with product revenue, net.
Licenses and sales of intellectual property
Licenses of Intellectual Property - We may enter into collaboration and licensing arrangements for research and development, manufacturing, and commercialization activities with collaboration partners for the development and commercialization of our product candidates, which have components within the scope of ASC 606. The arrangements generally contain multiple elements or deliverables, which may include (i) licenses, or options to obtain licenses, to our intellectual property or sale of our license, (ii) research and development activities performed for the collaboration partner, (iii) participation on joint steering committees, and (iv) the manufacturing of commercial, clinical or preclinical material. Payments pursuant to these arrangements typically include non-refundable, upfront payments, milestone payments upon the achievement of significant development events, research and development reimbursements, sales milestones, and royalties on product sales. The amount of variable consideration is constrained until it is probable that the revenue is not at a significant risk of reversal in a future period. The contracts into which we enter generally do not include significant financing components.
In determining the appropriate amount of revenue to be recognized as we fulfill our obligations under each of our collaboration and license agreements, we perform the following steps: (i) identification of the promised goods or services in the contract within the scope of ASC 606; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the
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transaction price to the performance obligations; and (v) recognition of revenue when (or as) we satisfy each performance obligation. As part of the accounting for these arrangements, we must use significant judgment to determine: a) the number of performance obligations based on the determination under step (ii) above; b) the transaction price under step (iii) above; c) the stand-alone selling price for each performance obligation identified in the contract for the allocation of transaction price in step (iv) above; and d) the measure of progress in step (v) above. We use judgment to determine whether milestones or other variable consideration, except for royalties on license arrangements, should be included in the transaction price.
For sales of license and intellectual property, that include sale-based royalties, including milestone payments based on a level of sales, we evaluate whether the royalties and sales-based milestones are considered probable of being achieved and estimate the amount of royalties to include over the contractual term using the expected value method and estimate the sales-based milestones using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated royalty and milestone value is included in the transaction price. Royalties and sales-based milestones for territories for which there is not regulatory approval are not considered probable until such regulatory approval is achieved. We evaluate factors such as whether consideration is outside of our control, timeline for when the uncertainty will be resolved and historical sales of COPIKTRA if applicable. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, we reevaluate the probability of achievement of all milestones subject to constraint and amount of royalty revenue to be received and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. At December 31, 2022, we determined $0.1 million of future potential royalties, we expect to receive pursuant to the Secura APA, were not constrained and have been recorded in the transaction price. As the consideration for future royalties is conditional, we recorded a corresponding contract asset for the expected future royalties. Portions of the contract asset are reclassified to accounts receivable when the right to consideration becomes unconditional.
Refer to Note 2. Significant Accounting Policies, Note 6. Product revenue reserves and allowances and Note 13. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of revenue.
Collaborative Arrangements
Collaborative Arrangements: Contracts are considered to be collaborative arrangements pursuant to GAAP when they satisfy the following criteria defined in ASC Topic 808, Collaborative Arrangements (ASC 808): (i) the parties to the contract must actively participate in the joint operating activity and (ii) the joint operating activity must expose the parties to the possibility of significant risk and rewards, based on whether or not the activity is successful. Payments received from or made to a partner that are the result of a collaborative relationship with a partner, instead of a customer relationship, such as co-development activities, are recorded as a reduction or increase to research and development expense, respectively.
Accrued and prepaid research and development expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued expenses. This process involves reviewing contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly in arrears for services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to CROs in connection with research and development activities for which we have not yet been invoiced.
We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant to quotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services
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provided and result in a prepayment of the research and development expense. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. To date, there have been no material differences between our estimates of such expenses and the amounts actually incurred.
Refer to Note 2. Significant Accounting Policies, and Note 4. Accrued expenses to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of accrued research and development expenses.
Stock-based compensation
For service-based awards, we recognize stock-based compensation expense for stock options, and restricted stock units (“RSUs”) issued to employees, directors and consultants based on the grant date fair value of the awards on a straight-line basis over the requisite service period. In addition, we issue shares under our employee stock purchase plan (“ESPP”) to employees. The fair value of our stock options and ESPP grants is estimated at the date of grant using the Black-Scholes option pricing model.
We have also granted performance-based RSUs and stock options with terms that allow the recipients to vest in a specific number of shares based upon the achievement of performance-based milestones as specified in the grants. Stock-based compensation expense associated with these performance-based RSUs and stock options is recognized if the performance condition is considered probable of achievement using management’s best estimates of the time to vesting for the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from our estimates, stock-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current and prior periods of a change in the estimated time to vesting for performance-based RSUs and stock options will be recognized as compensation cost in the period of the revision, and recorded as a change in estimate.
While the assumptions used to calculate and account for stock-based compensation awards represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, our stock-based compensation expense could vary significantly from period to period.
During the year ended December 31, 2022, we recorded $6.0 million of stock-based compensation expense. As of December 31, 2022, there was approximately $6.6 million of unrecognized stock-based compensation related to stock options, which are expected to be recognized over a weighted-average period of 2.6 years. As of December 31, 2022, there was approximately $4.3 million of unrecognized stock-based compensation related to RSUs, which are expected to be recognized over a weighted-average period of 2.8 years. See Note 2. Significant accounting policies and Note 8. Stock-based compensation to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of stock-based compensation.
Leases
Leases are accounted for in accordance with ASC Topic 842, Leases (“ASC 842”). This standard requires lessees to recognize in the statement of financial position a liability to make lease payments and a right-of-use asset representing its right to use the underlying asset for the lease term for both finance and operating leases.
At the inception of an arrangement, we determine whether the arrangement is or contains a lease based on the unique facts and circumstances within the arrangement. A lease is identified where an arrangement conveys the right to control the use of identified property, plant, and equipment for a period of time in exchange for consideration. Leases which are identified within the scope of ASC 842 and which have a term greater than one year are recognized on our consolidated balance sheets as right-of-use assets, lease liabilities and, if applicable, long-term
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lease liabilities. We have elected not to recognize leases with terms of one year or less on our consolidated balance sheets. Operating lease liabilities and their corresponding right-of-use assets are recorded based on the present value of lease payments over the expected remaining lease term. However, certain adjustments to the right-of-use asset may be required for items such as initial direct costs paid or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, we utilize our incremental borrowing rates to calculate the present value of lease payments. Incremental borrowing rates are the rates we incur to borrow on a collateralized basis over a similar term an amount equal to the lease payments in a similar economic environment. As of December 31, 2022, we have a right-of-use asset of $1.8 million and lease liability of $2.3 million which reflected on the consolidated balance sheets. See Note 2. Significant accounting policies and Note 6. Leases to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of leases.
RESULTS OF OPERATIONS
All financial information presented has been consolidated and includes the accounts of our wholly-owned subsidiaries, Verastem Securities Company and Verastem Europe GmbH. All intercompany balances and transactions have been eliminated in consolidation.
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year Ended December 31, | |||||||
| | | | | | | | | | |
| | 2022 | 2021 | 2020 | ||||||
| Revenue: | | | | | | | | | |
| Product revenue, net | | | — | | | — | | | 15,232 |
| License and collaboration revenue | | | — | | | — | | | 2,912 |
| Sale of COPIKTRA license and related assets | | | 2,596 | | | 1,447 | | | 70,000 |
| Transition services revenue | | | — | | | 606 | | | 372 |
| Total revenue | | 2,596 | | 2,053 | | 88,516 | |||
| Operating expenses: | | | | | | | | | |
| Cost of sales - product | | | — | | | — | | | 1,765 |
| Cost of sales - intangible amortization | | | — | | | — | | | 793 |
| Cost of sales - sale of COPIKTRA license and related assets | | | — | | | — | | | 31,187 |
| Research and development | | | 50,558 | | | 39,347 | | | 41,376 |
| Selling, general and administrative | | 24,975 | | 24,115 | | 62,755 | |||
| Total operating expenses | | 75,533 | | 63,462 | | 137,876 | |||
| Loss from operations | | (72,937) | | (61,409) | | (49,360) | |||
| Other income (expense) | | | 47 | | | — | | | (1,313) |
| Interest income | | 1,215 | | 181 | | 515 | |||
| Interest expense | | (2,137) | | (9,972) | | (15,794) | |||
| Loss on debt extinguishment | | | — | | | — | | | (1,580) |
| Net loss before income taxes | | (73,812) | | (71,200) | | (67,532) | |||
| Income tax expense | | | — | | | — | | | (194) |
| Net loss | | $ | (73,812) | | $ | (71,200) | | $ | (67,726) |
Comparison of the Year Ended December 31, 2022 to the Year Ended December 31, 2021
Sale of COPIKTRA license and related assets revenue. Sale of COPIKTRA license and related assets revenue for the year ended December 31, 2022 (the “2022 Period”) was $2.6 million compared to $1.4 million for the year ended December 31, 2021 (the “2021 Period”). Sale of COPIKTRA license and related assets revenue for the 2022 Period was comprised of one regulatory milestone for $2.5 million achieved by Secura’s sublicensee, CSPC, and $0.1 million related to royalties on COPIKTRA sales in the 2022 Period and future royalties expected to be received pursuant to the Secura APA that are not constrained. Sale of COPIKTRA license and related assets revenue for the 2021 Period primarily related to two regulatory milestones for $1.3 million achieved by Secura’s sublicensee and $0.2 million related to royalties we received and expected to be received pursuant to the Secura APA.
