Ocular Therapeutix (OCUL) investor relations material

Key developments in wet macular degeneration treatment

• AXPAXLI demonstrated superiority over anti-VEGF therapy in a phase III trial, marking the first such result in the field.

• The SOL-1 trial followed FDA-mandated design, using a head-to-head comparison with Eylea and achieving statistical significance on its primary endpoint.

• Disease control with AXPAXLI was unprecedented, with OCT data showing minimal retinal fluid fluctuation and fewer retinal hemorrhages compared to control.

• The trial's design and results position AXPAXLI as the only candidate with a potential superiority label, creating a high barrier for future competitors.

• Safety data showed imbalances in floaters and cataracts, but all findings were within expected ranges and did not impact vision.

Regulatory strategy and trial progression

• The regulatory approach is based on a single pivotal trial, aligned with recent FDA guidance making this the default for approval.

• Submission for approval is underway, leveraging prior FDA approvals of AXPAXLI's components and a Special Protocol Assessment (SPA) to expedite review.

• Ongoing SOLAR study, a non-inferiority trial with a different, more stable patient population, is expected to read out in Q1 2027.

• Sufficient safety and redosing data are available from SOL-1, and the company is confident in regulatory interactions and approval prospects.

• The company is well-capitalized, with $739 million in cash and runway through 2028.

Real-world application and future outlook

• AXPAXLI is expected to be adopted immediately upon approval, requiring no workflow changes for physicians and offering a higher price point.

• The drug is positioned for fixed six-month dosing, aligning with patient and physician preferences and providing a safety net for missed appointments.

• Additional pipeline progress includes a non-proliferative diabetic retinopathy program, with updates expected alongside regulatory milestones.

• The next 12–18 months will feature updates on FDA interactions, SOLAR trial progress, and other clinical catalysts.