Fulcrum Therapeutics, Inc. (FULC) announced Tuesday that the U.S. Food and Drug Administration has lifted the clinical hold on the Investigational New Drug or IND application for FTX-6058 for the potential treatment of sickle-cell disease or SCD.
In pre-market activity on Nasdaq, Fulcrum shares were gaining around 48.5 percent to trade at $5.82.
FTX-6058 is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development or EED that was discovered using FulcrumSeek, Fulcrum’s proprietary discovery engine. FTX-6058 has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD, a genetic disorder of the red blood cells caused by a mutation in the HBB gene.
The clinical-stage biopharmaceutical company focused on genetically defined rare diseases noted that the FDA on February 23 had 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 or PRC2.
In connection with the clinical hold, Fulcrum had suspended dosing in the Phase 1b trial of FTX-6058.
The company now said it plans to resume enrollment for patients with SCD.
Alex Sapir, Fulcrum’s president and chief executive officer, said, “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. 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.
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