Transition services revenue. Transition services revenue for the 2022 Period was $0.0 million compared to $0.6 million for the 2021 Period. Transition services revenue was comprised of the revenue recognized for us providing certain support functions to Secura pursuant to the transition services agreement, which was entered into
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in connection with the Secura APA (“Secura TSA”). The services were provided at a mutually agreed upon rate. The services were substantially completed in 2021 and there will not be revenue recorded in the future pursuant to the Secura TSA.
Research and development expense. Research and development expense for the 2022 Period was $50.6 million compared to $39.3 million for the 2021 Period. The $11.3 million increase from the 2021 Period to the 2022 Period was primarily related to an increase of $4.1 million in drug substance and drug product costs, an increase of $4.1 million in CRO costs, an increase of $1.5 million in consulting costs, an increase of $0.9 million in investigator fees, an increase of $0.5 million in personnel related costs, including non-cash stock-based compensation, an increase of $0.4 million in clinical supply costs and an increase of $0.8 million in other costs. The increase was partially offset by a decrease of $1.0 million in investigator sponsored trial costs.
Selling, general and administrative expense. Selling, general and administrative expense for the 2022 Period was $25.0 million compared to $24.1 million for the 2021 Period. The increase of $0.9 million from the 2021 Period to the 2022 Period primarily resulted from an increase of $1.1 million of commercial operations costs, an increase of $0.4 million in personnel related costs, including non-cash stock-based compensation and an increase of $0.4 million of other costs. The increase was partially offset by a decrease of $1.0 million of consulting and professional fees.
Other Income. Other income for the 2022 Period was less than $0.1 million compared to $0.0 million in the 2021 Period. Other income for the 2022 Period was comprised of a gain on the sale of fixed assets and changes in foreign currency exchange rates.
Interest income. Interest income for the 2022 Period was $1.2 million compared to $0.2 million for the 2021 Period. The increase of $1.0 million in interest income was primarily driven by an increase in interest rates on debt securities.
Interest expense. Interest expense for the 2022 Period was $2.1 million compared to $10.0 million for the 2021 Period. The decrease of $7.9 million from the 2021 Period to the 2022 Period was primarily driven by $7.8 million of non-cash interest expense recorded in the 2021 Period upon conversion of the 2020 Notes into common stock in July 2021. In addition, as a result of the conversion, there were no interest charges recorded for the 2020 Notes in the 2022 Period. The decrease is partially offset by the interest expense recorded pursuant to the Loan Agreement entered into with Oxford.
LIQUIDITY AND CAPITAL RESOURCES
Sources of liquidity
We have financed our operations to date primarily through public and private offerings of our common stock, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the upfront payments under our license and collaboration agreements with Sanofi, Yakult, and CSPC, the upfront payment under the Secura APA, the issuance of 2018 Notes in October 2018, the proceeds in connection with the PIPE, the Loan Agreement with Oxford, and issuance of our Series B Preferred Stock in January 2023. With the commercial launch of COPIKTRA in the United States in September 2018 through our ownership period ending in September 2020, we financed a portion of our operations through product revenue. As of September 30, 2020, we have sold our COPIKTRA license and no longer sell COPIKTRA in the United States. We expect to finance a portion of our business through potential future milestones and royalties received pursuant to the Secura APA.
As of December 31, 2022, we had $87.9 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
On March 10, 2023, SVB was closed by the California Department of Financial Protection and Innovation, which appointed the FDIC as receiver. On March 12, 2023, the Department of the Treasury, the Federal Reserve,
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and the FDIC announced that all depositors of SVB will be fully protected and have access to all their money starting March 13, 2023. As of March 13, 2023, our deposit balance at SVB was approximately $2 million. We are continually monitoring developments related to the recovery of uninsured funds at SVB.
Risks and uncertainties include those identified under Item 1A. Risk Factors, in this Annual Report on Form 10-K.
Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below (in thousands):
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | |||||||
| | | 2022 | | 2021 | | 2020 | |||
| Net cash (used in) provided by: | | | | | | ||||
| Operating activities | | $ | (63,673) | | $ | (53,502) | | $ | (33,506) |
| Investing activities | | 66,185 | | 87 | | | (47,363) | ||
| Financing activities | | 51,784 | | 6,885 | | | 69,630 | ||
| Increase (decrease) in cash, cash equivalents and restricted cash | | $ | 54,296 | | $ | (46,530) | | | (11,239) |
Operating activities. The use of cash in operating activities in the 2022 Period and 2021 Period resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges was $67.6 million and $54.1 million for the 2022 Period and 2021 Period, respectively. Non-cash charges were primarily related to stock-based compensation expense in the 2022 Period and stock-based compensation expense and non-cash interest, net in the 2021 Period. Our cash inflow from operating activities due to changes in operating assets and liabilities was $3.9 million and $0.6 million for the 2022 Period and 2021 Period, respectively. Cash inflow due to changes in operating assets and liabilities for the 2022 Period was primarily driven by an increase of $2.6 million in accounts payable, an increase of $0.7 million in deferred liabilities, a decrease of $0.7 million of prepaid expenses, other current assets and other assets, and a decrease of $0.5 million in accounts receivable. The decrease in prepaid expenses, other current assets, and other assets is exclusive of the cash received from PanCAN. Cash inflow due to changes in operating assets and liabilities for the 2021 Period was primarily driven by an increase of $1.8 million in accrued expenses, an increase of $0.6 million in accounts payable, partially offset by an increase in $1.6 million in prepaid expenses, other current assets, and other assets. Cash used in operating activities was $63.7 million and $53.5 million for the 2022 Period and 2021 Period, respectively.
Investing activities. The cash provided by investing activities for the 2022 Period primarily relates to the net maturities of investments of $66.2 million. The cash provided by investing activities for the 2021 Period primarily relates to the net maturities of investments of $0.3 million, partially offset by purchases of fixed assets of $0.2 million.
Financing activities. The cash provided by financing activities for the 2022 Period primarily represents $27.4 million of net proceeds received under our at-the market equity offering program, $24.1 million of net proceeds received from the Loan Agreement with Oxford, and $0.3 million of proceeds received related to exercise of stock options and employee stock purchase plan. The cash provided by financing activities for the 2021 Period primarily represents $6.7 million in net proceeds received under our at-the-market equity offering program, and $1.1 million of proceeds received related to exercise of stock options and employee stock purchase plan. This is partially offset by $0.9 million of payments for settlement of restricted stock for tax withholdings.
On March 25, 2022 (the “Closing Date”) we entered into a loan and security agreement (the “Loan Agreement”), with Oxford as collateral agent and a lender, and Oxford Finance Credit Fund III LP, as a lender (“OFCF III” and together with Oxford, the “Lenders”) pursuant to which the Lenders have agreed to lend us up to an aggregate principal amount of $150.0 million in a series of term loans (the “Term Loans”). The initial Term Loan of $25.0 million was funded at the Closing Date of the Loan Agreement, an additional $75.0 million will be available at our option upon achievement of certain milestones as outlined in Note 5. Debt to our consolidated financial statements included in this Annual Report on Form 10-K, and $50.0 million is subject to the Lenders’ sole
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discretion. In January 2023, we met the Term B Milestone as outlined in Note 5. Debt to our consolidated financial statements included in this Annual Report on Form 10-K and we expect to draw down an additional $15.0 million Term Loan in March 2023.
The Term Loans bear interest at a floating rate equal to (a) the greater of (i) the one-month CME Secured Overnight Financing Rate and (ii) 0.13% plus (b) 7.37%, which is subject to an overall floor and cap. Interest is payable monthly in arrears on the first calendar day of each calendar month. Beginning (i) April 1, 2024, if the Term B Loan (as defined in Note 5. Debt to our consolidated financial statements included in this Annual Report on Form 10-K) is not made, (ii) April 1, 2025, if the Term B Loan is made, or (iii) April 1, 2026, if the Term B Loan is made and either (A) avutometinib has received FDA approval for the treatment of LGSOC or (B) COPIKTRA has received FDA approval for the treatment of peripheral T-cell lymphoma (“PTCL”), we shall repay the Term Loans in consecutive equal monthly payments of principal, together with applicable interest, in arrears. All unpaid principal and accrued and unpaid interest with respect to each Term Loan is due and payable in full on March 1, 2027. As we have drawn the Term B Milestone, principal repayments will not be required to commence until at least April 1, 2025.
