On December 19, 2024, Opus Genetics, Inc. announced it reached an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for a Phase 3 clinical trial of oral APX3330. This trial will evaluate APX3330 for the treatment of moderate to severe non-proliferative diabetic retinopathy (NPDR).
The SPA agreement confirms that the proposed Phase 3 trial design, endpoints, and planned analyses are adequate to support a New Drug Application (NDA) submission, contingent on successful trial outcomes. The primary endpoint is a reduction in 3-step or greater worsening on the binocular diabetic retinopathy severity scale (DRSS) score.
Opus Genetics intends to seek a partner for APX3330 to fund its further development, allowing the company to focus resources on its gene therapy candidates for inherited retinal diseases. This SPA agreement helps de-risk certain regulatory aspects of the APX3330 program, making it a more attractive asset for potential partners.
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