Oculis Holding AG announced that the US Food and Drug Administration (FDA) has granted a special protocol assessment (SPA) agreement for PIONEER-1, the first registrational phase 3 trial evaluating Privosegtor (OCS-05) for optic neuritis. The SPA confirms that the study design and planned statistical analysis are acceptable to support a future new drug application, pending successful trial results and FDA review.

Privosegtor is a peptoid small molecule designed to cross the blood-brain and retinal barriers and is being developed as a neuroprotective therapy for optic neuritis and related neuro-ophthalmic conditions. PIONEER-1 will enroll patients with optic neuritis, including individuals with and without multiple sclerosis. The primary endpoint is the proportion of patients achieving at least a 15-letter improvement in low-contrast visual acuity at month 3. Participants will be followed for 12 months for safety and tolerability assessments.

The study design closely follows the phase 2 ACUITY trial, in which Privosegtor combined with steroids demonstrated improvements in low-contrast visual acuity through month 6, along with anatomical measures consistent with neuroprotection. The most common treatment-related adverse events were headache and acne, each occurring in 10.5% of participants. No serious drug-related adverse events or discontinuations were reported.

Privosegtor previously received breakthrough therapy designation from the FDA and PRIME designation from the European Medicines Agency for optic neuritis. RP