We are required to make a final payment of 5.0% of the original principal amount of the Term Loans that were drawn, payable at maturity or upon any earlier acceleration or prepayment of the Term Loans. We may prepay all, but not less than all, of the Term Loans, subject to a prepayment fee equal to (i) 3.0% of the principal amount of the applicable Term Loan if prepaid on or before the first anniversary date of the funding date of such Term Loan, (ii) 2.0% of the principal amount of the applicable Term Loan if prepaid after the first anniversary and on or before the second anniversary of the funding date of such Term Loan, and (iii) 1.0% of the principal amount of the applicable Term Loan if prepaid after the second anniversary of the applicable funding date of such Term Loan. All Term Loans are subject to a facility fee of 0.5% of the principal amount.
The Loan Agreement contains no financial covenants. The Loan Agreement includes customary events of default, including, among others, payment defaults, breach of representations and warrants, covenant defaults, judgment defaults, insolvency and bankruptcy defaults, and a material adverse change. The occurrence of an event of default could result in the acceleration of the obligations under the Loan Agreement, termination of the Term Loan commitments and the right to foreclose on the collateral securing the obligations. During the existence of an event of default, the Term Loans will accrue interest at a rate per annum equal to 5.0% above the otherwise applicable interest rate.
In connection with the Loan Agreement, we granted Oxford a security interest in all of our personal property now owned or hereafter acquired, excluding intellectual property (but including the right to payments and proceeds of intellectual property), and a negative pledge on intellectual property.
In August 2021, we entered into a sales agreement with Cantor Fitzgerald & Co. (“Cantor”) pursuant to which we can offer and sell up to $100.0 million of our common stock at the current market prices from time to time through Cantor as sales agent (“August 2021 ATM”). During the 2022 Period and 2021 Period, we sold 23,573,403 shares and 2,930,585 shares, respectively, under the August 2021 ATM for net proceeds of approximately $27.4 million, and $6.8 million, respectively (after deducting commissions and other offering expenses). As of December 31, 2022, we can issue an aggregate amount of $65.1 million of common stock under this program.
On October 17, 2018, we closed a registered direct public offering of $150.0 million aggregate principal amount of our 2018 issued 5.00% Convertible Senior Notes due 2048 (the “2018 Notes”), for net proceeds of approximately $145.3 million. The 2018 Notes are governed by the terms of a base indenture for senior debt securities (the “Base Indenture”), as supplemented by the first supplemental indenture thereto (the “2018 Notes Supplemental Indenture” and together with the Base Indenture, the “2018 Indenture”), each dated October 17, 2018, by and between us and Wilmington Trust, National Association (“Wilmington”), as trustee. The 2018 Notes are senior unsecured obligations of us and bear interest at a rate of 5.00% per annum, payable semi-annually in arrears on May 1 and November 1 of each year. The 2018 Notes will mature on November 1, 2048, unless earlier repurchased, redeemed or converted in accordance with their terms.
The 2018 Notes are convertible into shares of our common stock, par value $0.0001 per share, together, if applicable, with cash in lieu of any fractional share, at an initial conversion rate of 139.5771 shares of common
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stock per $1,000 principal amount of the 2018 Notes, which corresponds to an initial conversion price of approximately $7.16 per share of common stock. Upon conversion, converting noteholders will be entitled to receive accrued interest on their converted 2018 Notes.
We will have the right, exercisable at our option, to cause all 2018 Notes then outstanding to be converted automatically if the “Daily VWAP” (as defined in the 2018 Indenture) per share of our common stock equals or exceeds 130% of the conversion price, which equates to approximately $9.31 per share, on each of at least 20 “VWAP Trading Days” (as defined in the 2018 Indenture), whether or not consecutive, during any 30 consecutive VWAP Trading Day period commencing on or after the date we first issued the 2018 Notes.
In the fourth quarter of 2019, we entered into privately negotiated agreements to exchange approximately $121.7 million aggregate principal amount of the 2018 Notes for (i) approximately $66.9 million aggregate principal amount of 5.00% Convertible Senior Second Lien Notes due 2048 (the “2019 Notes”), (ii) an aggregate of approximately $12.1 million in 2018 Notes principal repayment and (iii) accrued interest on the 2018 Notes through the exchange date. As of March 31, 2020, all 2019 Notes have converted into shares of common stock and are no longer outstanding.
On November 6, 2020, we entered into a privately negotiated agreement with an investor who is a holder of our 2018 Notes to exchange approximately $28.0 million aggregate principal amount of 2018 Notes for approximately $28.0 million aggregate principal amount of newly issued 5.00% Convertible Senior Notes due 2048 (the “2020 Notes”). The issuance of the 2020 Notes closed on November 13, 2020. The 2020 Notes were governed pursuant to the Base Indenture between us and Wilmington dated as of October 17, 2018 as supplemented by the second supplemental indenture thereto dated as of November 13, 2020 (the “2020 Notes Supplemental Indenture” and together with the Base Indenture, the “2020 Indenture”).
We had the right, exercisable at our option, to cause all 2020 Notes then outstanding to be converted automatically if the “Daily VWAP” (as defined in the 2020 Indenture) per share of our common stock equals or exceeds 123.08% of the conversion price on each of at least 20 “VWAP Trading Days” (as defined in the 2020 Indenture), whether or not consecutive, during any 30 consecutive VWAP Trading Day period commencing on or after the date we first issued the 2020 Notes (“2020 Notes Mandatory Conversion Option”).
On July 1, 2021, we exercised our 2020 Notes Mandatory Conversion Option for the aggregate principal amount of $28.0 million of the 2020 Notes. On July 16, 2021, the aggregate principal of $28.0 million of 2020 Notes was converted into 8,615,384 shares of common stock. Upon conversion of the 2020 Notes, holders received a cash payment equal to the accrued and unpaid interest on the converted 2020 Notes. As a result, as of September 30, 2021, all 2020 Notes have converted into shares of common stock.
As of December 31, 2022 and 2021 there was $0.3 million aggregate principal amount outstanding of 2018 Notes.
Funding requirements
We expect to continue to incur significant expenses and may continue to incur operating losses. Refer to risk factor titled We have incurred significant losses since our inception. We may incur losses for the foreseeable future and may never achieve or maintain profitability within section Item 1A. Risk Factors for detailed activities which may drive our continued operating losses and expenses in future periods.
Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs and timing of commercialization activities for our product candidates for which we expect to receive marketing approval; |
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| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, progress, and results of our ongoing and potential future clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the extent to which we acquire or in-license other product candidates and technologies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs, timing, and outcome of regulatory review of our product candidates (including our efforts to seek approval and fund the preparation and filing of regulatory submissions); |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | revenue received from commercial sales our product candidates, should any of our other product candidates receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property rights, and defending intellectual property related claims; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to establish collaborations or partnerships on favorable terms, if at all; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | receipt of milestone payments and royalties pursuant to the Secura APA including timing of such receipt. |
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and through future milestones and royalties received pursuant to the Secura APA. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
CONTRACTUAL OBLIGATIONS AND COMMITMENTS
On April 15, 2014, we entered into a lease agreement for approximately 15,197 square feet of office and laboratory space in Needham, Massachusetts. The lease term commenced on April 15, 2014, and it was scheduled to expire on September 30, 2019. Effective February 15, 2018, we amended the lease agreement to relocate within the facility to another location consisting of 27,810 square feet of office space (the “Amended Lease Agreement”). The Amended Lease Agreement extends the expiration date of the lease from September 2019 through June 2025. Pursuant to the Amended Lease Agreement, the initial annual base rent amount is approximately $0.7 million, which increases during the lease term to $1.1 million for the last twelve-month period. As of December 31, 2022, the total future minimum lease payments under the agreement are $2.7 million through June 2025.
As discussed in Note 13. License, collaboration and commercial agreements to the consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K, we are party to several agreements to license intellectual property. The license agreements may require us to pay upfront license fees, ongoing annual license maintenance fees, milestone payments, minimum royalty payments, as well as reimbursement of certain patent costs incurred by the licensors, as applicable. As of December 31, 2022, we do not have any minimum contractual obligations in relation to these agreements because: there were no upfront license fees payable in future periods; no annual license maintenance fees; we cannot estimate if milestone and/or royalty payments will occur in future periods; and patent cost reimbursement costs are perpetual and the agreements are cancelable by us at any time upon prior written notice to the licensor.
