Ocular Therapeutix Showcases Progress On New Eye Drug That Could Change Treatment For Vision Loss

Author: Benzinga Newsdesk | September 30, 2025 06:22am

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.

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