EyePoint, Inc. (EYPT) Business

ITEM 1. BUSINESS

Overview

EyePoint, Inc. (Nasdaq: EYPT) is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative therapeutics to improve the lives of patients with serious retinal diseases. The Company's pipeline leverages its proprietary bioerodible Durasert E™ technology (Durasert E™) for sustained intraocular drug delivery. The Company’s lead product candidate, DURAVYU™1, is an investigational sustained delivery treatment for vascular endothelial growth factor (VEGF) mediated retinal diseases combining vorolanib, a selective and patent-protected tyrosine kinase inhibitor (TKI) with the Company's bioerodible Durasert E™ drug delivery technology. DURAVYU is currently being evaluated in Phase 3 pivotal trials (LUGANO and LUCIA) for wet age-related macular degeneration (wet AMD) and in Phase 3 clinical trials (COMO and CAPRI) for diabetic macular edema (DME). Additional pipeline programs include EYP-2301, razuprotafib, a TIE-2 agonist, formulated in Durasert E™ to potentially improve outcomes in serious retinal diseases. EyePoint is headquartered in Watertown, Massachusetts with a commercial manufacturing facility in Northbridge, Massachusetts.

DURAVYU brings a potential new multi-mechanism of action paradigm for the treatment of retinal diseases as vorolanib, the active drug in DRUAVYU™, acts through intracellular inhibition of all VEGF receptors, platelet-derived growth factor (PDGF) and pro-inflammatory interleukin 6 (IL-6)/JAK1 signaling for at least six months. Vorolanib has also demonstrated neuroprotection in an in-vivo model of retinal detachment.

DURAVYU is currently in Phase 3 clinical trials for the potential treatment of wet AMD and DME, the two largest retinal disease markets. Enrollment in the pivotal Phase 3 clinical trials for wet AMD is complete with data expected beginning in mid-2026. The first patient was dosed in the Phase 3 DME program in February 2026.

Additional pipeline programs include EYP-2301, razuprotafib, a TIE-2 agonist, formulated in Durasert E™ to potentially improve outcomes in serious retinal diseases.

The proven Durasert® drug delivery technology (Durasert®) has been safely administered to thousands of patient eyes across four products approved by the U.S. Food and Drug Administration (FDA). Durasert® enables the development of a solid drug cylinder that is delivered through a standard intravitreal (IVT) injection in the physician office, similar to current standard practice with FDA approved IVT treatments. A Durasert® insert is designed to provide consistent” sustained release of drug over a desired time period and can generally be tailored for each drug and disease indication. Durasert® products have been developed in both non-erodible and Durasert E™ bioerodible formulations.

In wet AMD, DURAVYU is currently being evaluated in the Phase 3 LUGANO and LUCIA clinical trials. The Phase 3 program was developed with feedback from both the FDA and European Medicines Agency (EMA). We initiated the pivotal trials leveraging learnings from our robust DAVIO and DAVIO 2 clinical trials, which achieved primary and secondary endpoints.

Key characteristics of the Phase 3 wet AMD pivotal program are as follows:

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Randomized, double-masked, aflibercept controlled, non-inferiority trials assessing the efficacy and safety of DURAVYU 2.7mg in patients with active wet AMD.

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Trials include approximately 75% treatment-naïve and 25% previously treated patients.

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The LUGANO trial enrolled 432 patients and the LUCIA trial enrolled 475 patients who were randomly assigned, 1:1, to a DURAVYU 2.7mg arm or an on-label aflibercept control arm. Randomization occurred on Day 1.

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Patients in the DURAVYU 2.7mg treatment arm will be re-dosed every six months for a total of four doses over the two-year trial.

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The primary endpoint is the difference in mean change in best corrected visual acuity (BCVA) from Day 1 to Week 52 and 56 (blended) versus aflibercept control. Secondary endpoints include safety, reduction in treatment burden, percentage of eyes free of supplemental aflibercept injections, and anatomical stability.

If the wet AMD Phase 3 clinical program is successful, we plan on filing a New Drug Application (NDA) with the U.S Food and Drug Administration (FDA) with the initial 56-week efficacy and safety data. A supplemental NDA (sNDA) is planned for the Year 2 safety data only, once complete.

1. DURAVYU™ has been conditionally accepted by the FDA as the proprietary name for EYP-1901. DURAVYU is an investigational product; it has not been approved by the FDA. FDA approval and the timeline for potential approval is uncertain.

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In DME, DURAVYU is currently being evaluated in global Phase 3 clinical trials (COMO and CAPRI). We initiated the pivotal trials leveraging learnings from the positive Phase 2 VERONA trial, which achieved primary and secondary endpoints. The Phase 3 program was developed with feedback from both the FDA and European Medicines Agency (EMA).

Key characteristics of the Phase 3 DME pivotal program are as follows:

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Randomized, double-masked, aflibercept controlled, non-inferiority trials assessing the efficacy and safety of DURAVYU 2.7mg in patients with active DME.

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Each trial will enroll approximately 240 patients, including both previously treated and treatment naïve patients, who will be randomly assigned to either a DURAVYU 2.7mg arm or an on-label 2mg aflibercept control arm. Randomization occurs on Day 1.

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Each trial plans to enroll approximately 75% treatment-naïve and 25% previously treated patients.

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Patients in the DURAVYU 2.7mg treatment arm will be re-dosed every six months for a total of four doses over the two-year trial.

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The primary endpoint is the difference in mean change in BCVA from Day 1 to Week 52 and 56 (blended) versus aflibercept control. Secondary endpoints include safety, reduction in treatment burden, percentage of eyes free of supplemental aflibercept injections, and anatomical stability.

DURAVYU Clinical Development and Pipeline Progress – During 2025, we achieved significant clinical and regulatory milestones for DURAVYU, our lead product candidate for the treatment of major retinal diseases, including DME and wet AMD.

Clinical Milestones in DME – On February 2, 2025, we announced that the Phase 2 VERONA clinical trial of DURAVYU in DME met its primary and secondary endpoints. Data at 24 weeks demonstrated meaningful and sustained improvements in visual acuity and anatomical control with a favorable safety profile. Notably, a subgroup analysis of supplement-free patients receiving the DURAVYU 2.7mg dose showed rapid visual improvement (+10.3 letters vs. +3.0 letters for aflibercept control) and significant reduction in CST (117.4 microns vs. 43.7 microns for control) by week 24.

Building on these results, in October 2025, we finalized the design for our pivotal Phase 3 program in DME, with the first patient dosed in the first quarter of 2026.

Pivotal Phase 3 Program in Wet AMD – We made substantial progress in our wet AMD Phase 3 program during 2025. We successfully completed enrollment in our two pivotal Phase 3 clinical trials: the LUGANO trial on May 27, 2025, and the LUCIA trial on July 29, 2025. These trials are designed to evaluate the efficacy and safety of DURAVYU as a long-acting treatment option for patients with wet AMD.

Scientific Advancements and Mechanism of Action (MOA) – In October 2025, we disclosed new preclinical data identifying vorolanib (the active agent in DURAVYU) as a multi-mechanism therapy. In addition to its established inhibition of VEGF, data demonstrated that vorolanib inhibits pro-inflammatory IL-6 signaling through the inhibition of Janus Kinase (JAK) receptors, specifically JAK-1. This dual-action profile—addressing both vascular permeability and inflammatory pathways—further differentiates DURAVYU from current standard-of-care treatments for wet AMD and DME.

Scientific Engagement –Throughout the year, we presented extensive datasets at major medical conferences, including the Association for Research in Vision and Ophthalmology (ARVO) and the Retina World Congress. These presentations included 24-month GLP repeat-dose toxicology studies and treatment burden assessments from the Phase 2 DAVIO 2 trial, which we believe further de-risks our clinical program and reinforces DURAVYU’s potential commercial success.

Pipeline

The following describes the stage of each of our programs:

DURAVYU – vorolanib in Durasert E™

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Wet AMD

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Phase 3 clinical trials (LUGANO/LUCIA) underway with data anticipated beginning in mid-2026

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DME

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Phase 3 global clinical trials (COMO/CAPRI) underway with first patient dosed in February 2026

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EYP-2301 – razuprotafib in Durasert E™

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Preclinical development including toxicity and pharmacokinetic (PK) studies.

Strategy

EyePoint is committed to developing and commercializing innovative therapies to improve the lives of patients with serious retinal diseases. The key elements of our strategy include:

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Advance DURAVYU through Phase 3 clinical development and, if successful, regulatory filings for wet AMD and DME

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Prepare for the potential commercial launch of DURAVYU in the United States in anticipation of successful trial outcomes and FDA approval

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Leverage our new state-of-the-art Northbridge, MA manufacturing facility to support the potential commercial launch of DURAVYU

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Advance DURAVYU into additional retinal disease indications

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Advance EYP-2301 into clinical development for serious retinal diseases

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Expand product pipeline through internal discovery, research collaborations, in-licensing arrangements, or acquisition

The Unmet Need in the Treatment of Retinal Disease – Duration of Action and a New Mechanism of Action (MOA)

Currently available large molecule anti-VEGF ligand blocking therapies for wet AMD and DME are frequently discontinued, due in part to the high treatment burden associated with regular injections. In addition, these anti-VEGF ligand blocking biologics only treat one aspect of disease pathogenesis, with inflammation still playing a key role. An unmet need remains for both comprehensive, durable treatment options and a new MOA to treat retinal exudative diseases like wet AMD and DME.

Inflammation has been identified to play a key role in the worsening of disease control in wet AMD and DME, suggesting the addition of an anti-inflammatory mechanism could potentially improve outcomes in patients with either condition. As an example, up to two thirds of patients still have active DME after anti-VEGF loading doses, and previous data has demonstrated a combination of corticosteroids with anti-VEGF treatment leads to better anatomical disease control compared to anti-VEGF treatment alone.

Vorolanib, the active drug in DURAVYU, acts through intracellular binding of all VEGF receptors thereby blocking all VEGF isoforms, the main driver of the proliferation of blood vessels that are the hallmark of wet AMD and DME. In vitro data showed a reduction in IL-6 activity of more than 50% associated with vorolanib, the active ingredient in DURAVYU, via inhibition of JAK1, suggesting anti-inflammatory properties. Accordingly, DURAVYU brings a potential new multi-mechanism of action paradigm for the treatment of retinal diseases as vorolanib, the active drug in DURAVYU, acts through intracellular inhibition of all VEGF receptors, PGDF and pro-inflammatory IL-6 signaling for at least six months.

Durasert® Technology

Durasert® has been safely administered to thousands of patient eyes across four products approved by the FDA. Durasert® enables the development of a solid drug cylinder that is delivered through a standard IVT injection in the physician office, similar to current standard practice with FDA approved anti-VEGF treatments. A Durasert® insert is designed to provide consistent, sustained release of drug over a desired time period and can generally be tailored for each drug and disease indication. Durasert® products have been developed in both non-erodible and bioerodible Durasert E™ formulations.

The four FDA-approved Durasert® products utilized a non-erodible formulation. In these products, the solid insert was coated with a non-erodible polymer layer and other design aspects to slow the rate and duration of drug release.

DURAVYU leverages a bioerodible formulation of the Durasert® technology, Durasert E™. In this structure, there are no non-erodible polymer layers utilized and the resulting insert consists of bioerodible matrix and the active drug, vorolanib. This formulation allows the solid insert to deliver a higher payload of drug (94%) for sustained delivery until the drug is fully released from the insert. By design, the core matrix maintains the drug release before it fully bio-erodes to avoid free-floating drug particles.

Our Durasert® technology is designed to provide sustained drug delivery for ophthalmic diseases and conditions with the following features:

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Sustained Delivery. The delivery of drugs for predetermined periods of time. We believe that uninterrupted, sustained delivery offers the opportunity to develop products that reduce the need for repeated dosing, thereby improving patient compliance and potential adverse effects from repeated administrations.

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Controlled Release Rate. The release of therapeutics for sustained release at a controlled rate. We believe that this feature allows us to develop products that deliver optimal concentrations of therapeutics over time and minimize variability in dosing during treatment.

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IVT Delivery. The delivery of therapeutics intravitreally can allow the natural barriers of the body to isolate and assist in maintaining appropriate concentrations locally to achieve the maximum therapeutic effect while minimizing unwanted systemic effects.

Our Product Candidates

DURAVYU for wet AMD and DME

DURAVYU is an investigational product deploying vorolanib, a selective and patent protected TKI, that potentially brings a new multi-mechanism of action and treatment paradigm for retinal diseases beyond existing anti-VEGF large molecule ligand blocking therapies. DURAVYU utilizes our bioerodible Durasert E™ technology.

Vorolanib acts through intracellular binding of all VEGF receptors thereby blocking all VEGF isoforms, the main driver of the proliferation of blood vessels that are the hallmark of wet AMD and other retinal diseases and also inhibits PDGF and pro-inflammatory IL-6 signaling. In addition to the safety and efficacy demonstrated in the DAVIO, DAVIO 2 and VERONA clinical trials, vorolanib has also demonstrated neuroprotection in an in-vivo model of retinal detachment.

The inhibition of PDGF by vorolanib may provide antifibrotic benefits and the inhibition of IL-6 signaling, by blocking JAK-1, may provide an anti-inflammatory effect. Prior to in-licensing by the Company, vorolanib was studied in Phase 1 and 2 clinical trials as an orally delivered therapy for the treatment of wet AMD and data from these trials demonstrated a positive clinical signal and no ocular toxicity.

DURAVYU has been evaluated in over 190 patients across Phase 1 and Phase 2 clinical trials with a favorable safety profile and no DURAVYU related local or systemic severe adverse events (SAE’s). The independent Data Safety Monitoring Committee (DSMC) completed its second scheduled review of the ongoing pivotal Phase 3 program evaluating DURAVYU for the treatment of wet AMD in November 2025 and recommended that both the LUGANO and LUCIA trials continue as planned, with no modifications to the protocol.

We have reported positive safety and efficacy data for DURAVYU in our Phase 2 DAVIO clinical trial and fully enrolled the pivotal Phase 3 clinical trials (LUGANO and LUCIA) for wet AMD with over 900 patients recruited across both trials. Data from the Phase 3 program is anticipated to be reported beginning in mid-2026.

