Fulcrum Therapeutics

Fulcrum Therapeutics is an early clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for rare genetically defined diseases. The company’s proprietary product engine integrates patient-derived cell models, genetic screening, and AI algorithms to identify new drug targets, and treatments for a range of diseases including muscle disorders, central nervous system disorders, and blood disorders. 

Fulcrum has two drug candidates in its pipeline that are at clinical trials; Losmapimod, which is in Phase 3 clinical trials and expects to complete enrolment in 2H 2023; was also granted fast-track designation by the FDA for the treatment of patients with facioscapulohumeral muscular dystrophy— a rare, progressive disease with no approved treatment. The second drug, FTX-6058, is a small molecule developed to increase the expression of fetal hemoglobin for treating sickle cell disease. The drug candidate is in Phase 1b clinical trials. Fulcrum is also conducting preclinical studies for treating beta-thalassemia, a blood disorder that reduces the production of hemoglobin, and drug screenings for other disorders and plans to submit an investigational drug application in 2023.  

Key partnerships and customers

The company has two collaboration and license agreements with 1) MyoKardia, a wholly-owned subsidiary of Bristol-Myers Squibb Company, to identify biological targets for a genetic form of heart disease, and 2) Acceleron, a biopharmaceutical company, to identify biological targets within the pulmonary disease space. 

Funding and financials

It went public in July 2019 on the Nasdaq Global Market under the “FULC” ticker, raising USD 72 million. In January 2023, Fulcrum Therapeutics announced a USD 125 million underwritten public offering consisting of 9.6 million common shares priced at USD 13 per share. The new funds were used towards general corporate expenses, R&D expenses, administrative expenses, and potential acquisitions. 

For 2022, the company reported a net loss of USD 109.9 million (up 36% YoY). As a clinical-stage biotechnology company, it is yet to generate any revenue from drug sales. However, collaboration revenue was USD 6.3 million (down 67.2% YoY). In 1H 2023, net losses narrowed by 19.1% YoY to USD 48.6 million while collaboration revenue reduced by 73.7% YoY to USD 1.2 million. As of June 30, 2023, cash, cash equivalents, and marketable securities stood at USD 278.2 million, as compared to USD 202.9 million as of December 31, 2022. which it claims is sufficient to operate until mid-2025.