Valo Health Suspends Development of OPL-0401 for Diabetic Retinopathy After Phase 2 Study

Valo Health has announced the results of its Phase 2 SPEC­TRA study evaluating OPL-0401 in patients with diabetic retinopathy (DR). The study, which enrolled 114 patients, did not meet its primary or secondary endpoints in the predefined primary population. As a result, Valo Health has decided to suspend the development of OPL-0401 and seek a partner to continue the program.

Study Design and Objectives

The SPEC­TRA study (NCT05393284) was a 24-week, multicenter, randomized, double-blind, placebo-controlled trial. It included patients with:

       • Moderately severe or severe non-proliferative diabetic retinopathy (NPDR)

       • Mild proliferative diabetic retinopathy (PDR)

The study aimed to evaluate the efficacy of OPL-0401 in improving the Diabetic Retinopathy Severity Score (DRSS).

       • Primary Endpoint: Proportion of patients achieving a 2-step or more improvement in DRSS in the study eye.

       • Key Secondary Endpoint: Proportion of patients achieving a 3-step or more improvement in binocular DRSS.

Neither endpoint reached statistical significance.

Safety and Tolerability

The treatment demonstrated a favorable safety profile:

       • Adverse Events: Mild and infrequent.

       • No Serious Adverse Events: No effects on liver, kidney, or heart function were observed.

Certain doses in the study showed potential evidence of preventing disease progression. However, these trends require further validation in future trials.

The Unmet Need in Diabetic Retinopathy

“There is a sig­nif­i­cant unmet med­ical need for patients suf­fer­ing from dia­bet­ic retinopa­thy, a lead­ing cause of blind­ness in the US, and cur­rent treat­ment options con­sist main­ly of inva­sive intrav­it­re­al injec­tions and laser treat­ment tar­get­ed to patients with more severe or advanced dis­ease,” said Brian M. Alexander, MD, MPH, Chief Exec­u­tive Offi­cer of Valo Health and CEO-Part­ner of Flag­ship Pio­neer­ing.

Dr. Alexander expressed disappointment in the results but highlighted the favorable safety profile and evidence of clinical activity in specific doses, noting the potential of OPL-0401 to benefit DR patients.

Strategic Focus: Leveraging the Opal Platform

Valo Health’s Opal Computational Platform is an AI-driven drug development engine designed to accelerate the discovery and development of innovative medicines.

“While the data were intrigu­ing, OPL-0401 did not incor­po­rate ele­ments of our plat­form in dis­cov­ery or devel­op­ment. As a result, we are look­ing to find a part­ner well posi­tioned to real­ize the clin­i­cal poten­tial OPL-0401 in DR and beyond. Mov­ing for­ward, Valo’s renewed strate­gic focus will be on oppor­tu­ni­ties that lever­age our Opal plat­form to dis­cov­er new ther­a­peu­tic tar­gets in real world data, val­i­date those tar­gets in human-cen­tric mod­els, and devel­op new med­i­cines with our AI enabled closed loop small mol­e­cule design,” Dr. Alexander stated.

Valo Health is seeking a partner to further explore the clinical potential of OPL-0401 for diabetic retinopathy and other indications.

Future Directions

While the SPEC­TRA study did not meet its primary endpoints, the data suggest promising trends in one dose group, particularly regarding DRSS improvements and disease progression prevention. These findings require further investigation in future clinical studies.

By shifting its focus to AI-enabled drug discovery, Valo Health aims to address critical unmet medical needs and develop next-generation therapies for patients.