FDA Supports Oculis’ Privosegtor for Acute Multiple Sclerosis Relapses

Oculis has received positive feedback from the U.S. Food and Drug Administration (FDA) supporting the clinical and regulatory development of Privosegtor for the treatment of acute multiple sclerosis (MS) relapses. The FDA Division of Neurology agreed that existing clinical data generated under the current investigational program can be cross-referenced for a new Investigational New Drug (IND) application, allowing the company to expand Privosegtor beyond optic neuritis into a broader MS population. Oculis plans to submit the IND during the fourth quarter of 2026.

The regulatory feedback marks an important step for Privosegtor, which is being developed as a neuroprotective therapy to limit nerve damage during acute neurological attacks, an area where no approved treatment currently exists.

Expanding Neuroprotection Beyond Optic Neuritis

Privosegtor is a novel peptoid small molecule that penetrates both the blood-brain barrier and the retinal barrier. Unlike current therapies that primarily suppress inflammation, the investigational agent is intended to protect neurons and axons from irreversible damage during acute disease episodes.

The expansion into acute MS relapses builds on findings from the Phase 2 ACUITY trial in optic neuritis (NCT04762017), an inflammatory condition frequently associated with new-onset MS or disease relapses. Because optic neuritis shares key pathological mechanisms with other acute MS relapses, it provides a clinically relevant model for evaluating neuroprotective therapies.

MS affects approximately 2.8 million people worldwide, including around 850,000 individuals in the United States. Although disease-modifying therapies reduce relapse frequency, an estimated 170,000 acute MS relapses still occur annually in the U.S. Recovery is often incomplete, contributing to cumulative neurological disability over time.

FDA Endorses Proposed Development Strategy

Following a pre-IND interaction, the FDA confirmed that Oculis may rely on data from the existing Privosegtor IND without conducting additional preclinical studies before initiating development in acute MS relapses.

The agency also supported several key elements of the proposed clinical program, including:

• Enrollment of patients experiencing optic neuritis as well as other ambulatory MS relapses.
• Evaluation of a primary efficacy endpoint at three months.
• Use of the same 3 mg/kg intravenous dose administered once daily for five consecutive days currently being evaluated in the ongoing PIONEER-1 registrational study.

These recommendations provide a clear regulatory pathway as the company prepares for clinical development in a broader MS population.

Phase 2 ACUITY Data Established Proof of Neuroprotection

The FDA feedback was supported by encouraging results from the completed Phase 2 ACUITY study.

Privosegtor demonstrated improvements in visual function while preserving retinal ganglion cell integrity, including the ganglion cell-inner plexiform layer (GCIPL) and retinal nerve fiber layer (RNFL). Treatment also reduced circulating neurofilament levels, an established biomarker of neuroaxonal injury.

Preclinical studies further supported its mechanism by showing preservation of retinal ganglion cells in animal models of optic neuritis, glaucoma, and multiple sclerosis, together with improvements in mobility in MS models.

The ongoing global PIONEER registrational program is now evaluating Privosegtor in optic neuritis and non-arteritic anterior ischemic optic neuropathy (NAION).

Executive Perspective

Oculis Chief Executive Officer Dr. Riad Sherif said the FDA’s feedback validates a regulatory pathway for Privosegtor in acute MS relapses, where no approved neuroprotective therapies are available. He noted that the expansion builds on positive Phase 2 findings in optic neuritis and reinforces the company’s strategy to establish a leading neuro-ophthalmology pipeline.

Neurologist Professor Amit Bar-Or of the Perelman School of Medicine at the University of Pennsylvania highlighted the need for therapies that improve neurological recovery after relapses. While immunomodulatory treatments reduce relapse frequency, he noted they rarely prevent the lasting neurodegeneration that contributes to long-term disability. A central nervous system-penetrant neuroprotective therapy such as Privosegtor could potentially improve recovery and long-term outcomes.

Path Forward

Oculis expects to submit an IND for Privosegtor in acute MS relapses during the fourth quarter of 2026. The company also plans to host a virtual R&D Day later this year to present additional details on the pivotal PIONEER registrational program and outline the development strategy for acute MS relapses.

Privosegtor has already received Breakthrough Therapy, Orphan Drug, and Priority Medicines (PRIME) designations for optic neuritis from U.S. and European regulators. If ongoing clinical studies confirm its neuroprotective benefits, the investigational therapy could become the first approved treatment that directly protects neurons during acute optic neuritis and multiple sclerosis relapses.

Reference

Oculis | Oculis Announces Positive FDA Pre-IND Feedback Supporting Privosegtor Development Strategy for Treatment of Acute Multiple Sclerosis Relapses