Oculis Announces Positive OCS-05 Phase 2 ACUITY Trial in Acute Optic Neuritis, Met Primary Safety Endpoint and Key Secondary Efficacy Endpoints Opening Development Pathways as a Potential First-in-Class Neuroprotective Therapy

Clinical Trial Results - OCS-05 achieved the primary safety endpoint and demonstrated statistically significant results on key secondary efficacy endpoints in the Phase 2 ACUITY trial for acute optic neuritis [1][3] - The trial showed a 43% improvement in Ganglion Cell-Inner Plexiform Layer (GCIPL) thickness and a 28% improvement in Retinal Nerve Fiber Layer (RNFL) thickness at month 3, with further improvements at month 6 [5][7] - OCS-05 demonstrated a favorable difference in Low Contrast Letter Acuity (LCVA) mean change from baseline of approximately 18 letters at month 3 and 15 letters at month 6 [8] Safety and Tolerability - OCS-05 showed a favorable safety and tolerability profile compared to placebo, with no drug-related serious adverse events (SAEs) or AEs leading to drug withdrawal or study discontinuation [6][9] - The most frequently reported drug-related AEs in the OCS-05 treatment group were headache and acne, each occurring in 10.5% of patients [9] Regulatory and Development Status - OCS-05 has received orphan drug designation from both the FDA and EMA for acute optic neuritis [8] - The Investigational New Drug (IND) application for OCS-05 has been cleared by the FDA, enabling the initiation of clinical development in the United States [9][10] Company and Pipeline Overview - Oculis is a global biopharmaceutical company focused on developing innovative treatments for eye care, with a pipeline including OCS-01, OCS-02, and OCS-05 [19] - The company has a strong financial position with approximately $105 to 110 million in cash, cash equivalents, and short-term investments [10] Market and Medical Need - Acute optic neuritis is a rare condition affecting up to 8 in 100,000 people worldwide, with no currently approved therapies specifically indicated for the condition [15] - There is a critical unmet need for neuroprotective therapies to preserve vision and prevent permanent visual impairment in acute optic neuritis patients [8][15]