Press release
Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for First Quarter 2025
― Enrollment complete in the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); initiated the 20 mg dose cohort
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"― Enrollment complete in the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); initiated the 20 mg dose cohort ― ― On track to provide clinical data from the 12 mg dose cohort in early Q3 2025 and the 20 mg dose cohort by the end of 2025 ― ― Ended Q1 2025 with $226.6 million in cash, cash equivalents, and marketable securities; cash runway into at least 2027 ― ― Conference call and webcast scheduled for 8:00 a.m. ET today ― CAMBRIDGE, Mass., May 01, 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 financial results for the first quarter of 2025 and provided a business update. “We are pleased with the continued momentum of our lead clinical program, pociredir, in development for the treatment of sickle cell disease, including the recent initiation of the 20 mg dose cohort” said Alex C. Sapir, Fulcrum’s president and chief executive officer. “The level of engagement that we’ve observed in the PIONEER trial is highly encouraging, with 16 patients enrolled in the 12 mg dose cohort and enrollment underway in the 20 mg dose cohort. We look forward to sharing data from the 12 mg dose cohort in early Q3 2025, and from the 20 mg dose cohort by the end of 2025. We believe that pociredir has the potential to increase fetal hemoglobin to levels that could ameliorate SCD symptomology and transform the standard of care with a once daily oral treatment option.” Recent Business Highlights Patient enrollment continues to progress in the Phase 1b PIONEER trial evaluating pociredir in patients with SCD. Enrollment is complete in the 12 mg dose cohort (n=16) with greater than 90% rates of adherence to study drug and no patient discontinuations to date. Based on the recommendation of the data monitoring committee, Fulcrum has initiated the 20 mg dose cohort and plans to share data from the 12 mg dose cohort in early Q3 2025 and from the 20 mg dose cohort by the end of 2025.Two abstracts have been accepted for presentation at the 2025 European Hematology Association (EHA) Congress in Milan, Italy, taking place on June 12-15, 2025. The abstracts, which will be published online on May 15, 2025, highlight precl...