Recursion’s oral drug candidate for cerebral cavernous malformation showed no improvements in patient- or physician-reported outcomes at 12 months. The biotech will engage with the FDA to determine the need for an additional study.
Recursion on Tuesday released topline data from its Phase II SYCAMORE trial, showing that its drug candidate REC-994 is largely safe to use in patients with cerebral cavernous malformation—though its efficacy profile leaves much to be desired.
After 12 months of treatment, both 200-mg and 400-mg doses of REC-994 showed similar adverse event profiles to placebo, indicating that the investigational treatment is largely safe and tolerable for patients with cerebral cavernous malformation (CCM).
However, SYCAMORE’s efficacy data were less promising. Magnetic resonance imaging showed a “trend toward reduced lesion volume” in patients who were given the higher, 400-mg dose of REC-994, compared with placebo. These trends improved over time, though Recursion reported no improvements in patient- or physician-reported outcomes at 12 months.
Recursion Chief R&D and Commercial Officer Najat Khan in a statement said the biotech is “encouraged” by these findings, which make REC-994 “the first investigational therapy to demonstrate safety” in CCM, while also demonstrating “promising trends in exploratory efficacy endpoints.”
“These results provide critical insights that will inform our next study design, including exploring study duration, higher doses, and a larger cohort of patients,” Khan said, noting that Tuesday’s readout “represents an early proof-of-platform milestone” for the biotech.
Recursion is seeking a meeting with the FDA “as soon as practical” to determine the way forward for REC-994, including an additional clinical trial, according to the company. The biotech will also present findings from SYCAMORE at an upcoming scientific congress and will submit them for publication in a peer-reviewed journal.
CCM is a rare neurovascular disease characterized by malformations of the spinal cord and the brain, often involving abnormally enlarged capillary cavities. The condition affects around 360,000 symptomatic patients, who are at heightened risk of seizures, headaches and progressive neurological deficits, as well as potentially fatal hemorrhagic strokes. There are currently no pharmacologic treatments for CCM.
REC-994, an orally available small molecule superoxide scavenger, was discovered using Recursion’s AI-powered drug discovery platform, which leverages “one of the world’s largest proprietary biological and chemical datasets,” according to the biotech’s website. The platform then makes use of machine-learning algorithms to produce an extensive searchable list of interactions from this database.
Recursion is also developing REC-2282, another orally available drug candidate that can penetrate the central nervous system, for the treatment of NF2-mutated meningiomas, and REC-4481, a non-ATP-competitive blocker of MEK1 and MEK 2 for familial adenomatous polyposis.
