SGK2

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.

Topline results anticipated in Q4 2024 for Phase 2 trial evaluating the safety and tolerability of OCS-05 in patients with Acute Optic Neuritis (AON) Oculis also aims to complete an IND submission for OCS-05 in the U.S. in 2024 OCS-05 has been granted orphan drug designation in the United States and Europe for AON, an indication for which there are no approved therapies May 08, 2024 -- Oculis Holding AG (Nasdaq: OCS; XICE: OCS) (“Oculis” or the “Company”), a global biopharmaceutical company purposefully driven to save sight and improve eye care, today announced the completion of enrollment in its multi-center, randomized, double-blind, placebo-controlled Phase 2 ACUITY trial evaluating the safety and tolerability of OCS-05 in patients with Acute Optic Neuritis (AON). Topline results are expected in Q4 2024. The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON. The study is ongoing across four (4) sites in France. At present, there are no approved therapies for AON, leaving a significant gap in medical care for treatments that offer neuroprotection and can prevent vision loss. OCS-05 has been granted orphan drug designation in both the United States and Europe, highlighting the importance of this unmet medical need. Riad Sherif, M.D., Chief Executive Officer of Oculis, commented: “We continue to execute on our strategic programs and are pleased to complete enrollment in the ACUITY trial, an important milestone for OCS-05 clinical program that keeps us on track for an anticipated topline readout in Q4 this year. In the meantime, we keep on working towards an IND submission for OCS-05 in the U.S. in 2024. We anticipate that a positive readout in this trial would support further development of OCS-05 as a potential first-in-class neuroprotective treatment with wide applicability in neuro-ophthalmic diseases such as AON, glaucoma, geographic atrophy, diabetic retinopathy and other diseases where protecting retinal neurons is key to preserving patients’ sight.” Sophie Bonnin M.D., Ph.D., Deputy Head of the Ophthalmology Department, at Rothschild Foundation Hospital, in Paris, and Scientific Advisor for the ACUITY trial, said: “AON is a rare disease characterized by acute inflammation and demyelination of the optic nerve. While corticosteroids are used to shorten the attack, there is no approved therapy for AON and unmet needs remain for therapies that can prevent vision loss after an acute episode of optic neuritis. The completed enrollment brings us a step forward in the development of OCS-05 as a potential neuroprotective candidate which could have a profound impact in ophthalmology, and we are eagerly awaiting the anticipated trial readout in Q4 of this year.” Pablo Villoslada M.D., Chair, Department of Neurology, Hospital del Mar, Barcelona, and Oculis Scientific Advisory Board member, said: “The completion of enrollment in the ACUITY trial in patients with an acute inflammatory-demyelinating disorder is very exciting. The novel and differentiated mechanism of action of OCS-05 triggers multiple beneficial effects on apoptosis, oxidation and inflammation which supports neuronal survival and repair. The results from this Phase 2 trial will provide an opportunity to further understand this mechanism of action in a clinical setting and explore its potential benefits for patients suffering from ophthalmic diseases where neuroprotection is needed.” OCS-05 is a serum-glucose corticoid kinase-2 (SGK-2) activator with the potential to become a neuroprotective therapy for acute optic neuritis and other neuro-ophthalmic diseases. In ophthalmology, this mechanism of action could potentially protect the nerve axons in conditions such as acute optic neuritis, to ultimately prevent vision loss. In animal models of neuroinflammation and neurodegeneration, OCS-05 has shown positive results in prevention of retinal ganglion cell damage and was associated with improvements in clinical function (disability). The Phase 2 ACUITY study in Acute Optic Neuritis (AON) is currently ongoing with results anticipated before the end of the year. OCS-05 is an investigational drug and has not received regulatory approval for commercial use in any country. The Phase 2 ACUITY is an ongoing randomized, double-blind, multi-center, two-arm, placebo-controlled study to evaluate the safety and tolerability of once daily OCS-05 intravenous infusion in patients with Acute Optic Neuritis (AON). Positive outcomes in this trial could support the development of the compound for potential application in the treatment of ophthalmic conditions where neuroprotection is needed. AON is a rare disease characterized by an acute inflammation of the optic nerve that can lead to permanent visual impairment. It affects up to 5 in 100,000 people worldwide each year and often represents the first sign of multiple sclerosis. It mainly occurs in adults between the age of 20 and 40 years and is more frequent in women (2:1). The acute inflammatory process of AON leads to the loss of myelin covering the optic nerve and the axons. At the onset, patients often suffer from ocular pain that increases with eye movement and vision loss. Once the inflammation recedes, remyelination often occurs but it is incomplete. Without the myelin sheath protecting the axon, neurons located in demyelinated segments become fragile and prone to death. Unfortunately, damaged axons cannot regrow, leading to permanent visual impairment. Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) purposefully driven to save sight and improve eye care. Oculis’ highly differentiated pipeline comprises multiple innovative product candidates in development. It includes OCS-01, a topical eye drop candidate for diabetic macular edema (DME) and for the treatment of inflammation and pain following cataract surgery; OCS-02, a topical biologic anti-TNFα eye drop candidate for dry eye disease (DED) and for non-infectious anterior uveitis; and OCS-05, a neuroprotective candidate for acute optic neuritis (AON). Headquartered in Switzerland and with operations in the U.S. and Iceland, Oculis’ goal is to improve the health and quality of life of patients worldwide. The company is led by an experienced management team with a successful track record and is supported by leading international healthcare investors. The content above comes from the network. if any infringement, please contact us to modify.