Oculis aces the Phase 2 clinical trial for OCS-01 treating DME

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Oculis, the Icelandic-Swiss clinical stage biopharmaceutical company recently presented some interesting results at the Angiogenesis, Exuadation and Degeneration 2020 Conference organized by the Bascom Palmer Eye Institute in Miami.

Their groundbreaking nanoparticle formulation, OCS-01, has successfully completed the Phase 2 (DX-211) clinical trial for treating diabetic macular edema (DME). According to their website [1]:

  • The Phase 2 (DX-211) study provides a clinical proof-of-concept of OCS-01 topical drug effect on DME
  • The study met its pre-defined 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 / unexpected ocular adverse events were observed
  • The results also provide a proof-of-concept of the nanoparticle technology as a potential innovative means to develop topical therapies for retinal diseases

OCS-01 is the brainchild of renowned Icelandic scientists, Thorsteinn Loftsson and Einar Stefánsson, co-founders of Oculis. They have long been studying drug/cyclodextrin complexes for ophthalmic drug delivery. Their invention, which Oculis now calls solubilizing nanoparticle (SNP) technology is based on the aggregation of drug/cyclodextrin complexes, which forms stable nanoparticles of the complex aggregates that can fall apart upon dilution. Figure 1 shows the steps of the formation of the nanoparticles. According to the available information, OCS-01 contains 1.5% dexamethasone. Oculis did not publish the type of cyclodextrin used, although, looking over Prof. Loftsson’s publications, it is not that difficult to figure out the identity of the cyclodextrin.

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Figure 1. The formation of drug/cyclodextrin inclusion complexes and their subsequent aggreation to nanoparticles.

Diabetic macular edema (DME), is practically an accumulation of fluid in the the macula part of the retina that controls the most detailed vision abilities. This accumulation is commonly caused by leaking blood vessels. DME develops in people suffering from diabetic retinopathy, which is a disease that damages the blood vessels in the retina, resulting visual impairment. It is a common problem for diabetics. Figure 2 shows a healthy macula and an inflammed one (pictured by optical coherence tomography). The best treatment for this disease is currently via an injection into the eye. OCS-01 is a suspension eye drop, thus would offer a simpler solution.

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Figure 2. A healthy macula (top) vs macula of a DME patient (bottom)

The DX-211 study was a prospective, multi-center, randomized, double-masked, parallel group, vehicle-controlled study. Type 1 or 2 diabetic patients were recruited, two main parameters were followed, their central macular thickness (CMT), and their best corrected visual acuity (BCVA). The people were treated with 1 drop 3 times per day for 12 weeks with either OCS-01 or with matching vehicle eye drops.

According to the results of the finished DX-211 trial, a total of 144 patients were randomized and 133 patients (92.3%) completed the study. After 12 weeks, in the OCS-01 group both CMT (-53.6 µm vs. -16.8 µm) and BCVA (+2.62 letters vs +1.04 letters) improved. OCS-01 proved statistically better than the vehicle. In the OCS-01 treated patients the intraocular pressure was slightly increased during the treatment, but that was consistent with known dexamethasone effects. The results clearly show that OCS-01 treatment improves the vision of DME patients and shows the objective biological effect on the retina as well.

Meanwhile another Phase 2 clinical study is being prepared for OCS-01 for treating inflammation and pain in post-cataract patients (DX-216) according to the available information in the Clinical Trials website [2].

Cyclodextrin News is sending their congratulations to Oculis for the good news. We are looking forward to their future success and hopefully soon there will be one more product on the market containing cyclodextrins.

References:

[1] Oculis announces positive OCS-01 Phase 2 data in patients with diabetic macular edema (DME); https://oculis.com/2020/02/10/a-phase-2-study-of-ocs-01-an-effective-topical-treatment-for-dme/

[2] OCS-01 in Treating Inflammation and Pain in Post-cataract Patients (DX-216); NIH, US National Library of Medicine; https://clinicaltrials.gov/ct2/show/NCT04130802

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