Bringing two technologies together for a best-in-class therapy
miRecule, Inc., a biotech based in Gaithersburg, Maryland, today announced a strategic collaboration and exclusive license agreement with pharmaceutical giant Sanofi to develop and commercialize a novel treatment for FSHD. Sanofi is a multinational corporation valued at $36 billion, making it the ninth largest pharmaceutical company in the world. The two companies will collaborate to combine miRecule’s anti-DUX4 RNA therapy with Sanofi’s NANOBODY technology to target the therapy to muscles. The companies aim to produce a “best-in-class” treatment to slow or stop the progression of FSHD by suppressing the expression of the DUX4 gene, the underlying cause of the disease.
“Under the terms of the collaboration agreement, miRecule will grant Sanofi an exclusive worldwide license to intellectual property rights to the FSHD therapy,” miRecule said. “miRecule and Sanofi will collaborate on research activities through lead candidate selection.” Once a lead candidate has been selected, Sanofi will assume sole responsibility for investigational new drug (IND) enabling studies—laboratory studies to produce data to demonstrate to the Food and Drug Administration (FDA) that the drug is likely to be safe, effective, and suitable to test in human clinical trials. If the FDA provides IND clearance, Sanofi would shepherd the drug through clinical trials, regulatory approval, and commercializing the therapy globally.
What does this partnership mean for patients?
The FSHD Society took an early interest in miRecule when its co-founder and CEO, Anthony Saleh, PhD, reached out after his own family was diagnosed with FSHD. The Society, Friends of FSH Research, and National Institute of Neurological Disorders and Stroke (NINDS, Grant 5R44NS119147-02) funded early-stage research. Last week, miRecule announced that SOLVE FSHD has agreed to invest $1 million toward the next phase of development.
The Society hoped that as miRecule hit its milestones, a large pharmaceutical company would become interested and provide the substantial resources and expertise needed to develop miRecule’s technology and take it to market. The partnership with Sanofi is great news because miRecule now has those needed resources.
"We are thrilled to enter our first major licensing transaction with a partner of Sanofi’s caliber and capabilities as a global leader in the development and commercialization of rare disease therapies,” said Saleh. “Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life.”
"We look forward to working with miRecule to bring together our two groundbreaking technologies synergizing in a best-in-class therapy designed to suppress the underlying cause of FSHD,” said Pablo Sardi Global Head of Rare and Neurologic Diseases Research, Sanofi. “We are excited to embark on this collaboration with miRecule as we work together to bring hope to the FSHD community.”
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Read miRecule’s press release here.
More about miRecule’s technology
Dr. Saleh described miRecule’s research at the 2020 FSHD Connect conference. Watch: