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Fulcrum Therapeutics Announces Positive 12-Week Results from the 20 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease
― Mean absolute fetal hemoglobin (HbF) increased by 12.2% (from 7.1% to 19.3%) at Week 12 (n=12), representing a rapid, robust, and clinically relevant
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"― Mean absolute fetal hemoglobin (HbF) increased by 12.2% (from 7.1% to 19.3%) at Week 12 (n=12), representing a rapid, robust, and clinically relevant response, with progression toward pan-cellular HbF induction as F-cells increased from 31% to 63% ― ― 7 of 12 patients (58%) achieved absolute HbF levels ≥20%; all patients achieved at least a 6.5% absolute increase in HbF ― ― Improvements in markers of hemolysis, improved erythropoiesis, and a >1 g/dL increase in total hemoglobin ― ― 7 of 12 patients (58%) reported zero VOCs during the treatment period ― ― Pociredir was generally well-tolerated, with no treatment-related serious adverse events (SAEs) ― ― Fulcrum plans to initiate a potential registration-enabling trial in the second half of 2026 ― ― Conference call and webcast scheduled for 8:00 a.m. ET today ― CAMBRIDGE, Mass., Feb. 24, 2026 (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 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD). “The 12-week data from the complete 20 mg cohort demonstrated robust and rapid HbF induction and progression toward pan-cellular distribution, accompanied by reductions in markers of hemolysis and associated improvements in anemia,” said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer. “Importantly, the HbF levels achieved are consistent with levels historically associated with reductions in sickling and hemolysis in sickle cell disease. These encouraging results in a severe patient population strengthen our conviction as we prepare for discussions with regulators regarding the design of the next study.” “The magnitude of HbF induction observed at 20 mg, together with the concomitant increase in F-cells and associated reductions in markers of hemolysis and improvements in anemia, is consistent with what we would expect from a therapy that may be capable of altering the underlying pathophysiology of sickle cell disease,” said Dr. Martin Steinberg, Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine at Boston University Chobanian & Avedisian School of Medicine. “Achieving HbF levels in this range rep...