We also reported positive 24-week data for DURAVYU in a phase 2 clinical trial (VERONA) for DME and have initiated pivotal Phase 3 clinical trials (COMO and CAPRI) with first patient dosed in February 2026.

DURAVYU for wet AMD - Market Opportunity

Wet AMD occurs when new, abnormal blood vessels grow under the retina. These vessels may leak blood or other fluids, causing scarring of the macula. This form of AMD is less common but much more serious. AMD is one of the major causes of vision loss of the total vision impairment globally.

As the proportion of people in the U.S. age 65 and older grows larger, more people are developing age-related diseases such as AMD. By 2050, the estimated number of people with later stages of AMD such as wet AMD is expected to more than double from 2.07 million to 5.44 million. White Americans are expected to continue to account for the majority of cases. However, Hispanics are expected to account for the greatest rate of increase, with a nearly six-fold rise in the number of expected cases from 2010 to 2050.

Age is the greatest risk factor for developing AMD and individuals aged 50+ are more prone to the disease. Among all AMD patients in the United States, wet AMD accounts for only 10% of cases, yet it alone accounts for 90% of legal blindness.

There are multiple short acting effective and safe treatments for wet AMD available on the market, including large molecule anti-VEGF IVT drugs marketed under the brands names Lucentis, Eylea, Eylea HD, Vabysmo, Beovu, and Avastin (off label). These treatments are injected in a physician’s office either monthly, bi-monthly or in some patients every three to four months, which can cause inconvenience and often leads to reduced compliance and poor outcomes. The branded drug, SUSVIMO™, a port delivery technology for ranibizumab, was approved by the FDA in 2021 for the treatment of wet AMD and requires an initial surgical placement of the port. In February 2025, Susvimo was approved for the treatment of DME.

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Separate published studies using real world data (one study in the U.S. and another that includes Canada, France, Germany, Ireland, Italy, the Netherlands, UK, and Venezuela) indicate that despite initial efficacy, approved wet AMD treatments still result in vision loss over time.

We believe that DURAVYU, if approved as a potential six-month sustained delivery therapy, has the potential to offer wet AMD patients a safe and effective treatment option with a new and complementary multi-MOA to current therapies.

DURAVYU for wet AMD - Clinical Development

In wet AMD, DURAVYU demonstrated positive data from the Phase 1 DAVIO and Phase 2 DAVIO clinical trials with clinically meaningful efficacy data with stable visual acuity and central subfield thickness (CST) and a favorable safety profile. The Phase 1 clinical trial (DAVIO) was a dose escalation trial that enrolled 17 wet AMD patients across four separate doses. The primary endpoint of the trial was safety, and key secondary endpoints were BCVA and CST measured by optical coherence tomography (OCT).

Safety and efficacy data for the Phase 1 DAVIO clinical trial included stable visual acuity (VA) and OCT and a clinically significant reduction in treatment burden of 75% at six months and 73% at 12 months with a median time to supplement of six months. The data also reported that 53% of patients in the trial did not require a supplemental anti-VEGF treatment up-to the six-month visit and 35% of patients did not require a supplemental anti-VEGF treatment up to twelve-months. There were no ocular SAEs reported, no drug-related systemic SAEs reported, and all ocular adverse events (AEs) were ≤ grade 2; the only grade 3 AE was not drug-related.

In July 2022, we updated the results of the Phase 1 DAVIO clinical trial through 12-months reporting continued positive safety and efficacy results. This included a continuation of a clinically significant reduction in treatment burden of 73% at 12 months. The data also reported that 30% of patients in the trial did not require a supplemental anti-VEGF treatment up-to the twelve-month visit.

DAVIO 2 was a multi-center randomized, double-masked controlled Phase 2 clinical trial of DURAVYU in previously treated patients with wet AMD. Originally designed to enroll 144 patients, the trial enrolled 160 patients in total due to strong investigator and patient interest. All enrolled patients were previously treated with a standard-of-care anti-VEGF therapy and were randomly assigned to one of two doses of DURAVYU (approximately 2 mg or 3 mg) or an aflibercept control. DURAVYU is delivered with a single intravitreal injection in the physician's office, similar to current FDA approved anti-VEGF treatments. The primary non-inferiority efficacy endpoint was change in BCVA compared to the aflibercept control, approximately six-months after the DURAVYU injection. Secondary endpoints include safety, reduction in treatment burden, mean change in CST as measured by OCT, the percent of eyes that remain free of supplemental anti-VEGF injections, and number of aflibercept injections in each group.

DAVIO 2 top line results at week 32 indicated:

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Both DURAVYU doses (2mg and 3mg) achieved all primary and secondary endpoints.

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Statistical non-inferiority in change in BCVA (at a confidence interval of 95%) compared to aflibercept control, at weeks 28 and weeks 32 combined. The 2mg and 3mg doses were only -0.3 and -0.4 letters different, respectively, versus on-label aflibercept. The lower limit of the non-inferiority margin is defined as a -4.5 letters by the FDA with 5 letters representing one line on the eye chart.

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Continued positive safety and tolerability profile with no DURAVYU-related ocular or systemic SAEs.

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89% and 85% reduction in treatment burden, respectively, for the 2mg and 3mg DURAVYU doses, when comparing the injections in the six months prior to entry into the study vs. the injections administered during the study following DURAVYU dosing.

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65% and 64% of eyes were supplement free up to six-months, respectively, for the 2mg and 3mg doses of DURAVYU.

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Both DURAVYU doses demonstrated strong anatomic control with OCT difference below 10 microns at week 32 compared to the aflibercept control.

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Patient discontinuation up to week 32 was low at 4% with no DURAVYU related discontinuation.

In the sub-group of patients who were supplement-free up to six months, the DURAVYU groups demonstrated numerical superiority in change in BCVA along with strong anatomic control compared to the aflibercept control group. This result confirms that the positive topline data from the Phase 2 DAVIO 2 trial were driven by DURAVYU and not by study eyes requiring supplemental injection. Visual and anatomical outcomes were not meaningfully influenced by differences in patient baseline BCVA, duration of wet AMD diagnosis, or historical treatment burden. DURAVYU outcomes were consistent and durable in a range of wet AMD patient types.

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In June 2024, we reported positive twelve-month safety and efficacy data from the Phase 2 DAVIO 2 clinical trial evaluating DURAVYU for the treatment of wet AMD including:

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Favorable safety profile – No DURAVYU related ocular or systemic SAEs reported.

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Best Corrected Visual Acuity (BCVA) – Statistically significant visual acuity outcomes with both DURAVYU arms change in visual acuity similar to aflibercept control arm through 12 months after a single injection of DURAVYU.

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Central Subfield Thickness (CST) – Strong anatomical control through 12 months after a single injection of DURAVYU.

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Supplement Free – After a single injection of DURAVYU, approximately half of the treated study eyes were anti-VEGF supplement free, while 22% of the eyes in the aflibercept control arm were administered a supplement despite these control eyes receiving mandated bi-monthly injections through 12 months.

The positive results from the DAVIO 2 clinical trial supported the initiation of the current global Phase 3 clinical trials, LUGANO and LUCIA in wet AMD.

LUGANO and LUCIA are global, randomized, double-masked, aflibercept controlled, non-inferiority Phase 3 trials assessing the efficacy and safety of DURAVYU in patients with active wet AMD including previously treated and treatment-naïve patients. The LUGANO trial enrolled 432 patients and the LUCIA trial enrolled 475 patients globally who were randomly assigned 1:1 to a 2.7mg dose of DURAVYU or an on-label aflibercept control. The LUGANO and LUCIA trials are the only sustained release wet AMD pivotal Phase 3 trials evaluating re-dosing in both trials. Patients in the DURAVYU treatment arm will receive an intravitreal injection of DURAVYU every six months, starting at month two of the trial with a total of four injections over the two-year trial. DURAVYU is delivered via a standard intravitreal injection in the physician's office, similar to current standard practice with FDA approved anti-VEGF treatments. The primary endpoint of the Phase 3 pivotal trials is the average change in BCVA at weeks 52 and 56 versus baseline. Secondary endpoints include safety, reduction in treatment burden, percentage of eyes free of supplemental aflibercept injections and anatomical results as measured by optical coherence tomography (OCT).

In May 2025, we presented multiple datasets at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, demonstrating DURAVYU’s potential real-world application in multiple retinal disease indications and de-risked trial designs that position DURAVYU for potential clinical and commercial success. Presentations included:

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An assessment of the treatment burden in wet AMD treated with DURAVYU versus aflibercept from the Phase 2 DAVIO 2 clinical trial

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Trial design of the global LUGANO and LUCIA pivotal Phase 3 trials in wet AMD

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A 24-month Good Laboratory Practice (GLP) repeat-dose toxicology study of vorolanib intravitreal insert

We successfully completed enrollment in our two pivotal Phase 3 clinical trials: the LUGANO trial on May 27, 2025, and the LUCIA trial on July 29, 2025. Data anticipated beginning in mid-2026.

DURAVYU for Diabetic Macular Edema - Market Opportunity

DME is a complication of diabetic retinopathy (DR), a common finding in diabetic patients. DR is caused by long-term damage to the retina’s small blood vessels. The leakage of fluid into the retina leads to swelling of the central retina, which is called the macula. If left untreated, DME can result in severe visual loss and even blindness. DME can occur at any stage of DR, although it is more likely to occur later with the disease’s progression.

Common signs and symptoms of DME include dark spots like a smudge on glasses or gaps that may appear in the vision, blurred vision, double vision, faded colors, or the affected person may find bright light or glare difficult. The American Academy of Ophthalmology (AAO) estimates that nearly 80% of Type 1 diabetics and 50% of Type 2 diabetics will develop DR after living with diabetes for 15 and 20 years, respectively.

Per the March 3, 2022, Journal of American Medical Association of Ophthalmology, DR is the leading cause of incident blindness in US adults aged 20 to 74 years old and DME can occur with any stage of DR. DR and DME affect 28.5% and 3.8%, respectively, of US adults, 40 years and older, with diabetes.

The most common treatments of DME are anti-VEGF drugs, corticosteroids, and laser photocoagulation. Topical nonsteroidal anti-inflammatory drugs (NSAIDs), in the form of eye drops, are sometimes used either before or after cataract surgery to prevent the development of macular edema. Currently, intravitreal anti-VEGF agents are the preferred first-line treatment for DME.

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DURAVYU for Diabetic Macular Edema - Clinical Development

In DME, DURAVYU demonstrated positive data from the VERONA Phase 2 clinical trial, which was a three-arm trial with two separate doses (low and high) of DURAVYU and an aflibercept control. VERONA evaluated DURAVYU as a potential six-month treatment in previously treated DME patients with active disease. The two DURAVYU doses were administered as a single injection on Day 1 following the aflibercept injection on the same visit.

In February 2025, we reported positive results for the Phase 2 VERONA clinical trial of DURAVYU for DME meeting primary and secondary endpoints.

Phase 2 VERONA results included:

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Both DURAVYU doses (1.34 mg and 2.7mg) met the primary endpoint of extended time to first supplemental injection versus aflibercept control.

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DURAVYU 2.7mg demonstrated an early and sustained improvement in both BCVA and CST as measured by OCT.

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BCVA improved +7.1 letters compared to baseline.

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CST improved 75.9 microns compared to baseline representing 74% more drying in DURAVYU eyes versus aflibercept control.

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Visual and anatomical gains were observed at week 4 demonstrating the immediate bioavailability of DURAVYU.

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73% of eyes in the DURAVYU 2.7mg arm were supplement-free versus 50% in the aflibercept control arm up to week 24 underscoring that the positive efficacy results were driven by treatment with DURAVYU and not supplemental injections.

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Over two-thirds reduction in treatment burden for the DURAVYU 2.7mg arm.

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DURAVYU favorable safety profile continues:

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No DURAVYU related ocular or systemic serious adverse events reported

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No cases of:

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Impaired vision

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Endophthalmitis

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Retinal vasculitis (occlusive or non-occlusive)

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Insert migration

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Intraocular inflammation (IOI)

24-week supplement-free patient subgroup analyses from the Phase 2 VERONA clinical trial demonstrate that DURAVYU 2.7mg significantly improved vision and fluid compared to the aflibercept control group, including:

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BCVA improvement of +10.3 letters versus +3.0 letters for aflibercept control

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CST improvement of 117.4 microns versus 43.7 microns for aflibercept control

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43% had an absence of DME compared to zero for the aflibercept control arm.

These results show that the positive data from the Phase 2 VERONA trial were driven by DURAVYU.

The 24-week topline results from the Phase 2 VERONA study in DME were presented at the Retina World Congress in May 2025, which highlighted DURAVYU’s potential to transform the treatment landscape in the second largest retinal disease market with its potential best-in-class safety and efficacy profile.

On October 14, 2025 we announced details for our pivotal Phase 3 program evaluating DURAVYU for the treatment of DME with first patient dosing anticipated in first quarter of 2026. In this announcement we shared new preclinical data that demonstrates vorolanib, the active drug in DURAVYU, inhibits IL-6 mediated inflammation through inhibition of all JAK receptors, in particular JAK-1, in addition to known blockage of VEGF mediated vascular permeability. This finding reinforces the early and sustained improvements observed through six months in the Phase 2 VERONA clinical trial and positions DURAVYU as a potential multi-MOA treatment.

In February 2026, the Company initiated two global, randomized, double-masked, non-inferiority Phase 3 clinical trials (COMO and CAPRI) evaluating the efficacy and safety of DURAVYU in patients with DME. These trials, developed with feedback from both the FDA and the EMA, will each enroll approximately 240 patients— roughly 75% treatment-naive and 25% previously treated—randomized 1:1 to receive either a 2.7mg dose of DURAVYU administered every six months or on-label aflibercept. The primary endpoint is the difference in mean change in BCVA from Day 1 to Week 52 and 56 (blended) versus aflibercept control. Secondary

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endpoints include safety, reduction in treatment burden, percentage of eyes free of supplemental aflibercept injections, and anatomical stability.

