Oculis' OCS-05: A Promising Neuroprotective Therapy for Acute Optic Neuritis

Oculis Holding AG (Nasdaq: OCS / ICX: OCS.IC), a global biopharmaceutical company, has announced positive topline results from its Phase 2 ACUITY trial for OCS-05, a neuroprotective therapy candidate for acute optic neuritis. The trial met its primary safety endpoint and achieved statistical significance on key secondary efficacy endpoints, opening development pathways for OCS-05 as a potential first-in-class neuroprotective therapy.

The Phase 2 ACUITY trial was a randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the safety, tolerability, and efficacy of OCS-05 in patients with acute optic neuritis. The trial randomized 36 patients with recent onset of unilateral acute optic neuritis with a demyelinating origin, of which 33 patients received treatment and were included in the pre-specified modified intent-to-treat (mITT) analysis.

Positive results from the ACUITY trial showed that OCS-05 achieved its primary safety endpoint, with no difference in the percentage of patients with abnormal ECG parameters between the OCS-05 arms and the placebo arm. Only two patients in the OCS-05 arms (2 and 3 mg/kg/day) and one patient in the placebo arm had a shift from normal to abnormal in any ECG measures between baseline and Visit 4 (Day 15), both equivalent to 12.5%. Events observed in the OCS-05 arms were mild and transient and qualified as not clinically significant by central review reading center.

Secondary efficacy endpoints assessed changes in retinal structure and visual function. Optical Coherence Tomography (OCT) imaging was used to objectively measure the thickness of two different retinal segments in the affected eye to evaluate the potential neuroprotective effects of OCS-05 compared to placebo: 1) Ganglion Cell-Inner Plexiform Layer (GCIPL) and 2) Retinal Nerve Fiber Layer (RNFL). Results showed:

* A 43% improvement in GCIPL thickness mean change from baseline in favor of OCS-05 (3mg/kg/day) + steroid compared to placebo + steroid at month 3, which was maintained through month 6 with p-values* of 0.049 and 0.052 at 3 and 6 months, respectively.
* A 28% improvement in RNFL thickness mean change from baseline in favor of OCS-05 (3mg/kg/day) + steroid compared to placebo + steroid at month 3, reaching 30% improvement at month 6 with p-values* of 0.045 and 0.033 at 3 and 6 months, respectively.

Secondary efficacy endpoint assessed changes in visual function, with changes in 2.5% ETDRS low contrast letter acuity (LCVA) measured to assess visual function improvement. Results showed:

* A favorable difference in LCVA mean change from baseline of approximately 18 letters at month 3 and approximately 15 letters at month 6 with OCS-05 (3 mg/kg/day) + steroid compared to placebo + steroid, with p-values^ of 0.004 and 0.012 at 3 and 6 months, respectively.

OCS-05 has received orphan drug designation from both the FDA and EMA, and its IND application has been cleared by the FDA. The Company reports approximately $105-110 million in cash and equivalents entering 2025.

The positive results from the ACUITY trial show that OCS-05 has the potential to be a first-in-class neuroprotective therapy for acute optic neuritis, a rare disease with a significant unmet medical need. The drug's clean safety profile, robust efficacy data, and potential to address unmet medical needs position it well for long-term market adoption and commercial success. As Oculis continues to advance its development pipeline, investors should keep a close eye on the progress of OCS-05 and other promising therapies in the Company's portfolio.