Ocugen submits NDA with FDA to initiate phase 1 clinical trial evaluating OCU200 for treatment of DME

The company noted the planned Phase 1 clinical study will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME. (Adobe Stock image)

Ocugen Inc. today announced that it has submitted an Investigational New Drug application (IND) with the FDA to initiate a Phase 1 clinical trial of OCU200, a fusion protein with a distinct mechanism of action (MOA), for the treatment of diabetic macular edema (DME).

According to a news release, the regulatory milestone fulfills the company’s goal to file the IND for OCU200 within the first quarter of 2023.

“Today’s achievement is an important step towards fulfilling our mission to bring novel therapeutics to address limitations of the current standard of care or unmet medical needs in hard-to-treat blindness diseases,” said Arun Upadhyay, PhD, chief scientific officer at Ocugen. “We are encouraged by the potential for OCU200 to provide a new treatment option for the significant percentage of people living with DME, including non-responders to the current standard of care.”

The company noted the planned Phase 1 clinical study will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME. This is a multicenter, open-label, dose-ranging study with 3 cohorts in the dose-escalation portion of the study and 1 cohort in the combination therapy portion of the study.

DME is one of the most common vision-threatening diseases occurring in people with diabetes and includes blurriness in vision and progressive vision loss as the disease progresses. Approximately 745,000 people in the United States are affected with DME, and this number is expected to further increase as the number of people with diabetes increases.

The company said in the news release it intends to pursue additional indications for OCU200 to potentially treat diabetic retinopathy and wet age-related macular degeneration, which combined affect nearly 9 million Americans.

OCU200 is a novel fusion protein consisting of human transferrin linked to human tumstatin. It exerts anti-proliferative, anti-inflammatory, and anti-oxidative effects by selective targeting to the retinal and choroidal tissues. OCU200 potentially showcases better bioavailability and tissue penetrance than tumstatin alone due to transferrin and provides distinct MOA binding through αVβ3 integrin pathways that can potentially reduce the number of injections for patients.

OCU200 can potentially be used for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. These diseases, combined, account for approximately 10 million cases in the U.S.