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X4 Announces Publication of Mavorixafor Clinical Data in ‘Blood’

Current TAP Partner


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.

In the study, mavorixafor 400 mg once daily was established as a therapeutically effective dose, demonstrating dose-dependent increases in patients’ WBCs, ANCs, ALCs, and AMCs. Mavorixafor significantly reduced the annualized infection rate, with further reductions observed on extended treatment. Mavorixafor effected a 75% reduction in the number of cutaneous warts. To date, mavorixafor has been well tolerated.

The full Blood manuscript is available here:

The efficacy and safety of mavorixafor, dosed once daily, is currently being evaluated in a global Phase 3 clinical trial in patients with WHIM syndrome, and in two Phase 1b clinical trials – in combination with ibrutinib in patients with Waldenström’s macroglobulinemia (WM), and as monotherapy in patients with severe congenital neutropenia (SCN).

The Leukemia & Lymphoma Society (LLS) partnered with X4 in March 2019 to accelerate the development of mavorixafor for the treatment of WM and a Phase 1b clinical trial was initiated in December 2019. Mavorixafor was selected for LLS’s Therapy Acceleration Program® (TAP), a strategic initiative where LLS builds business alliances and collaborations with biotechnology companies and academic researchers to speed the development of new therapies for blood cancers.