Ocular Therapeutix™ Investor Day to Highlight Exceptional AXPAXLI™ Progress Across SOL Program and Detail Registrational Trial Plans to Pursue a Diabetic Retinopathy Label with a Novel Primary Endpoint

About this update from Ocular Therapeutix, Inc.

SOL-1 superiority trial continues to demonstrate outstanding patient retention and protocol adherence, with no new or unexpected safety signals observed to date; topline data on track for 1Q 2026 Event to detail how robust SOL-R patient selection strategy, trial design, and potential SOL-1 success could drive confidence in SOL-R outcomes; topline data on track for 1H 2027 Will showcase planned HELIOS-2 and HELIOS-3 registrational trials to evaluate AXPAXLI in non-proliferative diabetic retinopathy (NPDR) using novel primary endpoint, aligned with FDA in HELIOS-2 Special Protocol Assessment (SPA) agreement To outline SOL-X open label extension program and AXPAXLI’s potential to improve long-term outcomes and expand the retinal vascular disease market The live event will begin at 2:00 PM ET in New York City with virtual access available BEDFORD, Mass. , Sept. 30, 2025 (GLOBE NEWSWIRE) -- Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”), an integrated biopharmaceutical company committed to redefining the retina experience, will host an Investor Day today where it will highlight outstanding progress in the SOL wet AMD registrational program, announce plans for a registrational program in non-proliferative diabetic retinopathy (NPDR), and share further details on how AXPAXLI™ (also known as OTX-TKI) is being positioned to redefine retina treatment. "I continue to be incredibly confident and enthusiastic about the future of Ocular Therapeutix ," said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix . "At our Investor Day, we will highlight how Ocular is uniquely positioned for success, anchored by AXPAXLI’s potential to have the first superiority label compared to a single dose of aflibercept (2 mg) in wet AMD, targeting a large market opportunity with significant potential for expansion, and seamless adoptability into clinical practice. A superiority label may allow physicians to avoid step therapy, enabling them to choose the optimal drug for their patients. We believe AXPAXLI is well-positioned to optimize retina practices worldwide, allowing physicians to see more patients, less often.” Dr. Dugel continued, “We are especially thrilled to unveil our plans for diabetic retinopathy (DR) with our HELIOS-2 and HELIOS-3 registrational trials. We have designed these trials to include a novel primary endpoint that we believe has the highest probability of success. This new endpoint is FDA-aligned with a Special Protocol Assessment (SPA) agreement for HELIOS-2. We will also share new details on our SOL-X open label extension study in wet AMD, and why we believe a positive SOL-1 readout could provide strong read through to SOL-R . All of this exceptional progress, coupled with the significant market opportunity, is the reason for our enthusiasm and continued confidence moving forward." Investor Day Highlights Wet Age-Related Macular Degeneration (wet AMD) Program Highlights SOL-1 (Phase 3, wet AMD) retention and protocol adherence continues to be exceptional, with topline data on track for 1Q 2026. The SOL-1 superiority trial, conducted under an SPA agreement with the U.S. Food and Drug Administration (FDA), has the potential to support the first label with a superiority claim over a single dose of aflibercept (2 mg) for any wet AMD product. Retention in the trial continues to be outstanding, with >95% of randomized subjects remaining on-study to date. Rescues reviewed under masking suggest >95% of rescue events have met protocol-defined criteria. Further, to ensure patient safety, the SOL-1 trial is overseen by an independent data and safety monitoring committee (DSMC) which has not identified any new or unexpected safety signals to date. Robust SOL-R (Phase 3, wet AMD) patient selection strategy, trial design, and potential SOL-1 success drive confidence in SOL-R outcomes, with topline data on track for 1H 2027. The SOL-R non-inferiority trial complements SOL-1 with the potential to provide data supporting the immediate adoption of AXPAXLI into clinical practice, if approved. Prior Phase 3 trials for other wet AMD drugs have shown that patients with persistent retinal fluid introduce variability and can disrupt non-inferiority trials. Therefore, SOL-R incorporates a comprehensive 24-week screening and loading phase to exclude subjects with early persistent fluid and to randomize subjects with less variability in visual acuity. In addition to patient selection, Ocular believes the