A startup developing a drug for a genetic muscular dystrophy has raised a new round of financing that it will use to bring the drug into clinical testing.
Cambridge, Massachusetts-based Fulcrum Therapeutics said Wednesday that it had closed an $80 million Series B funding round, led by Foresite Capital. Fidelity Management and Research Company, 6 Dimensions Capital, Casdin Capital, Sanofi Ventures, Section 32, NS Investments and “entities affiliated” with Leerink Partners participated as well, along with undisclosed institutional investors.
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The company’s lead program is a small-molecule drug it will develop for fascioscapulohumeral muscular dystrophy, or FSHD, an inherited form of muscular dystrophy. According to the FSH Society – which is one of Fulcrum’s patient community partners – FSHD is the most prevalent of the nine primary types of muscular dystrophy affecting adults and children, and estimates of its incidence have ranged from 1-in-20,000 people to 1-in-8,333. About 870,000 people worldwide are estimated to be affected, but there may be significantly more undiagnosed cases. About 95 percent of FSHD cases are Type 1 disease, or FSHD1, while the remaining 5 percent are FSHD2. The main difference between the two types is genetic, with FSHD1 being linked to deletions of D4Z4 units on chromosome 4, while FSHD2 is linked to mutations of the gene SMCHD1 on chromosome 18. About 2 percent of FSHD cases are of unknown genetic causes. However, despite differing genetic origins, FSHD1 and FSHD2 appear to be the same disease and have similar symptoms.
Fulcrum said it has started a clinical trial readiness study in partnership with the FSHD Clinical Trial Research Network, whose aim is to standardize a set of tools and measurements for the company’s future clinical trials.
In addition to the FSHD program, Fulcrum’s pipeline page also lists Duchenne muscular dystrophy as being in the discovery stage under neuromuscular disorders for which it is developing therapies. It also has discovery-stage programs in fragile X syndrome, Parkinson’s disease and sickle cell disease.
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