TAX LOSS CARRYFORWARDS
As of December 31, 2022, we had federal and state net operating loss (“NOL”) carryforwards of $436.6 million and $203.3 million, respectively, which are available to reduce future taxable income. We also had federal and state tax credits of $5.6 million and $1.9 million, respectively, which may be used to offset future tax liabilities. The net operating loss and tax credit carryforwards will expire at various dates through 2042, except for $240.9 million of federal net operating loss carryforwards which may be carried forward indefinitely. Net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax
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authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. At December 31, 2022, we recorded a 100% valuation allowance against our NOL and tax credit carryforwards of $133.9 million, as we believe it is more likely than not that the tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our tax carryforwards will be realized, net income would increase in the period of determination.
Based on our analysis under Section 382 of the Internal Revenue Code and similar provisions under state law, we believe that our federal net operating loss carryforwards, our state net operating loss carryforwards, our Research and Development (“R&D”) credits and our Orphan Drug (“OD”) credits will be limited as of December 31, 2022. The portion of federal NOL, state NOL, R&D credits and OD credits that were determined to be limited by Section 382 have been written off as of December 31, 2022. The remaining unused carryforwards remain available for future periods. Due to our full valuation allowance the write off of NOLs, R&D credits, and OD credits did not have any impact to the statements of operation and comprehensive loss for the 2022 Period and 2021 Period.
RECENTLY ADOPTED ACCOUNTING STANDARDS
Refer to Note 2. Significant Accounting Policies to our consolidated financial statements located in this Annual Report on Form 10-K for recently adopted accounting standards.
FY 2021 10-K MD&A
SEC filing source: 0001558370-22-004498.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and related notes appearing elsewhere in this Annual Report on Form 10-K. The following discussion contains forward-looking statements that involve risks and uncertainties. Our actual results and the timing of certain events could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those discussed below and as set forth under “Risk Factors.” Please also refer to the section under the heading “Forward-Looking Statements.”
OVERVIEW
We are a late stage development biopharmaceutical company, with ongoing registration directed trials, committed to advancing new medicines for patients battling cancer. Our pipeline is focused on novel anticancer agents that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, particularly RAF/MEK inhibition and FAK inhibition.
Our most advanced product candidates, VS-6766 and defactinib, are being investigated in both preclinical and clinical studies for treatment of various solid tumors, including, low-grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC), colorectal cancer (CRC), pancreatic cancer, uveal melanoma, and endometrial cancer. We believe that VS-6766 may be beneficial as a therapeutic as a single agent or when used together in combination with defactinib, other agents, other pathway inhibitors, or other current and emerging standard of care treatments in cancers that do not adequately respond to currently available therapies.
On August 10, 2020, we and Secura Bio, Inc. (Secura) signed an Asset Purchase Agreement (Secura APA) and on September 30, 2020, the transaction closed. Pursuant to the Secura APA, we sold our exclusive worldwide license for the research, development, commercialization, and manufacture in oncology indications of products containing duvelisib. Refer to Note 14. License, collaboration and commercial agreements for a detailed description of the terms and conditions of the Secura APA. With the transition of the duvelisib program to Secura, we are focusing our efforts on our lead product candidates, VS-6766 and defactinib.
Our operations to date have been organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA. We have financed our operations to date primarily through public offerings of our common stock, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules Capital, Inc. (Hercules) in March 2017, as amended, the upfront payments under our license and collaboration agreements with Sanofi, Yakult and CSPC, the upfront payment under the Secura APA, the issuance of the 2018 Notes in October 2018, and the proceeds in connection with the PIPE. After the U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue.
As of December 31, 2021, we had an accumulated deficit of $663.7 million. Our net loss was $71.2 million, $67.7 million, and $149.2 million, for the years ended December 31, 2021, 2020, and 2019, respectively. We expect to incur significant expenses and may continue to incur operating losses for the foreseeable future as a result of the continued research and development of VS-6766 and defactinib. As of December 31, 2021, we had cash, cash equivalents, and investments of $100.3 million. We expect our existing cash resources will be sufficient to fund our planned operations through at least 12 months from the date of the issuance of these consolidated financial statements.
We expect to finance the future development costs of our clinical product portfolio with our existing cash, cash equivalents and investments, through future milestones and royalties received pursuant to the Secura APA or through strategic financing opportunities that could include, but are not limited to, collaboration agreements, future offerings of our equity, or the incurrence of debt. However, there is no guarantee that any of these strategic or financing opportunities will be executed or executed on favorable terms, and some could be dilutive to existing stockholders. If we fail to obtain additional future capital, we may be unable to complete our planned preclinical
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studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities.
COVID-19 pandemic
The current COVID-19 pandemic has presented a substantial public health and economic challenge around the world and is affecting our employees, patients, communities and business operations, as well as the U.S. economy and financial markets. We have been carefully monitoring the COVID-19 pandemic and its impact on our operations. All employees who are able to work from home have been primarily working from home since mid-March 2020. Shortages in personnel in clinics and hospitals have cause some United States sites to institute limits on new clinical trials which could impact our ability to open new sites for our clinical trials. In addition, clinics in Europe and United States continue to have delays in startup activities due to the ongoing pandemic and the increase in COVID-19 variant infections. The full extent to which the COVID-19 pandemic will directly or indirectly impact our business, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted, including new information that may emerge concerning COVID-19, new variants, the actions taken to contain it or treat its impact and the economic impact on local, regional, national and international markets.
For additional information on the various risks posed by the COVID-19 pandemic, please read Item 1A. Risk Factors included within.
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FINANCIAL OPERATIONS OVERVIEW
Revenue
From our U.S. commercial launch of COPIKTRA on September 24, 2018 through our ownership period ending in September 2020 product revenue, net represented the gross sales of COPIKTRA in the United States less provisions for product sales allowances and accruals. These provisions included trade allowances, rebates, chargebacks and discounts, product returns and other incentives. We sold COPIKTRA to a limited number of specialty pharmacies and specialty distributors. The provisions for product sales and allowances fluctuated based on the mix of sales to either specialty pharmacy or specialty distributor customers. See “Critical Accounting Policies and Significant Judgements and Estimates” below and Note 2. Significant Accounting Policies located within the notes to the financial statements included within for more information on the components of net U.S. product sales of COPIKTRA.
License and collaboration revenue to date has been generated through our license and collaboration agreements for the development and commercialization of duvelisib with Sanofi in the Sanofi Territory, CSPC in China, and Yakult in Japan. The terms of these agreements contain multiple deliverables which may include (i) licenses, (ii) research and development activities, and (iii) the manufacture of finished drug product, active pharmaceutical ingredient (API), or development materials for a partner, which are reimbursed at a contractually determined rate. Payments to us may include (i) up‑front license fees, (ii) payments for research and development activities, (iii) payments for the manufacture of finished drug product, API or development materials, (iv) payments based upon the achievement of certain milestones, and (v) royalties on product sales.
Sale of licenses and related assets revenue to date have been generated through our sale of our duvelisib license and related assets to Secura. The sale included intellectual property related to duvelisib in oncology indications, certain existing duvelisib inventory, certain manufacturing equipment and, claims and rights under certain contracts pertaining to duvelisib including net contract prepaid balances.
Costs of sales - product
Cost of sales - product consisted of costs of COPIKTRA on which product revenue was recognized, royalties owed to Healthcare Royalty Partners (HCR) and Infinity we incurred as a result of such sales of COPIKTRA, and certain period costs. Certain of the costs of COPIKTRA units recognized as revenue during 2020 and 2019 were expensed prior to the September 2018 FDA marketing approval and, therefore, are not included in cost of sales during those periods period. There were no cost of sales – product in 2021.
Costs of sales - sale of COPIKTRA license and related assets
Cost of sales - sale of COPIKTRA license and related assets represent assets delivered to Secura as part of the sale pursuant to the Secura APA. This includes our intangible assets, certain duvelisib inventory, net duvelisib contract prepaid balances, and manufacturing equipment.
Research and development expenses
Research and development expenses consist of costs associated with our research activities, including the development of our product candidates. Our research and development expenses consist of:
| Column 1 | Column 2 | Column 3 |
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| ● | employee-related expenses, including salaries, benefits, travel, and stock-based compensation expense; |
| Column 1 | Column 2 | Column 3 |
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| ● | external research and development expenses incurred under arrangements with third parties, such as contract research organizations (CROs), clinical sites, manufacturing organizations and consultants, including our scientific advisory board; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | license fees; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | facilities, depreciation, and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities, depreciation of equipment, and laboratory supplies; and |
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Costs for certain development activities, such as clinical trial expenses, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, and information provided us by our vendors on their actual costs incurred or level of effort expended. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid expenses and other current assets or accrued expenses.
Our research and development expenses may increase significantly in future periods as we undertake costlier development activities for our existing and future product candidates, including larger and later-stage clinical trials.
