# CATALYST PHARMACEUTICALS, INC. (CPRX)

Informational only - not investment advice.

CIK: 0001369568
SIC: 2834 Pharmaceutical Preparations
SIC breadcrumb: [Manufacturing](/division/D/) > [Chemicals And Allied Products](/major-group/28/) > [SIC 2834 Pharmaceutical Preparations](/industry/2834/)
Latest 10-K filed: 2026-02-25
SEC page: https://www.sec.gov/edgar/browse/?CIK=1369568
Filing source: https://www.sec.gov/Archives/edgar/data/1369568/000119312526071525/cprx-20251231.htm

## Selected Fundamentals
| Metric | Value | Unit | FY | Filed |
| --- | ---: | --- | ---: | --- |
| Revenue | 588989000 | USD | 2025 | 2026-02-25 |
| Net income | 214326000 | USD | 2025 | 2026-04-30 |
| Assets | 1103979000 | USD | 2025 | 2026-02-25 |

## Financials

Annual standardized facts from SEC companyfacts as of latest extracted filing date 2026-04-30. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001369568.json. Derived margins are computed from the extracted annual SEC facts.

| Metric | 2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
| --- | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: | ---: |
| Revenue |  |  |  | 102,306,000 | 119,073,000 | 140,833,000 | 214,203,000 | 398,204,000 | 491,734,000 | 588,989,000 |
| Net income | -18,072,452 | -18,412,377 | -34,003,514 | 31,875,000 | 74,983,000 | 39,482,000 | 83,079,000 | 71,410,000 | 163,889,000 | 214,326,000 |
| Operating income | -19,280,201 | -18,679,636 | -35,295,165 | 31,823,000 | 41,303,000 | 52,385,000 | 101,838,000 | 86,812,000 | 195,124,000 | 257,778,000 |
| Diluted EPS |  |  | -0.33 | 0.30 | 0.71 | 0.37 | 0.75 | 0.63 | 1.31 | 1.68 |
| Assets | 41,706,853 | 85,387,430 | 60,449,962 | 112,376,230 | 192,354,000 | 237,788,000 | 375,630,000 | 470,114,000 | 851,411,000 | 1,103,979,000 |
| Liabilities | 2,397,923 | 4,423,618 | 9,666,153 | 24,746,274 | 22,756,000 | 30,957,000 | 75,209,000 | 82,233,000 | 123,779,000 | 149,711,000 |
| Stockholders' equity | 39,308,930 | 80,963,812 | 50,785,000 | 87,630,000 | 169,598,000 | 206,831,000 | 300,421,000 | 387,881,000 | 727,632,000 | 954,268,000 |
| Cash and cash equivalents | 13,893,064 | 57,496,702 | 16,559,000 | 89,512,000 | 130,237,000 | 171,445,000 | 298,395,000 | 137,636,000 | 517,553,000 | 709,171,000 |
| Net margin |  |  |  | 31.16% | 62.97% | 28.03% | 38.79% | 17.93% | 33.33% | 36.39% |
| Operating margin |  |  |  | 31.11% | 34.69% | 37.20% | 47.54% | 21.80% | 39.68% | 43.77% |

## Quarterly

Quarterly standardized facts from SEC companyfacts as of latest extracted filing date 2026-05-11. Source: https://data.sec.gov/api/xbrl/companyfacts/CIK0001369568.json.

Flow metrics use discrete quarter-length periods from 10-Q/10-Q/A filings. Q4 revenue and net income are derived only when annual FY and nine-month YTD facts exist for the same fiscal year; derived Q4 values are labeled. EPS Q4 is not derived.

| Quarter | End date | Revenue | Net income | Diluted EPS | Method |
| --- | --- | ---: | ---: | ---: | --- |
| 2022-Q2 | 2022-06-30 |  |  | 0.20 | reported discrete quarter |
| 2022-Q3 | 2022-09-30 |  |  | 0.20 | reported discrete quarter |
| 2023-Q2 | 2023-03-31 |  | 29,568,000 |  | reported discrete quarter |
| 2023-Q1 | 2023-03-31 |  |  | 0.26 | reported discrete quarter |
| 2023-Q2 | 2023-06-30 | 99,582,000 |  | 0.33 | reported discrete quarter |
| 2023-Q3 | 2023-06-30 |  | 37,762,000 |  | reported discrete quarter |
| 2023-Q3 | 2023-09-30 | 102,688,000 |  | -0.29 | reported discrete quarter |
| 2023-Q4 | 2023-12-31 | 110,568,000 | 34,844,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2024-Q1 | 2024-03-31 | 98,509,000 | 23,275,000 | 0.19 | reported discrete quarter |
| 2024-Q2 | 2024-03-31 |  | 23,275,000 |  | reported discrete quarter |
| 2024-Q3 | 2024-06-30 |  | 40,794,000 |  | reported discrete quarter |
| 2024-Q2 | 2024-06-30 | 122,710,000 |  | 0.33 | reported discrete quarter |
| 2024-Q3 | 2024-09-30 | 128,695,000 |  | 0.35 | reported discrete quarter |
| 2024-Q4 | 2024-12-31 | 141,820,000 | 55,936,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2025-Q1 | 2025-03-31 | 141,421,000 | 56,737,000 | 0.45 | reported discrete quarter |
| 2025-Q2 | 2025-03-31 |  | 56,737,000 |  | reported discrete quarter |
| 2025-Q3 | 2025-06-30 |  | 52,108,000 |  | reported discrete quarter |
| 2025-Q2 | 2025-06-30 | 146,563,000 |  | 0.41 | reported discrete quarter |
| 2025-Q3 | 2025-09-30 | 148,392,000 |  | 0.42 | reported discrete quarter |
| 2025-Q4 | 2025-12-31 | 152,613,000 | 52,698,000 |  | derived Q4 = FY annual - nine-month YTD |
| 2026-Q1 | 2026-03-31 | 149,390,000 | 63,734,000 | 0.50 | reported discrete quarter |

## Macro Cross-References
- [CPIAUCSL](/indicator/CPIAUCSL/): Consumer Price Index for All Urban Consumers: All Items in U.S. City Average
- [UNRATE](/indicator/UNRATE/): Unemployment Rate
- [FEDFUNDS](/indicator/FEDFUNDS/): Federal Funds Effective Rate
- [CES0500000003](/indicator/CES0500000003/): Average Hourly Earnings of All Employees, Total Private
- [DFEDTARU](/indicator/DFEDTARU/): Federal Funds Target Range - Upper Limit
- [DFEDTARL](/indicator/DFEDTARL/): Federal Funds Target Range - Lower Limit
- [DGS3MO](/indicator/DGS3MO/): Market Yield on U.S. Treasury Securities at 3-Month Constant Maturity
- [DGS2](/indicator/DGS2/): Market Yield on U.S. Treasury Securities at 2-Year Constant Maturity
- [DGS10](/indicator/DGS10/): Market Yield on U.S. Treasury Securities at 10-Year Constant Maturity
- [DGS30](/indicator/DGS30/): Market Yield on U.S. Treasury Securities at 30-Year Constant Maturity
- [T10Y2Y](/indicator/T10Y2Y/): 10-Year Treasury Constant Maturity Minus 2-Year Treasury Constant Maturity
- [CPILFESL](/indicator/CPILFESL/): Consumer Price Index for All Urban Consumers: All Items Less Food and Energy
- [CPIUFDSL](/indicator/CPIUFDSL/): Consumer Price Index for All Urban Consumers: Food
- [CPIENGSL](/indicator/CPIENGSL/): Consumer Price Index for All Urban Consumers: Energy
- [CUSR0000SAH1](/indicator/CUSR0000SAH1/): Consumer Price Index for All Urban Consumers: Shelter
- [PCEPI](/indicator/PCEPI/): Personal Consumption Expenditures: Chain-type Price Index
- [PCEPILFE](/indicator/PCEPILFE/): Personal Consumption Expenditures Excluding Food and Energy: Chain-type Price Index
- [PPIACO](/indicator/PPIACO/): Producer Price Index by Commodity: All Commodities
- [T10YIE](/indicator/T10YIE/): 10-Year Breakeven Inflation Rate
- [U6RATE](/indicator/U6RATE/): Total Unemployed, Plus All Marginally Attached Workers Plus Total Employed Part Time for Economic Reasons
- [PAYEMS](/indicator/PAYEMS/): All Employees, Total Nonfarm
- [CIVPART](/indicator/CIVPART/): Labor Force Participation Rate
- [EMRATIO](/indicator/EMRATIO/): Employment-Population Ratio
- [UNEMPLOY](/indicator/UNEMPLOY/): Unemployed
- [CE16OV](/indicator/CE16OV/): Employment Level
- [ICSA](/indicator/ICSA/): Initial Claims
- [JTSJOL](/indicator/JTSJOL/): Job Openings: Total Nonfarm
- [JTSQUR](/indicator/JTSQUR/): Quits: Total Nonfarm
- [GDPC1](/indicator/GDPC1/): Real Gross Domestic Product
- [A191RL1Q225SBEA](/indicator/A191RL1Q225SBEA/): Real Gross Domestic Product: Percent Change from Preceding Period
- [INDPRO](/indicator/INDPRO/): Industrial Production: Total Index
- [TCU](/indicator/TCU/): Capacity Utilization: Total Index
- [HOUST](/indicator/HOUST/): New Privately-Owned Housing Units Started: Total Units
- [PERMIT](/indicator/PERMIT/): New Privately-Owned Housing Units Authorized in Permit-Issuing Places: Total Units
- [RSAFS](/indicator/RSAFS/): Advance Retail Sales: Retail Trade
- [PCE](/indicator/PCE/): Personal Consumption Expenditures
- [DSPIC96](/indicator/DSPIC96/): Real Disposable Personal Income
- [PSAVERT](/indicator/PSAVERT/): Personal Saving Rate
- [M2SL](/indicator/M2SL/): M2
- [BOPGSTB](/indicator/BOPGSTB/): U.S. International Trade in Goods and Services: Balance
- [MSPUS](/indicator/MSPUS/): Median Sales Price of Houses Sold for the United States
- [HSN1F](/indicator/HSN1F/): New One Family Houses Sold: United States
- [RHORUSQ156N](/indicator/RHORUSQ156N/): Homeownership Rate in the United States
- [TTLCONS](/indicator/TTLCONS/): Total Construction Spending: Total Construction in the United States
- [RRVRUSQ156N](/indicator/RRVRUSQ156N/): Rental Vacancy Rate in the United States
- [TOTALSL](/indicator/TOTALSL/): Total Consumer Credit Owned and Securitized
- [REVOLSL](/indicator/REVOLSL/): Revolving Consumer Credit Owned and Securitized
- [DRCCLACBS](/indicator/DRCCLACBS/): Delinquency Rate on Credit Card Loans, All Commercial Banks
- [GDP](/indicator/GDP/): Gross Domestic Product
- [GPDI](/indicator/GPDI/): Gross Private Domestic Investment
- [GCE](/indicator/GCE/): Government Consumption Expenditures and Gross Investment
- [PCEC](/indicator/PCEC/): Personal Consumption Expenditures
- [NETEXP](/indicator/NETEXP/): Net Exports of Goods and Services
- [GFDEBTN](/indicator/GFDEBTN/): Federal Debt: Total Public Debt
- [GFDEGDQ188S](/indicator/GFDEGDQ188S/): Federal Debt: Total Public Debt as Percent of Gross Domestic Product
- [FYFSD](/indicator/FYFSD/): Federal Surplus or Deficit
- [FGRECPT](/indicator/FGRECPT/): Federal Government Current Receipts
- [FGEXPND](/indicator/FGEXPND/): Federal Government: Current Expenditures
- [MANEMP](/indicator/MANEMP/): All Employees, Manufacturing
- [USCONS](/indicator/USCONS/): All Employees, Construction
- [USTRADE](/indicator/USTRADE/): All Employees, Retail Trade
- [USFIRE](/indicator/USFIRE/): All Employees, Financial Activities
- [USGOVT](/indicator/USGOVT/): All Employees, Government
- [AWHAETP](/indicator/AWHAETP/): Average Weekly Hours of All Employees, Total Private
- [DGORDER](/indicator/DGORDER/): Manufacturers' New Orders: Durable Goods
- [NEWORDER](/indicator/NEWORDER/): Manufacturers' New Orders: Nondefense Capital Goods Excluding Aircraft
- [BUSINV](/indicator/BUSINV/): Total Business Inventories
- [EXPGS](/indicator/EXPGS/): Exports of Goods and Services
- [IMPGS](/indicator/IMPGS/): Imports of Goods and Services
- [IR](/indicator/IR/): Import Price Index (End Use): All Commodities
- [PPIFIS](/indicator/PPIFIS/): Producer Price Index by Commodity: Final Demand

