Vantage Biosciences announced today that it has successfully dosed the first patient in its Phase 2 clinical study of VX-01, an oral therapy being developed for non-proliferative diabetic retinopathy (NPDR). This milestone represents a significant advancement in the company's efforts to develop what could potentially become the first oral treatment for this widespread condition.
Diabetic retinopathy, affecting more than 100 million people globally, stands as one of the leading causes of blindness among working-age adults. The condition, if left untreated, can progress to more severe stages of diabetic eye disease with increased risk of permanent vision loss.
"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," said Alek Safarian, Co-Founder and Chairman of Vantage Biosciences. "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."
Novel Mechanism Targets Underlying Inflammation
VX-01 is an orally administered small molecule therapy specifically designed to target neurovascular inflammation associated with diabetic eye disease. The drug aims to address the underlying biological processes that contribute to microvascular complications in the retina, potentially slowing or preventing disease progression before irreversible vision loss occurs.
Current standard treatments for advanced diabetic retinopathy typically involve invasive procedures such as intravitreal injections or laser photocoagulation. An oral therapy that could effectively manage NPDR at earlier stages would represent a significant advancement in treatment paradigms for this condition.
Comprehensive Phase 2 Trial Design
The VX01-DR-201 study is a multi-center, double-masked, randomized, placebo-controlled trial designed to evaluate the efficacy of oral VX-01 in patients with moderate to severe NPDR without center-involved diabetic macular edema (CI-DME).
The trial will enroll patients across 27 clinical sites spanning the United States, Australia, and several Southeast Asian countries. Eligible participants will be randomized in a 1:1 ratio into one of two study cohorts, with all participants taking either VX-01 or placebo twice daily for 52 consecutive weeks. This treatment period will be followed by a 12-week post-treatment observation period to assess durability of response.
Results from this Phase 2 study are expected in 2027, providing critical data on the safety and efficacy of this novel approach to treating diabetic retinopathy.
Addressing a Significant Unmet Need
The development of VX-01 addresses a substantial unmet need in diabetic eye care. Early intervention in NPDR could potentially reduce the burden of more advanced diabetic eye disease, which often requires frequent clinic visits for invasive treatments.
"The current treatment landscape for diabetic retinopathy focuses heavily on managing advanced disease," noted Safarian. "Our approach with VX-01 aims to shift that paradigm toward earlier intervention, potentially preserving vision before significant damage occurs."
About Vantage Biosciences
Vantage Biosciences is a clinical-stage biotechnology company dedicated to developing innovative therapies for diabetic eye diseases. Founded by ALSA Ventures, the company maintains a commitment to scientific excellence and a patient-centered approach in advancing treatments that address significant unmet medical needs in diabetic eye disease.
The company is backed by ALSA Ventures, a biotech investment firm focused on the creation and development of breakthrough therapeutic companies. This Phase 2 trial represents a critical milestone in Vantage Biosciences' development pipeline as it works to bring novel therapies to patients with diabetic eye conditions.
