Ocular Therapeutix, Inc. (NASDAQ: OCUL) has announced significant clinical progress in its registrational program for AXPAXLI™ (axitinib intravitreal injection) in wet age-related macular degeneration (wet AMD) and plans to advance AXPAXLI in non-proliferative diabetic retinopathy (NPDR). The company shared these updates during its presentation at the 43rd Annual J.P. Morgan Healthcare Conference (JPM 2025). The progress underscores Ocular's commitment to addressing major causes of blindness with innovative retinal therapies.
Pravin U. Dugel, MD, Executive Chairman, President, and CEO of Ocular Therapeutix, emphasized the critical need for sustainable treatments in retinal diseases, stating, "Wet AMD is the leading cause of blindness in the U.S., but it does not need to be... Our mission at Ocular is to correct this." He highlighted the enthusiasm within the retina community for AXPAXLI, evidenced by the rapid enrollment in both SOL-1 and SOL-R studies.
SOL-1 and SOL-R Trials for Wet AMD
The AXPAXLI wet AMD registrational program includes two complementary studies: SOL-1 and SOL-R. These trials are strategically designed to de-risk patient populations, align with regulatory standards, enhance enrollment, and provide a broad evaluation of AXPAXLI’s durability, repeatability, and flexibility. The FDA has agreed that, together, these studies could support a potential New Drug Application (NDA) and label for AXPAXLI in wet AMD.
SOL-1, a Phase 3 superiority trial, completed randomization in December 2024, with over 300 subjects across more than 100 clinical trial sites in the U.S. and Argentina. This trial compares a single AXPAXLI injection to a single aflibercept (2 mg) injection in treatment-naïve wet AMD subjects, with a nine-month primary endpoint and up to two-year follow-up. Topline results are expected in Q4 2025.
SOL-R, another Phase 3 trial, has enrolled 311 subjects as of January 10, 2024. This non-inferiority trial compares repeat AXPAXLI injections every six months to repeat aflibercept (2 mg) injections every eight weeks, with a 56-week primary endpoint. SOL-R is designed to inform real-world treatment decisions and establish AXPAXLI’s safety and efficacy with repeat dosing.
Expansion into Non-Proliferative Diabetic Retinopathy (NPDR)
Building on positive Phase 1 HELIOS trial results, Ocular Therapeutix plans to seek FDA feedback in H1 2025 on the clinical trial design for AXPAXLI in NPDR. The HELIOS trial demonstrated that a single AXPAXLI injection resulted in no patients (N=13) developing a vision-threatening complication (VTC) at 48 weeks, compared to nearly 40% in the sham-treated group (N=8). Additionally, all patients in the AXPAXLI arm with non-center-involved diabetic macular edema (non-CI-DME) at baseline (N=8) showed improvement in their DME based on OCT image analysis at week 48, while none of the sham-treated subjects (N=3) experienced similar improvement.
Expert Commentary
Arshad M. Khanani, MD, MA, FASRS, Director of Clinic Research at Sierra Eye Associates, noted the high treatment burden associated with current wet AMD therapies, leading to discontinuation rates of 40-50%. He expressed enthusiasm for AXPAXLI's potential to reduce this burden with a durability of 6-12 months, stating, "If approved, I believe AXPAXLI will be rapidly adopted."
Patricio G. Schlottmann, MD, Director of the Research Department at the Charles Ophthalmic Center, echoed this sentiment, emphasizing the global impact of wet AMD and diabetic retinopathy and the need for more sustainable treatment regimens. He believes AXPAXLI has the potential to positively impact millions of patients worldwide.
About AXPAXLI
AXPAXLI™ (axitinib intravitreal injection), also known as OTX-TKI, is an investigational, bioresorbable, hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties. It is being evaluated for the treatment of wet AMD, diabetic retinopathy, and other retinal diseases.
