• Monday, October 5, 2020 @ 12:00 am

  • The study met its pre-specified efficacy endpoints and showed that OCS-01 eye drops were more effective than vehicle in reducing central macular thickness and improving visual acuity in patients with DME
  • No significant unanticipated ocular adverse events were observed
  • Phase 3 trial for OCS-01 in DME is in preparation following positive End-of-Phase 2 meeting with FDA
  • Results were presented by renowned retina specialist Prof. Ramin Tadayoni, M.D., Ph.D.

Oculis S.A. announced that the positive results from the successful Phase 2 (DX-211) clinical trial investigating OCS-01 for the treatment of Diabetic Macular Edema (DME) were presented at EURETINA 2020 Virtual during the Late Breaking & Reviews session on Saturday 3 October.

OCS-01 is a novel, high concentration, topical formulation of dexamethasone (ophthalmic suspension 1.5%) developed using Oculis’ proprietary Soluble NanoParticle technology (SNP). The SNP technology was developed to overcome the main barriers for the topical delivery of drugs to the eye: it enhances bioavailability of the active ingredient in the posterior segment of the eye and offers the potential to treat retinal diseases while minimizing the need for patients to undergo invasive ocular procedures.

The Phase 2 trial met its pre-specified efficacy endpoints and showed that OCS-01 eye drops were more effective than vehicle in reducing central macular thickness and improving visual acuity in patients with DME. No significant or unanticipated ocular adverse events were observed.

The DX-211 study was a prospective, multi-center, randomized, double-masked, parallel group, vehicle-controlled Phase 2 study. Type 1 or 2 diabetic patients with DME, central macular thickness (CMT) of ≥ 310 µm by SD-OCT and ETDRS best corrected visual acuity (BCVA) letter score ≤ 73 and ≥ 24 in the study eye were randomized to treatment with OCS-01 or matching vehicle eye drops, 3 times per day for 12 weeks. Efficacy was evaluated based on the change from baseline to week 12 of CMT and BCVA letter score. Safety was assessed in terms of adverse events and ophthalmology examination.

A total of 144 patients were randomized and 133 patients (92.3%) completed the study. Key results were:

  • Mean CMT showed a greater decrease from baseline in the OCS-01 group than the vehicle arm at week 12 ( -53.6 μm vs -16.8 μm, p=0.0115).
  • Mean change in ETDRS BCVA letter score from baseline to week 12 was higher in the OCS-01 group than the vehicle group (+2.62 letters vs +1.04 letters, p= 0.125).
  • P values met the pre-specified criteria in the protocol for statistical superiority of OCS-01 vs vehicle.
  • Ocular tolerability was not significantly different between the OCS-01 and vehicle groups with the exception of change in intraocular pressure (IOP).
  • IOP increases were more common with OCS-01 than vehicle during the treatment period consistent with known effects of topical steroids, including dexamethasone.

Since first reporting these data in February 2020 at Angiogenesis, Exudation, and Degeneration 2020 Conference, Oculis has announced positive Phase 2 results for OCS-01 in the treatment of inflammation and pain following ocular surgery. The Company has also successfully completed an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for OCS-01 and is currently planning Phase 3 trials for both indications, which are expected to begin in the first half of 2021.

The positive results were presented in a Late Breaking & Reviews session entitled “Oculis OCS-01 Phase 2 Study Results: A Topical Therapeutic for DME” by Ramin Tadayoni, M.D., Ph.D., renowned professor and ophthalmologist in the diagnosis and treatment of retinal diseases, retinovitreal surgery and ophthalmic imaging. Prof. Tadayoni is also a member of Oculis’ Scientific Advisory Board.

Ramin Tadayoni, M.D., Ph.D., Professor of Ophthalmology at University of Paris and Head of the department of Ophthalmology at Lariboisière University Hospital (Paris, France), commented: “The results of this clinical study clearly show that OCS-01 treatment has a clinical benefit in DME patients and is well tolerated. The fact that a topical treatment can improve the vision of DME patients as well as reduce inflammation of the retina is remarkable. If these results are confirmed in the planned Phase 3 trial, then OCS-01 could be transformational for DME patients, potentially making treatment more accessible and improving longer-term disease management, particularly for those at the early stages where a watch-and-wait strategy is currently employed. I look forward to the Phase 3 trial and hope that it can be swiftly and successfully completed.”

Riad Sherif, M.D., Chief Executive Officer of Oculis, said: “The positive clinical results with OCS-01 for treating DME and for treating inflammation and pain following ocular surgery, as well as the subsequent progress we have made during 2020, have been extremely gratifying. We believe these data clearly highlight the potential of this topical drug to be a truly innovative, non-invasive approach that would be welcomed by physicians and patients alike. We are fully committed to our mission to improve the sight and lives of patients through the development of transformative topical ophthalmic treatments using our innovative formulation technologies, and believe we have an exciting future ahead.”

Additional abbreviations:
SD-OCT - Spectral Domain Optical Coherence Tomography
ETDRS - Early Treatment Diabetic Retinopathy Study