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X4 Pharmaceuticals (XFOR) investor relations material
X4 Pharmaceuticals Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary10 Nov, 2025
Management and strategic direction
New management team joined three months ago, raising nearly $240 million to fund operations through phase III and potential launch.
Company restructured, C-suite removed, and focus shifted from WHIM commercialization to execution of the 4WARD phase III trial.
Emphasis on US trial site expansion and patient recruitment through advocacy groups and databases.
Sole focus is now on successful completion and execution of the 4WARD trial.
Lead program and market opportunity
Lead compound mavorixafor targets CXCR4, enabling release of neutrophils from bone marrow, and is administered orally.
Approved for WHIM syndrome, but main commercial focus is chronic neutropenia, a broader indication with an estimated 15,000 US patients.
Chronic neutropenia currently managed with G-CSF (40-45% of patients) or infection management; oral mavorixafor offers a competitive advantage.
WHIM data informs trial design and powering assumptions for chronic neutropenia studies.
Clinical development and trial design
Phase II data showed mavorixafor increases ANC as monotherapy and allows G-CSF dose reduction while maintaining ANC.
Main toxicity is mild to moderate GI symptoms, typically resolving within weeks.
4WARD phase III trial is double-blind, placebo-controlled, enrolling 176 patients, with two primary endpoints: ANC increase and reduction in Annualized Infection Rate.
Study includes independent adjudication of infection events and targets a one-third reduction in infection rate over one year.
Enrollment expected to complete by end of Q3 2026, with top-line data in H2 2027 and potential launch in 2028.
What specific actions accelerate FORWARD trial enrollment
Detail mavorixafor's competitive edge vs G-CSF
Elaborate on the chronic neutropenia market opportunity
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Next X4 Pharmaceuticals earnings date
Q4 202525 Mar, 2026
Next X4 Pharmaceuticals earnings date
Q4 202525 Mar, 2026
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X4 Pharmaceuticals Inc. is a biopharmaceutical company focused on developing therapies for rare diseases, particularly those involving immune system dysfunctions. The company leverages its expertise in small-molecule therapeutics to address conditions associated with abnormal white blood cell activity. The company is headquartered in Boston, Massachusetts, and its shares are listed on the NASDAQ.
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