Ocular Therapeutix (OCUL) investor relations material

Strategic program updates

• Two phase III programs, SOL-1 (superiority) and SOL-R (non-inferiority), are underway with readouts expected in Q1 2026 and H1 2027, respectively.

• Both studies are designed to be complementary and have been executed with a focus on de-risking and patient selection.

• SOL-1 targets treatment-naive, VEGF-dependent patients, while SOL-R focuses on stable patients, enhancing trial robustness.

• Over 95% of rescues in SOL-1 are on protocol, and patient retention exceeds 95%, both considered exceptional in retina studies.

• Regulatory submission is planned immediately after SOL-R, leveraging the 505(b)(2) pathway for efficiency.

Lead asset and clinical rationale

• AXPAXLI, a potent and selective TKI on a hydrogel platform, is being developed for wet AMD, diabetic retinopathy, and diabetic macular edema.

• The drug aims to address high dropout rates in wet AMD by offering a more sustainable, less frequent injection schedule.

• In diabetic retinopathy, annual injections could reduce vision-threatening complications from 40% to zero, based on early studies.

• The hydrogel platform ensures no residual material remains post-treatment, enhancing safety.

• AXPAXLI is positioned as a potential new standard of care in retinal diseases.

Clinical trial design and regulatory strategy

• SOL-1 is designed for a superiority label, following FDA guidelines and supported by a Special Protocol Assessment (SPA).

• The superiority label is expected to provide commercial advantages, including premium pricing and exemption from step therapy.

• SOL-R uses a unique, extended stabilization period and a singular endpoint at week 56 to de-risk outcomes.

• Rescue criteria in SOL-R were amended for comparability with previous studies and alignment with global regulators.

• The regulatory filing will benefit from both components of AXPAXLI already being FDA approved, expediting review.