AXPAXLI for Wet AMD - AXPAXLI aims to redefine the wet AMD market with its highly selective pan-VEGF inhibition and sustained drug delivery via ELUTYX technology[29, 31] - The SOL-1 trial is designed as a superiority study comparing a single dose of AXPAXLI to a single 2mg dose of aflibercept at Week 36, with topline data expected in Q1 2026[45, 48] - The SOL-R trial is designed as a non-inferiority study comparing AXPAXLI Q24W to aflibercept 2mg Q8W at Week 56, with a non-inferiority margin of -45 letters in BCVA[50] - As of January 10, 2025, 311 subjects were enrolled in the SOL-R trial across various stages of loading and randomization[53] - Phase 1 data showed 100% rescue-free rate per protocol at 6 months and 80% at 10 months in a US wet AMD trial with AXPAXLI treatment[39, 41, 111] AXPAXLI for Diabetic Retinopathy (DR) - In the U S, 72% of the 88 million DR patients have NPDR, but less than 1% of NPDR patients are treated[61] - Phase 1 HELIOS study of AXPAXLI in NPDR showed no disease progression to PDR or CI-DME in the AXPAXLI arm at Week 48, compared to 375% in the sham arm[72] Market and Financial Considerations - There are 16 million people with wet AMD in the U S [16] - The company's strong cash position of $3921 million is expected to fund operations into 2028[124]

Core Insights - Ocular Therapeutix has successfully completed enrollment for the SOL-R trial, the largest retinal TKI trial to date, with a target randomization of at least 555 subjects [1][2][6] - The SOL-1 trial, which is complementary to SOL-R, completed randomization in December 2024 and is on track for top-line readout in Q1 2026 [1][2] - Both trials are designed to support a New Drug Application (NDA) submission for AXPAXLI in wet age-related macular degeneration (wet AMD) [1][3][4] Company Overview - Ocular Therapeutix is a fully-integrated biopharmaceutical company focused on redefining the retina experience [14] - AXPAXLI, the investigational product candidate, is a bioresorbable intravitreal hydrogel incorporating axitinib, aimed at treating wet AMD and other retinal diseases [5][14] - The company is leveraging its proprietary ELUTYX technology in both AXPAXLI and its commercial product DEXTENZA [15] Clinical Trials - The SOL-R trial is a Phase 3 registrational study evaluating the safety and efficacy of AXPAXLI, involving approximately 100 sites across the U.S., Argentina, India, and Australia [6][8] - The primary endpoint of SOL-R is to demonstrate non-inferiority in mean change in best corrected visual acuity (BCVA) compared to aflibercept at Week 56, with a non-inferiority margin of -4.5 letters [8][11] - The SOL-1 trial aims to evaluate the proportion of subjects maintaining visual acuity at Week 36, with a focus on durability up to Week 52 [11][9] Market Context - Wet AMD is a leading cause of blindness, affecting approximately 14.5 million individuals globally and 1.7 million in the U.S. [12][13] - Current treatments face challenges such as the need for frequent injections and high patient discontinuation rates, highlighting the need for more durable therapies [13] - AXPAXLI has the potential to significantly reduce treatment burden and improve long-term vision outcomes for patients with wet AMD [3][4]