Fulcrum Therapeutics(US:FULC) Globenewswire·2025-12-02 13:30Core Viewpoint - Fulcrum Therapeutics is set to present new clinical data from the Phase 1b PIONEER trial of pociredir for sickle cell disease (SCD) at an investor event on December 7, 2025, which will also be shared at the 67th American Society of Hematology Annual Meeting [1][3]. Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules aimed at improving the lives of patients with genetically defined rare diseases, particularly in areas with high unmet medical needs [6]. - The company's lead clinical program, pociredir, is designed to increase fetal hemoglobin expression for the treatment of sickle cell disease [6][8]. Clinical Trial Information - The upcoming event will provide initial clinical data from the 20 mg dose cohort and full data from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD [3]. - Pociredir has shown proof-of-concept in initial data from the PIONEER trial, achieving absolute levels of fetal hemoglobin increases that may benefit patients [8]. - The drug has been generally well-tolerated in patients with SCD during the trial, with no treatment-related serious adverse events reported over three months of exposure [8]. Expert Involvement - The event will feature insights from Dr. Sheinei Alan, Director of the Inova Adult Sickle Cell Program, and Dr. Martin Steinberg, a prominent hematologist specializing in sickle cell disease [2][4][5]. - Dr. Alan leads multiple clinical trials and has contributed to the development of national guidelines for sickle cell care, while Dr. Steinberg has extensive research experience and has published over 450 articles on sickle cell disease [4][5]. Disease Context - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and the formation of sickle-shaped red blood cells, which can cause various serious health complications [9].