Ocugen Doses First Patient in Phase 1 Trial of OCU200 for Diabetic Macular Edema

Ocugen has announced that the first patient in its Phase 1 clinical trial for OCU200, a potential treatment for diabetic macular edema (DME), has been dosed. The trial represents a significant milestone in the development of this novel therapeutic candidate.

What is OCU200?

OCU200 is described as a recombinant fusion protein composed of two parts connected by a linker:

       • Tumstatin: The active component of the drug, acting as an anti-inflammatory and anti-VEGF (vascular endothelial growth factor) agent. It works by binding to integrin receptors, which play a key role in the pathogenesis of DME and related conditions.

       • Transferrin: This component targets the drug to the choroid and retina by binding to transferrin receptors on endothelial cells, enhancing delivery to the affected areas.

Insights from Ocugen Leadership

Shankar Musunuri, PhD, MBA, chairman, CEO, and co-founder of Ocugen, highlighted the innovative potential of OCU200.

“OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis,” Musunuri stated in a press release. “OCU200 holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.”

Details of the Phase 1 Clinical Trial

The ongoing Phase 1 clinical trial is a multicenter, open-label, dose-escalation study designed to evaluate the safety of OCU200. Key details include:

       • Dosing: The study involves three cohorts receiving low (0.025 mg), medium (0.05 mg), and high (0.1 mg) doses of OCU200.

       • Administration: Patients receive two intravitreal injections, administered six weeks apart.

       • Follow-Up: Participants will be monitored for six months following the final injection to assess safety and other outcomes.

Future Plans for OCU200

Ocugen has stated its intention to seek regulatory approval to use OCU200 as a first-line therapy for DME, diabetic retinopathy, and wet AMD. If successful, this could provide a new treatment option for patients, particularly for those who do not respond to existing anti-VEGF therapies.

This trial represents a critical step forward in the development of innovative treatments for retinal diseases and offers hope for improved patient outcomes in these challenging conditions.