DURAVYU Licenses

In February 2020, we entered into an Exclusive License Agreement (Equinox License Agreement) with Equinox Science, LLC (Equinox), relating to vorolanib, the active ingredient in DURAVYU. Pursuant to this agreement, Equinox granted us an exclusive, sublicensable, royalty-bearing right and license to certain patents and other Equinox intellectual property to research, develop, make, have made, use, sell, offer for sale and import the compound vorolanib and any pharmaceutical products comprising the compound for the prevention or treatment of wet AMD, DR and RVO (the Original Field) using our proprietary localized delivery technologies, in each case, throughout the world except China, Hong Kong, Taiwan and Macau (the Territory). On May 2, 2022, we entered into Amendment #1 to the Equinox License Agreement, pursuant to which the Original Field was expanded to cover the prevention or treatment of ophthalmology indications using the Company’s proprietary localized delivery technologies.

In consideration for the rights granted by Equinox, we (i) made a one time, non-refundable, non-creditable upfront cash payment of $1.0 million to Equinox in February 2020, and (ii) agreed to pay milestone payments totaling up to $50 million upon the achievement of certain development and regulatory milestones, consisting of (a) completion of a Phase 2 clinical trial for the compound or a licensed product, (b) the filing of a new drug application (NDA) or foreign equivalent for the compound or a licensed product in the United States, European Union, or United Kingdom and (c) regulatory approval of the compound or a licensed product in the United States, European Union, or United Kingdom.

We also agreed to pay Equinox tiered royalties based upon annual net sales of licensed products in the Territory. The royalties are payable with respect to a licensed product in a particular country in the Territory on a country-by-country and licensed product-by-licensed product basis until the later of (i) twelve years after the first commercial sale of such licensed product in such country and (ii) the first day of the month following the month in which a generic product corresponding to such licensed product is launched in such country. The royalty rates range from the high-single digits to low-double digits depending on the level of annual net sales. The royalty rates are subject to reduction during certain periods when there is no valid patent claim that covers a licensed product in a particular country.

On May 2, 2022, the Company entered into an Exclusive License Agreement (the Betta License Agreement) with Betta Pharmaceuticals Co., Ltd. (Betta), an affiliate of Equinox. Under the Betta License Agreement, the Company granted to Betta an exclusive, sublicensable, royalty-bearing license under certain of the Company’s intellectual property to develop, use (but not make or have made), sell, offer for sale, and import the Company’s product candidate, DURAVYU, an investigational sustained delivery intravitreal anti-VEGF treatment that combines a bioerodible formulation of the Company’s proprietary sustained-release technology with the compound vorolanib (the Licensed Product), in the field of ophthalmology (the Betta Field) in the Greater Area of China, including China, the Hong Kong Special Administrative Region, the Macau Special Administrative Region, and Taiwan (the Betta Territory). The Company retained rights under the Company’s intellectual property to, among other things, conduct clinical trials on the Licensed Product in the Betta Field in the Betta Territory.

In consideration for the rights granted by the Company, Betta agreed to pay the Company tiered, mid-to-high single-digit royalties based upon annual net sales of Licensed Products in the Betta Territory. The royalties are payable on a Licensed Product-by-Licensed-Product and region-by-region basis commencing on the first commercial sale of a Licensed Product in a region and continuing until the later of (i) the date that is twelve (12) years after first commercial sale of such Licensed Product in such region, and (ii) the first day of the month following the month in which a generic product corresponding to such Licensed Product is launched in the relevant region. The royalty rate is subject to reduction under certain circumstances, including when there is no valid claim of a licensed patent that covers a Licensed Product in a particular region.

EYP-2301

The Company is advancing EYP-2301 into pre-clinical development. EYP-2301 delivers razuprotafib, formulated in Durasert E™ to potentially improve outcomes in serious retinal diseases.

Manufacturing

The FDA carefully regulates the quality of pharmaceuticals. The main regulatory standard for ensuring pharmaceutical quality is the Current Good Manufacturing Practice (cGMPs) regulation for human pharmaceuticals. Manufacturing of our clinical trial materials (CTM) and of our commercial products is subject to these cGMPs which govern record-keeping, manufacturing processes and controls, personnel, quality control and quality assurance, among other activities. Incoming raw materials and components from suppliers are inspected upon arrival according to pre-specified criteria prior to use in the CTM or the commercial product. During

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product manufacture, in-process tests are conducted on intermediate products according to pre-specified criteria; testing is conducted on the finished product prior to its release. Our systems and our contractors are required to comply with cGMP requirements, and we assess compliance regularly through performance monitoring and audits. Our current manufacturing activities are focused exclusively on products designated for distribution and sale in markets outside the United States.

DURAVYU

Production, assembly, and packaging of DURAVYU Phase 3 CTM is done in the Class 10,000 clean rooms located at our Watertown, MA facility. We source the active pharmaceutical ingredient (API) vorolanib from one supplier, and various raw materials and components for both DURAVYU and its injector from third-party vendors. Our arrangements with these third parties include confidentiality, intellectual property, and supply provisions to protect our proprietary rights related to DURAVYU. In October 2024, we announced the grand opening of our commercial manufacturing facility in Northbridge, MA. The 41,000 square foot Good Manufacturing Process (cGMP) compliant commercial manufacturing facility was built to meet U.S. FDA and EMA standards and will support global manufacturing across the Company’s portfolio, including lead pipeline asset, DURAVYU upon potential regulatory approval. Manufacturing tech transfer of DURAVYU from Watertown to Northbridge began during 2025.

We source the active pharmaceutical ingredient (API) vorolanib from one supplier, and various raw materials and components for DURAVYU drug product manufacturing from third-party vendors. Injector and packaging components are purchased from several different vendors with the needles supplied by a third-party supplier. While we believe that multiple vendors are available for each component we purchase, we have historically sole-sourced each component. Our agreements with these third parties include confidentiality, intellectual property, and supply provisions to protect our proprietary rights related to DURAVYU.

YUTIQ®

Production, assembly, and packaging of YUTIQ® is done in the Class 10,000 clean rooms located at our Watertown, MA facility and we are supplying such product to our partner pursuant to our agreement with them. We source the API from Farmabios, needles from a third party supplier and various other raw materials and components for YUTIQ® from additional third-party vendors. While we believe that multiple vendors are available for each component we purchase, we have historically sole-sourced each component. The Company no longer produces YUTIQ® for sale in the US but continues to supply product for sale by its partner in China.

FDA Approved Products Licensed to Other Entities

YUTIQ® for posterior segment uveitis

YUTIQ® (fluocinolone acetonide intravitreal implant or FA 0.18 mg) for intravitreal injection, was approved by the FDA in October 2018 and commercially launched in the U.S. in February 2019. YUTIQ® is indicated for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye. YUTIQ® is a once every three-year treatment utilizing a non-erodible formulation of our proprietary Durasert® technology that is administered during a physician office visit. In May 2023 we licensed rights to YUTIQ® to ANI for $82.5 million with $75.0 million paid up-front and $7.5 million paid in equal quarterly installments in 2024. We are also entitled to low to mid double-digit royalty on ANI's related U.S. net sales above defined thresholds for the calendar years 2025-2028.

We have licensed clinical development, regulatory, reimbursement and distribution rights to YUTIQ® to Ocumension for Mainland China, Hong Kong, Macau, Taiwan, South Korea, and other jurisdictions across Southeast Asia. YUTIQ® was approved in China in 2022 and we are entitled to royalties on product sales by Ocumension.

ILUVIEN for DME

ILUVIEN is an injectable, sustained release micro-insert based on our Durasert® technology platform which delivers 0.19 mg of FA to the back of the eye for treatment of DME. DME is a disease suffered by diabetics where leaking capillaries cause swelling in the macula, the most sensitive part of the retina. DME is a leading cause of blindness in the working-age population in most developed countries. The ILUVIEN micro-insert is substantially the same micro-insert as YUTIQ®.

We originally licensed our Durasert® proprietary insert technology to ANI for use in ILUVIEN for the treatment of all ocular diseases (excluding uveitis). On July 10, 2017, we entered into an amended and restated collaboration agreement with ANI (the Amended ANI Agreement), pursuant to which we (i) expanded the license to ANI to our proprietary Durasert® sustained release drug delivery technology platform to include uveitis, including chronic non-infectious uveitis affecting the posterior segment of the eye, in EMEA and (ii) converted the net profit share arrangement for each licensed product (including ILUVIEN) under the original

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collaboration agreement with ANI (the Prior ANI Agreement) to a sales-based royalty on a calendar quarter basis commencing July 1, 2017, with payments from ANI due 60 days following the end of each calendar quarter.

Sales-based royalties started at the rate of 2% and increased, commencing December 12, 2018, to 6% on aggregate calendar year net sales up to $75 million and 8% in excess of $75 million. ANI's share of contingently recoverable accumulated ILUVIEN commercialization losses under the Prior ANI Agreement, capped at $25 million, are to be reduced as follows: (i) $10.0 million was cancelled in lieu of an upfront license fee on the effective date of the Amended ANI Agreement; (ii) for calendar years 2019 and 2020, 50% of earned sales-based royalties in excess of 2% will be offset against the quarterly royalty payments otherwise due from ANI; (iii) in March 2020, another $5 million was cancelled upon ANI's receipt of regulatory approval for ILUVIEN for the uveitis indication; and (iv) commencing in calendar year 2021, 20% of earned sales-based royalties in excess of 2% will be offset against the quarterly royalty payments due from ANI until such time as the balance of the original $25 million of recoverable commercialization losses has been fully recouped. On December 17, 2020, we sold our interest in royalties payable to us under our license agreement with ANI in connection with ANI's sales of ILUVIEN® to SWK Funding, LLC (SWK) in exchange for a one-time $16.5 million payment from SWK. On March 18, 2025, ANI announced that it completed the buyout of its 3.125% perpetual royalty obligation to SWK on worldwide net revenues of ILUVIEN® and YUTIQ® for a one-time payment of $17.25 million. Under the terms of the agreement, upon making the buyout payment, no further royalty is due to SWK on net revenues beginning January 1, 2025, forward. As a result, we terminated our royalty purchase agreement with SWK effective March 18, 2025.

Intellectual Property

We own or license patents in the U.S. and other countries. Our patents generally cover the design, formulation, manufacturing methods, and use of our sustained release therapeutics, devices and technologies. For example, we own and/or license U.S. and foreign patents and patent applications for our Durasert® technology. In addition, we own U.S. and foreign patents and patent applications covering other technologies, such as devices used to administer some of our products. Patents for individual products extend for varying periods according to the date of patent filing or grant and legal term of patents in the various countries where patent protection is obtained. The actual protection afforded by a patent, which can vary from country to country, depends upon the type of patent, the scope of its coverage, and the availability of legal remedies in the country. Patent term extension may be available in various countries to compensate for a patent office delay or a regulatory delay in approval of the product.

The last expiring patent covering the vorolanib compound licensed to us by Equinox Science and used in DURAVYU expires in September 2037, but the Company has a granted US patent for DURAVYU that expires in 2043. In addition, in this patent family, a patent has been granted in China with an expiration date in 2041) and in Taiwan (with an expiration date in 2042) and other patent applications are pending in this family. The Company has filed additional patent applications for technology relating to DURAVYU, that, if issued, could expire in 2043, and for a new injector designed for administration of Durasert®, that, if issued, could expire in 2046.

Under an Asset Purchase Agreement with Aerpio Pharmaceuticals Inc. (Aerpio) in 2021, we acquired all right title and interest in and to certain U.S. and ex-U.S. patents and applications relating to certain TIE-2 activating molecules, including razuprotafib. The acquired Aerpio patent portfolio now includes approximately 155 U.S. or ex-U.S. patents and pending applications that claim compositions of matter, pharmaceutical compositions and/or methods of use for both small molecule and mono and bi-specific antibody inhibitors of the protein tyrosine phosphatase (VE-PTP). One of the small molecules is razuprotafib. Some of the antibodies covered include both VE-PTP and VEGF binding domains. VE-PTP is a negative TIE-2 regulator that, when inhibited, can activate the TIE-2 pathway leading to downstream signaling that promotes vascular health, stability and decreases vascular permeability and inflammation associated with a number of posterior segment eye diseases. The patent claims for methods of use relate primarily to disease indications where activation of TIE-2 and associated vascular stabilization are potentially beneficial. The potential expiration dates of the patents and applications in this portfolio range from 2027 to 2041.

This patent expiration date range is estimated and this range and other patent expiration dates provided herein are based on certain assumptions, including that certain applications will be granted, all necessary fees will be paid and no terminal disclaimers or other limitations on expiration are required for certain patents or applications.

Human Capital Resources

To achieve our Company goals, it is critical to attract and retain top talent with experience across all functional areas crucial to executing on our strategy. To facilitate talent attraction and retention, our Company ensures a collaborative, safe and rewarding workplace, providing opportunities for our employees to grow and develop in their careers. We offer compensation and incentives that include market-competitive pay, equity grants, performance bonuses, healthcare benefits, retirement, and wellness programs,

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including paid time off and flexible work arrangements. We embrace our Company values and behaviors to build a culture that fosters collaboration, innovation, inclusivity, integrity and excellence.

As of February 27, 2026, we had 214 employees, all of which were full-time, all located in the United States. None of our employees are represented by a collective bargaining agreement and none are represented by labor union. During fiscal 2025 our voluntary turnover rate was 6.56%, which is below the average voluntary turnover rates for Boston-area biotech companies.

The success of our business is fundamentally connected to the well-being of our employees. Accordingly, we are committed to their health, safety, and wellness. We provide our employees and their families with access to a variety of innovative, flexible and convenient health and wellness programs, including benefits that provide protection and security so that they have peace of mind concerning events that may require time away from work, or that impact their financial well-being. We support their physical and mental health by providing tools and resources to help them improve or maintain their health status and encourage engagement in healthy behaviors. Depending on the nature of the work both remote and hybrid work arrangements are available.

We also provide robust compensation packages to meet the needs of our employees. In addition to competitive base salaries, these programs include annual discretionary bonus payments, equity awards, a 401(k) plan and employer match, an employee stock purchase program, tax advantaged health savings and flexible spending accounts, paid time off and family leave. Our broad-based equity programs include employees across all levels. The vesting conditions are set to facilitate the retention of employees with critical skills and experience and motivate employees to perform to the best of their abilities, while we achieve our objectives.

In order to promote long-term retention and maximize the potential of our employees, we invest in their professional and personal development. By offering needs-based supplemental training, management development, effective communications training and mentorship.

We survey our employees on a regular basis and report the results of those surveys back to employees and management while implementing programming to directly address the feedback.