The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete development of our product candidates or the period, if any, in which material net cash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs, including the uncertainty of:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | clinical trial results; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, rate of progress, and expense of our research and development activities, including preclinical research and clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the potential benefits of our product candidates over other therapies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to market, commercialize, and achieve market acceptance for any of our product candidates that we receive regulatory approval for; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the terms and timing of regulatory approvals; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the expense of filing, prosecuting, defending, and enforcing patent claims and other intellectual property rights. |
A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us to conduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or if we experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time on the completion of clinical development.
Selling, general, and administrative expenses
Selling, general, and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, in our executive, finance, legal, information technology, commercial, communication, human resources, and business development functions. Other selling, general, and administrative expenses include allocated facility costs, commercial costs, professional fees for legal, patent, investor and public relations, consulting, insurance premiums, audit, tax, and other public company costs.
Other, interest income and interest expense
Other expenses for the year ended December 31, 2020 and December 31, 2019, consists entirely of the mark-to-market adjustment of the bifurcated make-whole interest provision derivative liability related to the 2019 Notes. There was no other expense for the year ended December 31, 2021.
Interest income reflects interest earned on our cash, cash equivalents and available-for-sale securities.
Interest expense reflects interest expense due under both our term loan facility executed with Hercules and the Notes, as well as non-cash interest related to the amortization of debt discount and issuance costs.
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CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles (GAAP). The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued and prepaid research and development expenses, leases, stock-based compensation, revenue recognition, and collaborative agreements, described in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding and evaluating our financial condition and results of operations.
Revenue recognition
We recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services in accordance with ASC Topic 606 Revenue from Contracts with Customers (ASC 606).
Product revenue, net
Product Revenue, Net – From September 24, 2018 (the date of our U.S. commercial launch of COPIKTRA) through September 30, 2020 (the date we sold COPIKTRA to Secura), we sold COPIKTRA to a limited number of specialty pharmacies and specialty distributors in the United States. We recognized revenue on sales of COPIKTRA when a customer obtains control of the product, which occurs at a point in time (typically upon delivery). Product revenues are recorded at the wholesale acquisition costs, net of applicable reserves for variable consideration. Components of variable consideration include trade discounts and allowances, Third-Party Payer chargebacks and discounts, government rebates, other incentives, such as voluntary co-pay assistance, product returns, and other allowances that are offered within contracts between us and customers, payors, and other indirect customers relating to our sale of COPIKTRA. These reserves, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable or a current liability. These estimates take into consideration a range of possible outcomes based upon relevant factors such as customer contract terms, information received from third parties regarding the anticipated payor mix for COPIKTRA, known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which it is entitled with respect to sales made.
The amount of variable consideration which is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under contracts will not occur in a future period. Our analyses contemplate the application of the constraint in accordance with ASC 606. For the years ended December 31, 2020, and 2019, we determined a material reversal of revenue would not occur in a future period for variable consideration, and the transaction price was not reduced further. There was not any product revenue, net recorded for the year ended December 31, 2021. Actual amounts of consideration ultimately received could differ from our estimates. However, as of December 31, 2021, we do not have any reserve balances associated with product revenue, net.
Licenses and sales of intellectual property
Licenses of Intellectual Property - We may enter into collaboration and licensing arrangements for research and development, manufacturing, and commercialization activities with collaboration partners for the development
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and commercialization of our product candidates, which have components within the scope of ASC 606. The arrangements generally contain multiple elements or deliverables, which may include (i) licenses, or options to obtain licenses, to our intellectual property or sale of our license, (ii) research and development activities performed for the collaboration partner, (iii) participation on joint steering committees, and (iv) the manufacturing of commercial, clinical or preclinical material. Payments pursuant to these arrangements typically include non-refundable, upfront payments, milestone payments upon the achievement of significant development events, research and development reimbursements, sales milestones, and royalties on product sales. The amount of variable consideration is constrained until it is probable that the revenue is not at a significant risk of reversal in a future period. The contracts into which we enter generally do not include significant financing components.
In determining the appropriate amount of revenue to be recognized as we fulfill our obligations under each of our collaboration and license agreements, we perform the following steps: (i) identification of the promised goods or services in the contract within the scope of ASC 606; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) we satisfy each performance obligation. As part of the accounting for these arrangements, we must use significant judgment to determine: a) the number of performance obligations based on the determination under step (ii) above; b) the transaction price under step (iii) above; c) the stand-alone selling price for each performance obligation identified in the contract for the allocation of transaction price in step (iv) above; and d) the measure of progress in step (v) above. We use judgment to determine whether milestones or other variable consideration, except for royalties on license arrangements, should be included in the transaction price.
For sales of license and intellectual property, that include sale-based royalties, including milestone payments based on a level of sales, we evaluate whether the royalties and sales-based milestones are considered probable of being achieved and estimate the amount of royalties to include over the contractual term using the expected value method and estimate the sales-based milestones using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated royalty and milestone value is included in the transaction price. Royalties and sales-based milestones for territories for which there is not regulatory approval are not considered probable until such regulatory approval is achieved. We evaluate factors such as whether consideration is outside of our control, timeline for when the uncertainty will be resolved and historical sales of COPIKTRA if applicable. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, we reevaluate the probability of achievement of all milestones subject to constraint and amount of royalty revenue to be received and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. At December 31, 2021, we determined $0.2 million of future potential royalties, we expect to receive pursuant to the Secura APA, were not constrained and have been recorded in the transaction price. As the consideration for future royalties is conditional, we recorded a corresponding contract asset for the expected future royalties. Portions of the contract asset are reclassified to accounts receivable when the right to consideration becomes unconditional.
Refer to Note 2. Significant Accounting Policies, Note 6. Product revenue reserves and allowances and Note. 14. License, collaboration and commercial agreements to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of revenue.
Collaborative Arrangements
Collaborative Arrangements: Contracts are considered to be collaborative arrangements pursuant to GAAP when they satisfy the following criteria defined in ASC Topic 808, Collaborative Arrangements (ASC 808): (i) the parties to the contract must actively participate in the joint operating activity and (ii) the joint operating activity must expose the parties to the possibility of significant risk and rewards, based on whether or not the activity is successful. Payments received from or made to a partner that are the result of a collaborative relationship with a partner, instead of a customer relationship, such as co-development activities, are recorded as a reduction or increase to research and development expense, respectively.
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Accrued and prepaid research and development expenses
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued expenses. This process involves reviewing contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly in arrears for services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to CROs in connection with research and development activities for which we have not yet been invoiced.
We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant to quotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided and result in a prepayment of the research and development expense. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in us reporting amounts that are too high or too low in any particular period. To date, there have been no material differences between our estimates of such expenses and the amounts actually incurred.
Refer to Note 2. Significant Accounting Policies, and Note. 4. Accrued expenses to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of accrued research and development expenses.
Stock-based compensation
For service-based awards, we recognize stock-based compensation expense for stock options, and restricted stock units (RSUs) issued to employees, directors and consultants based on the grant date fair value of the awards on a straight-line basis over the requisite service period. In addition, we issue shares under our employee stock purchase plan (ESPP) to employees. The fair value of our stock options and ESPP grants is estimated at the date of grant using the Black-Scholes option pricing model.
We have also granted performance-based RSUs and stock options with terms that allow the recipients to vest in a specific number of shares based upon the achievement of performance-based milestones as specified in the grants. Stock-based compensation expense associated with these performance-based RSUs and stock options is recognized if the performance condition is considered probable of achievement using management’s best estimates of the time to vesting for the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from our estimates, stock-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current and prior periods of a change in the estimated time to vesting for performance-based RSUs and stock options will be recognized as compensation cost in the period of the revision, and recorded as a change in estimate.
While the assumptions used to calculate and account for stock-based compensation awards represent management’s best estimates, these estimates involve inherent uncertainties and the application of management’s judgment. As a result, if revisions are made to our underlying assumptions and estimates, our stock-based compensation expense could vary significantly from period to period.
During the year ended December 31, 2021 we recorded $7.7 million of stock-based compensation expense. As of December 31, 2021, there was approximately $10.7 million of unrecognized stock-based compensation related to stock options, which are expected to be recognized over a weighted-average period of 3.2 years. As of December 31, 2021, there was approximately $6.0 million of unrecognized stock-based compensation related to RSUs, which
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are expected to be recognized over a weighted-average period of 3.7 years. See Note 2. Significant accounting policies and Note 9. Stock-based compensation to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of stock-based compensation.
Leases
Effective January 1, 2019, we adopted ASC Topic 842, Leases (ASC 842). This standard requires lessees to recognize in the statement of financial position a liability to make lease payments and a right-of-use asset representing its right to use the underlying asset for the lease term for both finance and operating leases.
