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X4 Pharmaceuticals Announces Strategic Prioritization of Programs and Resources Extending Cash Runway Through Expected Key Clinical and Regulatory Milestones
Company now focused on advancing lead clinical candidate mavorixafor in chronic neutropenic disorders, including WHIM syndrome Important mavorixafor clinical
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Company now focused on advancing lead clinical candidate mavorixafor in chronic neutropenic disorders, including WHIM syndrome Important mavorixafor clinical milestones anticipated in 2H 2022, including readout from pivotal Phase 3 4WHIM trial and Phase 1b results across a range of chronic neutropenic disorders Recent capital raise, debt restructuring, revised company focus, and projected cost-reductions expected to extend cash runway into 3Q 2023 and accelerate mavorixafor WHIM NDA filing to early 2H 2023 BOSTON, July 20, 2022 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small-molecule therapeutics to benefit people with diseases of the immune system, today announced an update to its strategic priorities that includes streamlining resources to focus on advancing its lead clinical candidate, mavorixafor, in WHIM syndrome and other chronic neutropenic disorders, while progressing its clinical and pre-clinical oncology programs only via potential partnership(s). Strategic Actions Summary: Sharpening corporate focus and prioritizing resources towards immunodeficiency-related clinical programs: WHIM Syndrome: Data readout from the pivotal Phase 3 4WHIM trial of once-daily, oral mavorixafor in individuals 12 and older with WHIM (Warts, Hypogammaglobulinemia, Infections, & Myelokathexis) syndrome expected in the fourth quarter of 2022; U.S. New Drug Application (NDA) submission now anticipated early in the second half of 2023.Chronic Neutropenic (CN) Disorders: Results from the Phase 1b study of mavorixafor in individuals with CN disorders anticipated in the third quarter of 2022; data expected to confirm broad potential of mavorixafor to treat CN disorders beyond WHIM, to support discussions with the U.S. Food & Drug Administration (FDA) on next steps, and to inform the regulatory path forward.X4P-003: The candidate, a novel, small-molecule CXCR4 antagonist, has progressed to pre-clinical development and patent applications have been filed; further advancement dependent on the potential first approval of mavorixafor and lifecycle management of the company’s product portfolio. Progressing oncology efforts only via potential partnership(s) for the company’s clinical and pre-clinical programs: Waldenström’s macroglobulinemia (WM): Additional Phase 1b clini...