Press release
Fulcrum Therapeutics Announces Clinical Hold on FTX-6058 in Sickle Cell Disease
CAMBRIDGE, Mass., Feb. 24, 2023 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., Feb. 24, 2023 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that on February 23, 2023, the U.S. Food and Drug Administration (FDA) verbally informed the company that it has issued a full clinical hold regarding the Investigational New Drug (IND) application for FTX-6058 for the potential treatment of sickle-cell disease. The Agency indicated that it would provide a formal Clinical Hold Letter to the company within 30 days. The clinical hold was initiated by the Agency due to previously reported preclinical data. Fulcrum will suspend dosing in the Phase 1b trial of FTX-6058 and intends to work diligently with the Agency to resolve the hold as soon as possible. “Patient safety remains paramount to me. I am encouraged by the Agency’s willingness to work with us to clarify the therapeutic potential of FTX-6058. Fulcrum intends to address questions related to modulation of the PRC2 complex and the preclinical data,” said Robert J. Gould, Ph.D., Fulcrum’s interim president and chief executive officer. “We continue to have confidence in the benefit-risk profile of FTX-6058 and remain committed to our goal of providing a differentiated therapeutic option for people living with sickle cell disease.” About FTX-6058FTX-6058 is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED) that was discovered using FulcrumSeek™, Fulcrum’s proprietary discovery engine. Inhibition of EED leads to potent downregulation of key fetal globin repressors, including BCL11A, thereby causing an increase in fetal hemoglobin (HbF). FTX-6058 is being developed for the treatment of sickle cell disease (SCD) and other hemoglobinopathies. FTX-6058 is currently being evaluated in a Phase 1b multi-center open-label trial in people with SCD (NCT05169580). Initial data demonstrated proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. To date, FTX-6058 has been generally well-tolerated in people with SCD with up to three months of exposure, with no drug-related serious adverse events reported. FTX-6058 has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Desig...