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Recursion Pharma Receives Orphan Drug Designation for Hereditary Stroke Disorder

Recursion Pharmaceuticals, a drug discovery start-up company, announced receipt of an Orphan Drug Designation from the FDA for the use of Tempol for the treatment of Cerebral Cavernous Malformation, a hereditary stroke disease. Recursion expects this will be the first of many potential treatments identified using their proprietary drug discovery platform.

Salt Lake City, UT, October 05, 2015 --( 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.

About Recursion Pharmaceuticals - Recursion Pharmaceuticals, LLC is a drug discovery company founded in 2013 in Salt Lake City, Utah. Recursion uses a novel drug screening platform to efficiently repurpose and reposition drugs to treat rare genetic diseases. The company’s focus on rare genetic diseases is much-needed, as there are more than 5,000 such conditions that together affect millions of Americans, and more than 95% of these diseases have no approved therapy. Recursion’s novel drug screening platform combines experimental biology and bioinformatics in a massively parallel system to quickly and efficiently identify treatments for multiple rare genetic diseases. The core of the approach revolves around high-throughput automated screening using high-content assays in human cells, which allows the near simultaneous modeling of hundreds of genetic diseases. Rich data from these assays is probed using advanced statistical and machine learning approaches, and the effects of thousands of known drugs and shelved drug candidates can be investigated efficiently to identify those holding the most promise for the treatment of any one rare genetic disease.
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