## Latest quarter (10-Q)

Latest 10-Q source: https://www.sec.gov/Archives/edgar/data/1369568/000136956826000002/cprx-20260331.htm

Extracted between Part I Item 2 and the next Item 3/4 or Part II heading after HTML sanitization.
Confidence: high
Filing date: 2026-05-11
Report date: 2026-03-31

ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

Introduction

Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to provide an understanding of our financial condition, changes in financial condition and results of operations. The discussion and analysis is organized as follows:

•
Overview. This section provides a general description of our business and information about our business that we believe is important in understanding our financial condition and results of operations.

•
Basis of Presentation. This section provides information about key accounting estimates and policies that we followed in preparing our condensed consolidated financial statements for the first quarter of fiscal 2026.

•
Critical Accounting Policies and Estimates. This section discusses those accounting policies that are both considered important to our financial condition and results of operations and require significant judgment and estimates on the part of management in their application. All of our significant accounting policies, including the critical accounting policies, are also summarized in the notes to our interim condensed consolidated financial statements that are included in this report.

•
Results of Operations. This section provides an analysis of our results of operations for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025.

•
Liquidity and Capital Resources. This section provides an analysis of our cash flows, capital resources, off-balance sheet arrangements, and outstanding commitments.

•
Caution Concerning Forward-Looking Statements. This section discusses how certain forward-looking statements made throughout this MD&A and in other sections of this report are based on management’s present expectations about future events and are inherently susceptible to uncertainty and changes in circumstance.

OVERVIEW

We are a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases. We currently sell three commercial stage drug products, FIRDAPSE® (amifampridine), AGAMREE® (vamorolone), and FYCOMPA® (perampanel) in the United States. We are also currently seeking to further expand our product portfolio, with a focus on acquiring the rights to immediately and near-term accretive assets to treat rare (orphan) diseases across therapeutic areas, including clinical-stage opportunities with established proof of concept and a clear regulatory pathway to approval. With an unwavering patient focus embedded in everything we do, we are committed to providing innovative, best-in-class medications with the hope of making a meaningful positive impact on those affected by these conditions.

Currently, we have a total of 74 field personnel supporting FIRDAPSE® and AGAMREE® which includes Regional Account Managers, Area Business Directors, National Account Directors, Thought Leader Liaisons and Area Marketing Directors. This also includes 17 Patient Access Liaisons and insurance navigation support personnel who support both FIRDAPSE® and AGAMREE® and 15 Medical Science Liaisons who help educate the medical community about scientific literature concerning our drug products and the diseases they treat.

When we launched AGAMREE® in March 2024, we utilized the FIRDAPSE® commercial and medical field-based forces to market AGAMREE®. Effective April 1, 2025, we separated these field-based forces into two distinct units, one for each function expressly focused on supporting FIRDAPSE® and one for each function expressly focused on supporting AGAMREE®, respectively. This strategic change was made in an effort to allow us to better focus the support for each product. Further, we have two National Account Directors on our commercial team who exclusively focus on the oncology market for Lambert-Eaton Myasthenic Syndrome (LEMS) patients who also have cancer.

Recent Developments

On May 6, 2026, we entered into an Agreement and Plan of Merger (the Merger Agreement) with Angelini Pharma S.p.A., an Italian Società per azioni (Angelini Pharma or Parent) and Angelini Cielo Inc., a Delaware corporation and wholly-owned subsidiary of Parent (Merger Sub), providing for the merger of Merger Sub with and into the Company (the Merger), with the Company surviving the Merger as a wholly-owned subsidiary of Parent.

For further information about the Merger Agreement and the Merger, see the Current Report on Form 8-K that we filed with the SEC on May 7, 2026 reporting with more particularity on the terms of the Merger Agreement and the proposed Merger.

FIRDAPSE®

35

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On November 28, 2018, we received approval from the U.S. Food & Drug Administration (FDA) for our new drug application (NDA) for FIRDAPSE® Tablets 10 mg for the treatment of adult patients (ages 17 and above) with LEMS, and in January 2019, we launched FIRDAPSE® in the U.S. Further, on September 29, 2022, the FDA approved our supplemental NDA (sNDA) to expand the indicated age range for FIRDAPSE® Tablets 10 mg for the treatment of LEMS to include pediatric patients six years of age and older. Additionally, on May 30, 2024, the FDA approved our sNDA increasing the indicated maximum daily dosage of FIRDAPSE® tablets for the treatment of patients with LEMS from 80 mg to 100 mg. We are also planning to evaluate FIRDAPSE® in the future for the treatment of other diseases.

We sell FIRDAPSE® in the U.S. through a field-based force experienced in neurologic, central nervous system or rare disease products consisting at this time of approximately 26 field personnel, including sales (Regional Account Managers and Area Business Directors), National Account Directors and Thought Leader Liaisons. This includes two National Account Directors and three Thought Leader Liaisons who exclusively focus on the oncology market for LEMS patients with cancer. We also use non-personal promotion to reach the 20,000 neurologists who are potential LEMS treaters and the 16,000 oncologists who might be treating a LEMS patient who also has cancer (principally small cell lung cancer). Finally, we make available for online ordering a no-cost LEMS voltage gated calcium channel (VGCC) antibody diagnostic testing program for use by physicians who suspect that one of their patients may have LEMS and wish to reach a definitive diagnosis.

Further, we are continuing to expand our digital and social media activities to introduce our products and services to potential patients and their healthcare providers. We also work with several rare disease advocacy organizations (including the Myasthenia Gravis Foundation of America, the National Organization for Rare Disorders, and the LEMS Family Association) to help increase awareness and level of support for patients living with LEMS and to provide education for the physicians who treat this rare disease and the patients they treat.

On August 6, 2025, we reported that the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Small Cell Lung Cancer (SCLC) now include additions involving LEMS, amifampridine, and the tests for PQ- and N-type VGCC antibodies. The updated NCCN Guidelines® for SCLC relating to LEMS now include symptom specificity—characterized by proximal muscle weakness and autonomic dysfunction. Under "Signs and Symptoms of Small Cell Lung Cancer” (SCL-A 2 of 2), the guidelines recommend diagnosis through a neurological evaluation, ideally in consultation with a neurologist, which may include testing for PQ- and N-type VGCC antibodies. Additionally, under “Principles of Supportive Care” (SCL-D), the guidelines recommend that amifampridine should be considered as a treatment in consultation with neurology. We are currently working to increase awareness of these oncology clinical practice guidelines among oncology practices that treat patients with SCLC.

We are supporting the distribution in the U.S. of FIRDAPSE® through Catalyst Pathways®, our personalized treatment support program for patients who enroll in it. Catalyst Pathways® is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen required to reach an effective therapeutic dose. The program also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most drug products for ultra-orphan diseases are distributed and dispensed to patients. We believe that by using specialty pharmacies in this way, the task, which can be difficult, of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general.

In order to help patients with LEMS afford their medication, we, like other pharmaceutical companies that market drug products for orphan and ultra-orphan, rare diseases, have developed an array of financial assistance programs intended to reduce out-of-pocket costs in order to make FIRDAPSE® accessible and affordable. A co-pay assistance program has been designed to reduce commercial patients’ out of pocket costs to as little as $0 whenever possible (currently an average of less than $2 per month). Our co-pay assistance programs, including the one for FIRDAPSE®, are not available to patients enrolled in state or federal healthcare programs, including Medicare, Medicaid, Department of Veterans Affairs (VA), Department of Defense (DoD), or TRICARE. However, we have, at times, donated funds to one or more qualified, independent charitable foundations dedicated to providing assistance to any U.S. LEMS patients in financial need who meet those independent organizations' guidelines. In addition, we have a program in place to help patients who are uninsured and underinsured. Subject to compliance with applicable regulatory requirements, our goal is that no LEMS patient is ever denied access to their medication for financial reasons.

FIRDAPSE® is currently marketed for the treatment of LEMS in Canada through our exclusive sublicensee, KYE Pharmaceuticals, Inc. (KYE). We supply product to KYE at agreed upon prices and we are also eligible to earn sales milestones and sales royalties based on net revenues from sales of the product in Canada. Further, FIRDAPSE® is commercially available (since January 21, 2025) for the treatment of LEMS in Japan through our sublicensee for Japan, DyDo Pharma, Inc. (DyDo). We generate revenue from DyDo through the receipt of additional milestone payments, as such milestones are achieved, and a transfer price on the product supplied by us to DyDo (in lieu of royalties).

We control six U.S. patents for FIRDAPSE® that are listed in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book), the earliest of which expires in 2032 and the latest of which expires in 2037. Generic drug manufacturers were permitted to submit applications for the product challenging our patents starting in 2023 and the FDA is now permitted to approve such ANDA products following the expiration of our orphan drug exclusivity (ODE) on November 26, 2025, subject to any pending

36

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30-month stays of approval as required under the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Amendments).

