Oculis Secures FDA SPA for Phase 3 Optic Neuritis Trial Targeting $7B Market
OCS•FDA has granted Oculis a Special Protocol Assessment for its Phase 3 PIONEER-1 trial of Privosegtor in optic neuritis, confirming trial design and analysis to support an NDA submission. The study’s primary endpoint is a ≥15-letter low-contrast visual acuity gain at Month 3 with 12-month safety follow-up.
1. SPA Agreement Details
FDA has granted a Special Protocol Assessment for the PIONEER-1 Phase 3 study of Privosegtor in optic neuritis, formally confirming that the trial’s design and statistical analysis plan can support an NDA submission upon successful trial outcomes.
2. PIONEER-1 Trial Design
PIONEER-1 will enroll patients with optic neuritis, including those with and without multiple sclerosis, and measure the proportion achieving at least a 15-letter gain in low-contrast visual acuity at Month 3, with all participants followed for 12 months to assess safety and tolerability.
3. Prior Clinical Data and Designations
In the Phase 2 ACUITY trial, Privosegtor plus steroid showed substantial low-contrast visual acuity improvements at Month 3 that persisted through Month 6, and the therapy received FDA Breakthrough Therapy and EMA PRIME designations based on its neuroprotective potential and safety profile.
4. Market Potential and Next Steps
With an estimated $7 billion U.S. market for acute optic neuropathies, Oculis plans to initiate PIONEER-1 patient enrollment following SPA alignment and aims for NDA submission upon successful trial completion and regulatory review.
