Encouraging Phase Ib PIONEER Trial Results
Pociredir demonstrated a dose-dependent and clinically meaningful increase in fetal hemoglobin, improvement in key biomarkers of hemolysis, and reduction in vaso-occlusive crises for sickle cell disease patients. Additionally, it was well tolerated with all treatment AEs being grade 1.
Completion of Enrollment in 20mg Cohort
The company completed enrollment in the 20mg dose cohort with 12 evaluable patients. Over-enrollment in both 12mg and 20mg cohorts signals strong enthusiasm from participating physicians.
Strong Financial Position
Fulcrum ended the third quarter of 2025 with $200.6 million in cash, cash equivalents, and marketable securities, providing sufficient runway to fund operations into 2028.
Potential IND Submission
Fulcrum plans to submit an IND for bone marrow failure syndromes by the end of 2025, expanding their therapeutic focus beyond sickle cell disease.