Oculis Announces U.S. FDA Breakthrough Therapy Designation Granted to Privosegtor for Treatment of Optic Neuritis

Core Insights - Oculis Holding AG's neuroprotective candidate Privosegtor has received Breakthrough Therapy Designation from the FDA for the treatment of optic neuritis, highlighting its potential as a first-of-its-kind therapy in this area [1][7]. Company Overview - Oculis is a global biopharmaceutical company focused on innovations in neuro-ophthalmology and ophthalmology, with a late-stage clinical pipeline that includes Privosegtor, OCS-01, and Licaminlimab [15]. Product Details - Privosegtor is a novel peptoid small molecule designed to penetrate both the blood-brain and retinal barriers, potentially addressing significant unmet medical needs in optic neuropathies, with a market opportunity estimated at $7 billion in the U.S. alone [2][5]. - The drug has shown substantial improvements in visual function, with an average gain of 18 letters in Low Contrast Visual Acuity (LCVA) compared to placebo in the Phase 2 ACUITY trial [4][7]. Clinical Trial Insights - The ACUITY trial demonstrated that Privosegtor not only improved visual function but also preserved anatomical structures of the retina and optic nerve, indicating its neuroprotective potential [3][4]. - The trial involved 36 patients with acute optic neuritis, showing that those treated with Privosegtor alongside IV methylprednisolone experienced significant visual improvements [12]. Regulatory and Market Implications - Following a successful meeting with the FDA, Oculis initiated the PIONEER program, which includes three pivotal trials aimed at supporting registration plans for Privosegtor in optic neuritis and non-arteritic anterior ischemic optic neuropathy (NAION) [5][6]. - The unmet medical needs in these conditions are significant, as there are currently no approved neuroprotective therapies available [10][11].