Recursion is Granted Orphan Drug Designation for REC-4881 for the Potential Treatment of Familial Adenomatous Polyposis
Recursion (NASDAQ: RXRX) has received orphan drug designation from the U.S. FDA for its drug REC-4881, aimed at treating familial adenomatous polyposis (FAP). This designation supports the development of therapies for rare conditions and provides incentives such as tax credits and potential market exclusivity. REC-4881 is a small molecule MEK1/2 inhibitor intended to reduce tumor size in FAP patients, for whom no approved therapies currently exist. With approximately 50,000 cases in the US and Europe, the company plans a Phase 2 clinical trial to assess REC-4881's safety and efficacy within the next year.
- FDA granted orphan drug designation for REC-4881, benefiting drug development.
- REC-4881 targets a rare disease (FAP) with an unmet medical need.
- Incentives include tax credits and potential market exclusivity.
- None.
SALT LAKE CITY, Sept. 29, 2021 /PRNewswire/ -- Recursion (NASDAQ: RXRX), a clinical-stage biotechnology company decoding biology by integrating technological innovations across biology, chemistry, automation, machine learning and engineering, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for REC-4881 for the potential treatment of familial adenomatous polyposis (FAP). REC-4881 is an orally bioavailable, non-ATP-competitive allosteric small molecule inhibitor of MEK1 and MEK2 being developed to reduce tumor size in FAP patients.
The FDA designates orphan products to support the development and evaluation of new treatments for rare diseases. The designation qualifies the sponsor for incentives including tax credits for qualified clinical trials, exemption from user fees and potentially seven years of market exclusivity if the medicine is approved.
"The orphan drug designation for REC-4881 is an important addition to our work to develop this medicine to treat patients with FAP, for which there is significant unmet need," said Ramona Doyle, M.D. chief medical officer of Recursion. "I am pleased that the team continues to advance this medicine towards a Phase 2, randomized, double-blind, placebo-controlled study to evaluate safety, pharmacokinetics and efficacy in FAP patients, for which we expect to begin enrolling patients within the next three quarters."
FAP is a rare tumor syndrome with no approved therapies. In the US, France, Germany, Italy, Spain and the UK alone the disease affects approximately 50,000 patients. FAP is caused by autosomal dominant inactivating mutations in the tumor suppressor gene APC. FAP patients develop polyps and adenomas in the gastrointestinal tract throughout life. These growths have a high risk of malignant transformation and can give rise to invasive cancers of the colon, stomach, duodenum and rectal tissues. Standard of care for patients with FAP is colectomy, and without surgical intervention, affected patients will progress to colorectal cancer in adulthood. Post-colectomy, patients receive endoscopic surveillance every 6-12 months to monitor disease progression. While surgical management and surveillance have improved the prognosis for FAP patients, duodenal and desmoid tumors remain a major cause of death in patients with FAP following colectomy.
Learn more about Recursion and view its pipeline at Recursion.com/pipeline.
About Recursion
Recursion is a clinical-stage biotechnology company decoding biology by integrating technological innovations across biology, chemistry, automation, machine learning and engineering. Our goal is to radically improve the lives of patients and industrialize drug discovery. Central to our mission is the Recursion Operating System, which combines an advanced infrastructure layer to generate what we believe is one of the world's largest and fastest-growing proprietary biological and chemical datasets. We combine that with the Recursion Map, a suite of custom software, algorithms and machine learning tools that we use to explore foundational biology unconstrained by human bias and navigate to new biological insights. Learn more at www.Recursion.com, or connect on Twitter and LinkedIn.
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