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Sanofi licenses a preclinical genetic disease drug from an unknown biotech spun out of the NIH

2022-10-04

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In a new deal Tuesday morning, Sanofi is jumping into the antibody-RNA conjugate (ARC) space and elevating a little-known private biotech into the spotlight. Sanofi is licensing a preclinical ARC candidate from Gaithersburg, MD-based miRecule that’s designed to treat a genetic muscle disease known as facioscapulohumeral muscular dystrophy, or FSHD. Sanofi is getting exclusive worldwide rights for the program, known as MC-DX4, while miRecule gets an upfront and near-term milestones of about $30 million. Additional biobucks could bring the deal’s total value north of $400 million. For Sanofi, the total dollar figure represents a relatively modest bet on a technology that’s similar to antibody-drug conjugates but not quite the same. Rather than attach an antibody to a drug, the FSHD program will be paired with the Big Pharma’s “NANOBODY” tech to create a single compound. miRecule, meanwhile, gets its day in the limelight with its first licensing deal. The company, run by NIH vet Anthony Saleh, was spun out of the agency into an incubator in 2017 run by BioHealth Innovation. Prior to Tuesday, miRecule had posted only two press releases on its website: one detailing a $5.7 million seed round in May 2021, and another revealing an FSHD foundation had donated $1 million last week to advance the program. MC-DX4 is what miRecule describes as an anti-DUX4 RNA therapy, and the company noted in 2021 that this took the form of an antisense oligonucleotide. miRecule estimates there are about 40,000 patients living with FSHD in the US.

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