FDA Lifts Clinical Hold on Fulcrum Therapeutics’ FTX-6058 for Sickle Cell Disease

CAMBRIDGE, Mass., Aug. 22, 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 the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Investigational New Drug (IND) application for FTX-6058 for the potential treatment of sickle-cell disease (SCD).

“We are pleased with the FDA’s decision to lift the clinical hold and are eager to advance FTX-6058 through clinical development to address the significant unmet need in the sickle cell disease community,” said Alex C. Sapir, Fulcrum’s president and chief executive officer. “Based on the initial data from the Phase 1b trial, which showed increasing levels of HbF with each dose escalation, we believe in the potential of FTX-6058 to not only shift the current standard of care but importantly, offer these patients a differentiated oral option. We look forward to building on these results with plans to resume enrollment for patients with SCD.”

On February 23, 2023, the FDA placed the IND for FTX-6058 on clinical hold. In its communication, the Agency noted preclinical data previously submitted in April, October and December 2022, and non-clinical and clinical evidence of hematological malignancies observed with other inhibitors of polycomb repressive complex 2 (PRC2). In connection with the clinical hold, Fulcrum suspended dosing in the Phase 1b trial of FTX-6058 and worked diligently with the Agency to resolve the hold.

To learn more about the amended protocol and planned Phase 1b trial of FTX-6058, please see the program update presentation on the Investor Relations section of Fulcrum’s website at www.fulcrumtx.com or by clicking here. 

About FTX-6058
FTX-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. Initial data in SCD demonstrated proof-of-concept and achieved absolute levels of HbF increases associated with potential overall patient benefit. In clinical trials conducted prior to the clinical hold, FTX-6058 was generally well-tolerated in people with SCD with up to three months of exposure, with no serious treatment-related adverse events reported. FTX-6058 has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of SCD. To learn more about these studies please visit ClinicalTrials.gov.