Recursion Pharmaceuticals, a biotech start-up in Salt Lake City, today announced the receipt of Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the use of Tempol in the treatment of the genetic disorder Cerebral Cavernous Malformation (CCM). Recursion will accelerate its preclinical work to enable an Investigational New Drug Application to the FDA in the next 12 months.
Cerebral Cavernous Malformation is present in up to 1.5 million individuals in the United States, according to the Angioma Alliance, a patient advocacy organization for patients with the disease. The majority of those affected have few if any symptoms and are unaware they have the disease. However, approximately 60,000 Americans experience more severe symptoms, including seizures, vision and hearing loss, paralysis, other focal neurologic deficits and/or hemorrhagic stroke. “The patient community is excited by the possibility of a non-surgical treatment for CCM,” notes Angioma Alliance President Dr. Connie Lee. “We look forward to working with Recursion to bring Tempol into clinical trials.”
Tempol is the first of many treatments Recursion plans to advance to treat genetic diseases; the company leverages advances in both biology and computation to rapidly investigate the potential of thousands of mature molecules across many genetic disease models.
“Receiving this designation is an important step in our ongoing efforts towards developing the first non-surgical approved treatment for CCM,” said Chris Gibson, Co-Founder and CEO of Recursion. Though Tempol is not currently approved to treat any disease, it is a well-studied compound with well-described mechanism and toxicity in a number of models. “We are highly focused on repurposing both old drugs and shelved assets across many genetic diseases because of the translational acceleration that comes with finding new uses for compounds for which a great deal is already known, and the speed with which we’ve moved this project forward illustrates these benefits nicely.”
The Orphan Drug Act was passed in 1983 to incentivize research into diseases that impact fewer than 200,000 people in the United States. Under the Act, the FDA reviews applications to determine whether a particular drug is a good match for a rare disease, and provides market exclusivity, tax and other benefits if the drug is ultimately given marketing authorization.
Recursion Pharmaceuticals is an emerging biotechnology company based in Salt Lake City. Recursion combines experimental biology and bioinformatics with artificial intelligence in a massively parallel system to quickly and efficiently identify treatments for any disease which can be modeled at the cellular level. From its initial and continued focus on drug repurposing to treat rare diseases, Recursion has broadened its platform to probe rich data from high-throughput automated screens for a number of indications, including aging, inflammation, infectious disease, oncology, and diagnostics. Learn more at www.recursionpharma.com, or connect on Twitter, Facebook, and LinkedIn.