Valo Health’s Rho kinase (ROCK) 1 and 2 inhibitor has failed to reduce the severity of diabetic retinopathy in a phase 2 trial, leading the Flagship-founded biotech to drop the candidate.
The drug, OPL-0401, was evaluated in a study of 114 patients with various degrees of diabetic retinopathy, an eye condition caused by high blood sugar levels that can lead to blindness. The study failed its primary objective of showing a reduction in the severity of diabetic retinopathy after 24 weeks when compared to placebo.
Valo said the drug, which it licensed from Sanofi in 2021, was not linked to any serious adverse events and pointed out that “no adverse events related to liver, kidney or heart function were observed.”
Casting around for a silver lining in the efficacy data, Valo said that one of the dose groups that consisted of a “small number of patients” had demonstrated a “consistent trend across several key efficacy variables,” which “needs to be confirmed in future studies.”
Those studies won’t be conducted by Valo, however, which said in the Dec. 31 release that it had “made the strategic decision to suspend its development of OPL-0401 and seek a partner to further develop the program.”
“While the data were intriguing, OPL-0401 did not incorporate elements of our platform in discovery or development,” said CEO Brian Alexander, M.D., who joined Valo and Flagship in November. “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.”
Valo’s AI-powered Opal computational platform combines real-world patient data with human tissue modeling and machine learning models to speed up the process of discovering small molecules for new therapies. The aim is to provide an end-to-end solution that can develop drugs from the discovery process through to regulatory approval.
The platform has already attracted the attention of Novo Nordisk, which handed over $60 million in upfront cash back in September 2023 to turn Opal towards cardiovascular disease.
Based in Boston, Valo was founded by Flagship in 2020. Since then, the company has scooped up a couple of biotechs in the form of protein-therapeutics-focused Courier and cardiovascular-focused TARA Biosystems. Plans for Valo to go public via a special purpose acquisition company merger with Khosla Ventures Acquisition fizzled out in 2021.