Oculis Holding announced it will advance its novel neuroprotective candidate Privosegtor into a registrational development program following a positive meeting with the FDA.

Privosegtor, a new peptoid small molecule, is designed to become the first neuroprotective therapy for acute optic neuritis (AON) and non-arteritic anterior ischemic optic neuropathy (NAION)—two rare, vision-threatening neuro-ophthalmic diseases with no approved neuroprotective treatments.

Based on FDA feedback, Oculis will initiate the PIONEER program later this year, comprising three pivotal trials to support registration plans for Privosegtor in AON and NAION:

  • PIONEER-1 (AON) – Launching in Q4 2025

  • PIONEER-2 (AON) – Planned for 1H 2026

  • PIONEER-3 (NAION) – Expected start in mid-2026

The PIONEER-1 and PIONEER-2 studies will evaluate Privosegtor after the acute onset of optic neuritis in patients with and without multiple sclerosis (MS). Both trials will use low-contrast visual acuity (LCVA) at 3 months as the primary endpoint. The study design builds upon the successful phase 2 ACUITY trial, which demonstrated improvement in visual function and structural retinal preservation in AON patients.

The FDA also confirmed that Privosegtor can be evaluated in other neuro-ophthalmic indications, including NAION, under the current investigational new drug (IND) application. The PIONEER-3 study will mirror the design of the AON studies and assess Privosegtor following acute NAION onset. Running the three trials in parallel is expected to yield operational synergies, cost efficiencies, and accelerated timelines.

Acute optic neuritis (AON) often represents the first clinical manifestation of multiple sclerosis. While corticosteroids may shorten the duration of inflammation, they do not protect against permanent vision loss. Similarly, non-arteritic anterior ischemic optic neuropathy (NAION)—the most common cause of sudden optic nerve-related vision loss in older adults—lacks any proven pharmacological or surgical treatment that can improve outcomes.

Privosegtor aims to fill these gaps by delivering direct neuroprotection, preserving optic nerve axons and retinal ganglion cells to maintain visual function.

“The positive FDA meeting marks a significant milestone for our rapidly advancing neuroprotection candidate," said Riad Sherif, MD, Chief Executive Officer of Oculis. "The PIONEER program positions Oculis as a leader in ophthalmic neuroprotection drug development. With this milestone and the upcoming Diamond phase 3 DME program results, Oculis is now in a strong position with multiple pivotal studies targeting multi-billion-dollar markets.”