As a company our success is rooted in the diversity of our teams and our commitment to inclusion. We value diversity at all levels and continue to focus on extending our diversity and inclusion initiatives across our workforce – from working with managers to recruit diverse team members to the advancement of leaders from different backgrounds.

Competition

The market for products treating eye diseases is highly competitive and is characterized by extensive research efforts and rapid technological progress. Pharmaceutical, drug delivery, and biotechnology companies, as well as research organizations, governmental entities, universities, hospitals, other nonprofit organizations, and individual scientists, have developed and are seeking to develop drugs, therapies, and novel delivery methods to treat diseases targeted by our products and product candidates. Many of our competitors and potential competitors are larger, better established, more experienced, and have substantially more resources than we or our partners have. Competitors may reach the market earlier, may have obtained or could obtain patent protection that dominates or adversely affects our products and potential products, and may offer products with greater efficacy, lesser or fewer side effects, and/or other competitive advantages. We believe that competition for treatments of eye diseases is based upon the effectiveness of the treatment, side effects, time to market, reimbursement and price, reliability, ease of administration, dosing or injection frequency, patent position, and other factors.

Many companies have or are pursuing products to treat eye diseases that are or would be competitive with DURAVYU and other pipeline products. Some of these products and product candidates include the following:

FDA-approved LUCENTIS® (ranibizumab), EYLEA® (aflibercept 2mg), EYLEA®HD (aflibercept 8mg), VABYSMO® (faricimab) and off-label use of the cancer drug AVASTIN®(bevacizumab) are the leading treatments for wet AMD. Lucentis, Eylea, and Avastin are also used in the treatment of DR and DME. There are two FDA-approved Lucentis biosimilar mediations and two FDA-approved Eylea biosimilars (Opuviz™ and Yesafili™).

In 2021, the FDA approved Susvimo®(ranibizimab), a first-of-its-kind port delivery system (PDS) with ranibizumab for the treatment of patients with wet AMD. In 2025, Susvimo was approved for the treatment of DME and DR.

In January 2022, the FDA approved VABYSMO®(faricimab), a bispecific antibody targeting Ang-2 and vascular endothelial growth factor-A. Also in 2022, two ranibizumab biosimilars, Byooviz and Cimerli entered the market. The FDA also approved Beovu® brolucizumab injection on October 8, 2019.

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In August 2023, the FDA approved EYLEA® HD (aflibercept 8mg) for wet AMD, DME, and DR based on the pivotal PULSAR and PHOTON trials in which EYLEA® HD demonstrated clinically equivalent vision gains to EYLEA® (aflibercept 2 mg) that were maintained with fewer injections. In November 2025, EYLEA HD was approved for the treatment of macular edema following retinal vein occlusion, and a monthly (Q4W) dosing option across all existing indications was also approved.

In addition to FDA approved products, there are multiple investigational treatments in development.

REGENXBIO Inc., Adverum Biotechnologies, Inc., 4D Molecular Therapeutics (4DMT), Merck (EyeBio), Ollin Biosciences, Inc., as well as several others in early development, are advancing gene therapy treatments for retinal diseases, such as wet AMD and DME. REGENXBIO is developing ABBV-RGX-314, a gene therapy utilizing its NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment which inhibits VEGF. In October 2025, they completed patient enrollment in the ATMOSPHERE and ASCENT pivotal trials in wet AMD evaluating subretinal ABBV-RGX-314. They are also enrolling patients in AAVIATE, a Phase 2 trial for wet AMD evaluating suprachoroidal ABBV-RGX-314, and initiating a Phase 2b/3 trial evaluating suprachoroidal ABBV-RGX-314 in DR.

Adverum (acquired by Eli Lilly in December 2025) is developing Ixo-vec, a gene therapy utilizing an AAV.7m8 vector containing a gene encoding for a protein that expresses aflibercept. ARTEMIS is a Phase 3 trial currently underway evaluating intravitreal Ixo-vec in wet AMD.

4DMT is developing 4D-150 as an investigational genetic medicine using the intravitreal R100 vector to deliver a dual transgene payload (AFL and VEGF C RNAi) that inhibits VEGF A, B, C, and PIGF for the treatment of wet AMD and DME. In 2025, 4DMT initiated two Phase 3 trials in wet AMD (4FRONT-1 and 4FRONT-2), with plans to initiate a single global Phase 3 trial in DME in 2026.

Merck (EyeBio) is developing MK-3000, a trispecific antibody designed to agonize the Wingless-related integration site (Wnt) pathway. BRUNELLO and BAROLO are Phase 2b/3 trials that are currently underway in DME. Merck (EyeBio) is also developing MK-8748, a bispecific antibody which is a TIE-2-agonist and VEGF-antagonist. They are currently evaluating MK-8748 in wet AMD and DME in the second part of the Phase 1/2a RIOJA trial.

Ollin is developing OLN324, a small-format VEGF/Ang2 bispecific antibody. The Phase 1b JADE trial of OLN324 vs. faricimab in DME and wet AMD resulted in positive topline data. Ollin plans to move forwards with global Phase 3 studies in both DME and wet AMD in 2026.

AXPAXLI (formerly OTX-TKI) – Ocular Therapeutix, Inc.

Ocular Therapeutix, Inc. (Ocular Therapeutix) is developing AXPAXLI which utilizes axitinib, a TKI, formulated in a hydrogel and delivered through an intravitreal injection. In February 2026, Ocular Therapeutix announced positive results in their Phase 3 SOL-1 superiority clinical trial comparing a single AXPAXLI injection to a single aflibercept (2 mg) injection in treatment naïve wet AMD subjects with a nine-month primary endpoint.

Their Phase 3 SOL-R clinical trial is a non-inferiority trial comparing repeat AXPAXLI injections every six months to repeat aflibercept (2 mg) injections every eight weeks in wet AMD, with a 56-week primary endpoint. SOL-R reached its target enrollment of 555 patients in November 2025.

In August 2025, Ocular Therapeutix announced it received a SPA agreement with the FDA for its Phase 3 HELIOS-2 trial for AXPAXLI in NPDR. The HELIOS Phase 3 program in NPDR includes two studies: HELIOS-2 and HELIOS-3. In November 2025, the first patient was randomized in the HELIOS-3 trial.

Tarcocimab Tedromer (formerly KSI-301) – Kodiak Sciences, Inc.

Tarcocimab tedromer is an investigational anti-VEGF therapy. In July 2023, Kodiak Sciences, Inc. (Kodiak) announced its Phase 3 GLEAM and GLIMMER studies in treatment-naïve DME did not meet their primary efficacy endpoints of showing non-inferior visual acuity gains for tarcocimab compared to aflibercept. However, the Phase 3 DAYLIGHT study in wet AMD did meet its primary endpoint of non-inferior visual acuity gains for tarcocimab dosed monthly compared to aflibercept dosed every 8 weeks following 3 monthly loading doses.

In March 2025, Kodiak announced the completion of enrollment in its second registrational trial of tarcocimab in DR (GLOW2). The first registrational trial, GLOW1, met its primary endpoint in DR. As of November 2025, Kodiak had completed patient enrollment in DAYBREAK, a Phase 3 trial for tarcocimab and KSI-501, a bi-specific anti- IL-6 and VEGF trap molecule in

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wet AMD. Both GLOW2 and DAYBREAK are using tarcocimab's enhanced 50 mg/mL formulation containing both conjugated and unconjugated antibody that is intended to balance durability and immediacy. Topline data for GLOW2 is expected in the first quarter of 2026 and for DAYBREAK in the third quarter of 2026.

OCS-01 - Oculis Holding AG

OCS-01 1.5% ophthalmic suspension is a topical formulation of dexamethasone that utilizes novel solubilizing nanoparticle technology to enhance bioavailability and durability of the dexamethasone solution. DIAMOND is a 2-stage, double-masked, randomized, multicenter Phase 3 program that will evaluate the safety and efficacy of OCS-01 with 2 dosing regimens in comparison to vehicle alone in DME patients for 52 weeks. The Phase 3 DIAMOND-1 and DIAMOND-2 trials of OCS-01 eye drops in DME completed enrollment in April 2025 with topline data expected in the second quarter of 2026.

Government Regulation

We are subject to extensive regulation by the FDA and other federal, state, and local regulatory agencies. The Federal Food, Drug and Cosmetic Act (the FD&C Act), and FDA’s implementing regulations set forth, among other things, requirements for the testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record-keeping, reporting, distribution, import, export, advertising, and promotion of our products and product candidates. Although the discussion below focuses on regulation in the U.S., we currently out-license certain of our products and may seek approval for, and market, other products in other countries in the future. Generally, our activities in other countries will be subject to regulation that is similar in nature and scope to that imposed in the U.S., although there can be important differences. In the European Union, although significant aspects are addressed in a centralized way through the European Medicines Agency (the "EMA"), and the European Commission, country-specific regulation remains essential in many respects. The process of obtaining regulatory marketing approvals and the subsequent compliance with appropriate federal, state, local, and foreign statutes and regulations require the expenditure of substantial time and financial resources and may not be successful.

Development and Approval

Under the FD&C Act, FDA approval of an NDA is required before any new drug can be marketed in the U.S. NDAs require extensive studies and submission of a large amount of data by the applicant.

Pre-clinical Testing. Before testing any compound in human patients in the U.S., a company must generate extensive pre-clinical data. Pre-clinical testing generally includes laboratory evaluation of product chemistry and formulation, as well as toxicological and pharmacological studies in several animal species to assess the toxicity and dosing of the product. Certain animal studies must be performed in compliance with the FDA’s Good Laboratory Practice (GLP), regulations and the U.S. Department of Agriculture’s Animal Welfare Act.

Investigational New Drug (IND) Application. Human clinical trials in the U.S. cannot commence until an IND, application is submitted and becomes effective. A company must submit pre-clinical testing results to the FDA as part of the IND, and the FDA must evaluate whether there is an adequate basis for testing the drug in initial clinical studies in human volunteers. Unless the FDA raises concerns, the IND becomes effective 30 days following its receipt by the FDA, and the clinical trial proposed in the IND may begin. Once human clinical trials have commenced, the FDA may stop a clinical trial by placing it on “clinical hold” because of concerns about the safety of the product being tested, or for other reasons.

Clinical Trials. Clinical trials involve the administration of a drug to healthy human volunteers or to patients under the supervision of a qualified investigator. The conduct of clinical trials is subject to extensive regulation, including compliance with the FDA’s bioresearch monitoring regulations and Good Clinical Practice, or GCP, requirements, which establish standards for conducting, recording data from, and reporting the results of, clinical trials, and are intended to assure that the data and reported results are credible and accurate, and that the rights, safety, and well-being of study participants are protected. Clinical trials must be conducted under protocols that detail the study objectives, parameters for monitoring safety, and the efficacy criteria, if any, to be evaluated. Each protocol is reviewed by the FDA as part of the IND. In addition, each clinical trial must be reviewed and approved by, and conducted under the auspices of, an institutional review board (IRB), for each clinical site. Companies sponsoring the clinical trials, investigators, and IRBs also must comply with, as applicable, regulations and guidelines for obtaining informed consent from the study patients, following the protocol and investigational plan, adequately monitoring the clinical trial, and timely reporting of adverse events, or AEs. Foreign studies conducted under an IND must meet the same requirements that apply to studies being conducted in the U.S. Data from a foreign study not conducted under an IND may be submitted in support of an NDA if the study was conducted in accordance with GCP and the FDA is able to validate the data.

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A study sponsor is required to publicly post specified details about certain clinical trials and clinical trial results on government or independent websites (e.g., http://clinicaltrials.gov). Human clinical trials typically are conducted in three sequential phases, although the phases may overlap or be combined:

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Phase 1 clinical trials involve the initial administration of the investigational drug to humans, typically to a small group of healthy human subjects, but occasionally to a group of patients with the targeted disease or disorder. Phase 1 clinical trials generally are intended to evaluate the safety, metabolism and pharmacologic actions of the drug, the side effects associated with increasing doses, and, if possible, to gain early evidence of effectiveness.

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Phase 2 clinical trials generally are controlled studies that involve a relatively small sample of the intended patient population and are designed to develop initial data regarding the product’s effectiveness, to determine dose response and the optimal dose range, and to gather additional information relating to safety and potential AEs.

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Phase 3 clinical trials are conducted after preliminary evidence of effectiveness has been obtained and are intended to gather the additional information about dosage, safety and effectiveness necessary to evaluate the drug’s overall risk-benefit profile, and to provide a basis for regulatory approval. Generally, Phase 3 clinical development programs consist of expanded, large-scale studies of patients with the target disease or disorder to obtain statistical evidence of the efficacy and safety of the drug at the proposed dosing regimen.

The sponsoring company, the FDA, or the IRB may suspend or terminate a clinical trial at any time on various grounds, including a finding that the patients are being exposed to an unacceptable health risk. Further, success in early-stage clinical trials does not assure success in later-stage clinical trials. Data obtained from clinical activities are not always conclusive and may be subject to alternative interpretations that could delay, limit or prevent regulatory approval.

NDA Submission and Review. The FD&C Act provides two pathways for the approval of new drugs through an NDA. An NDA under Section 505(b)(1) of the FD&C Act is a comprehensive application to support approval of a product candidate that includes, among other things, data and information to demonstrate that the proposed drug is safe and effective for its proposed uses, that production methods are adequate to ensure its identity, strength, quality, and purity of the drug, and that proposed labeling is appropriate and contains all necessary information. A 505(b)(1) NDA contains results of the full set of pre-clinical studies and clinical trials conducted by or on behalf of the applicant to characterize and evaluate the product candidate.

Section 505(b)(2) of the FD&C Act provides an alternate regulatory pathway to obtain FDA approval that permits the filing of an NDA where at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference. The applicant may rely to some extent upon the FDA’s findings of safety and effectiveness for an approved product that acts as the reference drug and submit its own product-specific data — which may include data from pre-clinical studies or clinical trials conducted by or on behalf of the applicant — to address differences between the product candidate and the reference drug.

The submission of an NDA under either Section 505(b)(1) or Section 505(b)(2) generally requires payment of a substantial user fee to the FDA, subject to certain limited deferrals, waivers and reductions. The FDA reviews applications to determine, among other things, whether a product is safe and effective for its intended use and whether the manufacturing controls are adequate to assure and preserve the product’s identity, strength, quality, and purity. For some NDAs, the FDA may convene an advisory committee to seek insights and recommendations on issues relevant to approval of the application. Although the FDA is not bound by the recommendation of an advisory committee, the agency usually considers such recommendations carefully when making decisions.

