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X4 Pharmaceuticals, Inc. (Nasdaq: XFOR) is a late-stage clinical biopharmaceutical company committed to the development of novel therapeutics aimed at improving immune cell trafficking and reversing immune suppression. The company's core focus is on treating rare diseases and conditions resulting from immune system dysfunction, including cancer and genetic primary immunodeficiency diseases.
X4’s lead clinical candidate, mavorixafor, is a selective, small-molecule antagonist of the chemokine receptor CXCR4. Designed as an oral, once-daily therapy, mavorixafor targets immune cell mobilization and has shown promising results in clinical trials. It is currently under priority review by the FDA for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare primary immunodeficiency. The company’s recent NDA submission is supported by positive results from the global, pivotal 4WHIM Phase 3 clinical trial, which demonstrated mavorixafor's ability to reduce infection rates and improve immune cell counts without serious adverse events.
In addition to mavorixafor for WHIM syndrome, X4 is advancing its pipeline with ongoing investigations for mavorixafor in chronic neutropenic disorders and other immunodeficiencies. The company has also initiated preclinical development of other CXCR4 antagonists, including X4P-002 and X4P-003, to address different properties and mechanisms within the immune system.
Headquartered in Boston, Massachusetts, with a research center in Vienna, Austria, X4 leverages its deep understanding of CXCR4 biology to innovate and bring transformative therapies to patients with limited treatment options. The company's mission is driven by a commitment to improve the lives of those affected by rare and severe immune diseases through rigorous scientific research and strategic investment in its proprietary pipeline.
For more updates and detailed information, visit X4 Pharmaceuticals.
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