FDA feedback supports new testing path for therapy in MS relapses
Oculis plans late-2026 filing to study privosegtor in acute relapse events
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Privosegtor is given by intravenous infusion and is being developed as a potential neuroprotective treatment for acute MS relapses. (Image by iStock)
- Oculis plans to expand clinical testing of privosegtor as a potential neuroprotective treatment for acute multiple sclerosis relapses.
- MS relapses can cause new or worsening symptoms, including optic neuritis, vision loss, weakness, and balance problems.
- Oculis plans to file an investigational new drug application after receiving supportive FDA feedback on the proposed clinical program.
After receiving positive regulatory feedback from the U.S. Food and Drug Administration (FDA), Oculis is planning to expand clinical testing of its experimental therapy privosegtor as a potential treatment for acute relapses in people with multiple sclerosis (MS).
Privosegtor is already being investigated as a treatment for optic neuritis — inflammation of the optic nerve, which carries visual information from the eye to the brain. Optic neuritis is a common manifestation of MS that often occurs during a relapse.
According to the company, the FDA said Oculis could cross-reference existing privosegtor data from its open investigational new drug (IND) application in a new application seeking clearance to test the therapy in people experiencing acute MS relapses. Oculis plans to file the new IND application toward the end of this year.
“Our recent positive engagement with the FDA supports a regulatory pathway for privosegtor in acute MS relapses, an area of high unmet need with no currently approved neuroprotective options,” Riad Sherif, MD, CEO of Oculis, said in a company press release.
Acute MS relapses can leave lasting nerve damage
MS occurs when the immune system mistakenly attacks the protective covering around nerve fibers, causing a range of symptoms. When this inflammation affects the optic nerve, it results in optic neuritis.
Most people with MS are diagnosed with relapsing-remitting MS, in which periods of new or worsening symptoms, called relapses, are followed by periods of recovery. While existing disease-modifying therapies can reduce the risk of relapses, they do not prevent them entirely.
When relapses involve more debilitating symptoms, such as loss of vision, severe weakness, or balance problems, they are usually treated with high-dose corticosteroids to reduce inflammation and speed recovery.
However, corticosteroids do not prevent the lasting nerve damage that can occur during a relapse, and recovery is often incomplete, with some symptoms lingering after the relapse has resolved.
“While immunomodulators are increasingly deployed in practice to reduce the frequency of relapses in MS patients, recovery from an acute event is very rarely complete. The neurodegeneration that occurs during these acute attacks persists after the relapse, leading to an accumulation of disabilities,” said Amit Bar-Or, MD, chief of the MS division at the Perelman School of Medicine at the University of Pennsylvania.
Rather than targeting inflammation, privosegtor is designed to protect nerve cells and their long projections, called axons, from damage.
“Privosegtor, as a … neuroprotective agent, could potentially enhance recovery following relapses and improve long-term outcomes for people with MS,” Bar-Or added.
Clinical program builds on optic neuritis trial findings
Given by intravenous infusion, privosegtor has already been tested in combination with corticosteroids for optic neuritis. Data from the Phase 2 ACUITY trial (NCT04762017) showed that the 3 mg/kg dose of privosegtor plus corticosteroids outperformed corticosteroids alone at preserving retinal nerve structures and improving measures of vision in people with optic neuritis.
Oculis is now evaluating privosegtor in the Phase 3 PIONEER program, which includes the PIONEER-1 (NCT07623668) and PIONEER-2 trials for optic neuritis. Both studies are intended to support a potential approval application for the therapy. PIONEER-1 is being conducted under a special protocol assessment, meaning the FDA agreed that its design and planned analysis could support a future approval application if the trial is successful.
The program for acute MS relapses will build on data from these clinical studies. The FDA said Oculis would not need to conduct additional laboratory or animal studies before submitting the planned IND application.
The agency also supported the company’s proposed clinical development plan, including using the same regimen as in PIONEER-1 — 3 mg/kg once daily for five days — and assessing the primary efficacy outcome as early as three months.
“Building on positive Phase 2 ACUITY data in optic neuritis, a frequent type of MS relapse, we believe privosegtor has the potential to mitigate neuroaxonal damage and improve recovery following acute relapse events,” Sherif said. “We look forward to sharing further details on PIONEER and this new program … later this year.”
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