Our products and product candidates include products that combine drug and device components in a manner that meet the definition of a "combination product" under FDA regulations. The FDA exercises significant discretion over the regulation of combination products, including the discretion to require separate marketing applications for the drug and device components in a combination product. For a drug-device combination product for which CDER has primary jurisdiction, CDER typically consults with the Center for Devices and Radiological Health in the NDA review process. Whether reviewed under one application or separately, both the drug and device components of a drug-device combination product must satisfy the applicable regulatory requirements for marketing as if they were submitted for approval independently.

The FDA may determine that a Risk Evaluation and Mitigation Strategy (REMS), is necessary to ensure that the benefits of a new product outweigh its risks, and the product can therefore be approved. A REMS may include various elements, ranging from a medication guide or patient package insert to limitations on who may prescribe or dispense the drug, depending on what the FDA considers necessary for the safe use of the drug. Under the Pediatric Research Equity Act (PREA), certain applications for approval must also include an assessment, generally based on clinical study data, of the safety and effectiveness of the subject drug in relevant pediatric populations.

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Before approving an NDA, the FDA will inspect the facility or facilities where the product is manufactured. The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP, requirements and adequate to assure consistent production of the product within required specifications.

The FDA conducts a preliminary review of a submitted NDA to ensure the application is sufficiently complete for substantive review. Once the FDA accepts an NDA submission for filing — which occurs, if at all, within 60 days after submission of the NDA — the FDA’s goal for a non-priority review of an NDA is ten months. The review process can be and often is significantly extended, however, by FDA requests for additional information, studies, or clarification. The targeted action date can also be shortened to six months of the 60-day filing date for products that are granted priority review designation because they are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. The FDA reviews an NDA to determine, among other things, whether a product is safe and effective for its intended use and whether its manufacturing is cGMP-compliant to assure and preserve the product’s identity, strength, quality, and purity.

After review of an NDA and the facilities where the product candidate is manufactured, the FDA either issues an approval letter or a complete response letter (CRL), outlining the deficiencies in the submission. The CRL may require additional testing or information, including additional pre-clinical or clinical data, for the FDA to reconsider the application. Even if such additional information and data are submitted, the FDA may decide that the NDA still does not meet the standards for approval. Data from clinical trials are not always conclusive and the FDA may interpret data differently than the sponsor. FDA approval of any application may include many delays or never be granted. If FDA grants approval, an approval letter authorizes commercial marketing of the product candidate with specific prescribing information for specific indications.

Obtaining regulatory approval often takes a number of years, involves the expenditure of substantial resources, and depends on a number of factors, including the severity of the disease in question, the availability of alternative treatments, and the risks and benefits demonstrated in clinical trials. Additionally, as a condition of approval, the FDA may impose restrictions that could affect the commercial success of a drug or require post-approval commitments, including the completion within a specified time period of additional clinical studies, which often are referred to as “Phase 4” or “post-marketing” studies.

Post-approval modifications to the drug, such as changes in indications, labeling, or manufacturing processes or facilities, may require a sponsor to develop additional data or conduct additional pre-clinical studies or clinical trials, to be submitted in a new or supplemental NDA, which would require FDA approval.

Post-Approval Regulation

Once approved, drug products are subject to continuing regulation by the FDA. If ongoing regulatory requirements are not met, or if safety or manufacturing problems occur after the product reaches the market, the FDA may at any time withdraw product approval or take actions that would limit or suspend marketing. Additionally, the FDA may require post-marketing studies or clinical trials, changes to a product’s approved labeling, including the addition of new warnings and contraindications, or the implementation of other risk management measures, including distribution-related restrictions, if there are new safety information developments.

Good Manufacturing Practices. Companies engaged in manufacturing drug products or their components must comply with applicable cGMP requirements and product-specific regulations enforced by the FDA and other regulatory agencies. Compliance with cGMP includes adhering to requirements relating to organization and training of personnel, buildings and facilities, equipment, control of components and drug product containers and closures, production and process controls, quality control and quality assurance, packaging and labeling controls, holding and distribution, laboratory controls, and records and reports. The FDA regulates and inspects equipment, facilities, and processes used in manufacturing pharmaceutical products prior to approval. If, after receiving approval, a company makes a material change in manufacturing equipment, location, or process (all of which are, to some degree, incorporated in the NDA), additional regulatory review and approval may be required. The FDA also conducts regular, periodic visits to re-inspect equipment, facilities, and processes following the initial approval of a product. Failure to comply with applicable cGMP requirements and conditions of product approval may lead the FDA to take enforcement actions or seek sanctions, including fines, issuance of warning letters, civil penalties, injunctions, suspension of manufacturing operations, operating restrictions, withdrawal of FDA approval, seizure or recall of products, and criminal prosecution. Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements.

In addition to cGMP requirements, drug-device combination products are also subject to certain additional manufacturing and safety reporting regulations for devices. Specifically, the FDA requires that drug-device combination products comply with certain provisions of the Quality System Regulation (QSR), which sets forth the FDA’s manufacturing quality standards for medical devices.

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In addition to drug safety reporting requirements, the FDA also requires that we comply with some device safety reporting requirements for our drug-device combination product.

Advertising and Promotion. The FDA and other federal regulatory agencies closely regulate the marketing and promotion of drugs through, among other things, standards and regulations for direct-to-consumer advertising, advertising and promotion to healthcare professionals, communications regarding unapproved uses, industry-sponsored scientific and educational activities, and promotional activities involving the Internet. A product cannot be promoted before it is approved. After approval, product promotion can include only those claims relating to safety and effectiveness that are consistent with the labeling approved by the FDA. Healthcare providers are permitted to prescribe drugs for “off-label” uses — that is, uses not approved by the FDA and not described in the product’s labeling — because the FDA does not regulate the practice of medicine. However, FDA regulations impose restrictions on manufacturers’ communications regarding off-label uses. Broadly speaking, a manufacturer may not promote a drug for off-label use, but under certain conditions may engage in non-promotional, balanced, scientific communication regarding off-label use. Failure to comply with applicable FDA requirements and restrictions in this area may subject a company to adverse publicity and enforcement action by the FDA, the Department of Justice, or the Office of the Inspector General of the Department of Health and Human Services, as well as state authorities. This could subject a company to a range of penalties that could have a significant commercial impact, including civil and criminal fines and agreements that materially restrict the manner in which a company promotes or distributes a drug.

New Legislation. New legislation is passed periodically in Congress, or at the state level, that could significantly change the statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA.

Further, FDA revises its regulations and guidance in light of new legislation in ways that may affect our business or products. It is impossible to predict whether other changes to legislation, regulation, or guidance will be enacted, or what the impact of such changes, if any, may be.

Other Requirements. NDA holders must comply with other regulatory requirements, including submitting annual reports, reporting information about adverse drug experiences, reporting marketing status notifications, and maintaining certain records.

Hatch-Waxman Act

The Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act, establishes two abbreviated approval pathways for pharmaceutical products that are in some way follow-on versions of already approved products.

Generic Drugs. A generic version of an approved drug is approved by means of an abbreviated NDA, or ANDA, by which the sponsor demonstrates that the proposed product is the same as the approved, brand-name drug, which is referred to as the reference listed drug (RLD). Generally, an ANDA must contain data and information showing that the proposed generic product and RLD (i) have the same active ingredient, in the same strength and dosage form, to be delivered via the same route of administration, (ii) are intended for the same uses, and (iii) are bioequivalent. This is instead of independently demonstrating the proposed product’s safety and effectiveness, which are inferred from the fact that the product is the same as the RLD, which the FDA previously found to be safe and effective.

505(b)(2) NDAs. As discussed previously, products may also be submitted for approval via an NDA under section 505(b)(2) of the FD&C Act. Unlike an ANDA, this does not excuse the sponsor from demonstrating the proposed product’s safety and effectiveness. Rather, the sponsor is permitted to rely to some degree on information from investigations that were not conducted by or for the applicant and for which the applicant has not obtained a right of reference and must submit its own product-specific data of safety and effectiveness to an extent necessary because of the differences between the products. An NDA approved under 505(b)(2) may in turn serve as an RLD for subsequent applications from other sponsors.

RLD Patents. In an NDA, a sponsor must identify patents that claim the drug substance or drug product or a method of using the drug. When the drug is approved, those patents are among the information about the product that is listed in the FDA publication, Approved Drug Products with Therapeutic Equivalence Evaluations, which is referred to as the Orange Book. The sponsor of an ANDA or 505(b)(2) application seeking to rely on an approved product as the RLD must make one of several certifications regarding each listed patent. A “Paragraph I” certification is the sponsor’s statement that patent information has not been filed for the RLD. A “Paragraph II” certification is the sponsor’s statement that the RLD’s patents have expired. A “Paragraph III” certification is the sponsor’s statement that it will wait for the patent to expire before obtaining approval for its product. A “Paragraph IV” certification is an assertion that the patent does not block approval of the later product, either because the patent is invalid or unenforceable or because the patent, even if valid, is not infringed by the new product.

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Regulatory Exclusivities. The Hatch-Waxman Act provides periods of regulatory exclusivity for products that would serve as RLDs for an ANDA or 505(b)(2) application. If a product is a “new chemical entity,” or NCE — generally meaning that the drug contains no active moiety that has been approved by the FDA in any other NDA submitted under section 505(b) of the FD&C Act — there is a period of five years from the product’s approval during which the FDA may not accept for filing any ANDA or 505(b)(2) application for a drug with the same active moiety. An ANDA or 505(b)(2) application may be submitted after four years, however, if the sponsor of the application makes a Paragraph IV certification.

A product that is not an NCE may qualify for a three-year period of exclusivity if the NDA contains new clinical data (other than bioavailability studies), derived from studies conducted by or for the sponsor, that were necessary for approval. In that instance, the exclusivity period does not preclude filing or review of an ANDA or 505(b)(2) application; rather, the FDA is precluded from granting final approval to the ANDA or 505(b)(2) application until three years after approval of the RLD. Additionally, the exclusivity applies only to the conditions of approval that required submission of the clinical data.

Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD NDA holder and patent owner that the application has been submitted and provide the factual and legal basis for the applicant’s assertion that the patent is invalid or not infringed. If the NDA holder or patent owner files suit against the ANDA or 505(b)(2) applicant for patent infringement within 45 days of receiving the Paragraph IV notice, the FDA is prohibited from approving the ANDA or 505(b)(2) application for a period of 30 months or the resolution of the underlying suit, whichever is earlier. If the RLD has NCE exclusivity and the notice is given and suit filed during the fifth year of exclusivity, the regulatory stay extends to 7.5 years after the RLD approval. The FDA may approve the proposed product before the expiration of the regulatory stay if a court finds the patent invalid or not infringed or if the court shortens the period because the parties have failed to cooperate in expediting the litigation.

Patent Term Restoration. A portion of the patent term lost during product development and FDA review of an NDA is restored if approval of the application is the first permitted commercial marketing of a drug containing the active ingredient. The patent term restoration period is generally one-half the time between the effective date of the IND or the date of patent grant (whichever is later) and the date of submission of the NDA, plus the time between the date of submission of the NDA and the date of FDA approval of the product. The maximum period of restoration is five years, and the patent cannot be extended to more than 14 years from the date of FDA approval of the product. Only one patent claiming each approved product is eligible for restoration and the patent holder must apply for restoration within 60 days of approval. The U.S. Patent and Trademark Office (USPTO), in consultation with the FDA, reviews and approves the application for patent term restoration.

European and Other International Government Regulation

In addition to regulations in the U.S., we are subject to a variety of regulations in other jurisdictions governing, among other things, clinical trials and any commercial sales and distribution of our products. Whether or not we obtain FDA approval for a product, we must obtain the requisite approvals from regulatory authorities in foreign countries prior to the commencement of clinical trials or marketing of the product in those countries. Some countries outside of the U.S. have a similar process that requires the submission of a clinical trial application, or CTA, much like the IND prior to the commencement of human clinical trials. In the EU, for example, similar to the FDA a CTA must be submitted for authorization via the platform ‘Clinical Trials Information System (CTIS) to the competent national authority of each EU Member State (as well as Iceland, Norway and Liechtenstein) in which the clinical trial is to be conducted. Furthermore, the applicant may only start a clinical trial at a specific study site after the competent ethics committee, much like the IRB, has issued a favorable opinion. Once authorized in accordance with the EU Clinical Trials Regulation (Regulation (EU) No 536/2014 on clinical trials on medicinal products for human use), the clinical trial can be initiated in the authorizing EU Member States.

The EU Clinical Trials Regulation entered into force on January 31, 2022, repealing the previous EU Clinical Trials Directive (Directive (EC) 2001/20/EC) and the related national implementing provisions of the individual EU Member States. Applications through the CTIS are mandatory from January 31, 2023. Clinical trials authorized under the Clinical Trials Directive before January 31, 2023, can continue to be conducted under the EU Clinical Trials Directive until January 31, 2025. From January 31, 2025, any trials approved under the EU Clinical Trials Directive that continue running will need to comply with the EU Clinical Trials Regulation and their sponsors must have recorded information on them in the CTIS.

To obtain regulatory approval to commercialize a new drug under EU regulatory systems, we must submit an MAA, to the competent regulatory authority. In the EU, marketing authorization for a medicinal product can be obtained through a centralized, mutual recognition, or decentralized procedure, or via the national procedure of an individual EU Member State. A marketing authorization, irrespective of its route to authorization, may be granted only to an applicant established in the EU.

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The centralized procedure provides for the grant of a single marketing authorization by the European Commission that is valid for all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway. Under the centralized procedure, the Committee for Medicinal Products for Human Use, or the CHMP, established at the EMA is responsible for conducting the initial assessment of a product. The maximum timeframe for the evaluation of an MAA is 210 days. This period excludes clock stops during which additional information or written or oral explanations are to be provided by the applicant in response to questions posed by the CHMP. A request for accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of a major public health interest. There is no single definition of what constitutes major public health interest. This should be justified by the applicant and assessed by the CHMP on a case by case basis. Typically, the justification should include the major benefits expected and demonstrate that the medicinal product introduces new methods of therapy or improves on existing methods, thereby addressing to a significant extent public health unmet needs. If the CHMP accepts to review a medicinal product under the accelerated assessment procedure, the time limit of 210 days will be reduced to 150 days. It is, however, possible that the CHMP can revert to the standard time limit for the centralized procedure if it considers that it is no longer appropriate to conduct an accelerated assessment.