singular Week 56 primary endpoint in SOL-R is favorable as subjects will have received their most recent aflibercept or AXPAXLI injection eight weeks prior, at Week 48. Assuming positive data from its registrational trials, Ocular expects a New Drug Application (NDA) package would include SOL-1 two-year safety data (including re-dosing every six months in year two of the trial), along with SOL-R 56-week data. Ocular believes this NDA package would exceed the requirements for the FDA’s safety database at the maximum dose and most frequent cadence being proposed for marketing. Ocular plans to leverage the 505(b)(2) NDA pathway to potentially streamline the FDA review process. "The complementary SOL-1 and SOL-R trials have positioned Ocular to elegantly answer the questions that data purists like myself are looking for," said Adnan Tufail , MBBS, MD, FRCOphth, Consultant Ophthalmologist in Medical Retina Service at Moorfields Eye Hospital , London and Professor at The Institute of Ophthalmology , University College London . "What we really need in our clinical environments, worldwide, is a genuinely more durable therapy. Even with our best and latest therapies, when we begin to use them in the real-world setting, we see vision trailing off over time. If these trials both succeed, we will have seen that AXPAXLI was more durable than aflibercept (2mg) and sustained a benefit at a predictable low frequency of re-dosing, giving retina specialists the data and confidence needed to plan clinical use optimally to be able to reach more patients. I believe two successful readouts should set up AXPAXLI for strong and immediate adoption." SOL-X open-label wet AMD extension study to evaluate the potential of AXPAXLI to improve long-term outcomes and provide commercial advantages. Subjects who have completed two-year follow-up in either SOL-1 or SOL-R will have an opportunity to enroll in the SOL-X study for an additional three years. SOL-X outcomes may further expand AXPAXLI’s potential by highlighting the need to start AXPAXLI treatment early or risk worse long-term visual outcomes due to potential fibrosis and atrophy that may be seen with pulsatile treatments. By reducing the treatment burden and improving long-term outcomes, Ocular believes the data from SOL-X could increase both short-term and long-term patient retention significantly, thereby expanding the market opportunity. Diabetic Retinopathy (DR) Program Highlights Ocular announced plans to initiate two superiority registrational trials in non-proliferative diabetic retinopathy (NPDR), targeting a broad DR label: HELIOS-2 and HELIOS-3 (Phase 3, NPDR). Proactive anti-VEGF treatment of NPDR has been observed to benefit patients, but this treatment regimen is burdensome for a working-age patient population. The treatment burden is unsustainable for many, if not most, of these patients, as evidenced by less than 1% of the prevalent population currently receiving treatment. Ocular plans to target a broad label in DR by including subjects with non-center-involved diabetic macular edema (non-CI-DME) in its Phase 3 program. Based on the success of the HELIOS-1 trial, Ocular is preparing to initiate the HELIOS registrational program imminently with the goal of evaluating 6- and 12-month dosing intervals. HELIOS-2 is a two-arm superiority trial to be conducted in approximately 432 subjects (randomized 1:1) under an SPA agreement with the FDA. The trial is designed to evaluate a single AXPAXLI injection compared to a single ranibizumab (0.3 mg) injection, with a 52-week primary endpoint. Subjects will be re-treated at Week 52, then followed for safety until the end of Year 2. HELIOS-3 is a three-arm superiority trial to be conducted in approximately 930 subjects (randomized 1:1:1) designed to compare two dosing regimens of AXPAXLI to sham, with a 52-week primary endpoint. The first AXPAXLI arm will be treated at Day 1 and Week 24. The second AXPAXLI arm will be treated at Day 1 and will receive a sham injection at Week 24. The third arm will receive a sham injection at Day 1 and Week 24. This trial will conclude after the primary endpoint. While HELIOS-3 uses sham injections for masking, Ocular has not used sham injections in their ongoing registrational wet AMD trials where such use could influence outcomes as wet AMD trials rely on a subjective vision-based primary endpoint. DR trials, on the other hand, use an objective photographic DRSS primary endpoint and the use of sham injections for masking should not influence outcomes. Furthermore, similar to sham injections, the standard of care (SoC) in NPDR is largely watchful waiting, with

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