Key Takeaways

  • OCS-01 eye drops for the treatment of DME failed to meet the primary endpoint of improving best corrected visual acuity (BCVA) at Week 52 in both phase 3 DIAMOND-1 and DIAMOND-2 trials
  • OCS-01 demonstrated substantial and sustained reductions in retinal thickness versus vehicle across both studies
  • Based on the phase 3 results, Oculis does not plan to pursue an FDA regulatory filing for OCS-01 in DME 

Oculis' OCS-01 eye drops for the treatment of diabetic macular edema (DME) failed to meet the primary endpoint in two phase 3 trials.

The company announced topline results on Friday from its phase 3 DIAMOND-1 and DIAMOND-2 clinical trials evaluating OCS-01. The trials measured the mean change from baseline in best corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) letter score at Week 52. Both DIAMOND-1 and DIAMOND-2 did not demonstrate a statistically significant improvement in BCVA compared with vehicle control.

In addition, the key secondary endpoint assessing the proportion of patients achieving a gain of 15 or more ETDRS letters in BCVA was not met in either trial.

Despite the lack of visual acuity improvement, OCS-01 demonstrated a substantial and sustained reduction in retinal thickness, a key anatomical measure of disease activity. Optical coherence tomography (OCT) assessments showed consistent retinal thickness reductions compared with vehicle at all visits in DIAMOND-2 and at all visits except Week 52 in DIAMOND-1.

The DIAMOND (DIAbetic Macular edema patients ON a Drop) program consisted of two phase 3, double-masked, randomized, multicenter studies designed to assess the efficacy and safety of OCS-01 over a 52-week treatment period. More than 800 patients were enrolled across both pivotal trials at 119 investigational sites throughout the United States and several international locations.

The company reported that OCS-01 was generally well tolerated, with no unexpected adverse events observed during the studies. The safety profile remained consistent with findings from previous clinical trials.

As a result of the phase 3 outcomes, Oculis stated that it does not currently intend to pursue a FDA regulatory filing for OCS-01 as a treatment for DME.

“We are naturally disappointed that the substantial and sustained reduction in retinal thickness observed across both trials didn’t translate into BCVA improvement at Week 52,” said Riad Sherif, MD, Chief Executive Officer of Oculis. “In these two trials, our team partnered with 119 global sites across multiple countries and demonstrated excellent execution. We thank the patients, investigators, and all clinical experts who participated in the DIAMOND program.”

Dr. Sherif added that the company remains well positioned financially and plans to redirect resources toward other late-stage development programs.

“Our strong financial position allows us to execute on our robust late-stage development portfolio,” he said. “While we finalize the review of DIAMOND program data, we will strategically focus resources on advancing our late-stage portfolio, including the Privosegtor platform, starting with the PIONEER program for Privosegtor in optic neuropathies, and the PREDICT-1 trial for Licaminlimab to drive precision medicine in dry eye disease.”