Irrespective of the related procedure, at the completion of the review period the CHMP will provide a scientific opinion concerning whether or not a marketing authorization should be granted in relation to a medicinal product. This opinion is based on a review of the quality, safety, and efficacy of the product. Within 15 days of the adoption, the EMA will forward its opinion to the European Commission for its decision. Following the opinion of the EMA, the European Commission makes a final decision to grant or not a centralized marketing authorization. The centralized procedure is mandatory for certain types of medicinal products, including orphan medicinal products, medicinal products derived from certain biotechnological processes, advanced therapy medicinal products and medicinal products containing a new active substance for the treatment of certain diseases. This route is optional for certain other products, including medicinal products that are of significant therapeutic, scientific or technical innovation, or whose authorization would be in the interest of patients’ health at EU level.

Unlike the centralized authorization procedure, the decentralized marketing authorization procedure requires a separate application to, and leads to separate authorization by, the competent authorities of each EU Member State in which the product is to be marketed. This application process is identical to the application that would be submitted to the EMA for authorization through the centralized procedure and must be completed within 210 days, excluding potential clock-stops, during which the applicant can respond to questions. The reference EU Member State prepares a draft assessment and drafts of the related materials. The other concerned EU Member States must decide whether to approve the assessment report and related materials. If a concerned EU Member State cannot approve the assessment report and related materials due to concerns relating to a potential serious risk to public health, disputed elements will be referred to a coordination group and could thereafter be referred to the EMA, which could result in a decision from the European Commission, binding on all EU Member States.

The mutual recognition procedure is used in order to obtain marketing authorizations in several Member States where the medicinal product in question has already received a marketing authorization in any Member State at the time of application. The holder of a national marketing authorization may submit an application to the competent authority of an EU Member State requesting that this authority recognize the marketing authorization delivered by the competent authority of another EU Member State.

For our products and product candidates that combine drug and device components (‘combination products’), the rules applicable vary depending on the specific combination. If the principal intended action of the product is achieved by the drug, the product is considered a drug that includes a medical device. The entire product is regulated under EU pharmaceutical legislation and must obtain a marketing authorization in the terms explained above. For the device part of the combination, the MAA should include a CE Certificate of Conformity or Declaration of Conformity (for the case of Class I non-sterile, non-measuring, or non-reusable surgical instruments). If the device is not certified but would require notified body involvement to be certified if marketed separately, an opinion from an EU notified body on the conformity of the device with applicable requirements will be required. If, however, the device is co-packaged or obtained separately from the drug product, it must be CE-marked under, and will be governed by, the EU medical devices legislation (Regulation (EU) 2017/745 on medical devices or the previous Directives 90/385/EEC and 93/42/EEC). If the principal intended action in the product is achieved by the medical device (and the action of the drug is only ancillary to that of the device), the entire product is regulated as a medical device and should be CE-marked under the EU medical devices legislation.

Marketing authorization holders are subject to comprehensive regulatory oversight by the EMA and the competent authorities of the individual EU Member States both before and after grant of marketing authorization. This includes control of compliance by the entities with EU cGMP rules, which govern quality control of the manufacturing process and require documentation policies and procedures. The advertising and promotion of medicinal products are also subject to the EU Member States’ laws governing promotion of medicinal products, interactions with physicians and other healthcare professionals, misleading and comparative advertising and unfair commercial practices. Breaches of the rules governing the promotion of medicinal products in the EU could give rise to civil, criminal or administrative penalties, which may include fines and imprisonment.

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For other countries outside of the EU, such as countries in Eastern Europe, Latin America, or Asia, the requirements governing the conduct of clinical trials, product licensing, pricing, and reimbursement vary from country to country. Internationally, clinical trials are generally required to be conducted in accordance with GCP, applicable regulatory requirements of each jurisdiction and the medical ethics principles that have their origin in the Declaration of Helsinki.

Compliance

During all phases of development and in the post-market setting, failure to comply with applicable regulatory requirements may result in administrative or judicial sanctions. These sanctions could include the FDA’s imposition of a clinical hold on trials, refusal to approve pending applications, withdrawal of an approval, warning letters or untitled letters, product recalls, product seizures, total or partial suspension of production or distribution, product detention or refusal to permit the import or export of products, injunctions, fines, civil penalties or criminal prosecution. Third country authorities can impose equivalent penalties. Any agency or judicial enforcement action could have a material adverse effect on us.

Other Exclusivities

Pediatric Exclusivity. Section 505A of the FD&C Act provides for six months of additional exclusivity or patent protection if an NDA sponsor submits pediatric data that fairly respond to a Written Request from the FDA for such data. The data do not need to show that the product is effective in the pediatric population studied; rather, if the clinical trial is deemed to fairly respond to the FDA’s request, the additional protection is granted. If reports of requested pediatric studies are submitted to and accepted by FDA within the statutory time limits, whatever statutory or regulatory periods of exclusivity or Orange Book listed patent protection that cover the drug are extended by six months. This is not a patent term extension, but it effectively extends the regulatory period during which the FDA cannot approve an ANDA or 505(b)(2) application owing to regulatory exclusivity or listed patents. When any product is approved, we will evaluate seeking pediatric exclusivity as appropriate.

In the EU, Regulation No 1901/2006 (Pediatric Regulation) requires marketing authorization applications to include the results of all studies performed and details of all information collected in compliance with an EMA-approved Pediatric Investigation Plan (PIP), unless the EMA has granted either, a product-specific waiver, a class waiver, or a deferral for one or more of the measures included in the PIP. This PIP covers all subsets in a pediatric population. Medicinal products authorized with the results of studies from the agreed PIP included in the product information are eligible for a six-month extension period of qualifying Supplementary Protection Certificates (SPC).

The EU pharmaceutical legislation is currently under review. On April 26, 2023, the European Commission published its proposal to revise the EU pharmaceutical legislation (the “EU pharma package”), consisting of a new Directive and a new Regulation, which would revise and replace the existing general pharmaceutical legislation (Regulation 726/2004 and Directive 2001/83/EC) and the Pediatric Regulation and Regulation (EC) No 141/2000 on orphan medicinal products. In December 2025, the European Parliament and the Council of the European Union reached a provisional agreement on the EU pharma package. Among others, this provisional agreement contemplates certain changes to regulatory exclusivity periods (see in more detail below). The timeframe for the evaluation of an MAA is also expected to be reduced to 180 days (from the current 210 days). The 6-month SPC extension for when the MAA includes the results of all studies conducted in compliance with an agreed PIP is kept. The provisional agreement is still subject to the formal approval by the European Parliament and the Council of the European Union and subsequent publication in the Official Journal of the European Union (“OJEU”), likely to occur in the first or second quarter of 2026. After a transition period, the new legislation is expected to apply from mid-2028.

Orphan Drug Exclusivity. The Orphan Drug Act provides incentives for the development of drugs intended to treat rare diseases or conditions, which are diseases or conditions affecting less than 200,000 individuals in the U.S., or a disease or condition affecting more than 200,000 individuals in the U.S. but there is no reasonable expectation that the cost of developing and making the drug product would be recovered from sales in the U.S. If a sponsor demonstrates that a drug product qualifies for orphan drug designation, the FDA may grant orphan drug designation to the product for that use. The benefits of orphan drug designation include research and development tax credits and exemption from user fees. A drug that is approved for the orphan drug designated indication generally is granted seven years of orphan drug exclusivity. During that period, the FDA generally may not approve any other application for the same product for the same indication, although there are exceptions, most notably when the later product is shown to be clinically superior to the product with exclusivity. The FDA can revoke a product’s orphan drug exclusivity under certain circumstances, including when the product sponsor is unable to assure the availability of sufficient quantities of the product to meet patient needs. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same biologic for a different disease or condition.

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In the EU, medicinal products may be granted orphan designation if it can be established: (a) that they are intended to diagnose, treat or prevent life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU; or (b) that they are intended to diagnose, treat or prevent life-threatening, seriously debilitating or serious and chronic conditions and that, for economic reasons, would be unlikely to be developed without incentives; and (c) that no satisfactory method of diagnosis, prevention or treatment of the condition concerned exists, or, if such a method exists, the medicinal product would be of significant benefit to those affected by the condition. The application for orphan designation must be submitted to the EMA’s Committee for Orphan Medicinal Products and approved by the European Commission before an application is made for marketing authorization for the product. Once authorized, orphan medicinal product designation entitles an applicant to financial incentives such as reduction of fees or fee waivers. In addition, orphan medicinal products are entitled to ten years of market exclusivity following authorization. During this ten-year period, with a limited number of exceptions, neither the competent authorities of the EU Member States, the EMA, or the European Commission are permitted to accept applications or grant marketing authorization for other similar medicinal products with the same therapeutic indication. However, marketing authorization may be granted to a similar medicinal product with the same orphan indication during the ten-year period with the consent of the marketing authorization holder for the original orphan medicinal product, if the manufacturer of the original orphan medicinal product is unable to supply sufficient quantities, or if the second product is safer, more effective or otherwise clinically superior to the original orphan medicinal product. The period of market exclusivity may, in addition, be reduced to six years if it can be demonstrated on the basis of available evidence that the original orphan medicinal product is sufficiently profitable not to justify maintenance of market exclusivity.

As mentioned above, as part of the ongoing review of the EU pharmaceutical legislation, Regulation (EC) No 141/2000 on orphan medicinal products is expected to be repealed and replaced by a new regulation. Under the proposed text, the prevalence criterion of not more than five affected persons per 10,000 is maintained. With the purpose to expedite the orphan designation procedure, the EMA is given powers to itself adopt decisions granting, refusing and transferring an orphan designation. The duration of the market exclusivity will be reduced from the current 10 years to 9 years, except for well-established use orphan products which will benefit from 4 years. Also, “breakthrough orphan medicinal products” (those addressing a disease with no current available medicinal treatment) will benefit from up to 11 years of market exclusivity. The market exclusivity of the orphan medicinal product shall not prevent the submission, validation and assessment of an application for or granting a marketing authorization for a similar medicinal product, where the remainder of the duration of the market exclusivity is less than two years. The period of market exclusivity can be prolonged by an additional 12 months (except for well-established use orphan products) if at least two years before the end of the exclusivity period, the orphan marketing authorization holder obtains a marketing authorization for one or more new therapeutic indications for a different orphan condition. Such a prolongation may be granted twice. As mentioned above, this proposed legislative text is still subject to formal approval.

Data Exclusivity. In the EU, if a marketing authorization is granted for a medicinal product containing a new active substance, that product benefits from eight years of data exclusivity, during which generic marketing authorization applications referring to the data of that product may not be accepted by the regulatory authorities. The product also benefits from 10 years’ market exclusivity during which generic products, even if authorized, may not be placed on the market. The overall ten-year period will be extended to a maximum of 11 years if, during the first eight years of those ten years, the marketing authorization holder obtains an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies. As part of the review of the EU pharmaceutical legislation mentioned above, changes to regulatory exclusivity periods are expected. The regulatory data protection period is proposed to be kept at eight years, with one additional year of regulatory market protection. Pharmaceutical companies would be eligible for additional one-year periods of regulatory market protection: (i) if the particular product addresses an unmet medical need; (ii) if it contains a new active substance, fulfilling a combination of conditions on comparative clinical trials, clinical trials carried out in several Member States, and the obligation to apply for marketing authorization within 90 days after the submission of the first MAA outside the European Union; (iii) if the company obtains an authorization for one or more new therapeutic indications that bring a significant clinical benefit in comparison with existing therapies. The provisional agreement sets a cap of 11 years on the combined regulatory market protection period. As also mentioned above, this proposed legislative text is still subject to formal approval.

U.S. Healthcare Reform

The Patient Protection and Affordable Care Act, as amended, which we refer to as the Affordable Care Act, is a sweeping measure intended to expand healthcare coverage within the U.S., primarily through the imposition of certain health insurance mandates, the provision of subsidies to eligible individuals enrolled in plans offered on the health insurance exchanges, and expansion of the Medicaid program. This law substantially changed the way healthcare is financed by both governmental and private insurers and has significantly impacted the pharmaceutical industry. For further detail, please refer to the risk factor entitled “The Affordable Care Act and any changes in healthcare laws may increase the difficulty and cost for us to commercialize our future products in the U.S. and affect the prices we may obtain” set forth under the section titled “Risk Factors” in this Annual Report on Form 10-K.

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Some states have elected not to expand their Medicaid programs to certain individuals with an income of up to 133% of the federal poverty level, as is permitted under the Affordable Care Act. For each state that does not choose to expand its Medicaid program, there may be fewer insured patients overall, which could impact our sales of products and product candidates for which we receive regulatory approval, and our business and financial condition. Where patients receive insurance coverage under Medicaid, manufacturers may be required to pay Medicaid rebates on covered outpatient drugs, a decision that could impact manufacturer revenues.

Certain provisions of the Affordable Care Act have been subject to judicial challenges as well as efforts to modify them or to alter their interpretation and implementation. Additional legislative changes, regulatory changes, and judicial challenges related to the Affordable Care Act remain possible, but the nature and extent of such potential changes or challenges are uncertain at this time. It is unclear how the Affordable Care Act and its implementation, as well as efforts to modify or invalidate the Affordable Care Act, or portions thereof, or its implementation, will affect our business, financial condition, and results of operations. It is possible that the Affordable Care Act, as currently enacted or as it may be amended in the future, and other healthcare reform measures, including those that may be adopted in the future, could have a material adverse effect on our industry generally and on our ability to maintain or increase sales of our product candidates for which we receive regulatory approval or to successfully commercialize our product candidates.

