Sanofi, miRecule To Develop Muscular Dystrophy Candidate In $400+ Million Partnership

RNA developer miRecule has entered into a collaboration with Sanofi to develop an antibody-RNA conjugate (ARC) therapy for facioscapulohumeral muscular dystrophy (FSHD), a rare muscle-weakening disorder. 

Estimated to be worth over $400 million, the partnership will combine miRecule’s RNA discovery “DREAmiR” platform and Sanofi’s muscle-directed NANOBODY technology. 

Under terms of the agreement, Sanofi will obtain a global license for the ARC candidate and collaborate with miRecule on research activities through lead candidate selection. Once the candidate is selected, Sanofi will take over for IND enabling studies and subsequent development and commercialization globally. miRecule will receive an undisclosed upfront payment and could receive up to $400 million in milestones, $30 million of which are “near-term.” The biotech will also receive tiered royalties on potential global net sales of the product. 

Related Article: Sarepta Submits BLA for First-Ever Duchenne Muscular Dystrophy Gene Therapy

Developing A Therapy For FSHD

The second most common type of muscular dystrophy, FSHD affects more than 1 million individuals worldwide. In FSHD, misexpression of the DUX4 gene leads to overproduction of the DUX4 protein that is toxic to muscle cells. As a result, patients with FSHD suffer from progressive muscle deterioration. 

While there are no approved treatments for FSHD, several clinical candidates are edging towards the FDA finish line, such as Fulcrum Therapeutics’ small molecule losmapimod, which is currently undergoing a Phase 3 study. Roche also recently presented a study design for a Phase 2 study of an experimental anti-myostatin antibody in patients with FSHD.

Sanofi and miRecule’s candidate will attempt to suppress the underlying cause of FSHD in the muscle by linking miRecule’s anti-DUX4 RNA payload to Sanofi’s muscle-targeted, multivalent NANOBODY molecules via miRecule’s NAVIgGator linker. 

The pursuit of an FSHD therapy hits close to home for miRecule’s founder and CEO, Anthony Saleh as the disorder runs in his family.

“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,” he said. 

“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 an ideal partnership to bring forward our anti-DUX4 RNA therapy.”

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Joy Lin

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