At the inception of an arrangement, we determine whether the arrangement is or contains a lease based on the unique facts and circumstances within the arrangement. A lease is identified where an arrangement conveys the right to control the use of identified property, plant, and equipment for a period of time in exchange for consideration. Leases which are identified within the scope of ASC 842 and which have a term greater than one year are recognized on our consolidated balance sheets as right-of-use assets, lease liabilities and, if applicable, long-term lease liabilities. We have elected not to recognize leases with terms of one year or less on our consolidated balance sheets. Operating lease liabilities and their corresponding right-of-use assets are recorded based on the present value of lease payments over the expected remaining lease term. However, certain adjustments to the right-of-use asset may be required for items such as initial direct costs paid or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, we utilize our incremental borrowing rates to calculate the present value of lease payments. Incremental borrowing rates are the rates we incur to borrow on a collateralized basis over a similar term an amount equal to the lease payments in a similar economic environment. As of December 31, 2021, we have a right-of-use asset of $2.3 million and lease liability of $2.9 million which reflected on the consolidated balance sheets. See Note 2. Significant accounting policies and Note 7. Leases to our consolidated financial statements located in this Annual Report on Form 10-K for further discussion of leases.
RESULTS OF OPERATIONS
All financial information presented has been consolidated and includes the accounts of our wholly-owned subsidiaries, Verastem Securities Company and Verastem Europe GmbH. All intercompany balances and transactions have been eliminated in consolidation.
| | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|
| | | Year Ended December 31, | |||||||
| | | | | | | | | | |
| | 2021 | 2020 | 2019 | ||||||
| Revenue: | | | | | | | | | |
| Product revenue, net | | $ | — | | $ | 15,232 | | $ | 12,339 |
| License and collaboration revenue | | | — | | | 2,912 | | | 5,117 |
| Sale of COPIKTRA license and related assets | | | 1,447 | | | 70,000 | | | — |
| Transition services revenue | | | 606 | | | 372 | | | — |
| Total revenue | | 2,053 | | 88,516 | | 17,456 | |||
| Operating expenses: | | | | | | | | | |
| Cost of sales - product | | | — | | | 1,765 | | | 1,238 |
| Cost of sales - intangible amortization | | | — | | | 793 | | | 1,569 |
| Cost of sales - Sale of COPIKTRA license and related assets | | | — | | | 31,187 | | | — |
| Research and development | | | 39,347 | | | 41,376 | | | 45,778 |
| Selling, general and administrative | | 24,115 | | 62,755 | | 101,212 | |||
| Total operating expenses | | 63,462 | | 137,876 | | 149,797 | |||
| Loss from operations | | (61,409) | | (49,360) | | (132,341) | |||
| Other expense | | | — | | | (1,313) | | | (641) |
| Interest income | | 181 | | 515 | | 4,381 | |||
| Interest expense | | (9,972) | | (15,794) | | (20,608) | |||
| Loss on debt extinguishment | | | — | | | (1,580) | | | — |
| Net loss before income taxes | | | (71,200) | | | (67,532) | | | (149,209) |
| Income tax expense | | | — | | | (194) | | | — |
| Net loss | | $ | (71,200) | | $ | (67,726) | | $ | (149,209) |
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Comparison of the Year Ended December 31, 2021 to the Year Ended December 31, 2020
Product revenue, net. Product revenue net for the year ended December 31, 2021 (2021 Period) was $0.0 million compared to $15.2 million for the year ended December 31, 2020 (2020 Period). Product revenue, net consisted of net product sales of COPIKTRA in the United States. Pursuant to the Secura APA discussed in greater detail above within section Item 1. Business within heading Licenses and commercial agreements, we have sold our COPIKTRA license and as of September 30, 2020, we no longer sell COPIKTRA in the United States.
License and collaboration revenue. License and collaboration revenue for the 2021 Period was $0.0 million compared to $2.9 million for the 2020 Period. The 2020 Period license and collaboration revenue was comprised of Sanofi achieving two development milestones during the 2020 Period totaling $2.5 million and $0.4 million of duvelisib shipments to Sanofi, Yakult Honsha Co., Ltd. (Yakult), and CSPC Pharmaceutical Group Limited (CSPC).
Sale of COPIKTRA license and related assets revenue. Sale of COPIKTRA license and related assets revenue for the 2021 Period was $1.4 million compared to $70.0 million for the 2020 Period. The sale of COPIKTRA license and related assets revenue for the 2021 Period primarily related to two regulatory milestone for $1.3 million achieved by Secura’s sublicensee and $0.2 million related to royalties we received and expected to be received pursuant to the Secura APA. Sale of COPIKTRA license and related assets revenue for the 2020 Period was comprised of a $70.0 million upfront payment recognized under the Secura APA discussed in greater detail above within section Item 1. Business within heading Licenses and Commercial Agreements.
Transition services revenue. Transition services revenue for the 2021 Period was $0.6 million compared to $0.4 million for the 2020 Period. Transition services revenue in both periods consisted of us providing certain support functions pursuant to the Secura transition service agreement, which was entered into in connection with the Secura APA. The services were provided at a mutually agreed upon rate.
Costs of sales – product. Costs of sales – product for the 2021 Period was $0.0 million compared to $1.8 million for the 2020 Period. Pursuant to the Secura APA discussed in greater detail above within section Item 1. Business within heading Licenses and commercial agreements, we have sold our COPIKTRA license and as of September 30, 2020, we no longer sell COPIKTRA in the United States.
Cost of Sales – intangible amortization. Cost of sales – intangible amortization for the 2021 Period was $0.0 million compared to $0.8 million for the 2020 Period. Pursuant to the Secura APA discussed in greater detail above within section Item 1. Business within heading Licenses and commercial agreements, we have sold our COPIKTRA license, to which our intangible asset related. Therefore, there was no cost of sales – intangible amortization in the 2021 Period.
Cost of sales – sale of COPIKTRA license and related assets. Cost of sales – sale of COPIKTRA license and related assets for the 2021 Period was $0.0 million compared to $31.2 million for the 2020 Period. Cost of sales – sale of COPIKTRA license and related assets for the 2020 Period consisted of certain assets delivered to Secura under the Secura APA. For the 2020 Period, we recognized approximately $19.2 million, $6.0 million, $5.8 million and $0.2 million for the intangible asset, certain duvelisib inventory, net duvelisib contract prepaid balances and manufacturing equipment, respectively which were delivered to Secura as part of the sale.
Research and development expense. Research and development expense for the 2021 Period was $39.3 million compared to $41.4 million for the 2020 Period. The $2.1 million decrease from the 2020 Period to the 2021 Period was primarily related to a decrease of $3.0 million of license fees due to a non-refundable payment of $3.0 million to Chugai in the 2020 Period for the VS-6766 license described further above within section Item 1. Business within heading Licenses and commercial agreements, a decrease of $1.8 million in contract research organization (CRO) costs, a decrease of $1.7 million in consulting costs, and a decrease of $0.4 million in clinical supply costs. The decrease is partially offset by an increase of $2.2 million in investigator fees, an increase of $1.3 million in personnel related costs, including non-cash stock-based compensation, an increase of $0.7 million of drug substance and drug product costs, and an increase of $0.6 million of pre-clinical expenses. In future periods, we continue to expect expenses for VS-6766 and defactinib to increase as we have commenced our registration directed trials.
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Selling, general and administrative expense. Selling, general and administrative expense for the 2021 Period was $24.1 million compared to $62.8 million for the 2020 Period. The decrease of $38.7 million from the 2020 Period to the 2021 Period primarily resulted from a decrease of $20.1 million of personnel related costs, including non-cash stock-based compensation, as a result of reduced headcount, a decrease of $16.1 million in consulting and professional fees, primarily related to the support of commercial activities in 2020 Period and costs associated with the sale of COPIKTRA license and related assets to Secura, a decrease of $2.1 million in reduced commercial activities costs and a decrease of $0.4 million in reduced travel and other costs.
Other expense. Other expense for the 2020 Period of $1.3 million was for the mark-to-market adjustment related to the bifurcated make-whole interest provision derivative liability related to our 5.00% Convertible Senior Second Lien Notes due 2048 (the 2019 Notes). All 2019 Notes have converted to common stock as of March 31, 2020, and the derivative liability will no longer be remeasured. There was no other expense in the 2021 Period.
Interest income. Interest income for the 2021 Period was $0.2 million compared to $0.5 million for the 2020 Period. The decrease of $0.3 million from the 2020 Period to the 2021 Period was primarily due to lower investment cost basis and lower interest rates on investments.
Interest expense. Interest expense for the 2021 Period was $10.0 million compared to $15.8 million for the 2020 Period. The decrease of $5.8 million was primarily due to $8.1 million of non-cash interest expense recorded in the 2020 Period upon conversion of the 2019 Notes into common stock and decreased interest as a result of repayment of our term loan facility in November 2020. The decrease is partially offset by $7.8 million of non-cash interest expense recorded upon conversion of the 2020 Notes into common stock in the 2021 Period.
