Clear Regulatory Path for AXPAXLI™ in Wet AMD Through Ongoing SOL-1 and SOL-R Registration-Enabling Trials
Ocular to Host a Q2 2024 Conference Call and Webcast Today, August 7th, at 8:00 AM ET
BEDFORD, Mass., Aug. 07, 2024 (GLOBE NEWSWIRE) -- Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”, the “Company”), a biopharmaceutical company committed to improving vision in the real world through the development and commercialization of innovative therapies for retinal diseases and other eye conditions, today announced that the Company has received a written response from the U.S. Food and Drug Administration (FDA) that the Phase 3 SOL-R clinical trial is appropriate for use as the Company’s second adequate and well controlled study of AXPAXLI™ (axitinib intravitreal implant, also known as OTX-TKI) for the treatment of patients with wet age-related macular degeneration (wet AMD).
In a Type C written response, the FDA agreed that the SOL-R repeat dosing wet AMD study is appropriate as an adequate and well-controlled study in support of a potential New Drug Application (NDA) and product label. The FDA also noted that the use of one superiority study and one non-inferiority study is generally acceptable as the basis of an eventual NDA in wet AMD. Together, the Company intends the ongoing SOL-1 and SOL-R Phase 3 clinical trials to form the basis for the regulatory filing of AXPAXLI for wet AMD, assuming successful completion.
Ocular recently announced that the first subjects have been enrolled in the SOL-R non-inferiority study evaluating repeat dosing of AXPAXLI. Enrollment in the SOL-1 superiority study evaluating a single AXPAXLI implant continues to accelerate, with subjects who do not meet randomization criteria having an opportunity to be enrolled into SOL-R.
“We are very pleased that the FDA has found the SOL-R repeat dosing study to be acceptable as a registrational trial of AXPAXLI in wet AMD. This is an outstanding achievement for Ocular that clarifies the regulatory path for the two complementary Phase 3 studies in the AXPAXLI wet AMD program, SOL-1 and SOL-R,” said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. “Our Phase 3 program is designed to align with the FDA’s guidance and provide commercially meaningful data. Thanks to the efforts of our clinical team, we have accelerated enrollment in our first study, SOL-1. In addition, over a period of less than three months, the team was able to conceptualize SOL-R, activate clinical sites, enroll the first subjects, and has now obtained confirmatory FDA feedback regarding the registrational study. We look forward to continuing our collaboration with regulatory authorities as we work towards our goal of making AXPAXLI available to people with wet AMD.”
“Patients with wet AMD need new therapeutics that can provide long-term improvements in visual outcomes through durable and sustained suppression of VEGF. The initiation of the Phase 3 AXPAXLI program is great news for the retina community because it potentially brings us one step closer to having a new treatment paradigm for patients with wet AMD. In addition, the AXPAXLI program provides an opportunity to be part of innovative study protocols that have been accepted for registration-enabling studies by the FDA,” said Carl C. Awh, MD, FASRS, a Past-President of the ASRS and President of Tennessee Retina, a member of the Retina Consultants of America network. “Now that both SOL-1 and SOL-R are open for enrollment, people with wet AMD have two opportunities to participate in a clinical study for AXPAXLI. When I talk with my patients, they find it comforting to know that everyone who is randomized into each study is guaranteed to receive an active agent. Patients who are not eligible to be enrolled in SOL-1 are also enthusiastic to participate in SOL-R because of the ‘real-world’ repeat dosing element. I also feel the introduction of a third arm to ensure adequate masking, without the use of sham injections, enhances the integrity of the SOL-R study. I am glad to be part of two studies that have the potential to provide the retina community with important data on efficacy, durability of response, and dosing flexibility of AXPAXLI, and improve the care of people with wet AMD.”
SOL-1 is a superiority study comparing a single AXPAXLI implant to a single aflibercept (2 mg) injection, after both arms receive two aflibercept (2 mg) loading doses. SOL-R compares AXPAXLI dosed every six months (Q6M) to the current standard of care, aflibercept (2 mg) dosed every eight weeks. A third arm evaluating 8 mg aflibercept dosed Q6M is incorporated in the SOL-R study to ensure adequate masking, following the FDA’s recommendation that sham injections do not provide complete masking and therefore may elicit potential bias.
Conference Call and Webcast InformationOcular Therapeutix will host a conference call and webcast today at 8:00 AM ET to discuss recent business progress and financial results. To access the call, please dial: 1 (800) 343-4136 (United States) or 1 (203) 518-9843 (International) and reference the conference ID “OCULAR”. To access the webcast, please click here. The live and archived webcast can also be accessed by visiting the Ocular Therapeutix website on the Events and Presentations section of the Investor Relations page. A replay of the webcast will be archived for 90 days.
About AXPAXLIAXPAXLI™ (axitinib intravitreal implant, also known as OTX-TKI) is an investigational bioresorbable, hydrogel implant incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet AMD, diabetic retinopathy and other retinal diseases.
About the SOL-R studyThe registrational Phase 3 SOL-R trial (NCT06495918) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (2:2:1), three-arm study that will involve sites located in the U.S. and the rest of the world. The trial is intended to randomize approximately 825 subjects who are treatment naïve or diagnosed with wet AMD in the study eye within three months prior to enrollment.
The non-inferiority study reflects a patient enrichment strategy that includes multiple loading doses of aflibercept and monitoring to exclude those with significant retinal fluid fluctuations. In the first arm, subjects are randomized to receive a single dose of AXPAXLI at Day 1 and are re-dosed at Week 24. In the second arm, subjects receive aflibercept (2 mg) on-label every 8 weeks. In a third arm, subjects receive a single dose of aflibercept (8 mg) at Day 1 and are re-dosed at Week 24, aligned to the AXPAXLI treatment arm for adequate masking. Subjects in any arm meeting pre-specified rescue criteria will receive a supplemental dose of aflibercept.
The primary endpoint is non-inferiority in mean best corrected visual acuity (BCVA) change from baseline between the AXPAXLI and on-label aflibercept (2 mg) arms at one year.
About the SOL-1 studyThe registrational Phase 3 SOL-1 trial (NCT06223958) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (1:1), parallel group study that involves sites primarily located in the U.S. The trial is intended to randomize approximately 300 evaluable treatment-naïve subjects with a diagnosis of wet AMD in the study eye.
The superiority study begins with an eight-week loading segment prior to randomization, a 9-month treatment segment, followed by a safety follow-up. During the loading segment, subjects who have 20/80 vision or better and who satisfy other enrollment criteria receive two doses of aflibercept (at week -8 and week -4). Subjects that achieve visual acuity of 20/20 at Day 1 or gain at least 10 early treatment diabetic retinopathy (ETDRS) letters at Day 1 are then randomized to receive a single dose of AXPAXLI or a single dose of aflibercept and assessed monthly for the entire study. The clinical trial protocol requires that, during the study, subjects in any arm meeting pre-specified rescue criteria will receive a supplemental dose of aflibercept.
The primary endpoint of SOL-1 is the proportion of subjects who maintain visual acuity, defined as