With respect to ANDA filers, in January 2023, we received Paragraph IV Certification Notice Letters from three generic drug manufacturers (Teva Pharmaceuticals USA, Inc. and Teva Pharmaceuticals, Inc. (collectively Teva), Hetero USA, Inc. (Hetero), and Lupin Pharmaceuticals, Inc. (Lupin)) advising that they each had submitted an ANDA to the FDA seeking authorization from the FDA to manufacture, use or sell a generic version of FIR

[Excerpt truncated for page length; source filing is linked above.]

## Latest 10-K MD&A

Extracted between Item 7 and the next Item 7A/8 heading after HTML sanitization.
Confidence: high

Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion of our financial condition and results of operations should be read together with the Consolidated Financial Statements and related notes included elsewhere in this Annual Report on Form 10-K. This discussion and analysis may contain forward-looking statements that involve certain risks, assumptions and uncertainties that could cause actual results to differ materially from those implied or described by the forward-looking statements. Future results could differ materially from the discussion that follows for many reasons, including the factors described in Part I, Item 1A. “Risk Factors” in this Annual Report on Form 10-K, as well as those described in future reports filed with the SEC.

Introduction

Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to provide an understanding of our financial condition, changes in financial condition and results of operations. The discussion and analysis is organized as follows:

•
Overview. This section provides a general description of our business and information about our business that we believe is important in understanding our financial condition and results of operations.

•
Basis of Presentation. This section provides information about key accounting estimates and policies that we followed in preparing our consolidated financial statements for the 2025 fiscal year.

•
Critical Accounting Policies and Estimates. This section discusses those accounting policies that are both considered important to our financial condition and results of operations and require significant judgment and estimates on the part of management in their application. All of our significant accounting policies, including the critical accounting policies, are also summarized in the notes to our accompanying consolidated financial statements.

•
Results of Operations. This section provides an analysis of our results of operations for the three fiscal years presented in the accompanying consolidated statements of operations and comprehensive income.

•
Liquidity and Capital Resources. This section provides an analysis of our cash flows, capital resources, off-balance sheet arrangements, and our outstanding commitments, if any.

•
Caution Concerning Forward-Looking Statements. This section discusses how certain forward-looking statements made throughout this MD&A and in other sections of this report are based on management’s present expectations about future events and are inherently susceptible to uncertainty and changes in circumstance.

Overview

We are a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases. We currently sell three commercial stage drug products, FIRDAPSE® (amifampridine), AGAMREE® (vamorolone), and FYCOMPA® (perampanel). We are also currently seeking to further expand our product portfolio, with a focus on acquiring the rights to immediately and near-term accretive assets to treat rare (orphan) diseases across therapeutic areas, including clinical-stage opportunities with established proof of concept. With an unwavering patient focus embedded in everything we do, we are committed to providing innovative, best-in-class medications with the hope of making a meaningful positive impact on those affected by these conditions.

Currently, we have a total of 58 field personnel supporting FIRDAPSE® and AGAMREE®. We also have 19 patient assistance liaisons and insurance navigation support personnel who support both FIRDAPSE® and AGAMREE®. Finally, we have 19 medical science liaisons who help educate the medical community about scientific literature concerning our drug products and the diseases they treat.

When we launched AGAMREE® in March 2024, we utilized the FIRDAPSE® commercial and medical field-based forces to market AGAMREE®. In early 2025, we separated these field-based forces into two units, one for each function expressly focused on supporting FIRDAPSE® and one for each function expressly focused on supporting AGAMREE®. This strategic change was made in an effort to allow us to better focus the support for each product. This division of our field-based forces into two units became effective on April 1, 2025. Further, we have two national account directors on our commercial team who exclusively focus on the oncology market for LEMS patients who also have cancer.

FIRDAPSE®

On November 28, 2018, we received approval from the FDA for our new drug application (NDA) for FIRDAPSE® Tablets 10 mg for the treatment of adult patients (ages 17 and above) with Lambert-Eaton Myasthenic Syndrome (LEMS), and in January 2019, we launched FIRDAPSE® in the U.S. Further, on September 29, 2022, the FDA approved our supplemental NDA (sNDA) to expand the indicated age range for FIRDAPSE® Tablets 10 mg for the treatment of LEMS to include pediatric patients six years of age and older. Additionally, on May 30, 2024, the FDA approved our sNDA increasing the indicated

62

Table of Contents

maximum daily dosage of FIRDAPSE® tablets for the treatment of patients with LEMS from 80 mg to 100 mg. We believe that this most recent sNDA approval offers healthcare providers and patients greater flexibility in treatment regimens for the management of LEMS. We are also planning to evaluate FIRDAPSE® in the future for the treatment of other diseases.

We sell FIRDAPSE® in the U.S. through a field-based force experienced in neurologic, central nervous system or rare disease products consisting at this time of approximately 23 field personnel, including sales (Regional Account Managers and Area Business Directors), National Account Directors and thought leader liaisons. This includes two national account directors who exclusively focus on the oncology market for LEMS patients with cancer. We also use non-personal promotion to reach the 20,000 neurologists who are potential LEMS treaters and the 16,000 oncologists who might be treating a LEMS patient who also has cancer (principally small cell lung cancer). Finally, we make available for online ordering a no-cost LEMS voltage gated calcium channel (VGCC) antibody diagnostic testing program for use by physicians who suspect that one of their patients may have LEMS and wish to reach a definitive diagnosis.

Further, we are continuing to expand our digital and social media activities to introduce our products and services to potential patients and their healthcare providers. We also work with several rare disease advocacy organizations (including the Myasthenia Gravis Foundation of America, the National Organization for Rare Disorders, and the LEMS Family Association) to help increase awareness and level of support for patients living with LEMS and to provide education for the physicians who treat this rare disease and the patients they treat.

On August 6, 2025, we reported that the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Small Cell Lung Cancer (SCLC) now include new additions involving LEMS, amifampridine, and the tests for PQ- and N-type VGCC antibodies. The updated NCCN Guidelines® for SCLC relating to LEMS now include symptom specificity—characterized by proximal muscle weakness and autonomic dysfunction. Under "Signs and Symptoms of Small Cell Lung Cancer” (SCL-A 2 of 2), the guidelines recommend diagnosis through a neurological evaluation, ideally in consultation with a neurologist, which may include testing for PQ- and N-type VGCC antibodies. Additionally, under “Principles of Supportive Care” (SCL-D), the guidelines recommend that amifampridine should be considered as a treatment in consultation with neurology. We are currently working to get these guidelines into the standard of care followed by oncology practices that treat patients with SCLC.

We are supporting the distribution in the U.S. of FIRDAPSE® through Catalyst Pathways®, our personalized treatment support program for patients who enroll in it. Catalyst Pathways® is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen required to reach an effective therapeutic dose. The program also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most drug products for ultra-orphan diseases are distributed and dispensed to patients. We believe that by using specialty pharmacies in this way, the task, which can be difficult, of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general.

In order to help patients with LEMS afford their medication, we, like other pharmaceutical companies which market drug products for orphan and ultra-orphan, rare diseases, have developed an array of financial assistance programs to reduce out-of-pocket costs that makes FIRDAPSE® accessible and affordable. A co-pay assistance program has been designed to reduce commercial patients’ out of pocket costs to as little as $0 whenever possible (currently an average of less than $2 per month). Our co-pay assistance programs, including the one for FIRDAPSE®, are not available to patients enrolled in state or federal healthcare programs, including Medicare, Medicaid, Department of Veterans Affairs (VA), Department of Defense (DoD), or TRICARE. However, we have, at times, donated funds to one or more qualified, independent charitable foundations dedicated to providing assistance to any U.S. LEMS patients in financial need who meet those independent organizations' guidelines. In addition, we have a program in place to help patients who are uninsured and underinsured. Subject to compliance with applicable regulatory requirements, our goal is that no LEMS patient is ever denied access to their medication for financial reasons.

FIRDAPSE® is currently marketed for the treatment of LEMS in Canada through our exclusive sublicensee, KYE Pharmaceuticals, Inc. (KYE). We supply product to KYE at agreed upon prices and we are also eligible to earn sales milestones and sales royalties based on net revenues from sales of the product in Canada. Further, FIRDAPSE® is commercially available (since January 21, 2025) for the treatment of LEMS in Japan through our sublicensee for Japan, DyDo Pharma, Inc. (DyDo). We generate revenue from DyDo through the receipt of additional milestone payments, as such milestones are achieved, and a transfer price on the product supplied by us to DyDo (in lieu of royalties).

We control six U.S. patents for FIRDAPSE® that are listed in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book), the earliest of which expires in 2032 and the latest of which expires in 2037. Generic drug manufacturers were permitted to submit applications for the product challenging our patents starting in 2023 and the FDA is now permitted to approve such ANDA products following the expiration of our orphan drug exclusivity (ODE) on November 26, 2025, subject to any pending 30-month stays of approval as required under the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Amendments).

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With respect to ANDA filers, in January 2023, we received Paragraph IV Certification Notice Letters from three generic drug manufacturers (Teva Pharmaceuticals USA, Inc. and Teva Pharmaceuticals, Inc. (collectively Teva), Hetero USA, Inc. (Hetero), and Lupin Pharmaceuticals, Inc. (Lupin)) advising that they each had submitted an ANDA to the FDA seeking authorization from the FDA to manufacture, use or sell a generic version of FIRDAPSE® in the U.S. The notice letters each alleged that the six patents protecting FIRDAPSE® that are listed in the Orange Book in connection with FIRDAPSE® are not valid, not enforceable, and/or will not be infringed by the commercial manufacture, use or sale of the proposed product described in these ANDA submissions. Under the Federal Food, Drug, and Cosmetic Act (FDCA), as amended by the Drug Price Competition and Patent Term Restoration Act of 1984, we had 45 days from receipt of the notice letters to determine if there were grounds to bring a lawsuit and, if so, to commence patent infringement lawsuits against these generic drug manufacturers in a federal district court, which would trigger a statutory stay precluding the FDA from final approval of the subject ANDA until May 26, 2026 or entry of judgment holding the patents invalid, unenforceable, or not infringed, whichever occurs first in all cases (but not earlier than the expiration of orphan drug exclusivity on November 28, 2025). In that regard, after conducting the necessary due diligence, we filed lawsuits on March 1, 2023 in the U.S. District Court for the District of New Jersey against each of the three generic drug manufacturers who notified us of their ANDA submissions, thus triggering the stay.

Additionally, in October 2023, we received a Paragraph IV Certification Notice Letter from a fourth generic drug manufacturer (Inventia Healthcare Limited (Inventia)), and after conducting the necessary diligence we filed a similar lawsuit against that manufacturer in November 2023 in the U.S. District Court for the District of New Jersey. On July 30, 2024, we settled this patent litigation with Inventia. In this settlement, Inventia acknowledged both the validity of our FIRDAPSE® patents and also the infringement by the ANDA filer's product of our patents. As part of the settlement, Inventia also agreed not to commercialize its product until the earlier of all FIRDAPSE® patents expiration or the entry into the market of another ANDA product meeting certain conditions.

