Ocular Therapeutix (OCUL) Piper Sandler 36th Annual Healthcare Conference summary

Strategic focus and technology

• Company has redefined its identity, focusing on retinal therapeutics using a bioresorbable hydrogel with a potent TKI for wet AMD.

• Aims to address sustainability and chronic visual outcomes in wet AMD, targeting both patient dropout and long-term vision loss due to fibrosis.

• Believes long-term, sustained release of AXPAXLI can improve outcomes by avoiding oscillations from frequent injections.

• Positions itself as agnostic to biosimilars, emphasizing the unmet need for longer durability in treatment.

• Claims AXPAXLI offers 9–10 months of durability, surpassing current anti-VEGF therapies.

Clinical trial design and regulatory strategy

• Two phase III trials, SOL-1 (superiority) and SOL-R (non-inferiority), are designed to complement each other and follow FDA guidance.

• SOL-1 avoids sham controls, uses a unique design, and achieved a Special Protocol Assessment (SPA) agreement with the FDA.

• SOL-R is validated as a registrational study with a written Type C response from the FDA; both studies are required for approval.

• SOL-1 completed enrollment of over 300 patients ahead of schedule, with data readout expected in Q4 2025.

• Patient selection in both trials is highly tailored, with SOL-1 focusing on early-stage, responsive patients and SOL-R on stable patients.

Key endpoints and commercial positioning

• Primary endpoint for SOL-1 is rescue-free rate at nine months; expects a 50% delta between AXPAXLI and Eylea arms.

• Both studies are designed to answer clinician questions on durability, repeatability, and dosing flexibility.

• SOL-R includes three arms (AXPAXLI, Eylea, high-dose Eylea), with high-dose Eylea used for masking and commercial comparison.

• Data from high-dose Eylea arm, though not for statistical analysis, will be leveraged for commercial advantage.

• Plans to pursue a label showing superiority, dosing flexibility, and repeatability, aiming for strategic and commercial leadership.