Press release
Fulcrum Therapeutics to Present Initial Data from Phase 1b Trial of FTX-6058 in Adults Living with Sickle Cell Disease at the European Hematology Association (EHA) Hybrid Congress in Vienna, Austria
- FTX-6058 is the only oral hemoglobin F (HbF) inducer in clinical development -Data will include initial HbF changes, safety, tolerability, pharmacokinetics,
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"- FTX-6058 is the only oral hemoglobin F (HbF) inducer in clinical development -Data will include initial HbF changes, safety, tolerability, pharmacokinetics, and other pharmacodynamic measures from the ongoing 6 mg dose cohort of FTX-6058 in adults with sickle cell disease (SCD) - Investor webcast to be hosted on June 10th at 8 a.m. EDT CAMBRIDGE, Mass., May 12, 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 that it will be presenting two posters at the European Hematology Association (EHA) Hybrid Congress which will take place June 9-12, 2022, in Vienna, Austria. A third abstract has also been accepted for publication. The accepted abstracts are now available online through the EHA website at www.ehaweb.org. \"HbF is the only mechanism that has shown the ability to broadly improve clinical outcomes for patients with SCD— including anemia, vaso-occlusive crises, pain, fatigue, and acute chest syndrome,\" said Judy Dunn, Ph.D., president of research and development at Fulcrum. “This Phase 1b study was designed to provide proof-of-concept that FTX-6058 produces increases in HbF and could potentially be the first oral HbF inducer to address critical unmet needs in this population.\" FTX-6058 is an investigational oral small-molecule Embryonic Ectoderm Development Inhibitor (EEDI) that has demonstrated robust induction of fetal hemoglobin (HbF) in human cells and animal models of sickle cell disease (SCD). Clinically, in a multiple ascending dose (MAD) Phase 1 study in healthy volunteers, FTX-6058 induced HBG (hemoglobin subunit γ) mRNA in a dose-dependent manner. Translation of HBG mRNA is responsible for the production of HbF protein. In addition to the poster presentation, Fulcrum will host a virtual investor event on June 10th at 8 a.m. EDT to review these initial FTX-6058 data and provide a program update. Poster Presentation InformationDate: June 10, 2022Time: 4:30 p.m. CEST/10:30 a.m. EDT Title: Interim results of safety, tolerability, pharmacokinetics, and pharmacodynamics from an ongoingopen-label study investigating FTX-6058 in adults living with sickle cell diseasePresenter: Julie Kanter, M.D., Director of the UAB Adult Sickle Cell Clinic and Associate Professo...