Fulcrum Pill Lifts Fetal Hemoglobin 8.6 Points in Early Sickle Cell Test

Fulcrum Therapeutics said its experimental pill pociredir produced meaningful increases in fetal hemoglobin in adults with sickle cell disease, according to interim data from a 16-patient, 12-week cohort of the Phase 1b PIONEER study released Tuesday. Patients who received a 12 mg daily dose showed an average 8.6-percentage-point rise in fetal hemoglobin to 16.2%, with seven participants surpassing the 20% threshold that researchers associate with sharply lower pain-crisis rates. Total hemoglobin rose 0.9 g/dL, and indicators of red-blood-cell damage, including indirect bilirubin and lactate dehydrogenase, fell 37% and 28%, respectively. Pociredir was generally well tolerated: no treatment-related serious adverse events occurred and all drug-related side effects were Grade 1. Fulcrum said the results support further development of the once-daily oral therapy, which has FDA Fast Track and Orphan Drug status. Hematology specialists not involved in the study described the findings as encouraging but incremental, cautioning that the magnitude of benefit remains modest and that longer-term, placebo-controlled trials will be needed to confirm clinical impact on pain crises and overall outcomes.