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CAMBRIDGE, Mass. - Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a $419 million market cap biotechnology company, reported positive results from the 12 mg dose cohort of its Phase 1b PIONEER trial evaluating pociredir in sickle cell disease (SCD) patients, according to a press release statement. According to InvestingPro data, the company’s stock has shown strong momentum with a 91% return over the past six months.
The trial showed an absolute mean fetal hemoglobin (HbF) induction of 8.6% from baseline after 12 weeks of treatment, with 7 of 16 patients achieving absolute HbF levels exceeding 20%. The company noted that HbF levels of 20% are associated with approximately 90% of patients experiencing zero vaso-occlusive crises (VOCs) per year. InvestingPro analysis shows the company maintains a strong financial health score of 3.53, labeled as "GREAT," with notably high cash flow and growth metrics.
Patients treated with pociredir also demonstrated improvements in key markers of hemolysis, including a 37% mean decrease in indirect bilirubin and a 28% mean decrease in lactate dehydrogenase. Mean hemoglobin concentration increased by 0.9 g/dL from baseline.
The proportion of F-cells, or red blood cells containing HbF, increased from a mean of 34% at baseline to 67% at 12 weeks of treatment, which the company described as consistent with pan-cellular HbF induction.
The trial also showed a trend toward reduced VOC rates compared to patients’ VOC frequency over the 6-12 months prior to enrollment, with 8 of 16 patients reporting no VOCs during the 12-week treatment period.
Pociredir was generally well-tolerated with no treatment-related serious adverse events reported. All treatment-related adverse events were Grade 1.
Wally Smith, M.D., Director of the VCU Adult Sickle Cell Program, called the results "both impressive and encouraging" in the press release.
Pociredir has received FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD. The company plans to share additional observations after completion of the 4-week follow-up period at a future medical meeting. With analyst price targets ranging from $4 to $12 and the next earnings report due on August 6, investors seeking deeper insights can access comprehensive analysis through InvestingPro, which offers exclusive ProTips and detailed financial metrics for over 1,400 US stocks.
In other recent news, Fulcrum Therapeutics reported its Q1 2025 earnings, revealing a net loss of $17.7 million, which marks an improvement from the $26.9 million loss in Q1 2024. The company is preparing to present topline results from the 12 mg dose cohort of its Phase 1b PIONEER trial of pociredir for sickle cell disease. This presentation is scheduled for July 29, featuring insights from Fulcrum management and notable experts in the field. Analysts at Cantor Fitzgerald have maintained an Overweight rating on Fulcrum, citing potential upside for its sickle cell treatment. Meanwhile, Leerink Partners has upgraded Fulcrum’s stock to Outperform, setting a new price target of $12.00, based on promising results observed in the sickle cell trial. The 12 mg cohort data is anticipated to be available in early Q3 2025, with additional results from a 20 mg cohort expected later in the year. Despite a slight miss on EPS forecasts, Fulcrum’s strategic advancements in its lead program for sickle cell disease were highlighted as a positive development.
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