Oculis AG(US:OCS) Globenewswire·2026-01-06 09:00Core Insights - Oculis Holding AG announced that its neuroprotective drug, Privosegtor, has received Breakthrough Therapy Designation from the FDA for the treatment of optic neuritis, addressing significant unmet medical needs in this area [1][7]. Company Overview - Oculis is a global biotechnology company focused on innovation for treating eye diseases, with a pipeline that includes three key development drugs: Privosegtor, OCS-01, and Licaminlimab [15]. - The company is headquartered in Switzerland and has operations in the United States and Iceland, backed by leading global healthcare investment funds [15]. Drug Development and Market Opportunity - Privosegtor is a small molecule peptide mimic that can cross the blood-brain and retinal barriers, potentially becoming the first neuroprotective treatment for optic nerve disorders, with a combined market opportunity of over $7 billion in the U.S. alone [2][5]. - The ACUITY Phase 2 study demonstrated significant improvements in visual acuity with Privosegtor, showing an average increase of 18 letters on the ETDRS visual acuity scale compared to placebo after three months [4][7]. Clinical Trial Insights - The ACUITY study involved 36 eligible patients aged 18 to 60 with recent onset optic neuritis, comparing Privosegtor with placebo alongside intravenous steroids [12][13]. - Results indicated that Privosegtor not only improved visual acuity but also maintained the structural integrity of the retina and optic nerve, which typically suffer damage during acute optic neuritis [4][9]. Regulatory Status - The Breakthrough Therapy Designation is intended to expedite FDA review for drugs that show substantial improvement over existing treatments for serious conditions [14]. - Following a successful meeting with the FDA in 2025, Oculis initiated the PIONEER program, which includes three registration studies to support the approval of Privosegtor for optic neuritis and NAION [5][6].