Fulcrum Therapeutics, Inc. 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). 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 he he he 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. About FTX-6058 FTX-6058 is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED) that was discovered using FulcrumSeek(TM), Fulcrum's proprietary discovery engine. 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.