Ocular Therapeutix (OCUL) investor relations material

Strategic highlights and future plans

• NDA submission for AXPAXLI in wet AMD is planned for Q4 2026, leveraging the 505(b)(2) pathway and formal FDA alignment, aiming for accelerated review and 2027 launch.

• SOL1 demonstrated statistically significant superiority over aflibercept in wet AMD, with unmatched durability (up to 12 months) and strong safety profile, supporting a potential first-in-class, best-in-class label and annual dosing.

• SOLAR/SOL-R trial repurposed to pursue superiority versus high-dose Eylea at week 96, with efficacy data excluded from NDA to expedite approval; interim safety data will be included.

• Diabetic retinopathy program streamlined to prioritize HELIOS-3 as the sole registrational trial, focusing on annual dosing and reducing complexity.

• Commercial launch preparations are advanced, with strong physician and payer feedback indicating rapid adoption and premium pricing potential.

Regulatory and clinical development updates

• SOL1 is the first successful superiority phase III trial in wet AMD, meeting all FDA requirements for a single, adequate, well-controlled study under SPA, with a p-value of 0.0006.

• Confirmatory evidence includes mechanistic, pharmacodynamic, class consistency, animal model, and real-world data, all presented to the FDA and deemed compelling.

• Safety database will exceed 300 patients with 12-month exposure, combining SOL1 and interim SOLAR/SOL-R safety data, with a 120-day update to include year two SOL1 safety.

• Pre-NDA meeting in Q3 2026 will focus on operational aspects; content and data package already agreed with FDA in written minutes.

• High confidence in NDA acceptance and no anticipated major amendments or refusal to file, based on extensive FDA engagement and alignment.

Clinical trial design and endpoints

• SOL1 met its primary endpoint with a p-value of 0.0006, showing superiority in maintenance of vision at Week 36 and sustained effect at Week 52, with robust anatomical control and fewer interventions.

• SOL-R's primary endpoint is non-inferiority in mean BCVA change at Week 56; new key secondary endpoint will evaluate superiority of AXPAXLI vs aflibercept (8mg) in mean BCVA change at Week 96.

• Prevention of fibrosis and atrophy vs aflibercept (2mg) will also be evaluated at Week 96 for potential label differentiation.

• AXPAXLI demonstrated strong vision maintenance: ~74% rescue-free at 9 months, ~66% at 12 months, and ~90% of responders at 9 months maintained vision at 12 months.

• Safety profile is favorable, with no cases of endophthalmitis or vasculitis and a DSMC recommendation to continue the trial.