Ocular Therapeutix announced that it has reached an agreement with the US Food and Drug Administration on the design of a registrational trial for OTX-TKI (Axpaxli), an investigational therapy for nonproliferative diabetic retinopathy (NPDR). Under the Special Protocol Assessment (SPA), the trial design and primary endpoint are established in advance, providing a defined regulatory pathway for the company.

OTX-TKI is an investigational intravitreal implant that uses a bioresorbable hydrogel fiber platform to deliver axitinib, a tyrosine kinase inhibitor, directly into the eye over an extended period to maintain therapeutic drug levels for many months, potentially reducing or eliminating the need for frequent anti-VEGF injections. Data from the phase 1 HELIOS trial in NPDR were reported recently at the American Society of Retina Specialists meeting. OTX-TKI is also being studied for neovascular age-related macular degeneration (nAMD). RP