newsfilter
← Latest News
GlobeNewswire·

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

FULC
  • Mean absolute fetal hemoglobin (HbF) increased by 12.2% from 7.1% to 19.3% at Week 12 in the 20 mg cohort of the Phase 1b PIONEER trial (n=12).
  • F-cells increased from 31% to 63%, indicating progression toward pan-cellular HbF induction.
  • 58% of patients (7 of 12) achieved absolute HbF levels ≥20%, while all patients had at least a 6.5% absolute increase.
  • Markers of hemolysis improved, including a 40% decrease in indirect bilirubin, 34% decrease in lactate dehydrogenase, 26% decrease in RBC distribution width, and 42% decrease in reticulocyte counts.
  • Total hemoglobin increased by 1.1 g/dL, rising from 7.3 g/dL to 8.4 g/dL.
  • 58% of patients (7 of 12) reported zero vaso-occlusive crises (VOCs) during the 12-week treatment period.
  • Pociredir was generally well-tolerated with no treatment-related serious adverse events (SAEs) or discontinuations due to treatment-related adverse events.
  • Fulcrum Therapeutics plans to initiate a potential registration-enabling trial in the second half of 2026 following FDA feedback.
  • The company intends to engage with the European Medicines Agency (EMA) in mid-2026 for protocol assistance.
  • An open-label extension trial is being activated to evaluate the long-term safety and durability of response to pociredir.
  • A conference call to discuss these results was scheduled for 8:00 a.m. ET on February 24, 2026.