Oculis Announces Positive Phase 2 Results for OCS-05 in Acute Optic Neuritis

Oculis has reported positive topline results from its Phase 2 ACUITY trial evaluating OCS-05, a novel neuroprotective therapy, in patients with acute optic neuritis (AON). These findings represent a significant milestone in the development of therapies for neuro-ophthalmic diseases.

About OCS-05 and the ACUITY Trial

OCS-05 is a peptidomimetic small molecule candidate with the potential to be the first neuroprotective treatment for acute optic neuritis and other neuro-ophthalmic conditions.

Key Features of the ACUITY Trial

       • Study Design: A randomized, double-blind, placebo-controlled, multi-center trial.

       • Administration: Intravenous infusion of OCS-05 once daily (2 mg/kg/day or 3 mg/kg/day) for 5 days, in addition to steroid therapy.

       • Participants:

               - 36 eligible patients aged 18–60 with recent onset unilateral acute optic neuritis of demyelinating origin.

               - 33 patients received OCS-05 (either 2 mg/kg/day or 3 mg/kg/day) or placebo along with steroids.

Primary Safety Endpoint

The primary safety endpoint was assessed by measuring the percentage of patients experiencing shifts from normal baseline to abnormal electrocardiogram (ECG) parameters at any time between Day 1 and Day 15. Results showed:

       • No significant differences in ECG abnormalities between the OCS-05 treatment arms and placebo.

Key Efficacy Results

Neuroprotective Structural Benefits and Visual Function Improvement

OCS-05 demonstrated:

       • 43% improvement in ganglion cell and inner plexiform layer (GCIPL) thickness at Month 3 (compared to placebo), maintained through Month 6.

       • 28% improvement in retinal nerve fiber layer (RNFL) thickness at Month 3, increasing to 30% by Month 6, favoring OCS-05 (3 mg/kg/day) with steroid compared to placebo with steroid.

       • Improved low contrast visual acuity (LCVA) scores using the 2.5% ETDRS scale.

CEO Statement

Riad Sherif, MD, CEO of Oculis, highlighted the significance of the results:

"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."

Regulatory Status

OCS-05 has achieved notable regulatory milestones:

       • Orphan Drug Designation: Granted by the U.S. FDA and European Medicines Agency (EMA) for acute optic neuritis.

       • Investigational New Drug (IND) Clearance: Approved by the FDA.

Conclusion

The positive results from the ACUITY trial highlight OCS-05's potential as a groundbreaking treatment for acute optic neuritis, offering both structural and functional improvements. With ongoing development and regulatory support, OCS-05 could transform the treatment landscape for neuro-ophthalmic and neurological conditions.