Oculis’ Phase II trial showcases a novel neuroprotective approach to acute optic neuritis.
The future of acute optic neuritis (AON) could be moving away from focusing on inflammation.
Oculis (Zug, Switzerland) announced positive results for the Phase II ACUITY trial evaluating OCS-05, an investigational neuroprotective therapy for the disease.
This novel treatment showed significant improvements in both retinal structure and visual function, offering hope for patients with AON, a condition currently lacking targeted neuroprotective therapies. According to trial results, OCS-05, an advanced peptidomimetic small molecule, demonstrated a 43% improvement in ganglion cell-inner plexiform layer (GCIPL) thickness compared to placebo at 3 months, with the benefits sustained at 6 months.
The results position OCS-05 as a potential first-in-class neuroprotective treatment for acute optic neuritis and other neuro-ophthalmic conditions.
“These positive safety and efficacy results from ACUITY represent a significant milestone in bringing the first potential neuroprotective treatment in ophthalmology to patients,” said Dr. Riad Sherif, chief executive officer 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.”
Key trial results and highlights
The ACUITY trial, a double-blind, placebo-controlled study, enrolled 36 patients with acute optic neuritis of demyelinating origin. Patients received OCS-05 intravenously alongside standard steroid therapy.
OCS-05 showed a 28% improvement in retinal nerve fiber layer (RNFL) thickness by 6 months and significant visual function gains. Patients treated with OCS-05 (3 mg/kg/day) combined with steroids showed a favorable improvement in LCVA mean change from baseline, with patients gaining approximately 18 letters at 3 months and 15 letters at 6 months compared to placebo plus steroids. The results were statistically significant, with p-values of 0.004 at 3 months and 0.012 at 6 months.
The trial met its primary endpoint with no clinically significant differences in cardiac safety (electrocardiogram parameters) between OCS-05 and placebo groups. Adverse events were mild, transient, and non-clinically significant.
A shift in managing acute optic neuritis
AON is characterized by inflammation and demyelination of the optic nerve, often leading to vision loss.
Steroids are the current standard treatment, reducing inflammation in the optic nerve—but they fail to prevent long-term structural damage and visual impairment. OCS-05 is designed to go beyond inflammation, directly protecting the optic nerve and retinal structure.
Dr. Mark Kupersmith, an expert at the Icahn School of Medicine at Mount Sinai Hospital in New York, added: “These groundbreaking results represent an important advancement for acute optic neuritis patients. Steroids have been used to treat the inflammation seen in acute optic neuritis, but don’t prevent persistent visual impairments or reduce structural loss.`
“There remains a critical unmet need for neuroprotective therapies to preserve vision and the potential neuroprotective properties of OCS-05 observed in the ACUITY trial and its impact on visual function could offer significant hope for patients. These results, if replicated in larger clinical trials, could have profound implications, not only for this condition, but potentially for MS and other optic nerve disorders as well as glaucoma.”
Future development and expansion
The U.S. Food and Drug Administration (FDA) has cleared OCS-05’s Investigational New Drug (IND) application, which paves the way for clinical development in the U.S. Oculis also holds orphan drug designations for OCS-05 in both the U.S. and Europe, underscoring its potential to address this rare condition.
The company is planning additional studies to explore OCS-05’s applications in other neurodegenerative conditions, including multiple sclerosis, glaucoma, and optic nerve disorders.
Prof. Pablo Villoslada, chair of the Department of Neurology at Hospital del Mar, Pompeu Fabra University in Barcelona, Spain, and Oculis scientific advisory board member, said: “I am excited to see that the ACUITY results are consistent with the robust effects observed in animal models of neuroinflammation and neurodegeneration in the prevention of retinal ganglion cell damage and that OCS-05 has shown promising results in the improvement of vision. I look forward to the additional studies that can now be initiated for acute optic neuritis while further exploring the full potential of this promising neuroprotective candidate in both ophthalmology and neurology indications.”