In June 2024, Lupin converted five of its Paragraph IV Certifications in its ANDA to Paragraph III certifications acknowledging the validity and their ANDA’s infringement of five of those patents, the latest ending in 2034. We subsequently dismissed all of our claims against Lupin related to those five patents but maintained our claim against Lupin for the remaining Paragraph IV certification for U.S. Patent No. 10,626,088, which is the patent expiring in 2037.

On January 8, 2025, we reached a settlement with Teva in which Teva agreed not to market a generic version of FIRDAPSE® in the U.S. any earlier than February 25, 2035, if approved by the FDA, unless certain limited circumstances customarily included in these types of agreements occur. In accordance with the settlement agreement, the parties terminated all ongoing patent litigation between us and Teva regarding FIRDAPSE® patents pending in the U.S. District Court for the District of New Jersey. Later, on August 26, 2025, we reached a settlement with Lupin on substantially the same terms for market entry.

The pending FIRDAPSE® patent litigation against the remaining defendant, Hetero (relating to the FIRDAPSE® Orange Book-listed patents expiring in 2032, 2034 and 2037) remains ongoing. At this time, this trial is scheduled to start on March 23, 2026, which is prior to the expiration of the 30-month stay on FDA approval of Hetero’s ANDA under the Hatch-Waxman Amendments ending on May 26, 2026.

Since cases of this type are complex and the results of patent litigation with Paragraph IV challengers is always uncertain, there can be no assurance as to whether we will prevail in this litigation. As a result, there can be no assurance as to whether our currently ongoing litigation with Hetero will allow a generic version of FIRDAPSE® to be marketed in the U.S. prior to Teva's and Lupin's licensed entry into the market on February 25, 2035.

AGAMREE®

On June 19, 2023, we entered into a License and Collaboration Agreement (AGAMREE® License Agreement) and an Investment Agreement (Investment Agreement) with Santhera Pharmaceuticals Holding AG (collectively, Santhera). Under the AGAMREE® License Agreement, we contracted to obtain an exclusive North America license, manufacturing and supply agreement for Santhera’s investigational product candidate, AGAMREE®, a novel corticosteroid for the treatment of Duchenne muscular dystrophy, or DMD. Under the Investment Agreement, we agreed to make a strategic investment into Santhera.

Both transactions closed on July 18, 2023. Under the AGAMREE® License Agreement, upon closing we made a $75 million payment to Santhera in return for the exclusive North American license for AGAMREE®. Additionally, we hold the North American rights to any future approved indications for AGAMREE®. Finally, under our AGAMREE® License Agreement with Santhera, we agreed to purchase commercial supply of AGAMREE® from Santhera at agreed upon prices until we completed our process to transition to our own direct supplier. We are also in the process of transitioning final goods manufacturing to a U.S. location (estimated to be completed in 2026).

Concurrent with the closing of the AGAMREE® License Agreement, we made a strategic investment into Santhera in which we acquired 1,414,688 of Santhera’s ordinary shares (representing approximately 11.26% of Santhera’s outstanding ordinary shares immediately following the transaction) at an investment price of CHF 9.477 per share, with the approximately $15.7

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million USD in equity investment proceeds to be used by Santhera for Phase IV studies of AGAMREE® in DMD and future development of additional indications for AGAMREE®. On February 23, 2026, the closing price of Santhera’s common shares on the SIX Swiss Exchange was CHF 16.46 per share (approximately $21.33 USD based on then-current exchange rates).

On October 26, 2023, the U.S. FDA approved Santhera’s NDA for AGAMREE® for use in treating DMD in patients aged two years and older. Shortly thereafter, as part of the previously described transaction, Santhera transferred the approved NDA to us. Additionally, following approval of the NDA for the drug, we became obligated to make a milestone payment of $36 million to Santhera, which we paid during the fourth quarter of 2023. We may also be obligated to pay future regulatory and commercial milestone payments to Santhera tied to calendar year sales of AGAMREE® (the first such sales-based royalty was achieved in the fourth quarter of 2025 when our AGAMREE® net product revenues for 2025 exceeded $100 million), as well as commercial royalties.

When we launched AGAMREE® in March 2024, we utilized the FIRDAPSE® commercial and medical field-based forces to market AGAMREE®. In early 2025, we separated these field-based forces into two units, one for each function expressly focused on supporting FIRDAPSE® and one for each function expressly focused on supporting AGAMREE®. This strategic change was made in an effort to allow us to better focus the support for each product. This division of our field-based forces into two units became effective on April 1, 2025.

Since April 1, 2025, we have sold AGAMREE® in the U.S. through a dedicated field-based force of approximately 16, including sales (12 Regional Account Managers and two Area Business Directors) and two Area Marketing Directors. We are further supporting the distribution of AGAMREE® through our Catalyst Pathways® patient services program to ensure that patients have access to a dedicated, personalized support team that assists families through the AGAMREE® patient journey, from answering questions to coordinating financial assistance programs for eligible patients. Additionally, we have also donated to qualified, independent charitable foundations dedicated to providing assistance to DMD patients in financial need who meet the independent organization's guidelines. Subject to applicable regulatory requirements, our goal is to ensure that no DMD patient is ever denied access to their medication for financial reasons.

DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 people in the U.S. are affected by DMD, with approximately 70% of patients currently receiving a corticosteroid treatment. We believe that steroids are and will continue to remain the foundational therapy for DMD patients and dosed concomitantly with other therapies.

AGAMREE®’s unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid that we hope has the potential to demonstrate comparable efficacy to corticosteroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow vamorolone to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In that regard, we are currently enrolling up to 250 patients in our SUMMIT registry study to evaluate data about long-term patient safety and quality of life data from the use of our product, with the hope of offering a deeper understanding of the product's potential long-term benefits for patients, such as in the areas of stature, bone health, behavior, and cardiovascular health.

On October 13, 2023, Santhera announced that the European Union’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive position in favor of AGAMREE® for the treatment of DMD patients aged four and older. In its recommendation for approval, CHMP acknowledged that there was a positive benefit-risk profile of AGAMREE® in such patient population, including certain safety benefits of AGAMREE® compared to standard of care corticosteroids in the treatment of DMD. Further, on December 18, 2023, the European Commission (EC) granted to Santhera marketing authorization for AGAMREE® for the treatment of DMD in patients ages four years and older and on January 12, 2024 Santhera announced that AGAMREE® had received approval by the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom. Further, on January 15, 2024, Santhera announced that AGAMREE® was commercially launched in Germany. Additionally, on January 16, 2025, the National Institute for Health and Care Excellence (NICE) issued positive Final Guidance that recommends AGAMREE® for use in the National Health Service (NHS) in England, Wales and Northern Ireland for the treatment of DMD in patients four years of age and older and on February 13, 2025, Santhera announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement for AGAMREE® for the treatment of DMD. This agreement makes AGAMREE® the first product to receive an agreed federal price in Germany for the treatment of DMD in patients four years of age and older, independent of the underlying genetic mutation. Finally, on January 15, 2026, Santhera announced that the Swiss Agency for Therapeutic Products (Swissmedic) approved AGAMREE® for the treatment of DMD in patients four years of age and older.

We are currently taking the first steps seeking to expand the number of diseases that can be treated with AGAMREE®. In furtherance of that objective, we are currently conducting a Phase 1 study in healthy adults comparing a single dose of

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vamorolone, prednisone, and deflazacort, and studying the immunosuppressive effect of multiple ascending doses of AGAMREE®, which study will attempt to define the immunosuppressive dose of vamorolone for future indications and for the use of our product in conjunction with gene and cell therapies that are approved to treat DMD and require a concurrent immunosuppressive regimen of a corticosteroid when administered. We expect to have the results of both parts of this study by the end of the second quarter of 2026. Further, we are hopeful that the recent addition of DMD to the Recommended Uniform Screening Panel by the U.S. Department of Health and Human Services will help support earlier detection of the disease and more timely access to treatment options.

Further, we have established a joint steering committee with Santhera that is overseeing the lifecycle management and development of AGAMREE®. There can be no assurance that we can develop our product for the treatment of diseases other than DMD. Finally, we are currently in the process of validating a U.S. manufacturing site for the product, which we expect will be completed by the end of 2026.

In the U.S., AGAMREE® has New Chemical Entity exclusivity that expires in October 2028. AGAMREE® also has Orphan Drug Exclusivity expiring in October 2030. AGAMREE® is further protected by seven Orange Book listed patents expiring as early as May 28, 2029 and as late as July 16, 2040. The Company has also requested Patent Term Extension (PTE) and will update the relevant expiration date in the Orange Book upon a final determination by the U.S. Patent and Trademark Office (USPTO). On June 25, 2025, the FDA published a notice (Determination of Regulatory Review Period for Purposes of Patent Extension; AGAMREE®) in the Federal Register regarding the requested extension of patent numbers 8,334,279, 10,857,161, and 11,833,159, all of which currently expire on May 28, 2029, as listed in the FDA's Orange Book. With the publication of this notice, there is a 180-day period for third parties to submit comments and/or due diligence petitions to the FDA. If no comments or petitions are submitted within this period, the FDA will notify the USPTO so that the USPTO can then determine the length of the PTE for each patent for which an extension was requested. Upon completion of the PTE determination, USPTO will mail a Notice of Final Determination of PTE. We will then have one month from the mailing of the Notice of Final Determination to elect one of the three patents for an extension patent term.

The earliest a generic manufacturer could submit an ANDA for vamorolone is October 26, 2027. If we were to pursue a patent infringement action of any such ANDA challenges of any of AGAMREE®’s Orange Book patents, then the automatic statutory 30-month stay would prevent FDA approval of such ANDA until April 26, 2031.

On July 23, 2024 we entered into a license, supply and commercialization agreement with KYE, which is already our sublicensee for FIRDAPSE® in Canada, granting KYE the exclusive Canadian commercial rights to market AGAMREE® in Canada for DMD and other indications. Under the agreement, KYE was responsible for obtaining regulatory approval of the product from Health Canada and on April 8, 2025, we announced that Health Canada had accepted for review KYE's New Drug Submission (NDS) for AGAMREE®. Further, on October 2, 2025, KYE announced that Health Canada has approved AGAMREE® for the treatment of DMD in boys four years of age and older. Under our sublicense agreement with KYE for this product, we will supply product to KYE and also receive sales milestones and sales royalties based on net revenues from sales of the product in Canada.

