Fulcrum Therapeutics Inc. is a clinical-stage biopharmaceutical company that was founded in 2016. The company's focus is on developing therapies for genetically defined diseases. They use a proprietary technology platform called Product Engine, which integrates genomic data, CRISPR gene editing, and small molecule therapeutics. Fulcrum aims to identify and develop small molecule drugs that modulate gene expression to treat diseases caused by specific gene mutations.
Fulcrum's lead candidate is losmapimod, which is being evaluated in clinical trials for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder that causes progressive muscle wasting. Losmapimod targets the p38alpha/delta MAPK pathway, which has been implicated in FSHD pathology. Fulcrum is also developing several other small molecule therapeutics for diseases such as sickle cell disease and beta-thalassemia.
Fulcrum Therapeutics went public on July 18, 2019, with an initial public offering (IPO) of 4.5 million shares priced at $16 per share. The IPO raised approximately $72 million for the company. The shares were listed on the Nasdaq Global Select Market under the ticker symbol "FULC."
Since going public, Fulcrum has continued to advance its clinical pipeline. In October 2020, the company announced positive interim data from its Phase 2b clinical trial of losmapimod in FSHD, which showed a statistically significant improvement in muscle function compared to placebo. Fulcrum plans to continue developing losmapimod and other product candidates for additional indications.
Fulcrum's mission is to improve the lives of patients with genetically defined diseases through the development of targeted therapies. The company's approach of integrating genomic data, gene editing, and small molecule therapeutics has the potential to address a wide range of diseases caused by specific gene mutations.