Announcement • Aug 24

Oculis Holding AG Treats First Patient In PIONEER-1 Registrational Trial Of Privosegtor For Optic Neuritis

Oculis Holding AG has treated the first patient in the PIONEER-1 registrational trial evaluating Privosegtor for the treatment of optic neuritis (ON). The PIONEER program marks a key step toward potential first-in-class neuroprotection for optic neuritis and other neurological diseases, supported by positive Acuity Phase 2 data, FDA Breakthrough Therapy and EMA PRIME designations, and recent positive feedback from the FDA Neurology Division. Privosegtor, a novel peptoid small molecule that can cross the blood-brain and retinal barriers, could become the first neuroprotective therapy for ON, with broad potential applicability in other neuro-ophthalmic and neurological diseases. Following the successful Phase 2 ACUITY trial, Oculis launched the PIONEER (Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research) program, which includes two registrational trials in ON. The first registrational trial in the program, PIONEER-1, is evaluating Privosegtor in patients with acute-onset ON across a broad population, including patients with and without multiple sclerosis (MS). Oculis and its partners have been focused on establishing a robust, multidisciplinary network connecting emergency room physicians, ophthalmologists, and neurologists, as well as patient screening. This integrated approach aims to optimize operational flows from patient identification to treatment, capitalizing on peak enrolment opportunities during the fall, winter, and spring to align with the disease's natural seasonality. The primary endpoint of PIONEER-1 is the proportion of patients achieving at least a 15-letter gain from baseline, and the secondary endpoint is the mean change in low-contrast visual acuity (LCVA) at Month 3, both clinically meaningful functional endpoints in ON. The primary analysis will be conducted at Month 3, and patients will be followed through Month 12 to assess long-term safety and tolerability. Dosing and patient enrolment criteria will closely mirror those of the Phase 2 ACUITY trial, in which Privosegtor showed substantial improvements in vision at Month 3, which persisted through Month 6, as measured by LCVA, along with consistent anatomical and biological neuroprotective benefits compared with placebo. These positive findings supported the granting of Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of ON. Oculis received written agreement from the FDA, under a Special Protocol Assessment (SPA) agreement, confirming that the design and planned analysis of the PIONEER-1 trial will adequately address the objectives necessary to support an NDA submission in ON, subject to a successful outcome of the trial and review of all the data in the NDA submission. Building on the ACUITY Phase 2 dataset in ON and based on constructive pre-IND feedback from FDA’s Neurology division, Oculis is planning an IND submission for Privosegtor to treat acute MS relapses, anticipated in Fourth Quarter 2026. Privosegtor, a novel peptoid small-molecule candidate that crosses the blood-brain and retinal barriers, has the potential to become the first neuroprotective therapy for optic neuritis (ON) and other neuro-ophthalmic and neuro-axonal diseases. Positive results from the ACUITY Phase 2 trial showed Privosegtor’s neuroprotective potential, as evidenced by improvements in visual function, corroborated by anatomical preservation of the retina, including GCIPL and RNFL layers, and reduced neurofilament levels in the blood after an acute episode of optic neuritis. Consistent results were observed in animal models of glaucoma, optic neuritis, and multiple sclerosis (MS), where Privosegtor preserved retinal ganglion cells and was associated with improvements in mobility (clinical function disability) in the MS model. Privosegtor has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) as well as Orphan Drug designation from both the FDA and the EMA for ON. Privosegtor is currently being evaluated in Oculis’ PIONEER (Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research) program, which includes two registrational trials in ON and one registrational trial in non-arteritic anterior ischemic optic neuropathy (NAION). Building on the ACUITY Phase 2 dataset in ON and constructive FDA pre-IND feedback, Oculis is also planning an IND submission for Privosegtor for the treatment of acute MS relapses. Privosegtor is an investigational drug and has not received regulatory approval for commercial use in any country.

Oculis Holding AG Stock Price

OCS Share Price Performance

OCS Community Narratives

Trending Discussion

Recently Updated Narratives

Snowflake Analysis

Oculis Holding AG Key Details

About OCS

Recent OCS News & Updates

Oculis Holding AG Treats First Patient In PIONEER-1 Registrational Trial Of Privosegtor For Optic Neuritis

New minor risk - Shareholder dilution

Second quarter 2026 earnings: EPS and revenues exceed analyst expectations

Recent updates

Oculis Holding AG Competitors

Disc Medicine

Structure Therapeutics

Zai Lab

SELLAS Life Sciences Group