Other legislative changes relating to reimbursement have been adopted in the U.S. since the Affordable Care Act was enacted. For example, the Budget Control Act of 2011, among other things, created the Joint Select Committee on Deficit Reduction to recommend to Congress proposals for spending reductions. The Joint Select Committee did not achieve a targeted deficit reduction, which triggered the legislation’s automatic reductions. In concert with subsequent legislation, this has resulted in aggregate reductions to Medicare payments to providers. Sequestration is currently set at 2% through the first seven months of fiscal year 2033. As long as these cuts remain in effect, they could adversely impact payment for any products we may commercialize in the future. We expect that additional federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, and in turn could significantly reduce the projected value of certain development projects and reduce our profitability.

The Inflation Reduction Act of 2022 (IRA) includes several drug pricing policies that are intended to reduce costs for the Medicare program and its beneficiaries, as well as a variety of provisions on the environment and clean energy, corporate taxes, and other health care policies. For further detail, please refer to the risk factor entitled "The Inflation Reduction Act of 2022 and other changes in healthcare law may impact the prices we are able to obtain for our products and our obligations to make payments to the government” set forth under the section titled “Risk Factors” in this Annual Report on Form 10-K.

On July 4, 2025, the “One Big Beautiful Bill Act,” or OBBBA, was signed into law. The OBBBA is projected to decrease federal health care spending by approximately $1 trillion by reducing Medicaid spending and enrollment and making changes to federal Medicare spending. The law also made changes to ACA marketplace enrollment that are projected to decrease the number of individuals with marketplace coverage. It is unclear if these changes will impact demand for our products.

Additional legislative changes, regulatory changes, or guidance could be adopted, which may impact the marketing approvals and reimbursement for our product candidates. For example, there has been increasing legislative, regulatory, and enforcement interest in the United States with respect to drug pricing practices. There have been several Congressional inquiries and proposed and enacted federal and state legislation and regulatory initiatives designed to, among other things, bring more transparency to product pricing, evaluate the relationship between pricing and manufacturer patient programs, and reform government healthcare program reimbursement methodologies for drug products. Individual states in the United States have also enacted legislation and implemented regulations designed to control pharmaceutical product pricing, including by establishing Prescription Drug Affordability Boards (or similar entities) to review high-cost drugs and, in some cases, set upper payment limits, and by implementing marketing cost disclosure and transparency measures. If healthcare policies intended to curb healthcare costs are adopted or if we experience negative publicity with respect to pricing of our products or the pricing of drugs generally, the prices that we charge for any approved products may be limited, our commercial opportunity may be limited, and/or our revenues from sales of any commercialized products may be negatively impacted.

Coverage and Reimbursement

Sales of any of our product candidates, if approved and once commercialized, depend, in part, on the extent to which the costs of the product will be covered by Medicare and Medicaid, and private payors, such as commercial health insurers and managed care organizations. Third-party payors determine which drugs they will cover and the amount of reimbursement they will provide for a covered drug. In the U.S., there is no uniform system among payors for making coverage and reimbursement decisions. In addition, the process for determining whether a payor will provide coverage for a product may be separate from the process for setting the price

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or reimbursement rate that the payor will pay for the product once coverage is approved. Payors may limit coverage to specific products on an approved list, or formulary, which might not include all of the FDA-approved products for a particular indication.

In order to secure coverage and reimbursement for our products, we may need to conduct expensive pharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of the product, in addition to the costly studies required to obtain FDA or other comparable regulatory approvals. Even if we conduct pharmacoeconomic studies, our products may not be considered medically necessary or cost-effective by payors. Further, a payor’s decision to provide coverage for a product does not guarantee that an adequate reimbursement rate will be set, including because health care providers (HCPs) negotiate their own reimbursement directly with commercial payors.

In the past, payors have implemented reimbursement metrics and periodically revised those metrics as well as the methodologies used as the basis for reimbursement rates, such as average sales price, or ASP, average manufacturer price, or AMP, and actual acquisition cost. The existing data for reimbursement based on these metrics is relatively limited, although certain states have begun to survey acquisition cost data for the purpose of setting Medicaid reimbursement rates. CMS surveys and publishes retail pharmacy acquisition cost information in the form of National Average Drug Acquisition Cost files to provide state Medicaid agencies with a basis of comparison for their own reimbursement and pricing methodologies and rates.

We have participated in and, if we obtain approval to commercialize additional products, we expect to participate in, and would have certain price reporting obligations with respect to, the Medicaid Drug Rebate Program. This program would require us to pay a rebate for each unit of drug reimbursed by Medicaid. The amount of the “basic” portion of the rebate for each product is set by law as the larger of: (i) 23.1% of quarterly AMP, or (ii) the difference between quarterly AMP and the quarterly best price available from us to any commercial or non-governmental customer, or Best Price. AMP must be reported on a monthly and quarterly basis and Best Price is reported on a quarterly basis only. In addition, the rebate also includes the “additional” portion, which adjusts the overall rebate amount upward as an “inflation penalty” when the drug’s latest quarter’s AMP exceeds the drug’s AMP from the first full quarter of sales after launch, adjusted for increases in the Consumer Price Index-Urban. The upward adjustment in the rebate amount per unit is equal to the excess amount of the current AMP over the inflation-adjusted AMP from the first full quarter of sales. Rebates under the Medicaid Drug Rebate Program are no longer subject to a cap as of January 1, 2024. The rebate amount would be computed each quarter based on our report to CMS of current quarterly AMP and Best Price for our drugs, if commercialized. We would be required to report revisions to AMP or Best Price within a period not to exceed 12 quarters from the quarter in which the data was originally due. Any such revisions could have the impact of increasing or decreasing our rebate liability for prior quarters, depending on the direction of the revision. CMS has issued final regulations to implement the Medicaid Drug Rebate Program under the Affordable Care Act.

Federal law requires that any manufacturer that participates in the Medicaid Drug Rebate Program also participate in the Public Health Service’s 340B drug pricing program in order for federal funds to be available for the manufacturer’s drugs under Medicaid and Medicare Part B. The 340B program, which is administered by the Health Resources and Services Administration, or HRSA, requires participating manufacturers to agree to charge statutorily defined covered entities no more than the 340B “ceiling price” for the manufacturer’s covered outpatient drugs. These 340B covered entities include a variety of community health clinics and other entities that receive health services grants from the Public Health Service, as well as hospitals that serve a disproportionate share of low-income patients. The 340B ceiling price is calculated using a statutory formula, which is based on the AMP and rebate amount for the covered outpatient drug as calculated under the Medicaid Drug Rebate Program. Any changes to the definition of AMP and the Medicaid rebate amount under the Affordable Care Act or other legislation could affect our 340B ceiling price calculations and negatively impact our results of operations.

HRSA has issued a final regulation regarding the calculation of the 340B ceiling price and the imposition of civil monetary penalties on manufacturers that knowingly and intentionally overcharge covered entities. It is unclear how HRSA will apply its enforcement authority under this regulation. HRSA has also implemented a ceiling price reporting requirement related to the 340B program under which we would be required to report 340B ceiling prices to HRSA on a quarterly basis, which HRSA would then publish information to covered entities. Moreover, under final regulations HRSA has established an administrative dispute resolution (ADR) process for claims by covered entities that a manufacturer has engaged in overcharging, and by manufacturers that a covered entity violated the prohibitions against diversion or duplicate discounts. Such claims are to be resolved through an ADR panel of government officials rendering a decision that may be appealed to federal court. An ADR proceeding could subject us to onerous procedural requirements and could result in additional liability. In addition, legislation may be introduced that, if passed, would, for example, further expand the 340B program to additional covered entities or would require participating manufacturers to agree to provide 340B discounted pricing on drugs used in an inpatient setting.

Federal law also requires that a company that participates in the Medicaid Drug Rebate program report ASP information each quarter to CMS for certain categories of drugs that are paid under the Medicare Part B program. For calendar quarters beginning

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January 1, 2022, manufacturers are required to report the average sales price for certain drugs under the Medicare program regardless of whether they participate in the Medicaid Drug Rebate Program. Manufacturers calculate the ASP based on a statutorily defined formula as well as regulations and interpretations of the statute by CMS. CMS may use these submissions to determine payment rates for drugs under Medicare Part B. Starting in 2023, manufacturers must pay refunds to Medicare for single source drugs or biologicals, or biosimilar biological products, reimbursed under Medicare Part B and packaged in single-dose containers or single-use packages, for units of discarded drug reimbursed by Medicare Part B in excess of 10 percent of total allowed charges under Medicare Part B for that drug. Manufacturers that fail to pay refunds could be subject to civil monetary penalties of 125 percent of the refund amount. For more information about Medicare Part B, refer to the risk factor entitled “Our products and product candidates, if approved and commercialized, may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives which could harm our business” set forth under the section titled “Risk Factors” in this Annual Report on Form 10-K.

Statutory or regulatory changes or CMS guidance could affect the pricing of our approved products, once commercialized, and could negatively affect our results of operations. The IRA, among other things, requires the Secretary of Health and Human Services Secretary to negotiate, with respect to Medicare units and subject to a specified cap, the price of a set number of certain high Medicare spend drugs and biologicals per year with the first negotiated prices taking effect starting in 2026. The IRA established a Medicare Part B inflation rebate scheme, under which, generally speaking, manufacturers will owe rebates if the average sales price of a Part B drug increases faster than the pace of inflation. Failure to timely pay a Part B inflation rebate is subject to a civil monetary penalty. These or any other public policy changes could impact the market conditions for our product candidates. We further expect continued scrutiny on government price reporting and pricing more generally from Congress, agencies, and other bodies. For more information about Medicare Part B, refer to the risk factor entitled “Our products and product candidates, if approved and commercialized, may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives which could harm our business” set forth under the section titled “Risk Factors” in this Annual Report on Form 10-K.

In the U.S. Medicare program, certain outpatient prescription drugs may be covered under Medicare Part D. Medicare Part D is a voluntary prescription drug benefit, through which Medicare beneficiaries may enroll in prescription drug plans offered by private entities for coverage of certain outpatient prescription drugs. Part D plans include both stand-alone prescription drug benefit plans and prescription drug coverage as a supplement to Medicare Advantage plans provided for under Medicare Part C.

Coverage and reimbursement for covered outpatient drugs under Part D are not standardized. Part D prescription drug plan sponsors are not required to pay for all covered Part D drugs, and each drug plan generally can develop its own drug formulary that identifies which drugs it will cover and at what tier or level. Any formulary used by a Part D prescription drug plan must be developed and reviewed by a pharmacy and therapeutic committee. Although Part D prescription drug formularies must include drugs within each therapeutic category and class of covered Part D drugs, they have some flexibility to establish those categories and classes and are not required to cover all of the drugs in each category or class. Medicare Part D prescription drug plans may use formularies to limit the number of drugs that will be covered in any therapeutic class and/or impose differential cost sharing or other utilization management techniques.

Medicare Part D coverage may be available for any future product candidates for which we receive marketing approval and commercialize. However, in order for the products that we market to be included on the formularies of Part D prescription drug plans, we likely will have to offer pricing that is lower than the prices we might otherwise obtain. Changes to Medicare Part D that give plans more freedom to limit coverage or manage utilization, and other cost reduction initiatives in the program, could decrease the coverage and price that we receive for any approved products and could seriously harm our business.

In addition, manufacturers were required to provide to CMS a 70% discount on brand name prescription drugs utilized by Medicare Part D beneficiaries when those beneficiaries are in the coverage gap phase of the Part D benefit design, through December 31, 2024. The IRA sunset the coverage gap discount program starting in 2025 and replaced it with a new manufacturer discount program, under which manufacturers provide a 10% discount on a covered Part D drug where a beneficiary is in the initial phase of Part D coverage and a 20% discount where a beneficiary is in the catastrophic phase of Part D coverage. The IRA also makes other reforms to the Part D benefit, which could increase our liability under Part D. Further, the IRA establishes a Medicare Part D inflation rebate scheme, under which, generally speaking, manufacturers will owe additional rebates if the AMP of a Part D drug increases faster than the pace of inflation. Failure to timely pay a Part D inflation rebate is subject to a civil monetary penalty.

In order to be eligible to have our products paid for with federal funds under the Medicaid and Medicare Part B programs and purchased by certain federal agencies and grantees, we must participate in the U.S. Department of Veterans Affairs, (VA), Federal Supply Schedule, (FSS), pricing program. Under this program, we are obligated to make our “innovator” drugs available for procurement on an FSS contract and charge a price to four federal agencies — the VA, U.S. Department of Defense, (DoD), Public Health Service and U.S. Coast Guard — that is no higher than the statutory Federal Ceiling Price, (FCP). The FCP is based on the non-federal average manufacturer price, (Non-FAMP), which we calculate and report to the VA on a quarterly and annual basis. We

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also may participate in the Tricare Retail Pharmacy program, under which we would pay quarterly rebates on utilization of innovator products that are dispensed through the Tricare Retail Pharmacy network to Tricare beneficiaries. The rebates are calculated as the difference between the annual Non-FAMP and FCP.

Pricing and rebate calculations vary across products and programs, are complex, and are often subject to interpretation by us, governmental or regulatory agencies, and the courts. We could be held liable for errors associated with the submission of pricing data. In addition to retroactive Medicaid rebates and the potential for issuing 340B program refunds, if we are found to knowingly submit false AMP, Best Price, or Non-FAMP information to the government, we may be liable for significant civil monetary penalties per item of false information. If we are found to have made a misrepresentation in the reporting of our ASP, the Medicare statute provides for significant civil monetary penalties for each misrepresentation for each day in which the misrepresentation was applied. Our failure to submit monthly/quarterly AMP and Best Price data on a timely basis could result in a significant civil monetary penalty per day for each day the information is late beyond the due date. Such conduct also could be grounds for CMS to terminate our Medicaid drug rebate agreement, in which case federal payments may not be available under Medicaid or Medicare Part B for our covered outpatient drugs. Significant civil monetary penalties also could apply to late submissions of Non-FAMP information. Civil monetary penalties could also be applied if we are found to have charged 340B covered entities more than the statutorily mandated ceiling price or HRSA could terminate an agreement to participate in the 340B program, in which case federal payments may not be available under Medicaid or Medicare Part B for our covered outpatient drugs. In addition, claims submitted to federally-funded healthcare programs, such as Medicare and Medicaid, for drugs priced based on incorrect pricing data provided by a manufacturer can implicate the federal civil False Claims Act. Civil monetary penalties could be due if a manufacturer fails to offer discounts to beneficiaries under the Medicare Part D coverage gap discount program. Furthermore, under the refund program for discarded drugs, manufacturers that fail to pay refunds could be subject to civil monetary penalties of 125 percent of the refund amount.

