EMA grants PRIME status to new optic neuritis therapy privosegtor
Experimental therapy may help protect vision in MS-related condition
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Optic neuritis, a condition causing vision problems, is often linked to MS. (Image from iStock)
- Privosegtor received EMA PRIME designation for optic neuritis, a condition that can cause vision problems and is often linked to multiple sclerosis.
- This experimental neuroprotective therapy is designed to help protect optic nerves and preserve vision.
- Phase 2 results showed improvements in vision measures and signs of nerve protection.
The European Medicines Agency (EMA) has granted PRIME designation to Oculis‘ experimental treatment privosegtor for optic neuritis, a condition commonly seen in people with multiple sclerosis (MS).
Optic neuritis, marked by inflammation of the optic nerve that carries signals between the eyes and the brain, can lead to lasting vision problems. While also associated with other conditions, it is often one of the first signs of MS and can also occur during disease relapses.
PRIME designation supports faster development of promising therapies
EMA’s PRIME designation is intended to speed the development of therapies that may offer major benefits over existing options for conditions with unmet medical needs. The status provides Oculis with more frequent interactions with EMA experts, scientific advice, and may allow for an accelerated review process.
“EMA’s decision to grant PRIME designation highlights privosegtor’s compelling results and its potential as a first-in-class neuroprotective therapy for people experiencing optic neuritis,” Riad Sherif, MD, Oculis’ CEO, said in a company press release.
Up to 70% of people with MS are estimated to experience optic neuritis, which can lead to vision problems ranging from blurred vision to vision loss. While the condition may resolve on its own, corticosteroid treatment is often used to shorten the duration of the inflammatory attack, though some patients may still develop permanent vision loss.
Privosegtor, formerly known as OCS-05, is a small molecule being developed as a potential neuroprotective therapy aimed at preserving optic nerve function and vision in people with optic neuritis. It is administered via intravenous (into-the-vein) infusions.
“Despite the current use of corticosteroids to shorten the inflammatory attack after an acute episode of optic neuritis, there remains an unmet medical need for novel therapies that can prevent vision loss by providing neuroprotection,” Sherif said.
Phase 2 trial data support privosegtor’s potential benefits
According to Oculis, EMA’s designation was supported by results from the Phase 2 ACUITY clinical trial (NCT04762017). The study enrolled 36 people with acute optic neuritis in one eye caused by MS or other similar disorder marked by myelin loss.
Participants were within 12 days of their first symptoms of optic neuritis and were randomly assigned to receive one of two doses of privosegtor or a placebo for five days, on top of standard corticosteroids.
Results showed that those given the highest dose were able to read a mean of 18 more letters after three months, and 15 letters after six months, than those on a placebo. This was assessed with the low-contrast letter acuity test, which evaluates how well a person can read faint gray letters on a white background.
Privosegtor was also associated with preservation of retinal structure and other measures related to the optic nerve, as well as reduced levels of a nerve damage biomarker.
Following positive Phase 2 data, Oculis is now advancing privosegtor through its PIONEER program, which includes three global registrational trials.
Two of these — PIONEER-1 and PIONEER-2 — are evaluating the therapy for acute optic neuritis in people with and without MS, with PIONEER-1 underway. If positive, data could support regulatory applications seeking the approval of privosegtor for optic neuritis.
PIONEER program expands testing across optic nerve conditions
A third trial, called PIONEER-3, will evaluate the therapy in non-arteritic anterior ischemic optic neuropathy, a condition in which the optic nerve is damaged due to reduced blood flow.
“Through our PIONEER registrational program, we look forward to further progressing privosegtor’s late-stage clinical development globally and delivering on our commitment to redefine what’s possible for patients,” Sherif added.
Privosegtor also received orphan drug designation in the U.S. and the European Union, and breakthrough therapy designation in the U.S. for optic neuritis. These designations are intended to support and help expedite the development and review of the treatment.
