Ocular Therapeutix (OCUL) TD Cowen 46th Annual Health Care Conference summary

Key data and study outcomes

• Achieved a positive outcome in a first-ever superiority study in retina, meeting a primary endpoint never previously reached, with a highly significant p-value of 0.0006.

• Data consistency across 129 slides demonstrated superiority of the drug in all analyses, including rescue-free and ITT populations.

• OCT results showed 56% of patients maintained retinal stability within 30 microns for 9 months after a single injection, indicating unprecedented disease control.

• In aggressive subpopulations, AXPAXLI showed better disease control than EYLEA, with reversal of retinal hemorrhage rates at week 52.

• Nearly 80% of patients in SOL-1 were rescue-free, and the drug's effect duration aligns with its hydrogel design, peaking at 36-40 weeks and tapering by week 52.

Regulatory and commercial implications

• Expectation to secure the first and only superiority label in the field, providing protection from step therapy and supporting premium pricing.

• Superiority label anticipated to offer a long-term competitive moat, as future competitors are unlikely to pursue similar endpoints.

• Physicians value the ability to choose initial therapy and avoid step therapy hurdles, which a superiority label would enable.

• Confident in single trial approval pathway, with all regulatory criteria met and SPA in place; submission process already underway.

• Broad label expected for diabetic retinopathy and diabetic macular edema, covering all patients with these conditions.

Clinical practice and future outlook

• Drug expected to perform better in real-world settings than in clinical trials, especially for anti-VEGF-dependent patients.

• No workflow changes or new equipment needed for clinicians; drug offers improved durability and patient convenience.

• High-dose EYLEA is not expected to outperform or impact the commercial positioning of AXPAXLI; fewer than 10% of EYLEA HD patients reach 6 months between injections.

• Safety profile is favorable, with no concerning findings; floaters reported were drug particles, not affecting vision, and cataract rates were consistent with aging.

• Strong conviction in the drug's potential for broad impact in diabetic retinal disease, with no anticipated need for additional studies.