Opus Genetics has reached an agreement with the FDA on a Special Protocol Assessment (SPA) for a Phase 3 clinical trial evaluating oral APX3330 for the treatment of moderate to severe non-proliferative diabetic retinopathy (NPDR).

The SPA agreement reflects that the proposed Phase 3 trial design, endpoints, and planned analyses will be adequate to support a New Drug Application (NDA) submission for treatment of NPDR, subject to a successful outcome of the trial and review of all data in the NDA, the company said in a press release.

The agreed primary endpoint, according to the company, is a reduction in 3-step or greater worsening on the binocular diabetic retinopathy severity scale (DRSS) score, compared to placebo. In the previous Phase 2 ZETA-1 trial, oral APX3330 showed the potential to slow or prevent clinically meaningful progression of DR and demonstrated a favorable safety profile.

“This SPA agreement reflects our alignment with the FDA on the design of a Phase 3 trial for APX3330 and is a testament to the team’s developmental and regulatory acumen,” George Magrath, MD, Chief Executive Officer of Opus Genetics, said in the press release. "If successful in Phase 3 and subsequently approved, APX3330 has the potential to be a transformative treatment option for patients with NPDR. We believe that having this SPA in place will help de-risk certain regulatory aspects of this program. Our intention is to seek a partner for APX3330 to fund further development, as we focus our resources on advancing our gene therapy candidates for IRDs.”