16 July 2018 - - Salt Lake City, Utah-based biotechnology company Recursion Pharmaceuticals has received clearance from the US FDA of an investigational new drug application (iNDA) for a phase 1 clinical trial of REC-994 in the treatment of cerebral cavernous malformation (CMM), the company said.
REC-994 is a potent, selective superoxide dismutase mimetic. Its mechanism of action has preclinical proof of concept to impact lesion development in a mouse model of CCM. It has been granted orphan drug designation by the US FDA and the EMA.
CCM is a genetic disease resulting in collections of small blood vessels in the brain that are enlarged and irregular in structure and which lead to altered blood flow. While these malformations can occur anywhere in the body, CCMs occurring in the brain cause severe symptoms, including seizures, vision and hearing loss, paralysis, other focal neurologic deficits and/or hemorrhagic stroke.
Recursion Pharmaceuticals combines artificial intelligence, experimental biology, and automation to identify and de-risk potential drugs for diverse indications, including genetic disease, inflammation, immunology, and infectious disease.
The company applies causative perturbations to human cells to generate disease models and associated biological image data. Its database of biological images is then probed using advanced machine learning approaches, revealing drug candidates, mechanisms of action, and potential toxicity, with the eventual goal of decoding biology and advancing new therapeutics.