Fulcrum Therapeutics announced positive initial results from the 20 mg dose cohort of the Phase 1b PIONEER trial of Pociredir in Sickle Cell Disease.
The trial showed a clear dose-response with robust and clinically meaningful fetal hemoglobin induction at the Week 6 timepoint.
Pociredir demonstrated improvements in markers of hemolysis, anemia, and vaso-occlusive crisis reduction, while maintaining good tolerability.
Robust HbF Increase
Mean absolute HbF in the 20 mg cohort increased by 9.9% at Week 6, with 58% of patients achieving HbF levels ≥20%.
Dose-Response Relationship
>3.75-fold mean induction of HbF at Week 12 in the 20 mg cohort compared to the 12 mg cohort, indicating a clear dose-response.
Pan-Cellular HbF Induction
Consistent early evidence of pan-cellular HbF induction observed, supporting the inhibition of sickle hemoglobin polymerization.
- The results support the potential of pociredir as a best-in-class oral HbF inducer for Sickle Cell Disease.
- Induction of fetal hemoglobin remains a scientifically grounded strategy for treating SCD, with promising outcomes observed in the trial.
These positive initial results from the PIONEER trial highlight the efficacy and safety of Pociredir in treating Sickle Cell Disease, signaling a significant step forward in SCD therapeutic strategies.