Oculis Holding AG (NASDAQ:OCS) saw its stock price increase 4.2% on Thursday following an announcement that the U.S. Food and Drug Administration has issued a Special Protocol Assessment for the company's planned PIONEER-1 registrational trial of Privosegtor in optic neuritis.
The SPA confirms that the proposed trial design and the planned analyses for PIONEER-1 are adequate to meet the objectives required to support a future New Drug Application, provided the trial achieves a successful outcome and the FDA approves the full submission. The agreement formalizes the agency's view that the study structure is appropriate for registrational purposes under those conditions.
Privosegtor is described by the company as a peptoid small molecule that is capable of crossing both the blood-brain barrier and the retinal barrier. Oculis positions the compound as a potential first-in-class neuroprotective therapy for optic neuritis. Regulatory agencies have already given the drug accelerated review designations: it has received Breakthrough Therapy designation from the FDA and PRIME designation from the European Medicines Agency for the treatment of optic neuritis.
The PIONEER-1 Phase 3 study will enroll patients diagnosed with optic neuritis across a broad population, explicitly including individuals both with and without multiple sclerosis. The trial's primary endpoint is the proportion of patients who achieve at least a 15-letter improvement from baseline in low-contrast visual acuity at Month 3. Patients enrolled in PIONEER-1 will be followed for a total of 12 months to collect data on long-term safety and tolerability.
Oculis referenced earlier clinical data to contextualize the Phase 3 program. In the Phase 2 ACUITY trial, Privosegtor administered with a steroid showed improvements in vision at Month 3 that persisted through Month 6 when measured by low-contrast visual acuity. The Phase 2 safety profile noted the most common drug-related adverse events were headache and acne, each observed in two participants. The company reported no drug-related serious adverse events and no drug-related events that led to treatment or study discontinuations in that trial.
Oculis identifies itself as a global biopharmaceutical company focused on developing innovations to address unmet medical needs in ophthalmology and neuro-ophthalmology. The SPA for PIONEER-1 represents a regulatory milestone for the company as it moves Privosegtor into a registrational Phase 3 study designed to generate pivotal data for potential marketing approval.
Study details at a glance
- Trial name: PIONEER-1 Phase 3 registrational study
- Indication: Optic neuritis (broad population, with and without multiple sclerosis)
- Primary endpoint: Proportion of patients with >=15-letter gain in low-contrast visual acuity at Month 3
- Follow-up: 12 months for long-term safety and tolerability