Sanofi has joined the pile-on of drug developers targeting one of the biggest unmet needs in muscular dystrophy, committing to $30 million in upfront and near-term payments for global rights to miRecule’s preclinical prospect.
The deal will give Sanofi control of an anti-DUX4 RNA therapy that is in lead development as a treatment for facioscapulohumeral muscular dystrophy (FSHD). Sanofi plans to combine the therapy with its muscle-targeted nanobody technology to create an antibody-RNA conjugate (ARC). ARCs are designed to enable the targeted delivery of RNA.
Multiple technologies underpin the plan. Sanofi is relying on miRecule for the anti-DUX4 RNA therapy and the conjugation and formulation chemistry to join it to the nanobody. The Big Pharma is supplying its nanobody, part of a class of therapeutic proteins based on single-domain antibodies that it began developing through its takeover of Ablynx.
By bringing together the technologies, Sanofi is trying to create a best-in-class therapy that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. If the program is a success, the therapy could enable patients to resume their normal course of aging free from the effects of FSHD.
DUX4 has attracted multiple drug developers: Fulcrum Therapeutics is running a phase 3 trial of an oral small molecule designed to reduce expression of the gene; Arrowhead Pharmaceuticals has an RNAi prospect in preclinical development; and Epic Bio is seeking to suppress DUX4 expression using its gene expression modulation system.
Sanofi is betting $30 million in a mix of an upfront fee and near-term milestones to join the race. As the therapy advances, miRecule is in line to receive development, regulatory and commercial milestone payments that top out just short of $400 million, plus tiered royalties on global net sales.
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