Subjects with recent onset of unilateral acute optic neuritis were randomised in the trial. Credit: PeopleImages.com – Yuri A/Shutterstock.

Oculis Holding has reported that the randomised Phase II Acute OptiC NeUrITis of DemYelinating Origin (ACUITY) trial of OCS-05, in treating individuals with acute optic neuritis, met the primary endpoint of ‘safety’ and demonstrated ‘statistical significance’ in key secondary endpoints related to efficacy.

The double-blind, multi-centre, placebo-controlled trial is designed to assess the tolerability, efficacy, and safety of two different dosages of the neuroprotective candidate administered intravenously alongside steroids for a period of five days.

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A total of 36 subjects with recent onset of unilateral acute optic neuritis were randomised, and 33 of them received treatment, forming the basis for the pre-specified modified intent-to-treat (mITT) analysis.

The trial’s primary safety endpoint focused on the percentage of subjects experiencing a shift from normal to abnormal in electrocardiogram (ECG) parameters after receiving the study drug up to day 15. The outcomes indicated no significant difference in abnormal ECG parameters between the treatment arms.

Secondary endpoints evaluated changes in retinal structure using optical coherence tomography (OCT) imaging. The imaging assessed the thickness of the Ganglion Cell-Inner Plexiform Layer (GCIPL) and the Retinal Nerve Fiber Layer (RNFL).

The findings revealed a 43% improvement in GCIPL thickness and a 28% to 30% improvement in RNFL thickness for the OCS-05 group compared to placebo, with significant p-values at three and six months.

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Oculis CEO Riad Sherif said: “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.”

An orphan drug designation by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) has been granted to the therapy for this condition.

The investigational new drug (IND) application for OCS-05 has also been cleared by the US regulator, facilitating the initiation of clinical development in the US.

In August 2023, the company enrolled the first subject in the LEOPARD trial of OCS-01 eye drops for cystoid macular oedema (CME) treatment.