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Fulcrum Therapeutics (FULC) investor relations material
Fulcrum Therapeutics H.C. Wainwright 27th Annual Global Investment Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.H.C. Wainwright 27th Annual Global Investment Conference summary9 Sep, 2025
Strategic focus and pipeline
Emphasis on small molecule technology to modify gene expression in rare diseases, offering scalability and broad applicability compared to cell/gene therapies.
Robust discovery pipeline includes a new candidate for bone marrow failure syndromes, with IND filing planned and more details expected in 2026 and beyond.
Strong financial position with $214 million in cash as of Q2, providing operational runway into 2028.
Sickle cell disease landscape and unmet needs
Sickle cell disease affects 7.7 million globally, with 100,000 in the U.S. and 55,000 in Europe/UK.
Recent therapies, including Adakveo and Oxbryta, were withdrawn due to lack of efficacy or safety concerns.
Cell/gene therapies have limited uptake due to high costs, risks, and operational challenges, leaving significant unmet needs.
Posiradir clinical data and impact
Posiradir, an oral PRC2 inhibitor, increases fetal hemoglobin, which correlates with reduced vaso-occlusive crises (VOCs).
Phase 1B data: 16 patients on 12mg dose saw mean fetal hemoglobin rise from 7.6% to 16.2% in 12 weeks.
Nearly half of patients achieved >20% fetal hemoglobin, and six reached >70% pancellularity.
Markers of hemolysis decreased, and total hemoglobin increased by 0.9 g/dL.
50% reduction in expected VOCs; half of patients had no VOCs during the study period.
Expected HBF increase for 20mg cohort?
End of Phase 1B meeting: Registrational study design?
Posiradir's market access strategy vs. gene therapies?
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Next Fulcrum Therapeutics earnings date
Q3 20257 Nov, 2025
Next Fulcrum Therapeutics earnings date
Q3 20257 Nov, 2025
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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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