miRecule has announced a strategic collaboration and exclusive license agreement with Sanofi to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The collaboration marks miRecule’s first licensing transaction leveraging its proprietary DREAmiR platform.
FSHD is the second most common type of muscular dystrophy – affecting more than one million individuals worldwide with no approved treatments. Patients with causative genetic mutations experience lifelong deterioration of muscle function and progressive disability.
The collaboration will combine miRecule’s anti-DUX4 RNA therapy (discovered through its proprietary DREAmiR platform) with Sanofi’s proprietary muscle-targeted NANOBODY technology to join the two molecules into an ARC utilizing miRecule’s NAVIgGatorTM conjugation and formulation chemistry.
The potential resulting best-in-class therapy may become a disease-modifying treatment that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. This groundbreaking approach would enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive 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 and Sanofi will collaborate on research activities through lead candidate selection. Following candidate selection, Sanofi will assume sole responsibility for IND enabling studies and subsequent development and commercialization activities globally.
miRecule will receive an upfront payment and is eligible for near-term milestone payments, which combined could exceed $30 million. Additional development, regulatory, and commercial milestone payments could raise the total to nearly $400 million for advancement of the FSHD drug candidate, which will be exclusively developed and commercialized by Sanofi. miRecule is also eligible to receive tiered royalties on global net sales of the approved collaboration product.
Pablo Sardi, global head of Rare and Neurologic Diseases Research, Sanofi, said: “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. We hope that this will enable patients to live a life free from the debilitating symptoms of the disorder. We are excited to embark on this collaboration with miRecule as we work together to bring hope to the FSHD community.”
Anthony Saleh, founder and Chief Executive Officer, miRecule, said: “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. Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life. The philosophy driving our DREAmiR discovery platform focuses on patient centered drug development and strong scientific decision making. We believe Sanofi shares these core values on creating life changing therapies, making this is an ideal partnership to bring forward our anti-DUX4 RNA therapy.”
