Fulcrum Therapeutics reported positive results from the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease. The trial demonstrated robust and clinically meaningful absolute mean fetal hemoglobin (HbF) induction of 8.6% from baseline at 12 weeks of treatment, with 7 of 16 patients achieving absolute HbF levels greater than 20%.
Evidence of pan-cellular induction of HbF was observed, with F-cells increasing from a mean of 34% at baseline to 67% at 12 weeks of treatment. The data also showed meaningful improvements in key markers of hemolysis, including a 37% decrease in indirect bilirubin and a 28% decrease in lactate dehydrogenase, alongside a 0.9 g/dL mean increase in total hemoglobin.
Encouraging trends in vaso-occlusive crisis (VOC) reduction were noted, with 8 of 16 patients (50%) reporting no VOCs during the 12-week treatment period. Pociredir continued to be generally well-tolerated, with no treatment-related serious adverse events reported and all treatment-related adverse events limited to Grade 1. The Data Monitoring Committee recommended proceeding with the 20 mg dose cohort, which is now underway.
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