Focuses on the discovery and development of novel therapies for rare diseases and oncology.
X4 Pharmaceuticals, Inc. is a leading late-stage clinical biopharmaceutical firm dedicated to advancing innovative therapies for the treatment of rare diseases. At the core of its research, development, and commercialization efforts is mavorixafor, a small molecule inhibitor targeting the chemokine receptor CXCR4. Currently, mavorixafor is undergoing Phase III clinical trials for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, alongside Phase Ib trials for chronic neutropenia and Waldenström macroglobulinemia.
In addition to mavorixafor, X4 Pharmaceuticals is actively developing other promising candidates. X4P-002, another CXCR4 antagonist, is being investigated for its potential in treating various brain cancers. Meanwhile, X4P-003, also targeting CXCR4, shows promise for addressing CXCR4 disorders and primary immunodeficiencies, demonstrating the company's broad therapeutic focus.
X4 Pharmaceuticals has forged significant partnerships to bolster its research and development efforts. Notably, a licensing agreement with Abbisko Therapeutics Co., Ltd. enables the development, manufacturing, and commercialization of mavorixafor in combination with checkpoint inhibitors and other agents for oncology indications, expanding the therapeutic potential of its flagship product.
Founded in 2010 and headquartered in Boston, Massachusetts, X4 Pharmaceuticals is driven by a commitment to innovation and patient-centric care. Through its robust pipeline and strategic collaborations, the company strives to address critical unmet medical needs in rare diseases, positioning itself at the forefront of therapeutic advancements in the biopharmaceutical industry.