Expected to Enroll Approximately 300 Evaluable Subjects Primarily in the United States
Bedford, MA - Ocular Therapeutix, Inc. (NASDAQ: OCUL), a biopharmaceutical company focused on the formulation, development and commercialization of innovative therapies for diseases and conditions of the eye, today announced the initiation of its first pivotal clinical trial to evaluate OTX-TKI, the Company’s axitinib intravitreal implant, for the treatment of wet age-related macular degeneration (wet AMD). OTX-TKI is also being developed for the treatment of diabetic retinopathy and other retinal diseases.
"We are thrilled to announce the initiation of our pivotal trial evaluating OTX-TKI for the treatment of wet AMD and look forward to working with clinical sites across the US," said Antony Mattessich, CEO of Ocular Therapeutix. “With the activation of our first clinical site in the US, we believe we are on target to enroll our first subject before year end. The trial is a crucial step forward for our clinical program as we make progress toward our goal of bringing a transformative new treatment that can truly make a difference for wet AMD to patients coping with vision loss."
Ocular Therapeutix has requested a Special Protocol Assessment (SPA) from the U.S. Food and Drug Administration (FDA) regarding the design of the trial, which is designed as a superiority trial that will enroll approximately 300 evaluable wet AMD subjects who are treatment naïve in the study eye. The trial is designed to be a multi-center, parallel-group trial that will be run primarily at U.S. sites with subjects randomized to one injection of aflibercept or one implant of OTX-TKI followed by as needed supplemental anti-VEGF treatment based on pre-specified criteria. The safety and efficacy of OTX-TKI will be assessed by measuring best corrected visual acuity (BCVA) and central subfield thickness (CSFT) at 36 weeks. While this institutional review board (IRB) approval has been received, the Company does not intend to enroll a subject before the SPA feedback is received from the FDA.
Arshad M. Khanani, MD, MA, FASRS, Director of Clinical Research at Sierra Eye Associates in Reno, Nevada, has agreed to be the principal investigator for the OTX-TKI pivotal Phase 3 trial. Dr. Khanani is an internationally renowned retina specialist with extensive experience in clinical trial design for the treatment of age-related macular degeneration. He has served as a principal investigator for numerous clinical trials evaluating novel treatments for wet AMD and other retinal diseases.
“Anti-VEGF agents have revolutionized the treatment of wet AMD, but numerous real-world patients experience vision loss due to their inability to adhere to the demanding treatment regimen involving frequent injections,” said Dr. Khanani. “OTX-TKI has shown the potential to reduce treatment burden while maintaining vision and anatomy for patients with wet AMD. The OTX-TKI pivotal trial design is unique as it is based on the latest FDA guidance for wet AMD. As a field, we must continue to push for the approval of new therapies while making sure we appropriately manage our patients who participate in these clinical trials. I look forward to working with Ocular Therapeutix, the regulatory agency and the retina community to harmonize regulatory endpoints with clinical practice.”
David Brown, MD, FACS, a renowned leader in the retina community, is the co-chairman of the medical leadership board at Retina Consultants of America (RCA) which has agreed to participate in the trial. Dr. Brown has pioneered research and treatment in retina, publishing more than 300 national meeting presentations, abstracts and scientific papers, including many of the primary papers that established the use of anti-VEGF agents for AMD, diabetic retinopathy and retinal vein occlusion.
“This pivotal trial is thoughtfully designed to satisfy the FDA’s latest guidance in wet AMD, while also balancing the needs of subjects who enter it,” said Dr. Brown. “The sites at RCA are excited to be a part of the OTX-TKI program as we continue our mission to develop better treatments for patients in the fight against retinal blindness.”
The pivotal trial was initiated based on the previously announced positive 12-month top-line data from the Company’s 21-subject U.S.-based Phase 1 trial of OTX-TKI for the treatment of wet AMD. Data from this trial were presented at the Clinical Trials at the Summit 2023 Meeting held in Park City, Utah in June 2023. In that trial, subjects treated with a single implant of OTX-TKI were observed to have an 89% reduction in treatment burden compared to subjects treated with aflibercept while maintaining visual acuity and retinal thickness through 12 months. Subjects in the OTX-TKI arm had mean changes from baseline of -1.0 letters in BCVA and +20.2 μm in CSFT at 12 months, comparable to mean changes from baseline for subjects in the aflibercept arm of +2.0 letters and -2.2 μm. 60% of OTX-TKI subjects remained rescue-free up to 12 months. As of the April 14, 2023 data cut-off date, no drug-related ocular or systemic serious adverse events through 12 months were observed in the OTX-TKI arm.
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 age-related macular degeneration (wet AMD) and other retinal diseases.
About Ocular Therapeutix, Inc.
Ocular Therapeutix, Inc. is a biopharmaceutical company focused on the formulation, development and commercialization of innovative therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology. Ocular Therapeutix’s first commercial drug product, DEXTENZA®, is an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery and ocular itching associated with allergic conjunctivitis. Ocular Therapeutix’s earlier stage development assets include: OTX-TKI (axitinib intravitreal implant), currently in a Phase 1 clinical trial for the treatment of diabetic retinopathy and a pivotal Phase 3 trial for wet AMD; OTX-TIC (travoprost intracameral implant), currently in a Phase 2 clinical trial for the treatment of primary open-angle glaucoma or ocular hypertension; and OTX-CSI (cyclosporine intracanalicular insert) for the chronic treatment of dry eye disease and OTX-DED (dexamethasone intracanalicular insert) for the short-term treatment of the signs and symptoms of dry eye disease, both of which have completed Phase 2 clinical trials.