Fulcrum Therapeutics, Inc.
NASDAQ Global MarketCapital Structure
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in mil. unless spec.Working Capital
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in mil. unless spec.Growth Rates
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in mil. unless spec.Quarterly Revenue
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in mil. unless spec.Quarterly Earnings Per Share
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in mil. unless spec.Quarterly Dividends Per Share
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in mil. unless spec.- CEO
- Alexander C. Sapir
- Full Time Employees
- 45
- Sector
- Healthcare
- Industry
- Biotechnology
- Address
- 26 Landsdowne Street Cambridge MA United States of America 02139
- IPO Date
- Jul 18, 2019
- CIK
- 0001680581
- Website
- fulcrumtx.com
- Similar Companies
- Business
- Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecule therapeutics that modulate gene expression to treat genetically defined rare diseases. The company’s product portfolio includes its lead candidate, pociredir, an oral fetal hemoglobin inducer in Phase 1b clinical trials for the treatment of sickle cell disease (SCD) and beta-thalassemia. Fulcrum also develops losmapimod, an oral small molecule for facioscapulohumeral muscular dystrophy (FSHD). The company is advancing additional early-stage programs targeting bone marrow failure syndromes including Diamond-Blackfan anemia, 5q deletion syndrome, Shwachman-Diamond syndrome, and Fanconi anemia. Fulcrum’s drug candidates aim to address the root genetic causes of these diseases through gene expression modulation. Founded in 2016 and headquartered in Cambridge, Massachusetts, Fulcrum operates primarily in the United States but has established a significant global partnership with Sanofi for the development and commercialization of losmapimod outside the U.S. This strategic alliance, formed in 2024, includes an upfront payment of $80 million to Fulcrum and potential milestone payments over $975 million, with shared global development costs and tiered royalties. Fulcrum retains U.S. commercialization rights while leveraging Sanofi’s global commercial infrastructure for markets beyond the U.S. Recent company developments include promising clinical results announced in 2025 from the Phase 1b PIONEER trial of pociredir, showing dose-dependent increases in fetal hemoglobin with good safety and tolerability profiles, positioning pociredir as a potential transformative once-daily oral therapy for SCD. Enrollment for a higher-dose cohort has been completed with data expected by year-end 2025. Fulcrum is also preparing to submit an investigational new drug application (IND) in late 2025 for its programs targeting bone marrow failure syndromes. The company ended the third quarter of 2025 with over $200 million in cash, ensuring operational stability through 2028. Fulcrum’s core business segments encompass rare genetic and neuromuscular diseases, with an emphasis on developing disease-modifying therapies through precision small molecule approaches. Its pipeline reflects a focus on rare diseases with high unmet medical need, leveraging proprietary technology to identify targets for gene expression modulation and provide meaningful clinical benefits. This comprehensive approach positions Fulcrum Therapeutics as a specialized biopharmaceutical innovator advancing treatments for genetically defined rare diseases with a robust clinical pipeline and strategic global partnerships driving its commercialization efforts.
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