Loss on debt extinguishment. Loss on debt extinguishment for the 2020 Period of $1.6 million represents the loss recognized on early extinguishment of our Hercules term loan facility. On November 9, 2020, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Amended Loan Agreement with Hercules in an aggregate amount of $37.4 million (the Payoff Amount). The Payoff Amount, excluding accrued interest, exceeded the carrying amount of the Hercules debt on November 9, 2020 by $1.6 million which was recorded as a loss on debt extinguishment. There was no loss on debt extinguishment in the 2021 Period.
Income tax expense. Income tax expense for the 2020 Period of $0.2 million primarily related to state income tax as a result of the sale of COPIKTRA license and related assets. There was no income tax expense in the 2021 Period.
Restructuring. On February 27, 2020, we committed to an operational plan to reduce overall operating expenses, including the elimination of approximately 31 positions and other cost-saving measures.
In August 2020, in connection with the duvelisib sale to Secura pursuant to the Secura APA we committed to a strategic restructuring (August 2020 Restructuring). The August 2020 restructuring included a workforce reduction of approximately 41 positions primarily in our commercial operations department.
During the 2020 Period, we recorded an aggregate expense of $4.6 million for restructuring expenses, which is reflected in the consolidated statements of operation and comprehensive loss as selling general, and administrative expense and research and development expense of $4.1 million and $0.5 million, respectively, for one-time termination benefits for employee severance, benefits, and related costs. There were no restructuring expenses in the 2021 Period.
LIQUIDITY AND CAPITAL RESOURCES
Sources of liquidity
We have financed our operations to date primarily through public and private offerings of our common stock, sales of common stock under our at-the-market equity offering programs, our loan and security agreement executed with Hercules in March 2017, as amended, the upfront payments under our license and collaboration agreements with Yakult, CSPC, and Sanofi, the upfront payment under the Secura APA, the issuance of 2018 Notes
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in October 2018, and the proceeds in connection with the PIPE. With the commercial launch of COPIKTRA in the United States in September 2018, we had recently begun financing a portion of our operations through product revenue. As of September 30, 2020, in connection with the Secura APA, we no longer sell COPIKTRA in the United States. We expect to finance a portion of our business through future milestones and royalties received pursuant to the Secura APA.
As of December 31, 2021, we had $100.3 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
Risks and uncertainties include those identified under Item 1A. Risk Factors, in this Annual Report on Form 10-K.
Cash flows
The following table sets forth the primary sources and uses of cash for each of the periods set forth below (in thousands):
| | | | | | | | | | | |
|---|---|---|---|---|---|---|---|---|---|---|
| | | Year ended December 31, | ||||||||
| | | 2021 | | 2020 | | 2019 | ||||
| Net cash (used in) provided by: | | | | | ||||||
| Operating activities | | $ | (53,502) | | $ | (33,506) | | $ | (138,518) | |
| Investing activities | | 87 | | (47,363) | | 89,613 | | |||
| Financing activities | | 6,885 | | 69,630 | | (2,441) | | |||
| Decrease in cash, cash equivalents and restricted cash | | $ | (46,530) | | $ | (11,239) | | $ | (51,346) | |
Operating activities. The use of cash in 2021 Period and 2020 Period resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges was $54.1 million and $45.1 million for the 2021 Period and 2020 Period, respectively. Non-cash charges were primarily related to non-cash interest, net and stock-based compensation expense in both the 2021 Period and 2020 Period. Our cash inflow from operating activities due to changes in operating assets and liabilities was $0.6 million and $11.6 million for the 2021 Period and 2020 Period, respectively. Cash inflow (outflow) due to changes in operating assets and liabilities for the 2021 Period was primarily driven by an increase of $1.8 million in accrued expenses, an increase of $0.6 million in accounts payable, partially offset by an increase in $1.6 million in prepaid expenses, other current assets, and other assets, and an increase of $0.3 million in accounts receivable, net. Cash inflow due to changes in operating assets and liabilities for the 2020 Period was primarily driven by a decrease of $19.5 million in intangible assets and property, plant and equipment, a decrease of $3.1 million in inventory, and a decrease of $2.3 million in accounts receivable, net partially offset by a decrease of $8.0 million in accounts payable, and $5.0 million decrease in accrued expenses and other liabilities. The $20.0 million increase in cash used in operating activities for the 2021 Period compared to the 2020 Period was primarily due to increased net loss, and a net increase in the changes in the components of working capital which was primarily driven by the duvelisib sale to Secura in the 2020 Period. In the 2020 Period, we received $70.0 million of cash and recognized $70.0 million of sale of COPIKTRA license and related assets revenue and we expensed a total of $31.2 million for certain assets delivered to Secura upon finalization of duvelisib sale to Secura.
Investing activities. The cash provided by investing activities for the 2021 Period primarily relates to the net maturities of investments of $0.3 million, partially offset by purchases of fixed assets of $0.2 million. The cash provided by investing activities for the 2020 Period primarily reflects the net maturities of investments of $47.3 million.
Financing activities. The cash provided by financing activities for the 2021 Period primarily represents $6.7 million in net proceeds received under our at-the-market equity offering program, and $1.1 million of proceeds received related to exercise of stock options and employee stock purchase plan. This is partially offset by $0.9 million of payments for settlement of restricted stock for tax withholdings. The cash provided by financing activities for the 2020 Period primarily represents $93.8 million in net proceeds from sales of our common stock under the Purchase Agreement described below, $12.2 million in net proceeds received under our at-the-market equity
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offering program described below, and $3.0 million of proceeds received related to exercise of stock options and employee stock purchase plan. This is partially offset by $37.4 million for repayment of our Hercules Capital, Inc. (Hercules) term loan facility, $1.8 million of interest-make whole payments on the 2019 Notes and $0.3 million of payments for settlement of restricted stock for tax withholdings.
On February 27, 2020, we entered into a Securities Purchase Agreement with certain institutional investors in which we agreed to sell 46,511,628 shares of common stock at a purchase price of $2.15 per share, which represents 12.6% premium to the last reported sale price of our common stock of $1.91 per share on February 27, 2020. On March 3, 2020, the closing occurred. The aggregate proceeds net of underwriting discounts and offering costs were approximately $93.8 million.
In March 2017, we established an at-the-market equity offering program (2017 ATM) pursuant to which we were able to offer and sell up to $35.0 million of our common stock at then current market prices from time to time through Cantor Fitzgerald & Co. (Cantor) as sales agent. In August 2017, we amended our sales agreement with Cantor to increase the maximum aggregate offering price of shares of common stock that can be sold under the at-the-market equity offering program to $75.0 million.
During the 2021 Period, we sold zero shares under the 2017 ATM. During the 2020 Period, we sold 6,769,559 shares under the 2017 ATM for net proceeds of approximately $12.2 million (after deducting commissions and other offering expenses). Through September 30, 2021, we have sold a total of 18,287,913 shares under this program for net proceeds of approximately $59.6 million (after deducting commissions and other offering expenses).
In August 2021, we entered into a sales agreement with Cantor pursuant to which we can offer and sell up to $100.0 million of our common stock at the current market prices from time to time through Cantor as sales agent (August 2021 ATM). During the 2021 Period, we sold 2,930,585 shares under the August 2021 ATM for net proceeds of approximately $6.8 million (after deducting commissions and other offering expenses). As of December 31, 2021, we can issue an aggregate amount of $93.0 million of common stock under this program.
On March 21, 2017, we entered into a term loan facility of up to $25.0 million with Hercules. The term loan facility is governed by a loan and security agreement, dated March 21, 2017 (the Original Loan Agreement). The Original Loan Agreement was amended on January 4, 2018, March 6, 2018, October 11, 2018, April 23, 2019, and November 14, 2019 (the Amended Loan Agreement) to increase the total borrowing limit under the Original Loan Agreement from up to $25.0 million to up to $75.0 million, pursuant to certain conditions of funding.
On November 9, 2020, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Amended Loan Agreement with Hercules in an aggregate amount of $37.4 million (the Payoff Amount). The Payoff Amount includes the principal balance of $35.0 million, final payment fee of $1.8 million, prepayment penalty fee of $0.5 million, and accrued and unpaid interest of $0.1 million. On November 9, 2020 the Amended Loan Agreement was terminated along with Hercules’ commitment to provide funding under any future term loans. All liens on substantially all of our assets to secure the loans under the Amended Loan Agreement have been terminated and released.
On October 17, 2018, we closed a registered direct public offering of $150.0 million aggregate principal amount of our 2018 issued 5.00% Convertible Senior Notes due 2048 (the 2018 Notes), for net proceeds of approximately $145.3 million. The 2018 Notes are governed by the terms of a base indenture for senior debt securities (the Base Indenture), as supplemented by the first supplemental indenture thereto (the 2018 Notes Supplemental Indenture and together with the Base Indenture, the 2018 Indenture), each dated October 17, 2018, by and between us and Wilmington Trust, National Association, as trustee. The 2018 Notes are senior unsecured obligations of us and bear interest at a rate of 5.00% per annum, payable semi-annually in arrears on May 1 and November 1 of each year, beginning on May 1, 2019. The 2018 Notes will mature on November 1, 2048, unless earlier repurchased, redeemed or converted in accordance with their terms.
