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  • 12 February 2020
    X4 Pharmaceuticals Granted New Composition of Matter Patent for Late-Stage Clinical Candidate Mavorixafor

    X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced that the United States Patent and Trademark Office (USPTO) has issued United States Patent No. 10,548,889, which is expected to provide exclusivity of X4’s lead therapeutic candidate, mavorixafor (X4P-001), through 2038.

    “This new patent which covers critical compositions of matter enhances our already robust patent portfolio, further strengthening and extending the potential commercial horizon for mavorixafor,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We expect that this long patent runway will assist us in maximizing the full therapeutic potential of mavorixafor, which we are currently investigating across a number of rare disease indications.”

    About Mavorixafor

    X4 Pharmaceuticals’ lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in a Phase 3 clinical trial for the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor has demonstrated proof-of-concept in WHIM syndrome in a Phase 2 clinical trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile. Mavorixafor was recently granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with WHIM syndrome, and was granted orphan drug status by the FDA in 2018 and by the European Commission in 2019 for the treatment of WHIM syndrome. Mavorixafor is also being developed by X4 to treat Severe Congenital Neutropenia (SCN), Waldenström’s macroglobulinemia (WM), and clear cell renal cell carcinoma (ccRCC).