Fulcrum Therapeutics Inc
NASDAQ:FULC
Fulcrum Therapeutics Inc
Fulcrum Therapeutics, Inc. is a clinical stage biopharmaceutical company. The company is headquartered in Cambridge, Massachusetts and currently employs 104 full-time employees. The company went IPO on 2019-07-18. The firm focuses on improving the lives of patients with genetically defined rare diseases. The firm has developed a product engine which identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The firm is also engaged in developing drugs for the treatment for the root cause of muscle disorders, central nervous system disorders and blood disorders. The firm uses its proprietary product engine to identify and validate drug targets and develop product candidates to address diseases caused by the mis-expression of certain genes. Its product candidate includes losmapimod, is a small molecule that is developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058, is a small molecule designed to bind embryonic ectoderm development (EED).
Fulcrum Therapeutics, Inc. is a clinical stage biopharmaceutical company. The company is headquartered in Cambridge, Massachusetts and currently employs 104 full-time employees. The company went IPO on 2019-07-18. The firm focuses on improving the lives of patients with genetically defined rare diseases. The firm has developed a product engine which identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The firm is also engaged in developing drugs for the treatment for the root cause of muscle disorders, central nervous system disorders and blood disorders. The firm uses its proprietary product engine to identify and validate drug targets and develop product candidates to address diseases caused by the mis-expression of certain genes. Its product candidate includes losmapimod, is a small molecule that is developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058, is a small molecule designed to bind embryonic ectoderm development (EED).
Pociredir Progress: Fulcrum reported significant progress with its lead candidate, pociredir, for sickle cell disease, including positive data from the 12 mg cohort and completion of enrollment in the 20 mg cohort.
Upcoming Data: Data from the 20 mg cohort will be presented at the ASH conference in early December, with about half of patients completing the full 84-day treatment by the data cutoff.
Safety & Tolerability: Pociredir continues to show a strong safety profile, with only grade 1 adverse events and high patient adherence (>90%).
Open-Label Extension: Fulcrum is initiating an open-label extension study to allow longer-term evaluation of pociredir after positive feedback from trial investigators.
Financial Runway: Fulcrum ended Q3 with $200.6 million in cash and expects this will fund operations into 2028, covering anticipated clinical programs.
Other Programs: IND submission for bone marrow failure syndromes is planned for Q4 2025; preclinical oncology work was also highlighted.
Regulatory Plans: An end of Phase I meeting with the FDA for pociredir is planned in Q1 2026 to discuss next development steps.