The containment of healthcare costs has become a priority of federal, state and foreign governments, and the prices of drugs have been a focus in this effort. The U.S. government, state legislatures, and foreign governments have shown significant interest in implementing cost-containment programs to limit the growth of government-paid healthcare costs, including price controls, restrictions on reimbursement, and requirements for substitution of generic products for branded prescription drugs. For example, there have been several recent U.S. Congressional inquiries and proposed federal and state legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs, and reform government program reimbursement methodologies for drug products. The federal government has shown substantial interest in taking a variety of measures aimed at lowering U.S. prescription drug prices to align with the lowest prices available for the same drugs in comparably developed nations (so called “most favored nation” pricing). At the state level, some states have passed laws that regulate how manufacturers make the 340B Drug Pricing Program ceiling price available on the market.

There likely will continue to be proposals by legislators at both the federal and state levels, regulators, and third-party payors to contain healthcare costs. Thus, even if we obtain favorable coverage and reimbursement status for our products and any product candidates for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.

Different pricing and reimbursement schemes exist in other countries. In the EU, each EU Member State can restrict the range of medicinal products for which its national health insurance system provides reimbursement and can control the prices of medicinal products for human use marketed on its territory. As a result, following receipt of marketing authorization in an EU Member State, through any application route, the applicant is required to engage in pricing discussions and negotiations with the competent pricing authority in the individual EU Member State. The governments of the EU Member States influence the price of pharmaceutical products through their pricing and reimbursement rules and control of national healthcare systems that fund a large part of the cost of those products to consumers. Some EU Member States operate positive and negative list systems under which products may only be marketed once a reimbursement price has been agreed upon. To obtain reimbursement or pricing approval, some of these countries may require the completion of clinical trials that compare the cost-effectiveness of a particular product candidate to currently available therapies. Others adopt a system of reference pricing, basing the price or reimbursement level in their territories either on the pricing and reimbursement levels in other countries or on the pricing and reimbursement levels of medicinal products intended for the same therapeutic indication. Further, some EU Member States approve a specific price for the medicinal product or may instead adopt a system allowing companies to fix their own, but with direct or indirect controls on the profitability of the company placing the medicinal product on the market. The downward pressure on healthcare costs in general, particularly prescription drugs, has become more intense. As a result, increasingly high barriers are being erected to the entry of new products. In addition, we may face competition for our product candidates from lower-priced products in foreign countries that have placed price controls on pharmaceutical products. In addition, in some countries, cross-border imports from low-priced markets exert a commercial pressure on pricing within a country.

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Health Technology Assessment, or HTA, of medicinal products, however, is becoming an increasingly common part of the pricing and reimbursement procedures in some EU Member States. These EU Member States include France, Germany, Ireland, Italy, and Sweden. HTA is the procedure according to which the assessment of the public health impact, therapeutic impact and the economic and societal impact of use of a given medicinal product in the national healthcare systems of the individual country is conducted. HTA generally focuses on the clinical efficacy and effectiveness, safety, cost, and cost-effectiveness of individual medicinal products as well as their potential implications for the healthcare system. Those elements of medicinal products are compared with other treatment options available on the market.

The outcome of HTA regarding specific medicinal products will often influence the pricing and reimbursement status granted to these medicinal products by the competent authorities of individual EU Member States. The extent to which pricing and reimbursement decisions are influenced by the HTA of the specific medicinal product varies between EU Member States.

The EU HTA Regulation was adopted on December 13, 2021, and started to apply to all EU Member States from January 12, 2025.

The HTA Regulation provides that EU Member States will be able to use common HTA tools, methodologies, and procedures across the EU and sets the basis for permanent and sustainable cooperation at the EU level for joint clinical assessments. Individual EU Member States will continue to be responsible for drawing conclusions on the overall value of a new health technology for their healthcare system, and pricing and reimbursement decisions.

Healthcare Fraud and Abuse Laws

In addition to FDA restrictions on marketing of pharmaceutical products, if and when we commercialize our product candidates, our relationship with customers and third party payors will be subject to applicable anti-kickback, fraud and abuse, and other laws and regulations. These laws include, but are not limited to the following:

The federal Anti-Kickback Statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving remuneration, directly or indirectly, in cash or in kind, to induce or in return for purchasing, leasing, ordering or arranging for or recommending the purchase, lease or order of any healthcare item or service reimbursable, in whole or in part, under Medicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted to apply to arrangements between pharmaceutical manufacturers on one hand and prescribers, purchasers, and formulary managers on the other. A violation of the Anti-Kickback Statute may be established without proving actual knowledge of the statute or specific intent to violate it. The government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal civil False Claims Act.

The federal civil False Claims Act prohibits any person from, among other things, knowingly presenting, or causing to be presented, a false or fraudulent claim for payment of government funds, or knowingly making, using, or causing to be made or used, a false record or statement material to an obligation to pay money to the government or knowingly concealing or knowingly and improperly avoiding, decreasing, or concealing an obligation to pay money to the federal government. Actions under the False Claims Act may be brought by private individuals known as qui tam relators in the name of the government and to share in any monetary recovery.

The Health Insurance Portability and Accountability Act of 1996 and its implementing regulations (collectively HIPAA) prohibits, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program, including private third-party payors. HIPAA also prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement or representation, or making or using any false writing or document knowing the same to contain any materially false, fictitious or fraudulent statement or entry in connection with the delivery of or payment for healthcare benefits, items or services. We may obtain health information from third parties that are subject to privacy and security requirements under HIPAA and we could potentially be subject to criminal penalties if we, our affiliates, or our agents knowingly obtain individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA.

The majority of states, as well as many of the non-U.S. jurisdictions where we may operate, also have statutes or regulations similar to the federal anti-kickback and false claims laws, which apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payor. Several states now require pharmaceutical companies to report expenses relating to the marketing and promotion of pharmaceutical products in those states and to report gifts and payments to individual HCPs in those states. Some of these states also prohibit certain marketing-related activities including the provision of gifts, meals, or other items to certain HCPs. Other states have laws requiring pharmaceutical sales representatives to be registered or

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licensed, and still others impose limits on co-pay assistance that pharmaceutical companies can offer to patients. In addition, several states require pharmaceutical companies to implement compliance programs or marketing codes.

The Physician Payments Sunshine Act, implemented as the Open Payments program, and its implementing regulations, requires manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to CMS information related to direct or indirect payments and other transfers of value to physicians, physician assistants, nurse practitioners, clinical nurse specialists, certified nurse anesthetists, certified nurse-midwives, and teaching hospitals, as well as ownership and investment interests held in the company by physicians and their immediate family members. Many of the non-U.S. jurisdictions where we operate also have equivalent laws requiring us to report transfers of value to healthcare professionals.

Compliance with such laws and regulations will require substantial resources. Because of the breadth of these various fraud and abuse laws, it is possible that some of our business activities could be subject to challenge under one or more of such laws. Such a challenge could have material adverse effects on our business, financial condition and results of operations. In the event governmental authorities conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations, they may impose sanctions under these laws, which are potentially significant and may include civil monetary penalties, damages, exclusion of an entity or individual from participation in government health care programs, criminal fines and imprisonment, additional reporting requirements if we become subject to a corporate integrity agreement or other settlement to resolve allegations of violations of these laws, as well as the potential curtailment or restructuring of our operations. Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity.

Healthcare Privacy Laws

We may be subject to federal, state, and foreign laws and regulations governing data privacy and security of health information, and the collection, use, disclosure, and protection of health-related and other personal information, including state data breach notification laws, state health information and/or genetic privacy laws, and federal and state consumer protection laws, such as Section 5 of the FTC Act and the Health Breach Notification Rule, many of which differ from each other in significant ways, thus complicating compliance efforts. Compliance with these laws is complex, constantly evolving, and time consuming. Many of these state laws enable a state attorney general to bring actions and provide private rights of action to consumers as enforcement mechanisms. There is also heightened sensitivity around certain types of health information, such as sensitive condition information or the health information of minors, which may be subject to additional protections. Federal regulators, state attorneys general, and plaintiffs’ attorneys, including class action attorneys, have been and will likely continue to be active in this space.

The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues which may affect our business. Failure to comply with these laws and regulations could result in government scrutiny and enforcement actions and create liability for us (including the imposition of significant civil and/or criminal penalties), private litigation and/or adverse publicity that could negatively affect our business. We may obtain health information from third parties, such as HCPs who prescribe our products, and research institutions we collaborate with, who are subject to privacy and security requirements under HIPAA. Although we are not directly subject to HIPAA, other than potentially with respect to providing certain employee benefits, we could be subject to criminal penalties if we or our affiliates or agents knowingly obtain individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA.

In California, the California Consumer Privacy Act (CCPA) establishes certain requirements for data use and sharing transparency and provides California consumers (as defined in the law) certain rights concerning the use, disclosure, and retention of their personal data and nearly two dozen other states have enacted similar privacy laws or laws that broadly govern consumer data. For additional information, please refer to the risk factor entitled “If we fail to comply with data protection laws and regulations, we could be subject to government enforcement actions, which could include civil or criminal penalties, as well as private litigation and/or adverse publicity, any of which could negatively affect our operating results and business” set forth under the section titled “Risk Factors” in this Annual Report on Form 10-K. Health-specific consumer privacy laws were also passed in multiple states, including Washington and Nevada.

Outside the U.S., the legislative and regulatory landscape for privacy and data security continues to evolve. There has been increased attention to privacy and data security issues that could potentially affect our business, such as the EU General Data Protection Regulation (EU GDPR) and the UK General Data Protection Regulation (UK GDPR), which impose penalties for the most serious breaches of up to EUR 20 million/GBP 17.5 million or 4% of a company’s annual global revenue, whichever is greater. Both the EU and UK GDPR regulate the processing of personal data (including health data from clinical trials) and place certain obligations

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on the processing of such personal data including ensuring the lawfulness of processing personal data (including obtaining valid consent of the individuals to whom the personal data relates, where applicable), the processing details disclosed to the individuals, the adequacy, relevance and limitation of personal data to what is necessary in relation to the purposes for which they are processed, the sharing of personal data with third parties, the transfer of personal data out of the European Economic Area/UK to third countries including the U.S., contracting requirements (such as with clinical trial sites and vendors), the use of personal data in accordance with individual rights, the security of personal data and cybersecurity incident notifications. Data protection authorities from the different EU Member States and the UK may interpret the GDPR and applicable related national laws differently and impose requirements additional to those provided in the GDPR and that sit alongside the GDPR, as set out under applicable local data protection law. In addition, guidance on implementation and compliance practices may be issued, updated or otherwise revised. Enforcement by EU and UK regulators is generally active, and failure to comply with the GDPR or applicable EU Member State/UK local law may result in fines, amongst other things (such as notices requiring compliance within a certain timeframe). Further, the UK Government recently updated UK data protection law, which may result in changes to our business operations and potentially incur commercial cost.

European/UK data protection laws, including the EU and UK GDPR, generally restrict the transfer of personal data from the European Economic Area (EEA) and UK to the U.S. and most other countries (except those deemed to be adequate by the European Commission/UK Science and Technology Secretary of State as applicable) unless the parties to the transfer have implemented specific safeguards to protect the transferred personal data. On July 10, 2023, the European Commission adopted its adequacy decision for the EU-U.S. Data Privacy Framework, enabling personal data to be transferred from the EU to U.S. organizations that are certified under the Data Privacy Framework (DPF). The UK has formally approved multiple mechanisms for transferring UK data overseas: the International Data Transfer Agreement, the International Data Transfer Addendum to the SCCs, and UK binding corporate rules (BCRs), supported by a Transfer Risk Assessment. The UK also recognizes the UK-US Data Bridge for businesses in the U.S. that are certified under the UK Extension to the DPF as a valid transfer mechanism. The UK Information Commissioner’s Office has issued guidance on how to approach undertaking risk assessments for transfers of UK data to non-adequate countries outside the UK.

A failure to transfer EU or UK data under a valid transfer mechanisms could increase exposure to enforcement actions as described above and may affect our business operations and require commercial cost (including potentially limiting our ability to collaborate/work with certain third parties and/or requiring an increase in our data processing capabilities in the EU/UK). Further, the European/UK data protection laws (including laws on data transfers as set out above) may also be updated/revised, accompanied by new guidance and/or judicial/regulatory interpretations, which could entail further impacts on our compliance efforts and increased cost.

Foreign Corrupt Practices Act

In addition, the U.S. Foreign Corrupt Practices Act of 1977, as amended, (FCPA), prohibits corporations and individuals from engaging in certain activities to obtain or retain business or to influence a person working in an official capacity. It is illegal to pay, offer to pay or authorize the payment of anything of value to any official of another country, government staff member, political party or political candidate in an attempt to obtain or retain business or to otherwise influence a person working in that capacity.

Environmental Laws

Compliance with applicable environmental laws and regulations is expensive, and current or future environmental regulations may impair our research, development and production efforts, which could harm our business, prospects, financial condition or results of operations.

Corporate Information

We were incorporated under the laws of the state of Delaware on March 19, 2008, under the name New pSivida, Inc. Our predecessor, pSivida Limited, was formed in December 2000 as an Australian company incorporated in Western Australia. We subsequently changed our name to pSivida Corp. in May 2008,to EyePoint Pharmaceuticals, Inc. in March 2018 and EyePoint, Inc. in December 2025. Our principal executive office is located at 480 Pleasant Street, Suite C400, Watertown, Massachusetts 02472, and our telephone number is (617) 926-5000.

Additional Information

Our website address is www.eyepoint.bio. Information contained on, or connected to, our website is not incorporated by reference into this Annual Report on Form 10-K. Copies of this Annual Report on Form 10-K, and our annual reports on Form 10-K, proxy statements, quarterly reports on Form 10-Q, current reports on Form 8-K and, if applicable, amendments to those reports filed or furnished pursuant to Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended, are available free of charge

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through our website under “Investors – Financial Information – SEC Filings” as soon as reasonably practicable after we electronically file these materials with, or otherwise furnish them to, the SEC. The SEC maintains an Internet site that contains reports, proxy and information statements and other information regarding issuers that file electronically with the SEC at www.sec.gov.