Core Insights - The initiation of the HELIOS registrational program represents a significant advancement in the treatment of diabetic retinal disease, with AXPAXLI potentially addressing the needs of over 6 million NPDR patients in the U.S. who currently receive minimal therapy due to treatment burdens [2][3] - The HELIOS-2 and HELIOS-3 trials are designed to evaluate the efficacy of AXPAXLI in treating non-proliferative diabetic retinopathy (NPDR) and aim for a broad diabetic retinopathy label, including patients with non-center-involved diabetic macular edema (non-CI-DME) [2][3][12] - AXPAXLI is expected to provide durable efficacy with less frequent dosing, which could significantly improve patient adherence and outcomes compared to current treatment options that require frequent injections [2][3][12] Company Overview - Ocular Therapeutix, Inc. is an integrated biopharmaceutical company focused on redefining the retina experience through innovative treatments for retinal diseases, including AXPAXLI, which is currently in Phase 3 clinical trials for wet AMD and NPDR [14][15] - AXPAXLI is a bioresorbable intravitreal hydrogel that incorporates axitinib, a multi-target tyrosine kinase inhibitor with anti-angiogenic properties, aimed at treating various retinal diseases [4][5] HELIOS Program Details - The HELIOS program consists of two complementary superiority studies: HELIOS-2 and HELIOS-3, which will assess the safety and efficacy of AXPAXLI in approximately 432 and 930 subjects, respectively, with moderately severe to severe NPDR [6][9] - Both studies utilize a novel ordinal diabetic retinopathy severity score (DRSS) as the primary endpoint, focusing on changes in severity at Week 52 from baseline [8][11] - HELIOS-2 compares AXPAXLI dosed every 12 months to ranibizumab, while HELIOS-3 evaluates different dosing regimens of AXPAXLI against sham treatment [7][10] Industry Context - Diabetic retinopathy is a prevalent condition affecting over 103 million people globally, with a significant portion of NPDR patients remaining untreated due to the burdensome nature of existing therapies [12][13] - The HELIOS trials aim to address the unmet need for effective treatments with durable efficacy, potentially transforming care for diabetic retinal disease and improving patient outcomes [3][12]

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]