FYCOMPA®

On December 17, 2022, we entered into an agreement with Eisai Co., Ltd. (Eisai) for the acquisition of the U.S. rights to FYCOMPA® CIII. FYCOMPA® is a selective non-competitive antagonist of AMPA receptors, the major subtype of ionotropic glutamate receptors. It was the first, and still is the only, drug of its class to be approved for epilepsy. Studies suggest that AMPA receptor antagonism can lead to reduced overstimulation and anticonvulsant effects, as well as inhibiting seizure generation and spread. FYCOMPA® is a controlled substance and is approved with a boxed warning in its labeling. FYCOMPA® is used to treat certain types of focal onset seizures (seizures that involve only one part of the brain) in adults and children four years of age and older. It is also used in combination with other medications to treat certain types of primary generalized tonic-clonic seizures (also known as a “grand mal” seizure, a seizure that involves the entire body) in adults and children 12 years of age or older. Perampanel is in a class of medications called anticonvulsants. It works by decreasing abnormal electrical activity in the brain.

On January 24, 2023, we closed our acquisition of the U.S. rights to FYCOMPA®. In connection with the acquisition, we purchased Eisai’s regulatory approvals and documentation, product records, intellectual property, inventory, and other matters relating to the U.S. rights for FYCOMPA®, in exchange for an upfront payment of $160 million in cash. We also agreed to pay Eisai royalty payments on net sales only after all FYCOMPA® patents are expired with such royalty payments reduced after generic equivalents enter the market.

In conjunction with the closing of the asset purchase, we entered into two additional agreements, a Transition Services Agreement (TSA) and a Supply Agreement. Under the Supply Agreement, Eisai agreed to manufacture FYCOMPA® for us for at least seven years at prices listed in the Supply Agreement (to be updated on a yearly basis), and under the TSA, a U.S. subsidiary of Eisai provided us with certain transitional services (which transition services ended on December 31, 2023).

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Patent protection for FYCOMPA® tablets and oral solution was primarily derived from two patents listed in the FDA’s Orange Book. The first, U.S. patent no. 6,949,571 (the ‘571 patent), expired on May 23, 2025, including patent term extension. The second FYCOMPA® patent in the Orange Book is U.S. Patent No. 8,772,497 (the ‘497 patent), which will expire on July 1, 2026. The ‘497 patent, which covers certain polymorphic forms of the active pharmaceutical ingredient (API) used in both FYCOMPA® tablets and oral suspension. One of the Orange Book listed patents had been the subject of previous Paragraph IV certifications from three ANDA filers for the tablet formulation, which were not contested by Eisai prior to our acquisition of the drug. As a result, to our knowledge based on publicly available information, three ANDA filers for the tablet formulation have, to date, obtained approval for and are marketing a generic version of FYCOMPA® tablets and one ANDA filer has, to date, obtained approval for and is marketing a generic version of FYCOMPA® oral suspension. We will continue to sell FYCOMPA® tablets and oral suspension despite the loss of exclusivity, although we have ceased active marketing efforts for the product effective December 31, 2025.

Business Development

We continue to advance our strategic initiatives and portfolio expansion efforts, focusing on broadening and diversifying our rare (orphan) neurology product portfolio with innovative therapies that address critical unmet medical needs. In that regard, we are currently exploring immediate or near-term accretive, clinically differentiated and adequately de-risked opportunities, with a keen focus on rare (orphan) disease products across therapeutic areas and treatment modalities. These prospects include evaluating companies with existing commercial drug products or drugs in development, including clinical stage opportunities with established proof of concept, for potential licensing or acquisitions. We maintain a well-established U.S. presence, which remains the cornerstone of our commercial strategy, while continuously evaluating strategic opportunities to expand our global footprint.

We employ a disciplined, comprehensive, and exhaustive approach to identifying and evaluating opportunities that we believe will add significant value to our company over the near, mid, and long-term. However, no definitive agreements have been entered into to date to acquire the rights to any additional products, and there can be no assurance that any of the Company's business development initiatives will be successful.

Capital Resources

At December 31, 2025, we had cash and cash equivalents of approximately $709.2 million. Based on our current financial condition, including our profitability, cash flows generated from operations and forecasts of available cash, absent the use of cash to acquire potential business development opportunities, we believe that we have sufficient funds to support our operations for at least the next 12 months. There can be no assurance that we will continue to be successful in commercializing FIRDAPSE® and AGAMREE®, that our forecasts of revenues from sales of FYCOMPA® will be accurate now that generic competition has entered the market, or that we will continue to be profitable and cash flow positive. See “Item 7 - Management’s Discussion and Analysis of Financial Condition and Results of Operations” below for further information on our liquidity and cash flow.

Share Repurchase Program

On October 1, 2025, we announced that our Board of Directors has authorized a new share repurchase program to repurchase up to $200 million of shares of our common stock between October 1, 2025 and December 31, 2026. Repurchases under the new share repurchase program may be made through a variety of methods, including open market or privately negotiated purchases. The timing and amount of shares repurchased will depend on the stock price, business and market conditions, corporate and regulatory requirements, alternative investment opportunities, acquisition opportunities and other factors. We are not obligated to repurchase any specific amount of shares of common stock, and the new share repurchase program may be suspended or terminated at any time.

We are using existing cash on hand to fund our share repurchase program. We also believe that we can execute this share repurchase program without impairing the advancement of our business development strategy. In that regard, as of February 23, 2026, we have repurchased 1,740,713 shares of our outstanding common stock for an aggregate purchase price of approximately $39.9 million ($22.91 average price per share).

Basis of Presentation

Revenues

During the fiscal year ended December 31, 2025, we generated revenues from product sales of FIRDAPSE®, AGAMREE®, and FYCOMPA®. We expect these revenues to fluctuate in future periods based on our sales during such periods of our products.

We received approval from Health Canada on July 31, 2020, for FIRDAPSE® for the symptomatic treatment of LEMS and as of December 31, 2020, our sub-licensee KYE launched FIRDAPSE® in Canada. During the fiscal year ended December 31, 2025, revenues generated under our collaboration agreement with KYE were immaterial. In July 2024, we announced that we

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had entered into a collaboration agreement with KYE for the commercialization of AGAMREE® in Canada. On October 2, 2025, KYE issued a press release reporting that its NDS to commercialize AGAMREE® in Canada had been approved by Health Canada.

On September 24, 2024, we were informed by DyDo that it had received approval of its New Drug Application for the sale of FIRDAPSE® in Japan. Further, DyDo advised us that they launched FIRDAPSE® in Japan in January 2025.

We expect revenues from both the KYE and DyDo agreements to be immaterial in 2026, as distribution ramps up in each jurisdiction and KYE begins to market AGAMREE® in Canada.

Cost of Sales

Cost of sales consists of third-party manufacturing costs, freight, royalties, milestone payments, and indirect overhead costs associated with sales of our products. Cost of sales may also include period costs related to certain inventory manufacturing services, inventory adjustments charges, unabsorbed manufacturing and overhead costs and manufacturing variances.

Research and Development Expenses

Our research and development expenses consist of costs incurred for company-sponsored research and development activities, as well as support for selected investigator-sponsored research. The major components of research and development costs include acquired IPR&D, preclinical study costs, clinical manufacturing costs, clinical study and trial expenses, insurance coverage for clinical trials, consulting, and other third-party costs, salaries and employee benefits, stock-based compensation expense, supplies and materials, and allocations of various overhead costs related to our product development efforts.

Prior to January 2023, all of our research and development resources had been devoted to the development of FIRDAPSE®, and until such time as we acquire or license new products we currently expect that our future development costs will be attributable principally to the continued development of FIRDAPSE®, and AGAMREE®.

Our cost accruals for clinical studies and trials are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical study and trial sites and clinical research organizations (CROs). In the normal course of our business we contract with third parties to perform various clinical study and trial activities in the on-going development of potential products. The financial terms of these agreements are subject to negotiation and vary from contract to contract and may result in uneven payment flows. Payments under the contracts depend on factors such as the achievement of certain events or milestones, the successful enrollment of patients, the allocation of responsibilities among the parties to the agreement, and the completion of portions of the clinical study or trial or similar conditions. The objective of our accrual policy is to match the recording of expenses in our consolidated financial statements to the actual services received and efforts expended. As such, expense accruals related to preclinical and clinical studies or trials are recognized based on our estimate of the degree of completion of the event or events specified in the specific study or trial contract. We monitor service provider activities to the extent possible; however, if we underestimate activity levels associated with various studies or trials at a given point in time, we could be required to record significant additional research and development expenses in future periods. Preclinical and clinical study and trial activities require significant up-front expenditures. We anticipate paying significant portions of a study or trial’s cost before they begin and incurring additional expenditures as the study or trial progresses and reaches certain milestones.

Selling, General and Administrative Expenses

During 2019, we began to commit funds to developing our commercialization program for FIRDAPSE® and we have continued to incur substantial commercialization expenses, including sales, marketing, patient services, patient advocacy and other commercialization related expenses as we have continued our sales and marketing program for FIRDAPSE®. We are also now incurring substantial commercialization expenses for AGAMREE®, as we continue commercialization of this product. We expect that such expenses for FYCOMPA® will substantially decline as a result of declining sales resulting from the loss of exclusivity of the product with the entry of generic competitors for this product.

Our general and administrative expenses consist primarily of salaries and personnel expenses for accounting, corporate, compliance, and administrative functions. Other costs include administrative facility costs, regulatory fees, insurance, and professional fees for legal (including litigation) cost, IT, accounting, and consulting services.

Amortization of Intangible Assets

Amortization of intangible assets consists of the amortization of the FYCOMPA® product rights, which are amortized using the straight-line method over its estimated useful life of 5 years, the RUZURGI® product rights, which are amortized using the straight-line method over its estimated useful life of 14.5 years, and the AGAMREE® product rights, which are amortized using the straight-line method over its estimated useful life of 10.5 years.

Stock-Based Compensation

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We recognize expense for the fair value of all stock-based awards to employees, directors, and consultants in accordance with accounting principles generally accepted in the U.S. (U.S. GAAP). For stock options, we use the Black-Scholes option valuation model in calculating the fair value of the awards.

Income Taxes

Our effective income tax rate is the ratio of income tax expense over our income before income taxes. As of December 31 2025, 2024, and 2023, we had no federal net operating loss carry-forwards. Additionally, we had no state net operating loss carry-forwards in any such year.

Recently Issued Accounting Standards

For discussion of recently issued accounting standards, please see Note 2, "Basis of Presentation and Significant Accounting Policies," in the consolidated financial statements included in this report.

Critical Accounting Policies and Estimates

Our discussion and analysis of our financial condition and results of operations are based on our consolidated financial statements, which have been prepared in accordance with U.S. Generally Accepted Accounting Principles (GAAP). The preparation of these consolidated financial statements requires us to make judgments, estimates, and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, as well as the reported revenue and expenses during the reporting periods. We continually evaluate our judgments, estimates and assumptions. We base our estimates on the terms of underlying agreements, our expected course of development, historical experience and other factors we believe are reasonable based on the circumstances, the results of which form our management’s basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates.