The 2018 Notes are convertible into shares of our common stock, par value $0.0001 per share, together, if applicable, with cash in lieu of any fractional share, at an initial conversion rate of 139.5771 shares of common
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stock per $1,000 principal amount of the 2018 Notes, which corresponds to an initial conversion price of approximately $7.16 per share of common stock and represents a conversion premium of approximately 15.0% above the last reported sale price of our common stock of $6.23 per share on October 11, 2018. Upon conversion, converting noteholders will be entitled to receive accrued interest on their converted Notes.
We will have the right, exercisable at our option, to cause all 2018 Notes then outstanding to be converted automatically if the “Daily VWAP” (as defined in the 2018 Indenture) per share of our common stock equals or exceeds 130% of the conversion price, which equates to approximately $9.31 per share, on each of at least 20 “VWAP Trading Days” (as defined in the 2018 Indenture), whether or not consecutive, during any 30 consecutive VWAP Trading Day period commencing on or after the date we first issued the 2018 Notes.
On November 6, 2020, we entered into a privately negotiated agreement with an investor who is a holder of our 2018 Notes to exchange approximately $28.0 million aggregate principal amount of 2018 Notes for approximately $28.0 million aggregate principal amount of newly issued 5.00% Convertible Senior Notes due 2048 (the 2020 Notes). The issuance of the 2020 Notes closed on November 13, 2020. The 2020 Notes are governed pursuant to the Base Indenture between us and Wilmington dated as of October 17, 2018 as supplemented by the second supplemental indenture thereto dated as of November 13, 2020 (the 2020 Notes Supplemental Indenture and together with the Base Indenture, the 2020 Indenture).
We had the right, exercisable at our option, to cause all 2020 Notes then outstanding to be converted automatically if the “Daily VWAP” (as defined in the 2020 Indenture) per share of our common stock equals or exceeds 123.08% of the conversion price on each of at least 20 “VWAP Trading Days” (as defined in the 2020 Indenture), whether or not consecutive, during any 30 consecutive VWAP Trading Day period commencing on or after the date we first issued the 2020 Notes (2020 Notes Mandatory Conversion Option).
The initial conversion rate for the 2020 Notes was 307.6923 shares of our common stock per $1,000 principal amount of the 2020 Notes, which is equivalent to an initial conversion price of approximately $3.25 per share, representing an approximately 153.9% premium to the sale price of $1.28 per share of our common stock on November 5, 2020, as reported on the Nasdaq Global Market. The conversion rate was subject to adjustment from time to time upon the occurrence of certain events, including, but not limited to, the issuance of stock dividends and payment of cash dividends, but was not subject to adjustment for any accrued and unpaid interest.
Prior to November 1, 2023, we did not have the option to redeem the 2020 Notes. On or after November 1, 2023, we had the option to redeem the 2020 Notes, in whole or in part, at a cash redemption price equal to the principal amount of the 2020 Notes to be redeemed, plus accrued and unpaid interest, if any.
Unless we had previously called all outstanding 2020 Notes for redemption, the 2020 Notes were subject to repurchase by us at the holders’ option on each of November 1, 2023, November 1, 2028, November 1, 2033, November 1, 2038 and November 1, 2043 (or, if any such date is not a business day, on the next business day) at a cash repurchase price equal to the principal amount of the 2020 Notes to be repurchased, plus accrued and unpaid interest, if any.
The 2020 Notes were our senior unsecured obligations and were senior in right of payment to our future indebtedness that is expressly subordinated in right of payment to the 2020 Notes, and equal in right of payment with our existing and future indebtedness that is not so subordinated, and effectively subordinated to our existing and future indebtedness, to the extent of the value of the collateral securing such indebtedness. The 2020 Notes were structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables, and (to the extent we are not a holder thereof) preferred equity, if any, of our subsidiaries.
On July 1, 2021, we exercised our 2020 Notes Mandatory Conversion Option for the aggregate principal amount of $28.0 million of the 2020 Notes. On July 16, 2021, the aggregate principal of $28.0 million of 2020 Notes was converted into 8,615,384 shares of common stock. Upon conversion of the 2020 Notes, holders received a cash payment equal to the accrued and unpaid interest on the converted 2020 Notes. As a result, as of September 30, 2021, all 2020 Notes have converted into shares of common stock.
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As of December 31, 2021, there was $0.3 million aggregate principal amount outstanding of 2018 Notes compared to $0.3 million and $28.0 million aggregate principal amount outstanding of the 2018 Notes and 2020 Notes, respectively, for a total of $28.3 million aggregate principal amount outstanding as of December 31, 2020.
Funding requirements
We expect to continue to incur significant expenses and may continue to incur operating losses. Refer to risk factor titled We have incurred significant losses since our inception. We may incur losses for the foreseeable future and may never achieve or maintain profitability within section Item 1A. Risk Factors for detailed activities which may drive our continued operating losses and expenses in future periods.
Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs and timing of commercialization activities for our product candidates for which we expect to receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the scope, progress, and results of our ongoing and potential future clinical trials; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the extent to which we acquire or in-license other product candidates and technologies; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs, timing, and outcome of regulatory review of our product candidates (including our efforts to seek approval and fund the preparation and filing of regulatory submissions); |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | revenue received from commercial sales our product candidates, should any of our other product candidates receive marketing approval; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property rights, and defending intellectual property related claims; |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | our ability to establish collaborations or partnerships on favorable terms, if at all; and |
| Column 1 | Column 2 | Column 3 |
|---|---|---|
| ● | Receipt of milestone payments and royalties pursuant to the Secura APA including timing of such receipt. |
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements, and through future milestones and royalties received through the Secura APA. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
CONTRACTUAL OBLIGATIONS AND COMMITMENTS
On April 15, 2014, we entered into a lease agreement for approximately 15,197 square feet of office and laboratory space in Needham, Massachusetts. The lease term commenced on April 15, 2014, and it was scheduled to expire on September 30, 2019. Effective February 15, 2018, we amended the lease agreement to relocate within the facility to another location consisting of 27,810 square feet of office space (the Amended Lease Agreement). The Amended Lease Agreement extends the expiration date of the lease from September 2019 through June 2025. Pursuant to the Amended Lease Agreement, the initial annual base rent amount is approximately $660,000, which
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increases during the lease term to $1.1 million for the last twelve-month period. As of December 31, 2021, the total future minimum lease payments under the agreement are $3.7 million through June 2025.
As discussed in Note 14. License, collaboration and commercial agreements to the consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K, we are party to several agreements to license intellectual property. The license agreements may require us to pay upfront license fees, ongoing annual license maintenance fees, milestone payments, minimum royalty payments, as well as reimbursement of certain patent costs incurred by the licensors, as applicable. As of December 31, 2021, we do not have any minimum contractual obligations in relation to these agreements because: there were no upfront license fees payable in future periods; no annual license maintenance fees; we cannot estimate if milestone and/or royalty payments will occur in future periods; and patent cost reimbursement costs are perpetual and the agreements are cancelable by us at any time upon prior written notice to the licensor.
TAX LOSS CARRYFORWARDS
As of December 31, 2021, we had federal and state net operating loss (NOL) carryforwards of $410.6 million and $219.0 million, respectively, which are available to reduce future taxable income. We also had federal and state tax credits of $3.5 million and $1.3 million, respectively, which may be used to offset future tax liabilities. The net operating loss and tax credit carryforwards will expire at various dates through 2041, except for $214.9 million of federal net operating loss carryforwards which may be carried forward indefinitely. Net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. At December 31, 2021, we recorded a 100% valuation allowance against our NOL and tax credit carryforwards of $103.3 million, as we believe it is more likely than not that the tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our tax carryforwards will be realized, net income would increase in the period of determination.
Based on our analysis under Section 382 of the Internal Revenue Code and similar provisions under state law, we believe that our federal net operating loss carryforwards, our state net operating loss carryforwards, our Research and Development (R&D) credits and our Orphan Drug (OD) credits will be limited as of December 31, 2021. The portion of federal NOL, state NOL, R&D credits and OD credits that were determined to be limited by Section 382 have been written off as of December 31, 2021. The remaining unused carryforwards remain available for future periods. Due to our full valuation allowance the write off of NOLs, R&D credits, and OD credits did not have any impact to the statements of operation and comprehensive loss for the 2021 Period and 2020 Period.
RECENTLY ADOPTED ACCOUNTING STANDARDS
Refer to Note 2. Significant Accounting Policies to our consolidated financial statements located in this Annual Report on Form 10-K for recently adopted accounting standards.