Recursion Pharmaceuticals has announced positive top-line results from its Phase 2 SYCAMORE trial of REC-994, an AI-derived drug candidate for symptomatic cerebral cavernous malformation (CCM). The study met its primary safety endpoint and showed promising trends in MRI-based efficacy measures among participants receiving the highest dose.
SYCAMORE Trial Details
The SYCAMORE trial was a 12-month randomized, double-blind, placebo-controlled study assessing the safety, tolerability, and exploratory efficacy of REC-994 in patients with CCM. The trial enrolled 62 participants in June 2023, with 80% proceeding to a long-term extension study. Participants were administered either a placebo, a 200mg dosage of REC-994, or a 400mg dosage of REC-994.
Safety and Tolerability
The drug demonstrated a similar safety profile across the placebo and both 200mg and 400mg dosage arms, with no significant difference in the frequency and severity of adverse events. This suggests that REC-994 is well-tolerated in patients with CCM.
Efficacy Measures
Secondary efficacy endpoints suggested a reduction in lesion volume and hemosiderin ring size at the 400mg dose compared to placebo. However, improvements in patient or physician-reported outcomes were not observed at the 12-month mark. The trial was not powered to demonstrate statistical significance.
Expert Opinions
Dr. Jan-Karl Burkhardt, MD, the study's Principal Investigator, and Connie Lee, Psy.D., CEO of the Alliance to Cure Cavernous Malformation, both expressed optimism about the potential of REC-994 to address the unmet needs of CCM patients who currently have limited treatment options outside of surgery.
Next Steps
Recursion plans to engage with the FDA to discuss subsequent studies and intends to present trial data at an upcoming medical conference, as well as submit findings for publication in a peer-reviewed journal. REC-994 has previously received Orphan Drug Designation in the US and Europe.
About Cerebral Cavernous Malformation (CCM)
CCM is a neurovascular disorder characterized by vascular malformations in the brain and spinal cord, leading to seizures, neurological deficits, and possible hemorrhagic stroke. It affects an estimated 360,000 symptomatic individuals in the US and EU5, with a potentially larger undiagnosed population. Current treatment options are limited outside of surgery, highlighting the unmet medical need for effective therapies.
Recursion's Technology
Recursion leverages its Recursion OS, a platform that combines biology, chemistry, and machine learning to advance drug discovery and development. The company is headquartered in Salt Lake City and operates globally.
