Vantage Biosciences doses first patient in phase 2 study of VX-01, oral therapy for nonproliferative diabetic retinopathy

Vantage Biosciences has successfully dosed the first patient in its phase 2 clinical study evaluating VX-01, an oral therapy for the treatment of nonproliferative diabetic retinopathy (NPDR).

VX01-DR-201, is a phase 2, multicenter, double-masked, randomized, placebo-controlled study designed to evaluate the efficacy of an oral dose of VX-01 in patients with moderate to severe NPDR without center-involved diabetic macular edema (CI-DME). Participants will be randomly assigned 1:1 into 1 of 2 study cohorts and will take VX-01 or placebo twice daily for 52 consecutive weeks, followed by a 12-week posttreatment observation period.

VX-01 is an orally administered small molecule therapy designed to target neurovascular inflammation associated with diabetic eye disease and aims to slow or prevent disease progression in patients with NPDR before irreversible vision loss occurs.

Alek Safarian, cofounder and chairman of Vantage Biosciences, commented on the trial in a press release from the company, saying, “We are pleased to initiate patient dosing in this important phase 2 study, which evaluates VX-01 as a potential early intervention for diabetic retinopathy. By targeting the disease at an earlier stage, VX-01 has the potential to slow or prevent progression to more severe forms of diabetic retinopathy, reducing the likelihood that patients will require invasive treatments in the future.”

The company stated that the study is expected to read out in 2027 and is being run at 27 sites across the US, Australia, and a number of Southeast Asian countries.

Reference

Vantage Biosciences doses first patient in phase 2 clinical study of oral VX-01 for nonproliferative diabetic retinopathy. News release. Vantage Biosciences. April 11, 2025. Accessed April 11, 2025. https://www.globenewswire.com/news-release/2025/04/11/3059941/0/en/Vantage-Biosciences-Doses-First-Patient-in-Phase-2-Clinical-Study-of-Oral-VX-01-for-Non-Proliferative-Diabetic-Retinopathy.html