Ocular Therapeutix (OCUL) CTS 2025 presentation summary

Clinical program overview

• Two pivotal Phase 3 trials (SOL-1 and SOL-R) are evaluating OTX-TKI for wet AMD, with complementary designs to de-risk outcomes and regulatory path.

• SOL-1 is a superiority study comparing a single OTX-TKI dose to a single aflibercept 2mg dose, with the primary endpoint at week 36 based on visual acuity maintenance.

• SOL-1 fully randomized 344 subjects across over 100 sites in the US and Argentina, with topline data expected in Q1 2026.

• SOL-R is a non-inferiority study comparing OTX-TKI Q24W to aflibercept 2mg Q8W at week 56, with 555 subjects in a three-arm design.

• SOL-R enrollment closed in May 2025, with ongoing loading and randomization, and FDA alignment achieved for both trials.

OTX-TKI technology and mechanism

• OTX-TKI uses a proprietary bioresorbable hydrogel for sustained, localized axitinib delivery, enabling redosing intervals of 6–12 months.

• Axitinib is a highly potent, selective multi-target tyrosine kinase inhibitor, ~100x more potent for VEGFR-2 than sunitinib or vorolanib.

• The hydrogel platform allows for continuous, predictable drug release and complete bioresorption.

• Patent coverage for the technology extends through 2041.

Phase 1 clinical results in wet AMD (nAMD)

• Preclinical rabbit studies showed rapid and sustained reduction in vascular leakage after OTX-TKI injection.

• Phase 1 trials in Australia and the US demonstrated biological activity and durability, with evidence of efficacy in both treatment-naïve and previously treated nAMD patients.

• In the US Phase 1 trial, OTX-TKI reduced anti-VEGF treatment burden by 89% at 52 weeks, with 60% of patients rescue-free at one year.

• Changes in BCVA and CSFT were similar to aflibercept 2mg Q8W, and anatomic outcomes support 6–12 month durability.

• No drug-related ocular or systemic serious adverse events were reported; one moderate endophthalmitis event was related to mandated aflibercept, not OTX-TKI.