一次情報の記録
決定級トップライン結果
FULC: Pociredir 20 mgコホート、PIONEER SCD試験のWeek 6でHbF 9.9%上昇を示す
Fulcrumは鎌状赤血球症を対象としたpociredirの20 mgコホートにおける初期Phase 1b PIONEER結果を報告し、12 mgコホートでの5.6%に対し、Week 6で平均絶対HbFが9.9%増加する明確な用量反応を示した。58%の患者がWeek 6でHbF ≥20%に到達し、Week 12で3.75倍超のHbF誘導が観察された。Pociredirは治療関連の重篤な有害事象なく、概ね忍容性が良好であった。
主要事実
出典の抜粋
EX-99.1 2 fulc-ex99_1.htm EX-99.1 EX-99.1 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 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 ― ―