Fulcrum Therapeutics stock price plummets despite trial results announced

Investing.com – Fulcrum Therapeutics Inc. (NASDAQ:FULC) stock price dropped 20% on Tuesday after the company announced the 12-week results of the 1b PIONEER trial of pociredir in treating sickle cell disease at the 20 mg dose group.

The trial showed that, among 12 evaluable patients, the average absolute fetal hemoglobin increased by 12.2% at week 12, rising from 7.1% to 19.3%. Of the 12 patients, 7 had absolute HbF levels of at least 20%, and all patients experienced an absolute HbF increase of at least 6.5%.

The company reported improvements in hemolysis and erythropoiesis markers, with indirect bilirubin decreasing by 40%, lactate dehydrogenase decreasing by 34%, and average hemoglobin increasing from a baseline of 7.3 g/dL to 8.4 g/dL, a rise of 1.1 g/dL. The proportion of red blood cells containing HbF increased from an average of 31% at baseline to 63% at week 12.

During the 12-week treatment period, 6 cases of vaso-occlusive crises (VOC) were reported, while 7 out of 12 patients reported zero VOC. Based on medical records from 6-12 months prior to enrollment, approximately 16 VOC episodes were expected during treatment.

Pociredir was generally well tolerated, with no treatment-related serious adverse events reported as of the data cutoff date of December 23, 2025, and no discontinuations due to treatment-related adverse events. As of that date, pociredir had been administered to 148 adults, including 89 subjects who received up to 12 weeks of multiple-dose treatment.

Fulcrum plans to provide more details on the next trial design after receiving the FDA’s end-of-phase meeting minutes in the second quarter of 2026. The company intends to initiate a potential registration trial in the second half of 2026.

This article was translated with AI assistance. For more information, please see our Terms of Use.