Recursion's Phase 2 Brain Disease Trial Shows Limited Efficacy

Recursion has faced challenges with its innovative approach to drug discovery, particularly with its recent phase 2 trial of REC-994. This drug was evaluated for its potential to treat symptomatic cerebral cavernous malformation (CCM), a rare condition that involves lesions in the brain, increasing the risk of seizures, neurological problems, and potentially fatal strokes.

CCM is characterized by lesions that can lead to severe health issues. The initial version of Recursion’s platform suggested that superoxide scavengers might reduce neurological symptoms and slow lesion growth, leading to the development of REC-994. In a phase 2 trial, 62 patients were randomly assigned to receive either REC-994 or a placebo for assessment. Over a 12-month treatment period, the study showed that REC-994 had a consistent safety and tolerability profile compared to the placebo, thus meeting the primary endpoint of the trial.

However, the efficacy results were not as promising. MRI scans indicated a trend towards reduced lesion volume and hemosiderin ring size at the higher dose of REC-994 when compared to the placebo. Hemosiderin is a protein that typically surrounds more advanced, bleeding lesions and is detectable via MRI. Despite these findings, there were no significant improvements in patient-reported outcomes (PROs) or physician-led measures after 12 months of treatment. Najat Khan, Ph.D., chief R&D officer at Recursion, mentioned during an earnings call in August that MRI-based endpoints, PROs, and biomarkers were the primary efficacy areas the company was focusing on, highlighting the importance of PROs to patients.

Recursion remains optimistic about the future of REC-994 and plans to consult with the FDA about conducting an additional trial. The company is a leader in the quest to treat CCM but faces competition from other firms. Ovid Therapeutics is preparing to move its ROCK2 inhibitor into a phase 2 trial, and Neurelis is advancing a candidate through phase 1 studies.

Following the announcement of the trial results, Recursion’s share price opened down 10%, at $6.55. Despite the setback, the biotech company has several upcoming opportunities to enhance its reputation. Its pipeline includes multiple readouts and an anticipated merger with Exscientia, which is expected to bring additional assets and potentially improve the company's standing in the biotech field.