Agreement Reached on Primary Endpoint and Phase 3 Trial Design
Oral APX3330 is a Late-Stage Clinical Asset Available for Partnering
Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small-molecule drugs to treat other ophthalmologic disorders, announced that it has reached 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 agreed primary endpoint 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,” said George Magrath, MD, CEO of Opus Genetics. "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.”
Diabetic retinopathy is a progressive eye disease which results in vision impairment and blindness among adults with diabetes. It is the leading cause of blindness in working age adults and impacts approximately 10 million patients in the US.
About APX3330
APX3330 is a first-in-class, small molecule, oral inhibitor of the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). With a novel, multimodal mechanism of action, APX3330 modulates the downstream pathways regulated by Ref-1 – which involve angiogenesis (VEGF), oxidative stress (Nrf2) and inflammation (NFkB) – to restore homeostatic levels of angiogenenic, oxidative stress and inflammatory factors that are implicated across several ocular diseases, including DR, diabetic macular edema (DME), and age-related macular degeneration (AMD). APX3330 has shown a favorable tolerability profile in 12 clinical trials conducted in healthy, hepatitis, cancer, and diabetic subjects.