Recursion has stumbled through an early test of its tech-enabled approach to drug discovery, reporting a hit on its phase 2 primary endpoint of safety and tolerability but failing to wow on the efficacy front.
The study evaluated REC-994 in people with symptomatic cerebral cavernous malformation (CCM), a rare disease characterized by lesions that increase the risk of seizures, progressive neurological deficits and potentially fatal stroke. The first version of Recursion’s platform identified a superoxide scavenger as a molecule that may alleviate neurological symptoms and slow lesion accumulation, leading to REC-994.
Investigators put the idea to the test by randomizing 62 patients to take REC-994 or placebo in a phase 2 study. Recursion saw a similar safety and tolerability profile across the placebo group and two REC-994 doses over 12 months of treatment, causing the study to meet its primary endpoint.
The efficacy data are less compelling. Recursion said MRI scans showed “a trend towards reduced lesion volume and hemosiderin ring size” at the higher REC-994 dose compared to placebo. Hemosiderin is a protein that surrounds more advanced, bleeding cavernous malformations and is seen on MRI.
Recursion saw no improvements on patient-reported outcomes (PROs), or physician-led measures, after 12 months of treatment. Talking on an earnings call in August, Najat Khan, Ph.D., chief R&D officer at Recursion, said MRI-based endpoints and PROs, plus biomarkers, were the three efficacy areas the biotech was focusing on. Khan said the PROs “are extremely important to patients.”
The biotech continues to see a future for the molecule and plans to talk to the FDA about an additional trial. Recursion is at the forefront of efforts to treat CCM, but rivals are on its tail, with Ovid Therapeutics preparing to move a ROCK2 inhibitor into phase 2 and Neurelis pushing a candidate through phase 1.
Recursion’s share price opened down 10% at $6.55. The biotech has upcoming chances to improve perceptions, with its pipeline featuring multiple readouts and an agreed merger with Exscientia set to add more assets.