FDA, Opus Genetics reach special protocol assessment agreement for APX3330 trial

Key takeaways:

• The agreement deems the study’s design as supportive for a future new drug application.
• The primary endpoint will be a reduction of three steps or more on the binocular diabetic retinopathy severity scale.

Opus Genetics and the FDA have reached an agreement on a special protocol assessment for a phase 3 clinical trial investigating oral APX3330 for the treatment of moderate to severe nonproliferative diabetic retinopathy.

According to a company press release, the FDA deemed the trial’s design, endpoints and planned analyses to be adequate to support a new drug application submission for the treatment if the outcomes are successful. The agreed primary endpoint will be a reduction in the binocular diabetic retinopathy severity scale of three steps or more with the treatment.

The agreement is supported by results from the phase 2 ZETA-1 trial that showed a favorable safety profile and the potential to slow or prevent clinically meaningful progression of DR with APX3330.

“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, CEO of Opus Genetics, said in the 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.”

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