Fulcrum Therapeutics to Present New Clinical Data from the PIONEER trial of Pociredir in Sickle Cell Disease at the 67th American Society of Hematology Annual Meeting

Core Insights - Fulcrum Therapeutics is set to present new clinical data from the Phase 1b PIONEER trial of pociredir for sickle cell disease at the upcoming ASH Annual Meeting [1][2] - The company will also showcase preclinical data on its calmodulin pathway modulator program aimed at treating bone marrow failure syndromes [1] Presentation Details - The presentation titled "Pociredir, a novel oral once-daily fetal hemoglobin inducer: Results from the Phase 1b PIONEER study in adult participants with severe sickle cell disease and hydroxyurea intolerance or unresponsiveness" will occur on December 6, 2025, from 5:30 – 7:30 PM ET [2] - Another presentation titled "First-in-class small molecule calmodulin pathway modulators attenuate excess p53 activity and correct erythropoietic defects in models of diamond-blackfan anemia" will also take place on the same date and time [2] - Pociredir's poster has been selected for inclusion in the Poster Walk on Novel and Emerging Therapeutics in Erythrocyte and Iron Disorders on December 7, 2025 [2] Investor Event - Fulcrum will host a live and webcast investor event on December 7, 2025, at 7:00 AM ET in Orlando, featuring company leadership and medical experts [4] - The event will be accessible via the Investor Relations section of Fulcrum's website, with a recording available afterward [4] Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [5] - The lead clinical program, pociredir, aims to increase fetal hemoglobin expression for treating sickle cell disease [5][7] - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for sickle cell disease treatment [7] About Pociredir - Pociredir is an investigational oral small-molecule inhibitor of EED, leading to downregulation of fetal globin repressors and increased fetal hemoglobin levels [6][7] - Initial data from the PIONEER Phase 1b trial indicated proof-of-concept with well-tolerated results and no treatment-related serious adverse events reported [7] About Sickle Cell Disease - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [8] - Patients typically experience severe clinical consequences, including anemia, pain, infections, and reduced life expectancy [8]