Oculis Holding AG (Nasdaq: OCS / ICX: OCS.IC) announced positive topline results from its Phase 2 ACUITY trial, revealing that OCS-05, a neuroprotective candidate, met its primary safety endpoint and achieved statistical significance in key secondary efficacy endpoints for treating acute optic neuritis. The multi-center, double-blind, placebo-controlled trial evaluated the safety, tolerability, and efficacy of OCS-05 (2mg/kg/day or 3mg/kg/day) administered intravenously once-daily for five days in patients with acute optic neuritis also receiving steroids.
The ACUITY trial randomized 36 patients with recent onset of unilateral acute optic neuritis of demyelinating origin, with 33 patients receiving treatment included in the modified intent-to-treat (mITT) analysis. The primary endpoint focused on safety, assessing shifts in electrocardiogram (ECG) parameters. Secondary endpoints evaluated changes in retinal structure using optical coherence tomography (OCT) and visual function using low contrast letter acuity (LCVA).
Key Findings from the ACUITY Trial
The ACUITY trial demonstrated that OCS-05 achieved its primary safety endpoint, with no significant difference in abnormal ECG parameters between treatment arms. Two patients in the OCS-05 arms and one in the placebo arm showed shifts from normal to abnormal ECG measures, but these events were mild and transient.
Significant improvements were observed in secondary efficacy endpoints:
- Retinal Structure: A 43% improvement in Ganglion Cell-Inner Plexiform Layer (GCIPL) thickness was seen in the OCS-05 (3mg/kg/day) group compared to placebo at month 3 (p=0.049), maintained through month 6 (p=0.052). Retinal Nerve Fiber Layer (RNFL) thickness improved by 28% at month 3 (p=0.045), reaching 30% at month 6 (p=0.033).
- Visual Function: The OCS-05 (3 mg/kg/day) group showed an approximate 18-letter improvement in LCVA at month 3 (p=0.004) and 15 letters at month 6 (p=0.012) compared to placebo.
Expert Commentary
Riad Sherif, MD, CEO of Oculis, stated, "These positive safety and efficacy results from ACUITY represent a significant milestone in bringing the first potential neuroprotective treatment in ophthalmology to patients. The improvement in vision is especially encouraging, and the consistent improvement in retinal structure highlights the therapeutic potential of OCS-05 across multiple ophthalmic and neurological conditions."
Mark Kupersmith, MD, Professor at Icahn School of Medicine at Mount Sinai Hospital, noted, "Steroids have been used to treat the inflammation seen in acute optic neuritis, but don’t prevent persistent visual impairments or reduce structural loss. There remains a critical unmet need for neuroprotective therapies to preserve vision and the potential neuroprotective properties of OCS-05 observed in the ACUITY trial and its impact on visual function could offer significant hope for patients."
Regulatory and Development Plans
OCS-05 has received orphan drug designation from the FDA and EMA for acute optic neuritis. The FDA has also cleared the Investigational New Drug (IND) application for OCS-05, enabling clinical development in the United States. Oculis plans to advance OCS-05's development in acute optic neuritis and explore its potential in other neuro-ophthalmic indications.
About Acute Optic Neuritis
Acute optic neuritis is characterized by inflammation and demyelination of the optic nerve, leading to visual impairment. It affects approximately 8 in 100,000 people worldwide and often represents an early sign of multiple sclerosis. Currently, there are no approved therapies specifically for acute optic neuritis, highlighting the unmet need for neuroprotective treatments.
