Fulcrum Therapeutics

Historical price data

Fulcrum Therapeutics is pioneering a novel approach to drug discovery by targeting the mechanisms that control gene expression to treat genetically defined diseases. The company's lead program, losmapimod, is in Phase 3 development for facioscapulohumeral muscular dystrophy (FSHD), a debilitating muscle disorder with no approved therapies. Fulcrum's platform leverages functional genomics to identify master regulator proteins that can be targeted with small molecules, aiming to correct imbalanced gene expression and address the underlying cause of disease. The company's strategy focuses on high-unmet-need rare diseases where its technology can deliver transformative treatments.

Proprietary platform integrating functional genomics and computational biology to identify master regulator proteins that control disease-relevant gene expression, enabling development of small molecule therapies for genetically defined diseases.

10 drugs in pipeline, 2 in Phase 3

Total raised: $216M

Fulcrum faces limited direct competition in FSHD with losmapimod as the most advanced candidate, though companies like AavantiBio (Pfizer), Dyne Therapeutics, and Avidity Biosciences are pursuing alternative approaches. In sickle cell disease, the competitive landscape is crowded with gene therapies and other modalities, leading Fulcrum to pause FTX-6058 development. The company differentiates through its oral small molecule approach targeting gene regulation.