Ocular Therapeutix™ Announces Plans to Accelerate NDA Submission Timeline for AXPAXLI™ in Wet AMD

About this update from Ocular Therapeutix, Inc.

Ocular intends to submit AXPAXLI New Drug Application (NDA) for wet AMD shortly after year one data from SOL-1 , if positive SOL-1 topline data remain on track for 1Q 2026 Ocular plans to leverage the 505(b)(2) regulatory pathway for new drug approvals which has the potential to shorten the review timeline for AXPAXLI If approved, AXPAXLI could be the first TKI to be commercialized in wet AMD, with a potential superiority label and best-in-class durability BEDFORD, Mass. , Dec. 08, 2025 (GLOBE NEWSWIRE) -- Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”), an integrated biopharmaceutical company committed to redefining the retina experience, announced that following recent public statements from U.S. Food and Drug Administration (FDA) leadership and other recent interactions with the FDA’s Division of Ophthalmology , the Company now intends to submit a New Drug Application (NDA) for AXPAXLI™ (also known as OTX-TKI) for the treatment of wet age-related macular degeneration (wet AMD) following year one data, if positive, from its ongoing SOL-1 Phase 3 clinical trial for which data are on track for the first quarter of 2026. “At Ocular Therapeutix, we have always been courageous, opportunistic, and bold in our efforts to redefine retina. Based on recent developments, we now intend to submit our NDA for AXPAXLI in wet AMD shortly after SOL-1 year one data, assuming positive results. SOL-1 is the only ongoing Phase 3 retina trial currently being conducted under a Special Protocol Assessment (SPA) agreement. It is also the only current wet AMD trial exploring superiority compared to a single injection of aflibercept (2 mg) and the only wet AMD registrational trial that we are aware of that is being run completely in alignment with the FDA’s draft guidance and feedback. This triad of factors helps us make a compelling case for a truly differentiated NDA for AXPAXLI in the treatment of wet AMD,” said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix . “Recent FDA leadership comments on the potential for a single registrational trial for new product candidates stressed the importance that these studies be well powered, and well controlled. Being a superiority trial, SOL-1 is substantially powered compared to non-inferiority trials. Moreover, SOL-1 is well controlled, as the active and control arms have the same dosing cadence and do not use sham injections for masking, as per the FDA’s draft guidance and feedback. Since axitinib, the active component of AXPAXLI, is already approved in non-ophthalmic indications, we intend to leverage the 505(b)(2) pathway, which we believe has the potential to further accelerate our review timeline. With AXPAXLI’s potential to redefine treatment as the first TKI to market with differentiated and potentially best-in-class durability, we believe AXPAXLI can meaningfully and immediately change the standard of care.” The FDA has historically required two adequate and well-controlled clinical trials to demonstrate the safety and efficacy of ophthalmic product candidates, particularly for larger indications of use such as wet AMD. Recent statements from FDA leadership indicate that the Agency is potentially moving to requiring only a single registrational trial for approval, as long as the trial is adequately powered and controlled. Based on Ocular’s SPA agreement for SOL-1 , along with the trial’s superiority design, the Company plans to work with the FDA to submit its NDA for AXPAXLI in wet AMD following year one results from SOL-1 . The Company expects that additional data from the continuation of SOL-1 in year two, SOL-R , and SOL-X will help clinicians and payors appreciate the anticipated benefits of AXPAXLI's efficacy, safety, and durability, allowing them to seamlessly adopt AXPAXLI into clinical practice. The Company will engage further with the FDA regarding next steps on the regulatory pathway for AXPAXLI and will provide updates as appropriate. “Wet AMD remains one of the most challenging diseases we treat because the real-world burden of frequent injections is unsustainable for many, if not most, patients. We urgently need a therapy that delivers meaningfully better durability without adding complexity to clinical practice,” said Jeffrey S. Heier , MD, Chief Scientific Officer of Ocular Therapeutix . “AXPAXLI has the potential to transform how we manage wet AMD, with the goal of delivering truly differentiated durability of up to 12 months, with potential for better treatment adherence in the short term and improved visual outcomes over the long-term. Most importantly, retina specialists would not need to change anything about how they treat patients today – no surgery, no concomitant steroids, no new procedures. It is the same intravitreal injection procedure we use every day, but with the potential for the therapy to last far longer, with possibly better long-term outcomes. That simplicity is key to broad and rapid adoption. If successful, AXPAXLI could significantly reduce treatment discontinuation, improve long-term outcomes, and redefine the wet AMD treatment landscape.” Wet AMD remains a leading cause of blindness worldwide, affecting approximately 14.5 million individuals globally and 1.8 million in the United States alone. Despite advances in anti-VEGF therapy, many patients require frequent injections to maintain vision, and up to 40% discontinue treatment within the first year, leading to disease progression and vision loss. AXPAXLI is being developed to address this unmet need with the potential to extend dosing intervals to every 6 to 12 months and potentially provide superior and sustainable long-term visual outcomes. About AXPAXLI AXPAXLI™ (also known as OTX-TKI) is an investigational, bioresorbable, intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet AMD, diabetic retinopathy, and other retinal diseases. About the SOL-1 Study The registrational Phase 3 SOL-1 trial (NCT06223958) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (1:1), parallel group study that involves more than 100 clinical trial sites located in the U.S. and Argentina . In December 2024 , the trial completed randomization of 344 evaluable treatment-naïve subjects with a diagnosis of wet AMD in the study eye. The superiority study has an eight-week loading segment prior to randomization. During the loading segment, subjects who have 20/80 vision or better and a central subfield thickness (CSFT) of ≤500 μm receive two doses of aflibercept (2 mg) at Week -8 and Week -4. Subjects who achieve best corrected visual acuity (BCVA) of 20/20 at Day 1 or gain at least 10 early treatment diabetic retinopathy study (ETDRS) letters at Day 1 along with a CSFT of ≤350 μm are then randomized to receive a single dose of AXPAXLI or a single dose of aflibercept (2 mg). At Week 52 and at Week 76, all subjects are re-dosed with their respective initial treatment of AXPAXLI or aflibercept (2 mg). Subjects will be followed for safety until the end of Year 2. Throughout the study, subjects are assessed monthly. Trial subjects and designated study personnel will remain masked through the end of Year 2. The clinical trial protocol requires that, during the study, subjects in either arm meeting pre-specified rescue criteria will receive a supplemental dose of aflibercept (2 mg). The primary endpoint of SOL-1 is the proportion of subjects who maintain visual acuity, defined as a loss of

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