The amounts of assets and liabilities reported in our consolidated balance sheets and the amounts reported in our consolidated statements of comprehensive income are affected by estimates and assumptions, which are used for, but not limited to, the accounting for revenue recognition (including adjustments for government rebates), valuation of intangible assets and stock-based compensation. The accounting policies described below are not intended to be a comprehensive list of all of our accounting policies but represent the accounting estimates which involve a significant level of estimation uncertainty and have had or are reasonably likely to have a material impact on our financial condition or results of operations. In many cases, the accounting treatment of a particular transaction is specifically dictated by U.S. GAAP. There are also areas in which our management’s judgment in selecting any available alternative would not produce a materially different result. Our consolidated financial statements and the notes thereto included elsewhere in this report contain accounting policies and other disclosures as required by U.S. GAAP.

Revenue Recognition.

Revenue from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established. Components of variable consideration include trade discounts and allowances, product returns, provider chargebacks and discounts, government rebates, and other incentives, such as voluntary patient assistance, and other allowances that are offered within contracts with our customer, payors, and other indirect customers relating to the sale of our products. These reserves are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific 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 based on the terms of the respective underlying contracts.

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 the contract will not occur in a future period. Our analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates as of December 31, 2025, 2024 and 2023 and, therefore, the transaction price was not reduced further during the years ended December 31, 2025, 2024 and 2023. 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. Refer to Note 2, “Basis of Presentation and Significant Accounting Policies,” in the consolidated financial statements included in this report for further details on revenue recognition.

Valuation of Intangible Assets.

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We have acquired and continue to acquire significant intangible assets that we record at fair value at the acquisition date. Transactions involving the purchase or sale of intangible assets are usually based on a discounted cash flow analysis. The discounted cash flow model requires assumptions about the timing and amount of future net cash flows, risk, cost of capital and market participants. Each of these factors can significantly affect the value of the intangible asset. We engage independent valuation experts who review our critical assumptions and calculations for acquisitions of significant intangibles. We review intangible assets with finite lives for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Examples of events or changes in circumstances that may be indicative of impairment include events that result in significant decline in net selling price and/or significant loss of market share. If indicators of impairment exist, an impairment test is performed to assess the recoverability of the affected assets by determining whether the carrying amount of such assets exceeds the undiscounted expected future cash flows. If the affected assets are deemed not recoverable, we would estimate the fair value of the assets and record an impairment loss. Where cash flows cannot be identified for an individual asset, the review is applied at the lowest group level for which cash flows are identifiable.

Stock-Based Compensation.

We recognize stock-based compensation for the fair value of all share-based payments, including grants of stock options and restricted stock units. For stock options, we use the Black-Scholes option valuation model to determine the fair value of stock options on the date of grant. This model derives the fair value of stock options based on certain assumptions related to expected stock price volatility, expected option life, risk-free interest rate and dividend yield. Expected volatility is based on reviews of historical volatility of our common stock. The estimated expected option life is based upon the simplified method. Under this method, the expected option life is presumed to be the mid-point between the vesting date and the end of the contractual term. We will continue to use the simplified method until we have sufficient historical exercise data to estimate the expected life of the options. The risk-free interest rate assumption is based upon the U.S. Treasury yield curve appropriate for the expected life of our stock option awards. For the years ended December 31, 2025 and 2024, the assumptions used were an estimated annual volatility of 49.9% to 52.5% and 54.1% to 61.5%, respectively, expected holding periods of 4.5 to 5.0 years and 4.5 to 5.0 years, respectively, and risk-free interest rates of 3.6% to 4.4% and 3.7% to 4.7%, respectively.

Results of Operations

Years Ended December 31, 2025 and 2024

Revenues.

For the fiscal year ended December 31, 2025, we recognized total revenues of approximately $589.0 million, which included approximately $588.8 million in net product revenue (primarily in the U.S.), compared to total revenues of approximately $491.7 million, which included approximately $489.3 million in net product revenue (primarily in the U.S.), for the fiscal year ended December 31, 2024.

FIRDAPSE® net product revenue was approximately $358.4 million for the fiscal year ended December 31, 2025, compared to approximately $306.0 million for the fiscal year ended December 31, 2024.

AGAMREE® net product revenue was approximately $117.1 million for the fiscal year ended December 31, 2025, compared to approximately $46.0 million for the period between March 13, 2024 (date of commercial launch) and December 31, 2024.

FYCOMPA® net product revenue was approximately $113.3 million for the fiscal year ended December 31, 2025, compared to approximately $137.3 million for the fiscal year ended December 31, 2024.

The increase of approximately $99.5 million in net product revenue when comparing the fiscal years ended December 31, 2025 and 2024 was primarily due to the commercialization of AGAMREE® in March 2024 and related product sales. Additionally, FIRDAPSE® net product revenue increased by approximately $52.3 million or 17.1% from the fiscal year ended December 31, 2024 compared to the fiscal year ended December 31, 2025, which was primarily driven by an increase in sales volumes.

Further, FYCOMPA® net product revenue decreased by approximately $23.9 million or 17.4%, respectively, from the fiscal year ended December 31, 2024 compared to the fiscal year ended December 31, 2025, due to the generic entry following the loss of exclusivity in 2025. We expect that net product revenue for FYCOMPA® will likely continue to decrease in the future since generic competitors entered the market following the expiration of the '571 patent on May 23, 2025.

For the fiscal year ended December 31, 2025, we recognized approximately $0.2 million in license and other revenue, which consisted of royalties. For the fiscal year ended December 31, 2024, we recognized approximately $2.4 million in license and other revenue, which consisted primarily of a milestone payment of $2.1 million earned upon DyDo receiving product approval to commercialize FIRDAPSE® for the treatment of patients with LEMS in Japan.

Cost of Sales.

Cost of sales was approximately $87.3 million for the fiscal year ended December 31, 2025, compared to approximately $68.8 million for the fiscal year ended December 31, 2024. Cost of sales in all periods consisted principally of royalty payments,

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which are based on net revenue as defined in the applicable license agreements. For FIRDAPSE®, royalties are payable on the terms set forth below in Liquidity and Capital Resources—Contractual Obligations and Arrangements, and increase by 3% when net sales (as defined in the applicable license agreement) exceed $100 million in any calendar year. Cost of sales for FYCOMPA® for the fiscal year ended December 31, 2025 consisted of product costs and excludes the amortization of the FYCOMPA® intangible assets. Cost of sales for AGAMREE® for the fiscal year ended December 31, 2025 consisted of royalties payable on the terms set forth below in Liquidity and Capital Resources—Contractual Obligations and Arrangements, product costs and excludes the amortization of the AGAMREE® intangible asset. Royalties on sales of AGAMREE® in future years may increase as a percentage of net sales exceed certain amounts of net revenues over $100 million. See Note 13 of the "Notes to Consolidated Financial Statements" included elsewhere in this report.

Amortization of Intangible Assets.

Amortization of intangible assets was approximately $37.5 million for the fiscal year ended December 31, 2025 compared to $37.4 million for fiscal year ended December 31, 2024. Amortization of intangible assets consists of the amortization of the FYCOMPA® rights, which are amortized using the straight-line method over its estimated useful life of 5 years, the RUZURGI® rights, which are amortized using the straight-line method over its estimated useful life of 14.5 years and the AGAMREE® rights, which are amortized using the straight-line method over its estimated useful life of 10.5 years.

Each fiscal quarter, we review the value of our intangible assets to determine if they are impaired. If we determine one or more of our intangible assets are impaired during a future period we would record a charge in the amount of that impairment.

Research and Development Expenses.

Research and development expenses for the years ended December 31, 2025 and 2024 were approximately $12.7 million and $12.6 million, respectively, and represented approximately 4% of total operating costs and expenses. Research and development expenses for the years ended December 31, 2025 and 2024 were as follows (in thousands):

For the years ended December 31,

Change

2025

2024

$

%

Salary and benefit expense

$

4,567

$

3,905

662

17.0

Employee stock-based compensation

2,205

1,779

426

23.9

Research and clinical trial expense

4,855

5,813

(958

)

(16.5

)

Additional research and development expense

1,082

1,151

(69

)

(6.0

)

Total research and development expenses

$

12,709

$

12,648

61

0.5

Research and development expenses remained relatively consistent during the fiscal year ended December 31, 2025 when compared to the same period in 2024. During the fiscal year ended December 31, 2025, research and development expenses consisted of costs for company-sponsored research and development activities, support for selected investigator-sponsored research, and costs for development activities supporting our commercial products. Stock-based compensation expense includes a charge related to the retirement of a former executive officer, recorded during the second quarter of 2025, upon lapse of the applicable revocation period under the separation agreement with this former executive.

We expect that research and development activities may become more significant in the future if we seek to execute on the development of additional indications for FIRDAPSE® and AGAMREE® and on our portfolio expansion efforts.

Selling, General and Administrative Expenses.

Selling, general and administrative expenses for the years ended December 31, 2025 and 2024 were approximately $193.8 million and $177.7 million, respectively, and represented approximately 58% and 60% of total operating costs and expenses, respectively. Selling, general and administrative expenses for the years ended December 31, 2025 and 2024 were as follows (in thousands):

For the years ended December 31,

Change

2025

2024

$

%

Selling

$

122,240

$

111,344

10,896

9.8

General and administrative

48,938

45,924

3,014

6.6

Employee stock-based compensation

22,573

20,472

2,101

10.3

Total selling, general and administrative expenses

$

193,751

$

177,740

16,011

9.0

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For the fiscal year ended December 31, 2025, selling, general and administrative expenses increased by approximately $16.0 million when compared to the same period in 2024. The increases in selling and general and administrative expenses were primarily attributable to an increase in employee compensation related to annual merit increases and an increase in headcount. Further, general and administrative expenses increased due to consulting fees related to multiple business initiatives, including business development activities, which were offset due to decreases in contributions to 501(c)(3) organizations supporting patient assistance programs.

We expect that selling, general, and administrative expenses will continue to be substantial in future periods as we continue to sell FIRDAPSE® and AGAMREE® and as we take other steps in an effort to continue to expand our business, offset in part by reduced selling, general, and administrative expenses as more generic FYCOMPA® becomes available in the marketplace.

Stock-Based Compensation.

Total stock-based compensation for the years ended December 31, 2025 and 2024 was $24.8 million and $22.3 million, respectively. During the years ended December 31, 2025 and 2024, grants were principally for stock options and restricted stock units related to year-end bonus awards and grants to new employees.

Other Income, Net.

We reported other income, net in all periods, primarily relating to interest on our investment of our cash and cash equivalents of approximately $25.7 million and $21.1 million for the fiscal years ended December 31, 2025 and 2024, respectively. The increase in other income, net for the fiscal year ended December 31, 2025 when compared to the same period in 2024 was primarily due to higher invested balances and an increase in the fair value of our investment in Santhera. Since Santhera’s shares are traded on the SIX Swiss Exchange, they have a readily determinable fair value, and as a result the investment is measured quarterly, at fair value, with changes reported in other income, net.

