Ocular Therapeutix (OCUL) investor relations material

Strategic Positioning and Vision

• AXPAXLI is positioned to redefine retina treatment by targeting the first superiority label versus anti-VEGF therapies, aiming for market expansion in wet AMD and diabetic retinopathy, with a focus on best-in-class durability, seamless adoption, and broad label coverage.

• The company emphasizes a bold, confident approach, making all decisions to maximize clinical, regulatory, and commercial success, and leveraging a product profile that requires no workflow changes or new equipment.

• Expansion into diabetic retinopathy is a key growth area, targeting a largely untreated segment and leveraging innovative endpoints for regulatory efficiency.

• The ELUTYX hydrogel technology underpins AXPAXLI and other pipeline products, supporting future innovation.

• The company is committed to advancing its pipeline, including OTX-TIC for glaucoma and DEXTENZA for ocular inflammation and pain.

Clinical Development, Trial Design, and Regulatory Milestones

• The SOL program includes three pivotal trials: SOL-1 (superiority in wet AMD), SOL-R (non-inferiority/adoptability), and SOL-X (long-term outcomes), all under FDA SPA agreements and designed for regulatory acceptance.

• SOL-1 is the only ongoing superiority trial in wet AMD, with >95% patient retention, strong protocol adherence, and topline data expected Q1 2026.

• SOL-R uses robust patient selection and masking strategies, with topline data expected 1H 2027, and is designed to complement SOL-1 for NDA submission.

• SOL-X is a long-term extension study evaluating safety, disease modification, and sustained benefit over three years.

• NDA submission will include SOL-1 two-year safety data and SOL-R 56-week data, aiming to exceed FDA safety requirements and leverage the 505(b)(2) pathway.

Diabetic Retinopathy Program and Innovations in Endpoints

• HELIOS-2 and HELIOS-3 Phase 3 trials in NPDR use a novel ordinal ≥2-step DRSS primary endpoint, aligned with FDA under SPA, enabling smaller, faster, and less risky studies.

• HELIOS-2 is a two-arm superiority trial versus ranibizumab, and HELIOS-3 is a three-arm trial versus sham, both with 52-week primary endpoints.

• The ordinal endpoint captures both improvement and worsening, increasing statistical power and clinical relevance, and is expected to become the gold standard for DR studies.

• The HELIOS-1 Phase 1 trial demonstrated consistent efficacy and safety, with significant DRSS improvement and DME prevention.

• The registrational program is designed to support a broad DR label, including both prevention and treatment of DME.