Sanofi, miRecule Ink Muscular Dystrophy Research Deal Worth up to $430M

Sanofi_Pat Greenhouse/The Boston Globe via Getty

Pat Greenhouse/The Boston Globe via Getty Images

miRecule inked a research partnership and licensing deal with Sanofi worth up to $430 million that will advance a best-in-class antibody-RNA conjugate (ARC) to treat facioscapulohumeral muscular dystrophy (FSHD), the companies announced Tuesday.

Under the terms of the deal, miRecule grants Sanofi an exclusive global license to intellectual property rights to the FSHD therapeutic. They will then collaborate on the research up to choosing the lead candidate. At that point, Sanofi will take over all Investigational New Drug-enabling work and development and commercialization.

Sanofi will pay miRecule $30 million in upfront payments and near-term milestones. Additional biobucks for development, regulatory, and commercial milestones could approach $400 million. miRecule is also eligible for tiered royalties on global net product sales if approved.

This is the first major licensing deal for miRecule, said the company's founder and CEO Anthony Saleh. "Our goal since initiating this program has been to develop a treatment to allow FSHD patients to live a normal life."

Saleh added that the company's driver in developing its DREAmiR discovery platform was patient-focused drug development and "strong scientific decision-making."

miRecule launched its program in 2019, which included funding from the FSHD Society, the Friends of FSH Research and the National Institute of Neurological Disorders and Stroke grants. In September, SOLVE FSHD invested $1 million in the company to support its ARC for FSHD.

Eva Chin, Ph.D., executive director of SOLVE FSHD, stated the organization was excited to support miRecule and to "be part of this unique and cutting-edge approach to a disease-modifying therapy for FSHD." She also mentioned miRecule's goal is aligned with Chip Wilson's mission of "being a catalyst towards a cure for FSHD."

miRecule's DREAmiR platform leverages genomic and outcome data from thousands of patients to determine related genetic modifications that cause the disease. The researchers used this data to develop an RNA therapeutic to directly target and repair the genetic abnormality. ARCs attach the RNA drug to an antibody that targets delivery directly to diseased tissues.

In addition to its FSHD program, miRecule also has programs for head and neck cancer (MC-30) and a discovery program for diffuse large B-cell lymphoma.

The Need for Treatment

FSHD affects more than one million people globally, and no approved treatments exist. The second-most-common form of muscular dystrophy, patients with the genetic mutations that cause the disease have lifelong muscle function deterioration and progressively worsening disability. 

In children, it affects the muscles of the face, shoulders, upper arms and lower legs. Symptoms typically appear before 20 years of age. Most children with FSHD can still walk, but as the disease worsens, it causes weakness in other body parts.

FSHD is caused by mutations in the DUX4 gene, which is normally inactive in most cells but is activated in FSHD. The disease affects both boys and girls, and either parent can transmit it to their children. 

A parent with the FSHD gene has a 50% chance of passing it on to each of their children, according to Johns Hopkins Medicine. It can also occur spontaneously if there is a mutation in the parent's sperm or egg, but not in the rest of the parent's cells, or from sporadic mutations.

"We look forward to working with miRecule to bring together our two groundbreaking technologies synergizing in a best-in-class therapy designed to suppress the underlying cause of FSHD," stated Pablo Sardi, global head of rare and neurologic diseases research, Sanofi.

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