Key Takeaways

  • Oculis received FDA Special Protocol Assessment (SPA) agreement for its Phase 3 PIONEER-1 trial of Privosegtor in optic neuritis
  • Privosegtor, a novel neuroprotective small molecule designed to cross the blood-brain and retinal barriers, showed positive Phase 2 results with meaningful improvements in visual function and favorable safety data
  • The company believes Privosegtor could become the first neuroprotective therapy for optic neuritis

Oculis announced that the FDA has granted a Special Protocol Assessment (SPA) agreement for PIONEER-1, the first registrational phase 3 trial evaluating Privosegtor for the treatment of optic neuritis (ON).

The SPA agreement confirms that the design and planned statistical analysis of the PIONEER-1 study are acceptable to support a future new drug application (NDA), contingent on successful trial results and FDA review of the completed submission package.

Privosegtor is a novel peptoid small molecule designed to cross both the blood-brain and retinal barriers, positioning it as a potential first-in-class neuroprotective therapy for optic neuritis. The therapy may also have broader applications across neuro-ophthalmic and neurological diseases.

The FDA’s SPA decision follows positive outcomes from the phase 2 ACUITY trial and marks another milestone in Oculis’ PIONEER clinical development program, which includes two pivotal studies intended to support global registration efforts for Privosegtor in ON.

“The FDA SPA agreement for the PIONEER-1 trial, following Breakthrough Therapy and PRIME designations from the FDA and EMA, clarifies our path to NDA and validates our scientific approach,” said Riad Sherif, MD, Chief Executive Officer of Oculis. “With a potential $7 billion U.S. market in acute optic neuropathies, Privosegtor aims to address a critical gap in neuroprotection in neuro-ophthalmology and beyond.”

The PIONEER-1 phase 3 study will assess Privosegtor in a broad population of optic neuritis patients, including individuals with and without multiple sclerosis (MS). The trial’s primary endpoint is the proportion of patients achieving at least a 15-letter improvement from baseline in low-contrast visual acuity (LCVA) at Month 3. Participants will be monitored for 12 months to evaluate long-term safety and tolerability. The trial’s dosing regimen and enrollment criteria are expected to closely reflect those used in the phase 2 ACUITY study.

In the ACUITY trial, Privosegtor combined with steroids demonstrated substantial improvements in vision at Month 3 that were sustained through Month 6, as measured by LCVA. The treatment also showed consistent anatomical and biological markers of neuroprotection compared with placebo plus steroids.

According to the company, the most common drug-related adverse events were headache and acne, each reported in two participants (0.5%). No drug-related serious adverse events or treatment discontinuations were observed.