▎WuXi AppTec Content Team Editor
miRecule recently announced a strategic collaboration and exclusive license agreement with Sanofi to develop and commercialize an antibody RNA-conjugated drug (ARC) for the treatment of facioscapulohumeral muscular dystrophy disease (FSHD). This is the first licensing transaction for miRecule’s proprietary DREAmiR platform, with a total value of nearly $400 million.
FSHD is an inherited, progressively wasting disorder that commonly affects the muscles of the face, scapula, and upper arm. It is the second most common muscle wasting disorder and has no approved treatments. People with FSHD usually develop muscle weakness and atrophy in the eyes, mouth, shoulders, upper arms, and calves by age 20. As the disease progresses, FSHD may spread to the abdominal and buttock muscles, and about 20% of patients will need to use a wheelchair to walk by age 50. The FSH Society estimates that about 870,000 people worldwide have the disease. FSHD usually progresses slowly and is not fatal, but it can seriously affect the quality of life of patients, some of whom even require a ventilator to function normally. About 95% of FSHD patients are caused by abnormal expression of DUX4 gene due to chromosome 4 mutation.
Image source: 123RF
This collaboration will combine miRecule’s DUX4-targeting RNA therapywith Sanofi’s proprietary muscle-targeting NANOBODY Technology. The two molecules will be combined with a chemical formulation through miRecule’s NAVIgGator conjugation technology to become an ARC drug. This potential “best-in-class” drug may have the effect of modifying the disease process and is expected to restore the debilitating and abnormal aging process in FSHD patients due to disease progression.
Under this partnership agreement, miRecule will grant Sanofi exclusive worldwide rights to FSHD therapies. The two companies will collaborate on research in pilot project screening. After the selection of the pilot project is completed, Sanofi will be responsible for the preparation of the new drug clinical trial (IND) application, as well as the subsequent global development and commercialization activities. miRecule will receive an upfront payment from Sanofi and be eligible for future milestone payments estimated at nearly $400 million.
“We are excited to enter into our first large licensing deal. Our goal since the beginning of this project has been to develop a therapy that will enable people with FSHD to lead normal lives. DREAmiR discovers The platform focuses on the development of patient-centred therapeutics and makes decisions based on a solid scientific foundation. We believe that Sanofi shares our values of creating treatments that can change patients’ lives, and this collaboration will bring this An RNA therapy targeting DUX4 will be brought to patients.”
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