Recursion’s Phase 2 Trial for Cerebral Cavernous Malformation Shows Limited Efficacy Despite Meeting Safety Goals

Trial Overview:
Recursion Pharmaceuticals conducted a Phase 2 SYCAMORE trial to evaluate the safety, tolerability, and exploratory efficacy of REC-994, an orally bioavailable small molecule, in patients with symptomatic cerebral cavernous malformation (CCM).

Safety and Tolerability:
The trial met its primary endpoint of safety and tolerability, with REC-994 demonstrating a similar adverse event profile to placebo across both 200mg and 400mg doses after 12 months of treatment.

Efficacy Data:
While MRI-based secondary efficacy measures showed a trend towards reduced lesion volume and hemosiderin ring size in patients receiving the 400mg dose compared to placebo, there were no improvements in patient- or physician-reported outcomes at 12 months.

Future Plans:
Recursion plans to engage with the FDA to discuss the need for an additional clinical study, potentially exploring longer study durations, higher doses, and larger patient cohorts.

Background on CCM:
Cerebral cavernous malformation is a rare neurovascular disorder affecting approximately 360,000 symptomatic individuals in the US and EU5, with limited treatment options beyond surgical interventions.

Recursion's Platform:
REC-994 was discovered using Recursion’s AI-powered drug discovery platform, which leverages a large proprietary biological and chemical dataset and machine-learning algorithms.

Market Impact:
Despite meeting safety goals, Recursion's stock declined by 16.6% due to the mixed outcomes and limited efficacy data.