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CAMBRIDGE, Mass. - Fulcrum Therapeutics, Inc. (NASDAQ:FULC) announced Tuesday that its experimental sickle cell disease treatment pociredir showed promising results in a Phase 1b clinical trial, with patients experiencing increased fetal hemoglobin levels and improvements in key disease markers. The announcement has contributed to the company’s strong recent performance, with InvestingPro data showing a 91% surge in share price over the past six months.
The company reported data from the 12 mg dose cohort of its PIONEER trial, which included 16 patients who completed a 12-week treatment period. Patients experienced a mean increase of 8.6% in fetal hemoglobin and improvements in markers of hemolysis, along with a 0.9 g/dL increase in total hemoglobin.
The drug was generally well-tolerated with no treatment-related serious adverse events, and all treatment-related adverse events were classified as Grade 1, according to the company’s press release statement.
"We believe that this data demonstrates that pociredir has the potential to increase fetal hemoglobin to levels that could ameliorate SCD symptomology," said Alex C. Sapir, Fulcrum’s President and Chief Executive Officer.
Pociredir is an oral small-molecule inhibitor designed to increase the expression of fetal hemoglobin for treating sickle cell disease. The drug has received FDA Fast Track designation and Orphan Drug Designation.
The company is continuing to evaluate a higher 20 mg dose cohort and plans to share data from this group by the end of 2025.
Fulcrum reported it had $214.1 million in cash, cash equivalents, and marketable securities as of June 30, 2025, compared to $241.0 million at the end of 2024. According to InvestingPro analysis, the company maintains a strong financial health score of 3.53 (rated as "GREAT"), with liquid assets significantly exceeding short-term obligations as evidenced by a current ratio of 28.71. The company posted a net loss of $17.3 million for the second quarter, compared to net income of $55.4 million in the same period last year when it received an $80 million payment from Sanofi.
The company stated its current cash position is expected to fund operations into 2028. Investors seeking deeper insights into Fulcrum’s financial outlook can access comprehensive analysis and additional ProTips through InvestingPro, which notes the company’s upcoming earnings report on August 6, 2025, as a potential catalyst for stock movement.
In other recent news, Fulcrum Therapeutics reported positive results from the 12 mg dose cohort of its Phase 1b PIONEER trial for the drug pociredir in sickle cell disease patients. The trial showed a mean increase in fetal hemoglobin levels, with several patients achieving levels associated with reduced vaso-occlusive crises. Fulcrum plans to present these topline results during a conference call and webcast, featuring experts from the Inova Fairfax and VCU Adult Sickle Cell Programs. Meanwhile, Cantor Fitzgerald maintained its Overweight rating on Fulcrum, citing potential benefits from the sickle cell treatment. Leerink Partners upgraded Fulcrum’s stock to Outperform, with a new price target of $12.00, based on promising trial outcomes and potential improvements with different dosing. The analysts highlighted the progress under CEO Alex Sapir, especially after overcoming previous challenges like an FDA clinical hold. Fulcrum’s continued focus on advancing its clinical trials has generated significant interest among investors. These developments reflect the company’s ongoing efforts to bring effective treatments to market.
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