StemCell Therapy

Evotec and co-investors support Dutch Facio Therapies BV with 4.8m to push development of Dux4 inhibitors to treat facioscapulohumeral dystrophy (FSHD), one of the most common forms of muscular dystrophy.

With its investement alongside unnamed Australian and North American investors, Evotec enters the field of musculoskeletal diseases.

Currently there is no causative treatment for the muscle wasting disease that affects 700,000 people worldwide. In a patient cell-based screening, Evotec and Facio Therapies (Leiden) have already identified some FSHD candidate drugs, which block the overexpression of the DUX4 protein in skeletal muscle cells that causes muscle atrophy and oxidative stress, hallmarks of the disease. About 20% of people with FSHD end up in a wheelchair.

Facio announced it will use the proceeds to select pre-clinical lead candidates for further development. According to the NIH, the field of muscular dystrophies received US$80m in funding in 2017. One tenth of the amount has been channeled into FSHD research.

Some companies have already started clinical testing of candidate drugs for treating FSHD. aTyr Pharma, Inc. is in Phase I/IIa testing of the protein drug Resolaris in early onset FSHD. Acceleron Pharma, Inc. has recently started Phase II testing of ACE083, an inhibitor of proteins of the TGF-beta family.

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Evotec invests in Facio Therapies - European Biotechnology - European Biotechnology

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