The components of other income, net were as follows (in thousands):

For the years ended December 31,

2025

2024

Interest income, net

$

24,765

$

16,064

Net gains recognized during the period on equity

   securities

972

5,075

Total other income, net

$

25,737

$

21,139

Income Taxes.

Our effective income tax rate was approximately 24.4% and 24.2% for fiscal years ended December 31, 2025 and 2024, respectively. Differences in our effective tax and the statutory federal income tax of 21% are driven by state income taxes and anticipated annual permanent differences offset by equity compensation deductions. Our effective tax rate is affected by many factors, including the number of stock options exercised in any period, and our effective tax rate is likely to fluctuate in future periods (and may be higher than it was in the 2025 fiscal year).

We had no material uncertain tax positions as of December 31, 2025 and 2024.

Net Income.

Our net income was approximately $214.3 million in the year ended December 31, 2025 ($1.75 per basic and $1.68 per diluted share) as compared to approximately $163.9 million in the year ended December 31, 2024 ($1.38 per basic and $1.31 per diluted share).

Years Ended December 31, 2024 and 2023

The information comparing results of operations for the year ended 2024 compared to 2023 was included in our Annual Report on Form 10-K for 2024 filed with the SEC on February 26, 2025.

Liquidity and Capital Resources

Since our inception, we have financed our operations primarily through revenues from product sales and multiple offerings of our securities. At December 31, 2025 we had cash and cash equivalents aggregating $709.2 million and working capital of $746.9 million. At December 31, 2024 we had cash and cash equivalents aggregating $517.6 million and working capital of $502.9 million. At December 31, 2025, substantially all of our cash and cash equivalents were deposited with two financial institutions, and such balances were in excess of federally insured limits. Further, as of such date, substantially all such funds were invested in money market accounts.

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On September 8, 2023, we filed a shelf registration statement with the SEC to sell up to $500 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the 2023 Shelf Registration Statement). The 2023 Shelf Registration Statement (file no. 333-274427) became effective upon filing. On January 9, 2024, we completed a public offering of 10 million shares of our common stock under the 2023 Shelf Registration Statement, raising net proceeds of approximately $140.7 million.

Based on our current financial condition, including our profitability, cash flows generated from operations and forecasts of available cash, absent the use of cash to acquire potential business development opportunities, we believe that we have sufficient funds to support our operations for at least the next 12 months from the date of this report. There can be no assurance that we will remain profitable or that we will be able to obtain any additional funding that we may require in the future.

In the future, we may require additional working capital to support our operations depending on our future success with FIRDAPSE®, AGAMREE®, and FYCOMPA® sales, or the products we may acquire and continue to develop and whether our results continue to be profitable and cash flow positive. We may also need to raise additional capital to fund product acquisitions that are valued at more than our available cash. There can be no assurance as to the amount of any such funding that will be required for these purposes or whether any such funding will be available to us if and when it is required.

In that regard, our future funding requirements will depend on many factors, including:

•
the cost of diligence in seeking potential acquisitions and of the completion of such acquisitions, if any future acquisitions occur;

•
future clinical trial results;

•
the scope, rate of progress and cost of our clinical trials and other product development activities;

•
the terms and timing of any collaborative, licensing and other arrangements that we may establish;

•
the cost and timing of regulatory approvals;

•
the cost and delays in product development as a result of any changes in regulatory oversight applicable to our products;

•
the amount of net revenues that we report from sales of FIRDAPSE®, AGAMREE®, and FYCOMPA®;

•
the effect of competition and market developments;

•
the cost of filing and potentially prosecuting, defending and enforcing any patent claims and other intellectual property rights; and

•
the extent to which we acquire or invest in other products and the size of those investments.

We may raise additional funds through public or private equity offerings, debt financings, corporate collaborations or other means. We also may seek governmental grants for a portion of the required funding for our clinical trials and preclinical trials. We may further seek to raise capital to fund additional product development efforts or product acquisitions, even if we have sufficient funds for our planned operations. Any sale by us of additional equity or convertible debt securities could result in dilution to our stockholders. There can be no assurance that any such required additional funding will be available to us at all or available on terms acceptable to us.

Cash Flows.

Net cash provided by operating activities was $208.7 million and $239.8 million, respectively, for the years ended December 31, 2025 and 2024. During the year ended December 31, 2025, net cash provided by operating activities was primarily attributable to our net income of $214.3 million, an increase of $24.2 million in accrued expenses and other liabilities, $24.8 million in stock-based compensation and $37.9 million in amortization of intangible assets and depreciation. This was partially offset by increases of $61.0 million in accounts receivable, net, $18.1 million in inventory, net and $0.2 million in prepaid expenses and other current assets, decreases of $5.4 million in accounts payable and $0.4 million in operating lease liability, $6.8 million in deferred taxes and $0.6 million in non-cash expenses. The increase in accounts receivable, net, primarily relates to a change in the payment terms resulting from the renegotiation of a contract between us and a customer. Under the revisions to the contract with this customer, among other changes, we are now paying reduced fees to the customer (which are recorded as a reduction in gross-to-net expenses), but the customer is paying amounts due on its obligations to us on a monthly basis rather than a semi-monthly basis (as required under the previous contract terms). As a result of this change, the payment of approximately $27.0 million that we would have received, based upon the previous due date, at the end of December 2025 under the previous contract terms was received on January 2, 2026.

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During the year ended December 31, 2024, net cash provided by operating activities was primarily attributable to our net income of $163.9 million, increases of $1.8 million in accounts payable and $53.8 million in accrued expenses and other liabilities, $22.3 million in stock-based compensation and $37.8 million in amortization of intangible assets and depreciation. This was partially offset by increases of $12.0 million in accounts receivable, net, $3.9 million in inventory, net and $8.5 million in prepaid expenses and other current assets, a decrease of $0.4 million in operating lease liability, $9.4 million in deferred taxes and $5.6 million in non-cash expenses.

Net cash used in investing activities during the year ended December 31, 2025 was $0.1 million and consisted of purchases of property and equipment. Net cash used in investing activities during the year ended December 31, 2024 was $0.6 million and consisted of purchases of property and equipment.

Net cash used in financing activities during the year ended December 31, 2025 was $17.0 million, consisting primarily of repurchases of common stock, partially offset by proceeds from the exercise of stock options. Net cash provided by financing activities during the year ended December 31, 2024 was $140.7 million, consisting primarily of proceeds from the issuance of common stock.

Contractual Obligations and Arrangements.

We have entered into the following contractual arrangements with respect to sales of FIRDAPSE®:

•
Payments due under our license agreement for FIRDAPSE®. The following details the royalties under our license agreement:

•
Royalties to our licensor for seven years from the first commercial sale of FIRDAPSE® equal to 7% of net sales (as defined in the FIRDAPSE® License Agreement) in each country for any calendar year for sales up to $100 million, with the rate increasing to 10% of net sales for any total net sales in excess of $100 million in North America; and

•
Royalties to the third-party licensor of the rights sublicensed to us for seven years from the approval of the U.S. NDA for FIRDAPSE® at 7% of U.S. net sales (as defined in the license agreement between BioMarin (since transferred to SERB S.A.) and the third-party licensor) in any calendar year and after that 7th anniversary of the U.S. approval, royalties at 3.5% of U.S. net sales in any calendar year until the earlier of the 12th anniversary of the U.S. approval or the entry of a U.S. generic competitor. All royalty obligations to the third-party licensor for non-U.S. sales have concluded.

Further, we will pay royalties to our licensor on net sales in Japan equal to a similar percentage to the royalties that we are currently paying for non-U.S. sales under our original FIRDAPSE® License Agreement for North America.

For the years ended December 31, 2025 and 2024, we recognized an aggregate of approximately $56.2 million and $47.3 million, respectively, of royalties payable under these license agreements, which is included in cost of sales in the accompanying consolidated statements of operations and comprehensive income.

•
Payments due to Jacobus. In connection with our July 2022 settlement with Jacobus, we agreed to pay the following consideration to Jacobus:

•
$30 million of cash, of which $10 million was paid at the closing of the settlement on July 11, 2022, $10 million was paid on the first anniversary of closing and $10 million was paid on the second anniversary of closing; and

•
An annual royalty on Catalyst’s net sales (as defined in the License and Asset Purchase Agreement between Catalyst and Jacobus) of amifampridine products in the U.S. equal to: (a) for calendar years 2022 through 2025, 1.5% (with a minimum annual royalty of $3.0 million per year), and (b) for calendar years 2026 through the expiration of the last to expire of Catalyst’s FIRDAPSE® patents in the U.S., 2.5% (with a minimum annual royalty of $5 million per year); provided, however, that the royalty rate may be reduced and the minimum annual royalty may be eliminated under certain circumstances.

•
Summary of changes to royalty obligations related to FIRDAPSE® subsequent to the balance sheet date:

•
On January 25, 2026, we completed seven years from the date of first commercial sale of FIRDAPSE® in the U.S. On that date, the royalty on net U.S. sales that we previously paid to our immediate licensor at a tiered rate of 7-10% of net U.S. sales of FIRDAPSE® expired. Also, on January 1, 2026, as part of our acquisition and license agreement regarding RUZURGI® with Jacobus, the royalty rate we pay Jacobus on net U.S. sales of FIRDAPSE® and RUZURGI® increased from 1.5% to 2.5%. In addition to these two changes, there was also another change in FIRDAPSE® royalties owed by us on net U.S. sales that occurred in November 2025. On November 28, 2025, due to seven years passing from the date of the FDA approval of FIRDAPSE®, the

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royalty on net U.S. sales of FIRDAPSE® that we owe to our immediate licensor to satisfy their royalty obligations to their third-party licensor decreased from 7% of net U.S. sales to 3.5%. As a result, the overall royalty rate beginning on January 26, 2026, that we will pay to our upstream licensors for net U.S. sales of FIRDAPSE® will be 6%, which is down from a previous maximum rate of 18.5%.

For the years ended December 31, 2025 and 2024, we recognized an aggregate of approximately $5.3 million and $4.4 million, respectively, of royalties payable to Jacobus.

We have entered into the following contractual arrangements with respect to sales of FYCOMPA®:

•
Payments due under our asset purchase agreement for FYCOMPA®. In connection with our asset purchase agreement with Eisai Co., Ltd. (Eisai), we agreed to pay the following consideration to Eisai:

•
We paid at closing a $160 million upfront cash payment, plus $1.6 million for reimbursement of certain prepayments.

•
Royalties commencing on the expiration of the last patent for the product for each calendar year during the royalty term equal to 12% on net sales greater than $10 million and less than $100 million, 17% on net sales of greater than $100 million and less than $125 million and 22% on net sales greater than $125 million prior to the date of generic entry. Upon the entry of generic competition, these royalties will be reduced to 6% on net sales greater than $10 million and less than $100 million, 8.5% on net sales of greater than $100 million and less than $125 million and 11% on net sales greater than $125 million.

