Ocular Therapeutix Receives FDA Agreement Under Special Protocol Assessment for its First Pivotal Clinical Trial of OTX-TKI in Wet AMD

Ocular Therapeutix, Inc. recently announced it had received written agreement regarding the overall design from the US FDA under a Special Protocol Assessment (SPA) for the company’s ongoing pivotal Phase 3 clinical trial for AXPAXLI (axitinib intravitreal implant), for the treatment of wet age-related macular degeneration (wet AMD), which the company refers to as the SOL trial.

The Ccompany initiated the SOL trial in September 2023 and expects to enroll approximately 300 evaluable wet AMD subjects who are treatment naïve in the study eye in the trial. The SOL trial is designed to be a multi-center, parallel-group trial run primarily at US sites, with subjects randomized to one injection of aflibercept or one implant of AXPAXLI followed by supplemental anti-VEGF treatment based on pre-specified criteria. With the agreement under the SPA, the Company will begin enrolling patients in the SOL trial and expects to dose the first subject by year-end.

AXPAXLI 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 and other retinal diseases. AXPAXLI is also referred to by its laboratory code, OTX-TKI.

Wet age-related macular degeneration (wet AMD) is a leading cause of severe, irreversible vision loss affecting approximately 14 million individuals globally and 1.6 million in the US alone (2022 Market Scope Retinal Pharmaceuticals Market Report). Wet AMD causes vision loss due to abnormal new blood vessel growth and hyperpermeability and associated retinal vascularity in the macula, which is primarily stimulated by local upregulation of vascular endothelial growth factor (VEGF). Without prompt and continuous treatment to control this exudative activity, patients develop irreversible vision loss. With proper treatment, patients may maintain visual function for a period of time and may temporarily regain lost vision. Challenges with current therapies include repeated intraocular injections every 1 to 4 months, treatment-related adverse events, patient compliance, and lack of vision improvement.

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 ELUTYX. 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: AXPAXLI (axitinib intravitreal implant), currently in a pivotal Phase 3 trial for wet AMD and a Phase 1 clinical trial for the treatment of diabetic retinopathy; 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.