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A visual acuity test showed an 18-letter improvement by Month 3 with a 15-letter improvement retained by Month 6 on Oculis’ peptidomimetic small molecule compared to placebo.\n Oculis’ peptidomimetic small molecule has been shown to help protect the eyes of patients with inflammation of the optic nerve in a phase 2 study.The ACUITY trial in France analyzed 33 patients who had started to lose their vision as a result of unilateral acute optic neuritis with a demyelinating origin. These individuals received either a 2-mg/kg/day or a 3-mg/kg/day dose of the drug, dubbed OCS-05, or placebo intravenously once-daily for five days alongside steroid treatment.The trial hit its primary, heart-safety-based endpoint by demonstrating that patients receiving OCS-05 were no more likely to have a high electrocardiogram measurement than the placebo cohort at Day 15, Oculis said in a Jan. 6 release.There were no drug-related serious adverse events, with the most common adverse events in the OCS-05 cohorts being headache and acne, which affected two patients each.When it came to the secondary, efficacy-based endpoints, imaging showed that when compared to placebo, the 3-mg/kg/day dose was linked to a 43% improvement in the thickness of one part of the retina, called the ganglion cell-inner plexiform layer, and a 28% improvement in thickness of another, known as the retinal nerve fiber layer, at Month 3.A visual acuity test showed an 18-letter improvement by Month 3 with a 15-letter improvement retained by Month 6 compared to placebo, Oculis added. There are no therapies approved specifically for acute optic neuritis, a rare condition of acute inflammation of the optic nerve that tends to affect young adults and can involve retinal thinning leading to permanent reduction in vision loss. While steroids are used to treat inflammation and aid recovery, they can’t offer the neuroprotection needed to prevent loss of vision, Oculis pointed out in the release.The neuroprotective potential of OCS-05 means the Swiss biotech is already considering turning the therapy toward other neuro-ophthalmic conditions such as glaucoma and diabetic retinopathy.“These positive safety and efficacy results from ACUITY represent a significant milestone in bringing the first potential neuroprotective treatment in ophthalmology to patients,” Oculis CEO Riad Sherif, M.D., said. “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.”“We are excited to further advance OCS-05’s development in acute optic neuritis, while actively exploring its potential in additional neuro-ophthalmic indications with the aim to deliver a first-in-class neuroprotective treatment option to patients,” Sherif added.With up to $110 million to play with, Oculis said it is off to a “strong start in 2025” and has its sights set on launching a U.S. trial of OCS-05. The company’s pipeline also includes the likes of OCS-01, a high-concentration dexamethasone eye drop that is currently undergoing a phase 3 trial in diabetic macular edema.

Oculis kicked off the year on a positive note with a mid-stage success in a rare ophthalmic condition, setting the stage for clinical development in the US. The Swiss drugmaker’s serum glucocorticoid kinase-2 activator OCS-05 met the primary endpoint in the Phase 2 ACUITY trial in 36 patients in France with acute optic neuritis who were taking steroids, according to a Monday release . There was no difference between the two treatment arms in the percentage of subjects with abnormal electrocardiogram parameters, the company said. Oculis also said the 3 mg/kg dose of the drug was effective, producing a statistically significant 18-letter improvement in low contrast letter acuity compared with placebo at three months. Treated patients also saw a 43% improvement in ganglion cell-inner plexiform layer thickness versus placebo and a 28% improvement in retinal nerve fiber layer thickness. Mark Kupersmith, vice chair of translational research at Icahn School of Medicine, said in the release that if the Phase 2 data are replicated in larger studies, they could have “profound implications, not only for this condition, but potentially for [multiple sclerosis] and other optic nerve disorders as well as glaucoma.” In November, Stifel analysts wrote that each of Oculis’ programs have blockbuster potential. The FDA has cleared Oculis’ IND application for OCS-05, allowing the start of clinical development in the US. The biotech currently has around $105 million to $110 million in cash, equivalents and short-term investments to help fund this work. While the company plans to stick with an IV formulation of OCS-05 for further development in acute optic neuritis, it is also working on a topical version of the drug for neurotrophic keratitis which is expected to enter IND-enabling studies this year. “We are thinking about other, I would say, more friendly formulations for chronic diseases such as [intravitreal] and subcutaneous,” CEO Riad Sherif told analysts and investors on a Monday call. The Phase 2 success is a welcome change for the Zug-based drugmaker, which faced a series of setbacks in 2024. In June, Oculis’ candidate for dry eye disease missed statistical significance in a mid-stage test. Two months later, the company was forced to pull the plug on a second Phase 3 trial for its lead eye drop program for inflammation and pain following ocular surgery due to an undisclosed “third-party administrative error.” But Oculis said at the time that it plans to go ahead with an FDA filing based on data from a previous late-stage trial and a Phase 2 study. Editor’s note: This article was updated to add comments from a company call.