Recursion, a biotechnology company that combines artificial intelligence (AI), experimental biology, and automation to discover and develop drugs at scale, has received United States Food and Drug Administration (FDA) approval to start Phase I study of REC-994 in cerebral cavernous malformation.

CCM, a genetic disease, is present in up to 1.5 million individuals in the United States, according to Angioma Alliance, a patient advocacy organization for patients with the disease. Cerebral cavernous malformations (CCMs) are collections of small blood vessels in the brain that are enlarged and irregular in structure which lead to altered blood flow.

REC-994 is a well-characterized, small molecule that has been granted orphan designation by the U.S. FDA for the treatment of symptomatic CCM and by the European Medicines Agency for the treatment of familial CCM. Later this year, Recursion plans to initiate the company’s first clinical trial to evaluate the safety and pharmacokinetics of REC-994 as an oral treatment for CCM.

REC-994 is a potent, selective superoxide dismutase mimetic, a mechanism of action that has preclinical proof of concept to impact lesion development in a mouse model of CCM.

Chris Gibson, Ph.D., Co-founder and CEO of Recursion, said, “REC-994 was discovered using a basic machine learning system that was the seed for Recursion’s AI-powered drug discovery platform. As we embark on this important milestone of initiating our first clinical trial, we are further emboldened in our goal of building a fully integrated drug discovery and development organization that leverages computational tools to accelerate every step in the process of bringing new medicines to patients.”

“As we enter the clinic with our first program, we will continue to build tools to accelerate and scale the discovery of both repurposed and novel drugs, to advance important new therapies for rare genetic diseases, as well as unlock therapeutic targets in multifactorial diseases in inflammation and immunology,” he added.

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