Oculis (OCS) Study Result summary

Study design and objectives

• Phase II ACUITY/EQUATE trial was a randomized, double-blind, placebo-controlled, multicenter study evaluating OCS-05 in acute optic neuritis, with safety as the primary endpoint and neuroprotective efficacy as secondary endpoints.

• 36 patients were randomized; 33 received OCS-05 and were included in the modified intent-to-treat analysis.

• Patients received daily IV infusions of OCS-05 (2mg/kg or 3mg/kg) plus steroid or placebo plus steroid for five days, followed by six months of monitoring.

• Key efficacy endpoints included preservation of retinal ganglion cells (GCIPL), axon health (RNFL), and visual function (LCVA).

• OCS-05 has orphan drug designation in the US and Europe for acute optic neuritis.

Key efficacy results

• OCS-05 arm showed 43% greater preservation of GCIPL thickness at three and six months compared to steroid alone (p=0.049 and 0.052).

• RNFL thickness was preserved by 28% at month 3 and 30% at month 6 with OCS-05 (p=0.045 and 0.033).

• Functional improvement: 18-letter gain in low contrast visual acuity (LCVA) at three months, sustained at six months (p=0.004 and 0.012).

• Efficacy endpoints were achieved based on a prespecified statistical analysis plan.

• Lower relapse rate of MS or CNS inflammatory disorders in OCS-05 arm (10–10.5%) versus steroid alone (35–35.7%).

Safety and tolerability

• No difference in cardiac safety or drug-related serious adverse events between OCS-05 and placebo arms; all ECG events were mild and transient.

• No adverse events led to withdrawal or discontinuation.

• Most common treatment-related adverse events included mild skin, gastrointestinal, nervous system disorders, headache, and acne.

• OCS-05 showed a favorable safety and tolerability profile compared to placebo.