Ocular Therapeutix (OCUL) H.C. Wainwright 6th Annual Ophthalmology Virtual Conference summary

Regulatory alignment and submission plans

• Achieved alignment with FDA on NDA submission for AXPAXLI in wet AMD, confirming the package meets all evidentiary standards for single-trial approval.

• Submission is planned for Q4 this year, following an operational pre-NDA meeting in Q3 focused on submission logistics, not content.

• FDA has agreed to accept the package as complete, with compelling data and consistent guidance throughout the process.

• The 505(b)(2) pathway is confirmed, leveraging prior approvals of both drug components to expedite review.

• Commercial and CMC infrastructure is already in place, with readiness for both wet AMD and diabetic retinal disease launches.

Clinical trial results and product profile

• SOL-1 trial met all FDA evidentiary standards, including proper masking, powering, statistical significance (P<0.0006), and safety exposure in over 300 patients.

• Demonstrated superiority to aflibercept 2 mg in durability, with 69% of patients rescue-free at one year and strong OCT stability.

• Safety profile is transparent and robust, with no endophthalmitis or vasculitis; floaters observed were drug particles, not clinically significant.

• Redosing experience is extensive, supporting anticipated label flexibility for dosing every 6–12 months.

• Expectation is for a first-in-class superiority label with broad applicability across wet AMD and other VEGF-driven diseases.

Market adoption and financial outlook

• Anticipates broad adoption across all patient types, including treatment-naive, frequent injectors, and stable patients seeking fewer visits.

• No anticipated need for additional commercial build-out; experienced team in place, including leadership from prior successful retina launches.

• Well-capitalized with $667 million in cash, financing expected to last into 2028 and through launch.

• Expects the drug to outperform in real-world settings compared to phase III, with potential for use in all eligible patients.

• Position described as highly competitive, with regulatory and commercial readiness validated by years of FDA collaboration.