Fulcrum Therapeutics (NASDAQ:FULC) announced positive results from the 12 mg dose cohort of Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD). The trial demonstrated a significant 8.6% absolute mean increase in fetal hemoglobin (HbF) after 12 weeks of treatment, with 7 of 16 patients achieving HbF levels above 20%.
Key findings include pan-cellular HbF induction with F-cells increasing from 34% to 67%, meaningful improvements in hemolysis markers, and a 0.9 g/dL increase in total hemoglobin. The drug showed encouraging trends in reducing vaso-occlusive crises (VOCs) and maintained a favorable safety profile with no treatment-related serious adverse events.
The study involved 135 adults across multiple cohorts, including 103 healthy subjects and 32 SCD patients, with all treatment-related adverse events being Grade 1.
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