Ocular Therapeutix (OCUL) Jefferies London Healthcare Conference 2025 summary

Clinical development highlights

• Two pivotal phase III trials, SOL-1 (superiority) and SOL-R (non-inferiority), are underway for a lead retinal disease program, with SOL-1 data expected in Q1 2026 and SOL-R in H1 2027.

• SOL-1 targets treatment-naive, VEGF-dependent patients without fibrosis, aiming for a superiority label, while SOL-R focuses on stable patients for non-inferiority.

• Both trials feature rigorous patient selection and high retention, with over 95% protocol adherence and patient retention in SOL-1.

• The SOL-1 trial is designed per FDA guidelines and has received a Special Protocol Assessment (SPA), supporting regulatory alignment.

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

Product and market opportunity

• The lead asset, AXPAXLI, is a potent, selective TKI on a hydrogel platform, targeting wet AMD, diabetic retinopathy, and diabetic macular edema.

• AXPAXLI aims to address high unmet needs by reducing treatment burden to once yearly, potentially setting a new standard of care.

• For diabetic retinopathy, the target population is 3.5 times larger than wet AMD, with less than 1% currently treated.

• A novel ordinal endpoint for diabetic retinopathy has FDA agreement and SPA validation, potentially enabling a broad label.

• The commercial team is experienced in ophthalmology, and no partnerships are currently planned for launch due to strong capitalization and expertise.

Competitive positioning and strategic outlook

• AXPAXLI is positioned to be the first with a superiority label versus anti-VEGF, offering dosing flexibility and repeatability.

• The superiority label is expected to provide premium positioning, reimbursement advantages, and immunity from step therapy and pricing pressures.

• Long-term data and IP protection through 2044 further strengthen the competitive moat.

• The company expects a broad disease-based label for diabetic retinal diseases, covering both diabetic retinopathy and diabetic macular edema.

• No additional diabetic retinopathy studies are anticipated due to the comprehensive label strategy.