Oculis Announces Positive Topline Results from Phase 1 of DIAMOND Phase 3 Trial of OCS-01 Eye Drops for Diabetic Macular Edema

May 22, 2023, 10:30 AM Eastern Time

Zug, Switzerland and BostonMay 23, 2023PR Newswire -- Oculis (Nasdaq: OCS), an international biopharmaceutical company dedicated to improving vision and protecting eyes, today announced positive topline results from the first stage of the Phase III clinical trial DIAMOND for OCS-01 eye drops in treating diabetic macular edema (DME). DME is a leading cause of vision loss and blindness in diabetic patients, affecting approximately 37 million people worldwide, with a significant number of patients left untreated due to the lack of convenient treatment options.

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DIAMOND is a Phase III clinical trial that is double-blind, randomized, multicenter, and conducted in two phases to evaluate the efficacy and safety of OCS-01 eye drops in patients with DME. The primary objective of the first phase is to select the optimal dosing regimen. The first phase was conducted across 39 clinical centers in the United States and Europe, where 148 patients were randomly assigned in a 2:1 ratio to receive either OCS-01 (n=100) or placebo (n=48). This included a 6-week loading period with six administrations per day, followed by a 6-week maintenance period with three administrations per day.

Phase 1 met the primary efficacy endpoint, with a statistically significant improvement in mean BCVA score from baseline to Week 6 in the treatment group compared to the placebo group, as measured by the "Early Treatment Diabetic Retinopathy Study" chart (BCVA ETDRS) (OCS-01: 7.2 letters, placebo: 3.1 letters, p=0.007), indicating a significant increase in visual acuity in the treatment group. The efficacy sustained until Week 12 was also statistically significant (OCS-01: 7.6 letters, placebo: 3.7 letters, p=0.016). Additionally, at Week 6, a higher proportion of patients in the OCS-01 group achieved a BCVA improvement of ≥15 letters (OCS-01: 25.3%, placebo: 9.8%, p=0.015), which persisted until Week 12 (OCS-01: 27.4%, placebo: 7.5%, p=0.009).

In this 3-month trial, OCS-01 met both clinical efficacy endpoints (improvement in BCVA score and the proportion of patients gaining 3 lines of vision). Drug approval will require these metrics to be achieved over a 12-month treatment period.

In the OCS-01 treatment group, there was a statistically significant reduction in central subfield thickness (CST) at week 6 (OCS-01: -63.6µm, placebo: +5.5µm, p<0.0001). The reduction in retinal thickness persisted until week 12 (-61.6µm vs. -16µm, p=0.004).

OCS-01 was well-tolerated, with no unexpected adverse events observed.

The OCS-01 development program will proceed to Phase 2 as planned, including two global trials, each enrolling approximately 350-450 patients. Oculis expects to initiate Phase 2 of the DIAMOND trial in the second half of this year.

Riad Sherif, M.D., Chief Executive Officer of Oculis, said:"I am very pleased, extremelyAffectedEncouragingly, in the first phase of this trial, OCS-01...SignificantThe method achieved both primary and secondary endpoints.InBefore OCS-01, there has never been an eye drop treatment for DME.ThePositiveResults. OCS-01 has now been validated in two different studies, demonstrating consistent and reproducible results.PositiveResults. We stillWillFocus on advancing with high priorityPhase III Clinical TrialDIAMONDTrialThePhase 2. This important milestone has the potential to bring us closer toFirst ApplicationEye DropsLiquidTreatmentDMEThisSevere"One step closer to blinding diseases."

Arshad M. Khanani, MD, Director of Clinical Research at Sierra Eye Associates in Reno, Nevada, and Principal Investigator of the DIAMOND Clinical Trial, commented:"AsPhase III Clinical TrialDIAMONDTestThe mainResearchPatient, seeing the positive results of the Phase 1 trial is encouragingVeryExcitement. DME patients starting with OCS-01Eye DropsAfter 6 weeks of treatment, BCVA improved by 7.2 letters and CST decreased by 63.6µm, which is clinically significant for both the treating physician and the patient. OCS-01 has demonstrated thesePositiveThe non-invasive treatment, if approved, has the potential to benefit a large number of DME patients. I look forward to including patients in the second phase of this trial."

David S. Boyer, MD, Clinical Professor of Ophthalmology at the Keck School of Medicine of the University of Southern California, and Principal Investigator of the DIAMOND Clinical Trial, said:"DMEAndIncreased Permeability and InflammationAll related. Currently, anti-VEGFAs an effective anti-permeability drug, but with no effect on inflammation. Therefore, a considerable proportion of patients use anti-VEGFThe therapeutic effect is poor. If approved, OCS-01 has the potential to complement current treatments.Used forTreating refractory patients. Moreover, as an external preparation drug, it also has the potential to becomeDMEFirst-line treatment drugs. In short, I believe that OCS-01 may have an impact in DME.Make it becomeA Real Game-ChangerPatient。"