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X4 Pharmaceuticals Announces Publication of Mavorixafor Phase 2 Clinical Data for Treatment of WHIM Syndrome in ‘Blood’ - the Official Journal of the American Society of Hematology
Manuscript further details positive clinical results and long-term tolerability of mavorixafor in ongoing Phase 2 study Company continues to advance
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Manuscript further details positive clinical results and long-term tolerability of mavorixafor in ongoing Phase 2 study\n Company continues to advance mavorixafor in Phase 3 pivotal trial in WHIM using optimal dose and key endpoints from Phase 2 trial CAMBRIDGE, Mass., Sept. 02, 2020 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced the publication of comprehensive safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy data of mavorixafor from an ongoing Phase 2, open-label, dose-escalation and extension study in adult patients with genetically confirmed WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome. The manuscript, published in Blood, the official journal of the American Society of Hematology, expands on previously presented data. Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals commented on the news: “The publication of our positive Phase 2 safety and efficacy data in a prestigious journal like Blood not only elevates the visibility of the severely underserved patient population suffering from WHIM syndrome, but also provides key third-party validation of our clinical strategy, including the selection of the dose, biomarkers, and clinical endpoints for our ongoing pivotal Phase 3 clinical trial. The published results continue to reinforce our belief that by down-regulating CXCR4/CXCL12 signaling, mavorixafor has the potential to be the first disease-modifying therapy for the more than 3,500 estimated diagnosed and undiagnosed WHIM patients in the U.S. We are eager to continue exploring mavorixafor’s compelling clinical profile and, if approved, bring this promising therapy to patients as expeditiously as possible.” About the Phase 2 Trial. The Phase 2 clinical trial enrolled eight patients with a pathogenic CXCR4 mutation, an absolute neutrophil count (ANC) ≤400/μL and/or absolute lymphocyte count (ALC) ≤650/μL. Patients were given doses escalating from 50 mg up to 400 mg mavorixafor orally once daily based on the threshold-adjusted area under the curve for ANC and ALC over 24-hours (AUCANC and AUCALC). Primary objectives evaluated safety, tolerability, and the dose required to achieve ...