Ann: Central Nervous System Drug Program Added to PYC Pipeline, page-34

Sanofi has recently struck a collaboration and licensing deal for a preclinical, CNS antisense drug.

The strategic collaboration and exclusive global license agreement was made with miRecule Inc., a small, privately-owned, Maryland-based biotech. The aim is to develop and commercialize a best-in-class antibody-RNA conjugate (ARC) for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is the second most common type of muscular dystrophy, affecting more than one million individuals worldwide. There are no approved treatments.

miRecule’s preclinical antisense therapy for FSHD (MC-DX4), targets and suppresses expression of the toxic DUX4 protein in muscle tissue which is the cause of lifelong deterioration of muscle function and progressive disability in 95% of FSHD patients. The collaboration will combine MC-DX4 with Sanofi’s proprietary muscle-targeted NANOBODY technology to join the two molecules into an ARC, utilizing miRecule’s conjugation and formulation chemistry.

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 US$30 million. Additional development, regulatory, and commercial milestone payments could raise the total to nearly US$400 million. miRecule is also eligible to receive tiered royalties on global net sales of the approved collaboration product.

https://mirecule.com/2022/10/04/mir...acioscapulohumeral-muscular-dystrophy-fshd-2/

https://www.globenewswire.com/news-...rapies-for-Cancer-and-Muscular-Dystrophy.html

https://endpts.com/sanofi-licenses-...-from-an-unknown-biotech-spun-out-of-the-nih/