Press release
Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial
- Achieved up to 6.3% hemoglobin F (HbF) induction in initial subjects in first cohort; HbF was increasing at last measured timepoint - Supports
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"- Achieved up to 6.3% hemoglobin F (HbF) induction in initial subjects in first cohort; HbF was increasing at last measured timepoint - Supports proof-of-concept that FTX-6058 is a novel oral HbF inducer - FTX-6058 was generally well tolerated; no serious treatment emergent adverse events (TEAEs) reported - Data to be presented as a poster at European Hematology Association (EHA) Hybrid Congress; highlights to be presented at the Foundation for Sickle Cell Disease Research Symposium (FSCDR) on June 11, 2022 - Fulcrum to review results during an investor conference call, including guest KOL Dr. Julie Kanter, at 8:00 a.m. ET today CAMBRIDGE, Mass., June 10, 2022 (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 clinical proof-of-concept data from the ongoing Phase 1b trial of FTX-6058 for the treatment of sickle cell disease (SCD). Initial data from the first subjects to receive a 6 mg dose of FTX-6058, an oral HbF inducer, showed a rapid and robust induction of HbF, and subjects achieved increases of up to 6.3% over baseline. HbF levels were increasing at the last measured time point, and maximal levels of HbF may have not yet been achieved. Increases in HbF have been shown to reduce the frequency or severity of a broad range of SCD symptoms, including vaso-occlusive crises (VOC), anemia, pain, infection, stroke, and others. Based on a large body of genetic, clinical, and observational evidence showing the effects of higher levels of HbF in patients with SCD, the induction of HbF by 5-10% over baseline is associated with reduced disease burden and improved clinical outcomes. These initial data showing that FTX-6058 increases HbF levels by up to 6.3% support its potential to become a transformative therapy for people living with SCD. “People with sickle cell disease have a tremendous need for therapeutic options that can reduce morbidity and mortality and improve their quality of life,” said Julie Kanter, M.D., associate professor of Hematology and Oncology and director of the Adult Sickle Cell Program at the University of Alabama at Birmingham. “These initial data from FTX-6058 showing measurable increases in HbF induction are very encouraging. Achieving significant absolute inc...