Recursion Reports Phase 2 REC-994 Data Meeting Safety and Tolerability Endpoint for CCM

Recursion, a leading clinical-stage TechBio company, has announced the top-line results of their SYCAMORE trial, a year-long Phase 2 randomized double-blind study evaluating the safety, tolerability, and exploratory efficacy of REC-994 in patients with symptomatic Cerebral Cavernous Malformation (CCM). The results revealed that REC-994 met its primary endpoint of safety and tolerability, showing comparable profiles across placebo and two dosage arms (200mg and 400mg) in terms of adverse events after a year of treatment.

The trial also explored secondary efficacy endpoints using magnetic resonance imaging (MRI). Notable trends were observed at the highest dosage of 400mg, which indicated a reduction in lesion volume and hemosiderin ring size compared to the placebo. Moreover, improvements in these trends appeared to be time-dependent at the 400mg dose. However, neither patient nor physician-reported outcomes showed improvements at the 12-month mark.

Recursion plans to meet with the FDA to discuss the next steps for another clinical study. The company aims to present these data at a future medical conference and intends to submit the results for publication in a scientific journal.

Dr. Jan-Karl Burkhardt, the Principal Investigator of the study and Division Head of Cerebrovascular Surgery at the University of Pennsylvania, emphasized the importance of these findings. He acknowledged the initial data as a significant step and stressed the need for future studies involving a longer duration and a larger patient cohort. Connie Lee, Psy.D., founder and CEO of the Alliance to Cure Cavernous Malformation, also expressed optimism, noting that this promising start brings hope to thousands of families who currently rely solely on surgical options for treatment.

Najat Khan, Ph.D., Chief R&D Officer and Chief Commercial Officer of Recursion, voiced the company's encouragement by the study's success in meeting its primary safety endpoint and in showing promising trends in exploratory efficacy endpoints. The insights gained will shape the design of future studies, including considerations of study duration, higher doses, and a larger patient cohort. Khan highlighted this milestone as the first of several key clinical readouts for Recursion, marking an early proof of concept for the company's evolving Recursion OS platform, which aims to revolutionize drug discovery and development.

CCM is a neurovascular condition affecting approximately 360,000 symptomatic individuals in the US and EU5, with potentially over 1 million patients worldwide. It is characterized by enlarged capillary cavities in the spinal cord and brain, leading to risks of seizures, headaches, neurological deficits, and hemorrhagic stroke. Current treatments are limited to non-pharmacologic interventions like microsurgical resection and stereotactic radiosurgery, which are not always feasible or curative due to the lesions' location.

REC-994, an orally bioavailable small molecule designed as a superoxide scavenger, aims to address this high unmet need. Developed using the foundational technology of the Recursion OS, REC-994 has shown preclinical activity in CCM models and demonstrated tolerability for chronic dosing in Phase 1 trials. The drug has received Orphan Drug Designation in both the US and Europe for symptomatic CCM.

The Phase 2 SYCAMORE trial, a randomized, double-blind, placebo-controlled study, evaluated two doses of REC-994 in 62 participants with CCM, who completed 12 months of treatment and are now part of a long-term extension study. The trial's primary endpoint was safety and tolerability, with secondary efficacy endpoints including MRI-based measures, clinician and patient-reported outcomes, and selected biomarkers.

Recursion leverages its Recursion OS platform to integrate technology, biology, and chemistry, aiming to advance the future of medicine. The company is headquartered in Salt Lake City, with additional offices in Toronto, Montréal, London, and the San Francisco Bay Area.