Vantage Biosciences Begins Phase 2 Trial of Oral VX-01 for Diabetic Retinopathy

On April 11, 2025, Vantage Biosciences, a clinical stage biotechnology company, announced a significant advancement in its efforts to combat diabetic eye diseases. The company has successfully administered the first dose to a patient in its Phase 2 clinical trial of VX-01, a pioneering oral treatment designed to address non-proliferative diabetic retinopathy (NPDR).

Diabetic retinopathy is a condition that impacts over 100 million people globally, representing a major cause of blindness among adults of working age. In the absence of timely medical intervention, NPDR can progress into more severe stages, escalating the risk of significant vision impairment.

This recent development marks a critical milestone for VX-01, which could potentially become the first oral medication available for NPDR. This treatment is specifically crafted to target the inflammatory processes that drive the progression of NPDR.

Alek Safarian, Co-Founder and Chairman of Vantage Biosciences, expressed satisfaction with the commencement of the patient dosing in the Phase 2 study. He highlighted the potential of VX-01 as an early intervention strategy for diabetic retinopathy. By addressing the disease in its initial stages, VX-01 aims to halt or slow progression towards more advanced forms, potentially reducing the future need for invasive treatments.

The Phase 2 study is anticipated to deliver results in 2027 and is currently underway at 27 locations across the United States, Australia, and several Southeast Asian countries. This is a multi-center, double-masked, randomized, placebo-controlled trial. It is intended to assess the efficacy of the oral VX-01 dose in patients diagnosed with moderate to severe NPDR, excluding those with center-involved diabetic macular edema (CI-DME). Participants are randomized into two groups in a 1:1 ratio, receiving either VX-01 or a placebo twice daily for 52 weeks, with a subsequent 12-week observation period following the treatment.

VX-01 operates as an orally administered small molecule therapy that targets neurovascular inflammation associated with diabetic eye disease. By intervening in the biological processes that lead to microvascular complications in the retina, VX-01 aims to delay or prevent the advancement of NPDR before patients experience irreversible vision loss.

Vantage Biosciences, established by ALSA Ventures, is committed to the development of cutting-edge treatments for diabetic eye diseases. The company adopts a patient-focused approach, striving for scientific excellence in addressing unmet medical needs in the field of diabetic eye disease.

Backed by ALSA Ventures, a prominent biotech investment firm, Vantage Biosciences is at the forefront of creating and advancing therapeutic companies that deliver breakthrough treatments.