•
Concurrently with the acquisition, the parties entered into two related agreements: (i) a short-term TSA for commercial and manufacturing services (to which transition services ended on December 31, 2023) and (ii) a long-term Supply Agreement for the manufacturing of FYCOMPA®. Under the TSA, Eisai provided certain commercial and manufacturing services to the Company for a transition period following the closing of the acquisition. Further, under the Supply Agreement, Eisai will manufacture FYCOMPA® for the Company for a period of seven years (or such longer period as is set forth in the Supply Agreement) following the closing of the acquisition.

We have entered into the following contractual arrangements with respect to AGAMREE®:

•
Payments due under our license agreement for AGAMREE®. In connection with our acquisition from Santhera:

•
At closing we paid a $75 million initial cash payment.

•
In the fourth quarter of 2023, following regulatory approval of Santhera’s NDA for AGAMREE® by the FDA, we paid a regulatory milestone payment of $36 million. We are also obligated to pay additional regulatory milestone payments upon regulatory approval by the FDA in the U.S. of an NDA for the product for the first, second, and third additional indications in the amounts of $50 million, $45 million, and $45 million, respectively.

•
We may be obligated to pay Santhera sales-based milestones of up to $105 million, which includes a sales-based milestone payment of up to $12.5 million upon achievement of revenues in the calendar year in which revenues exceed $100 million (which was achieved in the fourth quarter of 2025), and pay royalties if the applicable amount of net sales of all products in the territory in a single calendar year fall within the range of one or more of the net sales threshold levels set forth in the AGAMREE® License Agreement.

•
At signing, we were obligated to purchase all of our finished goods requirements for products solely from Santhera at a set supply price until January 1, 2026, but the parties agreed upon an amendment to the license agreement that allowed us to start the process for creating our own supply chain to manufacture Agamree earlier and we expect to complete that process by the end of 2026.

•
Simultaneously with entering into the license agreement, we made a strategic equity investment into Santhera by acquiring 1,414,688 of Santhera’s ordinary shares (representing approximately 11.26% of Santhera’s outstanding ordinary shares immediately following the transaction) at an investment price of CHF 9.477 per share (corresponding to a mutually agreed volume-weighted average price prior to signing), with the approximately $15.7 million USD in equity investment proceeds, inclusive of the approximately $13.5 million USD fair value of the investment in Santhera and approximately $2.2 million USD of transaction costs included in acquired in-process research and development, to be used by Santhera for Phase IV studies in DMD and further development of additional indications for AGAMREE®.

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For the years ended December 31, 2025 and 2024, we recognized an aggregate of approximately $13.8 million and $5.1 million, respectively, of royalties payable under this license agreement, which is included in cost of sales in the accompanying consolidated statements of operations and comprehensive income.

We also have entered into the following contractual arrangements:

•
Purchase commitment. We have entered into a purchase commitment for FIRDAPSE® with a contract manufacturing organization for approximately $0.5 million per year. The agreement expires in December 2026. We also entered into a purchase commitment for AGAMREE® with a contract manufacturing organization in May 2025 for approximately $5.4 million, which we have fulfilled.

•
Lease for office space. We operate our business in leased office space in Coral Gables, Florida. We lease approximately 10,700 square feet of office space and we pay annual rent of approximately $0.5 million.

Off-Balance Sheet Arrangements.

We do not have any off-balance sheet arrangements as such term is defined in rules promulgated by the SEC.

Caution Concerning Forward-Looking Statements

This report contains “forward-looking statements”, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding our expectations, beliefs, plans or objectives for future operations and anticipated results of operations. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, “believes”, “anticipates”, “proposes”, “plans”, “expects”, “intends”, “may”, and other similar expressions are intended to identify forward-looking statements. Such statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other achievements to be materially different from any future results, performances or achievements expressed or implied by such forward-looking statements. Factors that might cause such differences include, but are not limited to, those discussed in the section entitled “Item 1A – Risk Factors.”

•
Whether we will be able to continue to successfully market and sell FIRDAPSE® and AGAMREE®, and continue to sell FYCOMPA® while maintaining full compliance with applicable federal and state laws, rules and regulations;

•
Whether we will be able to continue to attract and retain the qualified personnel necessary to run our business;

•
Whether we can continue to market our drug products on a profitable and cash flow positive basis;

•
Whether any revenue or earnings guidance that we provide to the investment community will turn out to be accurate;

•
Whether we will prevail in our currently pending Paragraph IV litigation with Hetero USA, Inc. regarding our FIRDAPSE® patents that expire in 2032, 2034, and 2037, which matter is currently scheduled to go to trial starting on March 23, 2026;

•
Whether our estimates of the size of the market for FIRDAPSE® for the treatment of LEMS will prove to be accurate, and whether we can continue to increase FIRDAPSE® net product revenues as we have done in past periods;

•
Whether the daily dose of FIRDAPSE® taken by patients changes over time and how that affects our net product revenues for FIRDAPSE® in future periods;

•
Whether we will continue to be able to locate LEMS patients who are undiagnosed or are misdiagnosed with another disease, including LEMS patients who also have small cell lung cancer;

•
Whether the addition of FIRDAPSE® to the NCCN Clinical Practice Guidelines for small cell lung cancer results in an increase in patients being treated with FIRDAPSE®;

•
Whether patients will discontinue from the use of our products at rates that are higher than historically experienced or higher than we forecast;

•
Whether new patients for our existing and future drug products can be successfully titrated to stable therapy.

•
Whether we will be able to demonstrate, to the satisfaction of the FDA and third-party payors, that AGAMREE® offers advantages compared to other corticosteroids or competitor’s products;

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•
Whether DMD patients transitioning to current or future approved gene therapy treatments will delay initiating use of AGAMREE® while waiting for access to such gene therapy, or stop their AGAMREE® therapy during the course of their gene therapy treatment;

•
Whether steroids will continue to be the foundational standard of care for the treatment of DMD as new DMD drugs are approved for commercialization in the future;

•
Whether we will be able to continue to successfully sell FYCOMPA® now that generic competition for FYCOMPA® tablets and oral suspension have entered the market;

•
Whether reduced revenue resulting from generic FYCOMPA® entering the market will require us to impair all or a portion of our intangible asset for FYCOMPA®;

•
The extent to which payors will continue to provide coverage and reimburse for our products at the price that we charge for our products;

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The ability of our third-party suppliers and contract manufacturers to continue to supply sufficient product to meet our customers’ needs in a timely manner;

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The impact on our profits and cash flow of adverse changes in reimbursement and coverage policies or regulations from government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators, or the impact of pricing pressures enacted by industry organizations, the federal government or the government of any state, including as a result of increased scrutiny over pharmaceutical pricing or otherwise;

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Changes in the healthcare industry and the effect of political pressure from and actions by the current Administration, Congress and/or medical professionals seeking to reduce prescription drug costs, and changes to the healthcare industry occasioned by any future changes in laws relating to the pricing of drug products, including changes made in the Inflation Reduction Act of 2022, changes (if any) to be made by the current Administration (including the possibility of seeking to impose Most Favored Nation pricing on drug companies or to impose a 100% tariff on branded or patented pharmaceutical products unless a company is "building" a manufacturing plant in the United States) and/or the current Congressional administrations, changes to the review and approval process at the FDA, imposing tariffs on imported product, or changes in the healthcare industry generally;

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The potential impact of tariffs on our cost of sales for products that are manufactured, in whole or in part, outside of the U.S.;

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The potential impact on our business of “most favorite nation”, or Most Favored Nation pricing, such as proposed in the GUARD and GLOBE Medicare demonstration projects, on what we will realize from the sale of our drug products if Most Favored Nation pricing becomes applicable to even a portion of our drug products;

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The ability of our third-party suppliers and contract manufacturers to maintain compliance with current Good Manufacturing Practices;

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The ability of those third parties that distribute our products to maintain compliance with applicable law;

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Our ability to maintain compliance with applicable rules relating to our patient assistance programs for our products;

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The scope and strength of our intellectual property and the outcome of challenges to our intellectual property, and, conversely, whether any third-party intellectual property presents unanticipated obstacles for FIRDAPSE® or AGAMREE®;

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Whether there will be a post-closing review by antitrust regulators of our previous or future Paragraph IV patent settlements or our previous acquisition transactions, and the outcome of any such reviews if they were to occur;

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Whether we will be able to acquire additional drug products under development, complete development required to commercialize such products, and thereafter, if such products are approved for commercialization, successfully market such products;

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Whether the FDA will approve generic versions of FIRDAPSE® following the expiration of our orphan drug exclusivity on November 26, 2025, subject to any pending 30-month stays of approval as required under the Drug Price Competition and Patent Term Restoration Act of 1984;

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Whether our patents will be sufficient to prevent generic competition for AGAMREE® after our orphan drug exclusivity for this product expires;

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Whether our clinical studies of AGAMREE® and our SUMMIT registry study of DMD patients being treated with AGAMREE®, will be successful and the impact, if any, of the results of these studies on our business;

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Assuming we prevail in our currently ongoing patent litigation with Hetero, whether we are able to successfully develop additional indications for FIRDAPSE® and obtain the ability to commercialize FIRDAPSE® for these additional indications;

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Whether we and Santhera Pharmaceuticals Holding AG can successfully develop additional indications for AGAMREE®, and obtain the ability to commercialize AGAMREE® for these additional indications.

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The state of the economy generally;

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The impact on our business of a prolonged U.S. government shutdown;

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The scope, rate of progress and expense of our clinical trials and studies, pre-clinical studies, proof-of-concept studies, and our other drug development activities, and whether any trials and studies we undertake will be successful;

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Our ability to complete any clinical trials and studies that we may undertake on a timely basis and within the budgets we establish for such trials and studies;

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Whether FIRDAPSE® and AGAMREE® will be successfully commercialized in Canada on a profitable basis through KYE Pharmaceuticals, Inc., our sublicensee for the products in Canada;

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Whether FIRDAPSE® will be successfully commercialized in Japan on a profitable basis by DyDo Pharma, Inc., our sublicensee for the product in Japan;

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The impact on sales of FIRDAPSE® in the U.S. if an amifampridine product is purchased in Canada for use in the U.S.;

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Whether any efforts we undertake to expand the reach of FIRDAPSE® into other global regions will be successful;

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System failures or security or data breaches due to cyber-attacks, or cyber intrusions, including ransomware, phishing attacks and other malicious intrusions whether it occurs directly to us or indirectly through third parties; and

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Our ability to enhance our systems, processes, and procedures to appropriately support the growing complexity and scale of our business.

Our current plans and objectives are based on assumptions relating to the continued sale of FIRDAPSE®, AGAMREE® and FYCOMPA® and on our plans to seek to acquire or in-license additional drug products. Although we believe that our assumptions are reasonable, any of our assumptions could prove inaccurate. Considering the significant uncertainties inherent in the forward-looking statements we have made herein, which reflect our views only as of the date of this report, you should not place undue reliance upon such statements. We undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
