Fulcrum Therapeutics’ pociredir shows promise in sickle cell disease trial. The company’s clinical-stage biopharmaceutical, valued at $482 million, has seen its stock drop 19% despite positive initial results from the Phase 1b PIONEER trial.
The trial found that patients in the 20 mg cohort experienced a mean absolute fetal hemoglobin increase of 9.9% at Week 6. Seven out of 12 patients achieved absolute HbF levels of 20% or higher, exceeding previous results.
Improvements were also seen in markers of hemolysis and anemia, with patients showing a decrease in indirect bilirubin, lactate dehydrogenase, and reticulocyte counts.
The drug demonstrated a favorable safety profile with no treatment-related serious adverse events reported across 148 adults who have received the drug. Fulcrum plans to report updated results from the full 12-week treatment period in Q1 2026.
Fulcrum’s stock has been boosted by rival Agios Pharmaceuticals’ disappointing sickle cell trial results, and H.C. Wainwright raised its price target for Fulcrum from $12 to $18, maintaining a Buy rating.
Source: https://www.investing.com/news/company-news/fulcrum-reports-positive-results-for-sickle-cell-drug-pociredir-93CH-4394706