Privosegtor optic neuritis clinical trial design gains FDA agreement
PIONEER-1 is first of two studies planned to support approval bid
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The FDA agreed to the design and planned analysis of PIONEER-1, a Phase 3 trial testing privosegtor in people with optic neuritis. (Image from iStock)
- The FDA agreed to the design of a Phase 3 trial testing privosegtor for optic neuritis.
- Optic neuritis, often linked to multiple sclerosis, involves optic nerve inflammation that can cause vision loss.
- PIONEER-1 will assess whether privosegtor can improve vision after acute optic neuritis.
The U.S. Food and Drug Administration (FDA) has formally agreed that the design and planned analysis of an upcoming Phase 3 trial testing privosegtor in people with optic neuritis could support a future application seeking the therapy’s approval if the trial succeeds.
Optic neuritis, or inflammation of the optic nerve that carries information between the eyes and the brain, is a common vision problem in multiple sclerosis (MS) that can lead to permanent vision problems or vision loss.
The PIONEER-1 trial will evaluate the therapy’s safety and efficacy in adults with optic neuritis in a broad population, including those with and without MS. This is the first of two registrational trials that Oculis is planning to support a potential approval application for privosegtor in optic neuritis.
The special protocol assessment (SPA) agreement follows recent designations granted by the FDA and European Medicines Agency (EMA) to privosegtor to accelerate its development and review for optic neuritis.
FDA agreement may support future approval bid
“The FDA SPA agreement for the PIONEER-1 trial, following Breakthrough Therapy and PRIME designations from the FDA and EMA, clarifies our path to [regulatory submission] and validates our scientific approach,” Riad Sherif, MD, Oculis’ CEO, said in a company press release.
Optic neuritis is one of the most common vision problems in MS, with up to 70% of patients experiencing it at some point during the disease. It can lead to problems ranging from blurred vision to complete vision loss.
Privosegtor, formerly known as OCS-05, is an investigational small molecule designed to protect nerve cells and preserve vision in people with acute optic neuritis. The therapy is thought to act on pathways involved in nerve cell development, survival, and repair.
In the completed Phase 2 ACUITY trial (NCT04762017), which enrolled people with acute optic neuritis, privosegtor plus steroid treatment improved measures of visual function and eye health while being generally well tolerated.
“Privosegtor has demonstrated compelling results in the treatment of optic neuritis with improvement of visual function combined with positive anatomical and biological measures of nerve cell preservation,” said Mark Kupersmith, MD, Oculis’ chief medical advisor of neuro-ophthalmology and professor at Icahn School of Medicine at Mount Sinai Hospital.
Based on the positive Phase 2 results, Oculis advanced privosegtor into its PIONEER registrational program. The program includes two registrational trials in optic neuritis and one in non-arteritic anterior ischemic optic neuropathy, or NAION.
PIONEER program includes two optic neuritis trials
PIONEER-1 and PIONEER-2 are intended to support an approval application for optic neuritis. A third trial, called PIONEER-3, will evaluate the therapy in NAION, a condition in which the optic nerve is damaged due to reduced blood flow.
PIONEER-1 will enroll adults ages 18 to 50 who are experiencing their first episode of acute optic neuritis in one eye and can start treatment within 12 days of symptom onset. Participants will be randomly assigned to receive either privosegtor or a placebo, each in combination with standard corticosteroid treatment.
Treatment will be given once daily for five consecutive days. At each visit, participants will first receive methylprednisolone, a corticosteroid commonly used for acute optic neuritis, followed by an infusion of privosegtor or placebo. They will then be followed for about one year.
The main goal is to determine how many patients have at least a 15-letter improvement after three months on a vision test called low-contrast letter acuity. This test measures how well a person can read faint gray letters on a white background.
Researchers will also assess the therapy’s safety and tolerability over one year.
“I look forward to continuing our collaborative work with Oculis to further advance this promising candidate through late-stage clinical development,” Kupersmith said.
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