Ocugen to move forward with dosing of second cohort of phase 1 clinical trial of OCU200

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Ocugen announced the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial convened following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort.

The first patient in the clinical trial of OCU200 was dosed at the beginning of 2025. OCU200 is a novel fusion protein consisting of 2 human proteins, tumstatin and transferrin, with the potential to treat diabetic macular edema (DME), administered intravitreally. The DSMB noted that no serious adverse events related to OCU200 have been reported to date.

The OCU200 phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in 3 cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). Patients will receive 2 doses, 6 weeks apart, and patients will be followed for up to 6 months.

Huma Qamar, MD, MPH, chief medical officer at Ocugen commented on the trial in a press release from the company.

“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Qamar. “There remains a considerable unmet medical need for the 30% to 40% of DME patients who do not respond to current anti-VEGF therapies. OCU200 holds the promise of potentially benefiting all DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) patients.”

Ocugen noted it intends to complete the Phase 1 OCU200 clinical trial in the second half of 2025 and to provide preliminary safety and efficacy updates throughout the year.

References:

1. Data and safety monitoring board reviews cohort 1 safety data and approves dosing cohort 2 in the clinical trial of OCU200—a novel fusion protein for diabetic macular edema. Globe Newswire. Published March 18, 2025. Accessed March 20, 2025. https://www.globenewswire.com/news-release/2025/03/18/3044471/0/en/Data-and-Safety-Monitoring-Board-Reviews-Cohort-1-Safety-Data-and-Approves-Dosing-Cohort-2-in-the-Clinical-Trial-of-OCU200-a-Novel-Fusion-Protein-for-Diabetic-Macular-Edema.html