Press release
Fulcrum Therapeutics Announces Positive Initial Results from the 20 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease at the 67th American Society of Hematology Annual Meeting
― Clear dose-response observed, with a robust and clinically meaningful fetal hemoglobin (HbF) induction at the Week 6 timepoint (n=12): mean absolute HbF in
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"― Clear dose-response observed, with a robust and clinically meaningful fetal hemoglobin (HbF) induction at the Week 6 timepoint (n=12): mean absolute HbF in the 20 mg cohort increased by 9.9% at Week 6 (vs. 5.6% at Week 6 in the 12 mg cohort); 7 of 12 patients in the 20 mg cohort (58%) achieved absolute HbF levels ≥20% ― ― >3.75-fold mean induction of HbF at Week 12 in the 20 mg cohort among patients who reached the Week 12 visit as of November 11, 2025 data cutoff (n=6), compared to a 2.4-fold induction at Week 12 in the 12 mg cohort ― ― Consistent early evidence of pan-cellular HbF induction, improvements in markers of hemolysis and anemia, and encouraging trends in vaso-occlusive crisis (VOC) reduction ― ― Pociredir continued to be generally well-tolerated, with no treatment-related serious adverse events (SAEs) ― ― Fulcrum to host investor event at 7:00 a.m. ET December 7, 2025 ― CAMBRIDGE, Mass., Dec. 06, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today reported positive initial results from the ongoing 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD). “We are highly encouraged by these initial data from the 20 mg cohort, which show clear evidence of a dose-response and build on the strong profile established with the 12 mg cohort,” said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer. “At just six weeks of treatment, we have observed robust and clinically meaningful increases in fetal hemoglobin with the majority of patients achieving absolute HbF levels ≥20%. These results reinforce pociredir’s potential as a best-in-class, once-daily oral HbF inducer. Importantly, pociredir continues to demonstrate a favorable safety profile with no treatment-related SAEs reported.” “These data reinforce that induction of fetal hemoglobin remains one of the most scientifically grounded strategies for treating SCD,” said Dr. Martin Steinberg, Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine at Boston University Chobanian & Avedisian School of Medicine. “The clear dose-response observed with the 20 mg cohort, including robust early increases in HbF and evidence suggesting pan-c...