A phase 2 clinical trial has assessed the effectiveness and safety of OCS-05, a peptidomimetic small molecule developed by Oculis, for patients with optic nerve inflammation. The ACUITY study, conducted in France, focused on individuals with unilateral acute optic neuritis (AON) associated with demyelination.
The trial included 33 participants who experienced vision loss due to AON. Over a five-day period, the participants received daily intravenous doses of either 2 mg/kg or 3 mg/kg of OCS-05, or a placebo, alongside conventional steroid therapy. According to Oculis, the study’s primary safety objective was met, as there were no significant differences in high electrocardiogram readings between the OCS-05 and placebo groups on Day 15.
“These positive safety and efficacy results from ACUITY represent a significant milestone in bringing the first potential neuroprotective treatment in ophthalmology to patients,” stated Riad Sherif, M.D., CEO of Oculis. “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.”
Secondary endpoints related to efficacy indicated structural improvements in the retina. By Month 3, patients receiving the 3 mg/kg dose exhibited a 43% increase in the thickness of the ganglion cell-inner plexiform layer and a 28% increase in the retinal nerve fiber layer compared to the placebo group. Additionally, visual acuity assessments showed an 18-letter improvement by Month 3, with a 15-letter gain maintained at Month 6.
Adverse events associated with the drug were minimal. No severe side effects directly linked to OCS-05 were reported, and the most common adverse events—headache and acne—affected two patients in the treatment groups.
Acute optic neuritis is a rare inflammatory condition that predominantly affects young adults and can result in retinal thinning and long-term vision impairment. While steroids effectively manage inflammation, they do not provide the neuroprotective benefits necessary to prevent vision loss. OCS-05 is being explored as a potential solution to address this unmet need by offering both anti-inflammatory and neuroprotective properties.
The potential applications of OCS-05 extend beyond optic neuritis, as Oculis is investigating its use in other neuro-ophthalmic conditions, including glaucoma and diabetic retinopathy. “We are excited further to advance OCS-05’s development in acute optic neuritis, while actively exploring its potential in additional neuro-ophthalmic indications to deliver a first-in-class neuroprotective treatment option to patients,” Sherif added.
With funding of up to $110 million, Oculis plans to launch a U.S. clinical trial for OCS-05. The company is also continuing to develop other treatments in its pipeline, such as OCS-01, a high-concentration dexamethasone eye drop currently in phase 3 trials for diabetic macular edema.
