Oculis Advances Privosegtor After FDA Feedback

Oculis Holding has announced it will initiate a registrational development program for Privosegtor, a novel neuroprotective small molecule, following a positive Type C meeting with the U.S. Food and Drug Administration (FDA). The candidate is being developed for two rare and vision-threatening neuro-ophthalmic conditions: acute optic neuritis (AON) and non-arteritic anterior ischemic optic neuropathy (NAION), both of which currently lack approved neuroprotective therapies.

PIONEER Program to Include Three Pivotal Trials

Based on regulatory alignment with the FDA, Oculis plans to initiate the PIONEER program, consisting of three pivotal trials:

• PIONEER-1 (AON) – Expected launch in Q4 2025

• PIONEER-2 (AON) – Planned for H1 2026

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

The PIONEER-1 and PIONEER-2 trials will assess Privosegtor in patients with AON, including those with and without multiple sclerosis (MS). The primary endpoint for both will be low-contrast visual acuity (LCVA) measured at three months. The study designs are informed by the phase 2 ACUITY trial, which showed positive outcomes in visual function and retinal structural preservation.

Regulatory Pathway Open for Broader Indications

In addition to AON, the FDA has confirmed that NAION can be studied under the current investigational new drug (IND) application. The upcoming PIONEER-3 trial will apply a similar design to the AON studies and evaluate Privosegtor following acute NAION onset. Running all three trials in parallel is expected to create operational and cost efficiencies while accelerating timelines.

Addressing Significant Unmet Needs

AON often serves as the initial clinical presentation of MS. While corticosteroids can reduce the duration of inflammation, they do not prevent permanent vision loss. NAION, meanwhile, is the leading cause of sudden optic nerve-related vision loss in older adults and lacks any proven pharmacologic or surgical interventions. Privosegtor is designed to offer direct neuroprotection, targeting optic nerve axons and retinal ganglion cells to preserve visual function in both diseases.

Company Outlook

“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 data in DME, Oculis is now in a strong position with multiple pivotal studies targeting multi-billion-dollar markets.”