Sanofi Enters Strategic Collaboration with MiRecule for Antibody-RNA Conjugate to Treat FSHD
October 4, 2022
Sanofi entered a strategic collaboration and exclusive license agreement with MiRecule to develop and commercialize a best-in-class antibody-RNA conjugate for the treatment of facioscapulohumeral muscular dystrophy.
The collaboration marks miRecule’s first licensing transaction leveraging its proprietary DREAmiR discovery platform.
Facioscapulohumeral muscular dystrophy (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 with Sanofi’s proprietary muscle-targeted NANOBODY technology to join the two molecules into an antibody-RNA conjugate (ARC) utilizing MiRecule’s NAVIgGator conjugation and formulation chemistry that selectively targets and suppresses the underlying cause of FSHD in muscle tissue to enable FSHD patients to resume their normal course of aging free from the debilitating effects of this progressive disease.
Under the terms of their 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.
“Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life,” said Anthony Saleh, founder and CEO of MiRecule.
Saleh has a strong commitment to developing a therapy to treat FSHD as the disorder runs in his family. MiRecule started its program in 2019 with strong support from the patient and academic community, receiving funding from the FSHD Society, the Friends of FSH Research, and National Institute of Neurological Disorders and Stroke.
Author: Rare Daily Staff
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