Fetal Hemoglobin (HbF) Induction
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Fulcrum Therapeutics (NasdaqGM:FULC) Earnings Call Presentation
2025-12-07 12:00
Pociredir 20mg Cohort Data Highlights - The 20 mg cohort data raises the bar on Pociredir's best-in-class potential for treating sickle cell disease[14, 17] - At Week 6, the 20 mg cohort (n=12) showed a 99% mean absolute increase in HbF[17] - 58% of patients (7/12) in the 20 mg cohort achieved ≥20% HbF at their latest study visit[17] - In the 20 mg cohort, the six patients who completed the 12-week treatment period achieved >375-fold induction of HbF[17] Clinical Improvements - The study observed a 28% reduction in Lactate Dehydrogenase (LDH) and a 37% reduction in indirect bilirubin, indicating consistent reductions in markers of hemolysis with the 20 mg dose[71, 72] - There was a 31% reduction in Mean Absolute Reticulocyte Count (ARC) and a 22% reduction in Mean Red Cell Distribution Width (RDW-CV) with the 20 mg dose, indicating consistent improvements in red blood cell morphology and erythropoiesis[75] - Mean hemoglobin increased from 73 g/dL to 81 g/dL in the 20 mg cohort[81] - 67% of patients (8/12) reported no vaso-occlusive crises (VOCs) during the 12-week treatment period[86] Safety and Tolerability - Pociredir at 20 mg was generally well-tolerated, with 23% of patients (3/13) reporting treatment-related adverse events[90] - There were no serious treatment-related adverse events in the 20 mg cohort[90]