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Fulcrum Therapeutics
Fulcrum Therapeutics (FULC) investor relations material
Fulcrum Therapeutics 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary14 Jan, 2026
Strategic focus and pipeline
Focused on developing oral small molecules to modify gene expression for rare diseases, with a primary emphasis on sickle cell disease.
Lead asset, pociredir, is a fetal hemoglobin inducer with Fast Track and Orphan designation, and patents extending to 2040.
Early-stage pipeline includes discovery efforts in other benign hematological conditions and oncology.
Strong cash position of $352 million, providing runway into at least 2029.
Sickle cell disease landscape and unmet need
Sickle cell disease affects 7.7 million globally, with significant prevalence in the US and Europe, primarily impacting Black and brown populations.
Patients experience chronic pain, acute vaso-occlusive crises (VOCs), and a substantial reduction in life expectancy.
Recent withdrawals of Adakveo and Oxbrita, and limited uptake of gene therapies, highlight persistent unmet needs.
Oral, once-daily fetal hemoglobin inducers are positioned as a promising alternative due to ease of use and efficacy.
Clinical data and efficacy of pociredir
Phase Ib study tested 2mg, 6mg, 12mg, and 20mg doses in sickle cell patients, focusing on safety, HbF induction, hemolysis markers, anemia, and VOCs.
20mg cohort showed robust HbF increase from 7% to 16.9% in 6 weeks; over half of patients exceeded 20% HbF after 12 weeks.
All patients achieved at least a 6.5% HbF increase, correlating with meaningful reductions in VOCs.
Markers of hemolysis (LDH, bilirubin, reticulocytes, red cell distribution width) decreased, and total hemoglobin increased by 0.8g/dL in 6 weeks.
Observed 5 VOCs in 12 patients over 12 weeks, versus 16 expected, with 8 patients experiencing no VOCs.
Pociredir accelerated approval strategy
Pociredir's competitive advantage
Pociredir long-term efficacy and safety
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Next Fulcrum Therapeutics earnings date
Q4 202527 Feb, 2026
Fulcrum Therapeutics
Next Fulcrum Therapeutics earnings date
Q4 202527 Feb, 2026
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Frequently asked questions
Fulcrum Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on developing therapies to treat genetically defined rare diseases. The company leverages its proprietary product engine to identify and target genetic mechanisms underlying diseases. Fulcrum’s primary areas of focus include muscle and central nervous system disorders, with therapeutic candidates aimed at modulating gene expression to address the root causes of these conditions. The company is advancing its treatments through clinical trials, working to provide new options for patients with limited or no available therapies. The company is headquartered in Cambridge, Massachusetts, and its shares are listed on the NASDAQ.
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