Fulcrum Therapeutics stock falls after sickle cell disease trial results

Published 29/07/2025, 12:20
© Reuters.

Investing.com -- Fulcrum Therapeutics Inc (NASDAQ:FULC) stock erased earlier gains and declined 17% on Tuesday after the company announced results from the 12 mg dose cohort of its Phase 1b PIONEER trial of pociredir in sickle cell disease.

The Cambridge, Massachusetts-based biopharmaceutical company reported that the trial showed an absolute mean fetal hemoglobin induction of 8.6% from baseline at 12 weeks of treatment, with 7 of 16 patients achieving absolute HbF levels greater than 20%. Despite these results, which the company characterized as "positive," investors appeared to view the data less favorably, sending shares lower.

The trial also demonstrated evidence of pan-cellular induction of HbF, with an increase in F-cells from a mean of 34% at baseline to 67% at 12 weeks. The company noted improvements in key markers of hemolysis coupled with a 0.9 g/dL mean increase in total hemoglobin, along with encouraging trends in vaso-occlusive crisis reduction compared to baseline.

"We are highly encouraged by these data from the 12 mg cohort of the PIONEER trial and believe they represent an important milestone in our mission to transform the treatment of sickle cell disease," said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer.

The company reported that pociredir continued to be generally well-tolerated with no treatment-related serious adverse events. All treatment-related adverse events were Grade 1, according to the press release.

Through the completion of the 12 mg dose cohort, pociredir has been dosed in 135 adults, including 76 subjects in multiple dose cohorts up to 12 weeks. Fulcrum indicated that additional observations after completion of the 4-week follow-up period for the 12 mg dose cohort will be shared at a future medical meeting.

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