OCS-01 improves macular thickness, visual acuity in patients with DME, according to Phase 2 data

Oculis S.A. announced that the Phase 2 DX-211, a randomized, double-blind, multicenter and vehicle controlled clinical trial assessing the safety and efficacy of topical OCS-01 in patients with diabetic macular edema (DME) (NCT05343156) has been published by the Acta Ophthalmologica journal.

In addition, a new subgroup analysis of this study was also presented Friday by Prof. Ramin Tadayoni at the 22nd EURETINA Congress in Hamburg, Germany.

According to the company, the dataset published shows OCS-01 to be superior to vehicle in the primary efficacy analysis of BCVA change from baseline with a between group difference of +1.58 ETDRS letters at the pre-specified significance level of 0.15.

Riad Sherif, MD, CEO of Oculis noted that the company was pleased by these data from the Phase 2DX-211 study, which further reinforce the potential of OCS-01 to provide patients with a potentially transformative treatment of DME as the first non-invasive, eye drop, option.

“An effective topical treatment for DME could expand the proportion of patients treated, especially those with recent onset DME, as well as provide a treatment option to general ophthalmologists prior to referral to a retina specialist and thereby, improving access to treatment,” he said in a statement. “We look forward to the results of the ongoing Phase 3 DIAMOND trial as Oculis seeks to deliver life-changing treatments that can be accessed globally for the world’s most prevalent ocular diseases.”

According to the company’s news release, mean CMT showed a statistically significant greater decrease from baseline in the OCS-01 group compared to vehicle at all post-baseline time points up to and including Week 12 (-54µm OCS-01 vs. -17µm vehicle) and reversed towards baseline when treatment stopped at 12 weeks, demonstrating the pharmacological effect of OCS-01 on retinal edema. OCS-01 was well tolerated and no significant or unanticipated ocular adverse events were observed.

Subgroup analysis in patients with BCVA baseline ≤ 65 letters, presented at EURETINA 2022, showed greater improvements in both CMT (-77.4µm OCS-01 vs. -23.1µm vehicle) and BCVA (+2.9 ETDRS letters in OCS-01 vs vehicle) at week 12.

Approximately 37 million people are affected by DME worldwide, representing around 7% of the large and growing diabetes patient population. DME is the build-up of fluid (edema) in the macula (the macula is important for the sharp, straight-ahead vision that is used for reading, recognizing faces, and driving). DME is the most common cause of vision loss among people with diabetic retinopathy and a leading cause of new cases of blindness in US adults. Although treatment options currently exist, all of them are invasive (injections or implants) and represent a significant burden for patients and caregivers.

OCS-01 is a novel, high concentration, preservative free, topical formulation of dexamethasone that has the potential to be the first topical eye drop and non-invasive treatment for DME. OCS-01 opens up the possibility of treating DME patients at all stages of disease with an eye drop. In addition, OCS-01 could increase the accessibility to treatment of those patients without easy access to specialists; together, this could significantly reduce the burden to the health care system and improve patient outcomes and quality of life.

OCS-01 is currently under investigation for the treatment of DME in the ongoing Phase 3 DIAMOND trial to confirm these findings in a larger patient population.

Prof. Ramin Tadayoni, MD, PhD, professor of ophthalmology at Université de Paris, pointed out in the news release the development of effective topical eye drops for posterior segment disorders such as DME would offer easier, non-invasive options than those currently available, and could be widely adopted across all socioeconomic and geographic boundaries.

“This would allow earlier treatment in DME, reduce the burden on both patients and their caregivers with potentially less injections per year if combined with current standard of care, increase adherence and ultimately, improve outcomes worldwide,” Tadayoni said in the news release. “OCS-01 is the most developed and promising topical therapy for DME to date, and I look forward to its further development as a potentially transformative treatment for patients in need.”