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  • 30 July 2019
    X4 Pharmaceuticals Receives Orphan Drug Designation from European Commission for Mavorixafor for Treatment of WHIM Syndrome

    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 European Commission (EC), based on a favorable recommendation from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation (ODD) to mavorixafor (X4P-001) for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. In October 2018, mavorixafor was granted ODD for the treatment of WHIM syndrome by the U.S. Food and Drug Administration. The exact prevalence of WHIM syndrome is unknown, however, in the United Statesalone there are between 15,000 and 100,000 patients classified as having a primary immunodeficiency disease of unknown origin – of which WHIM syndrome is one.

    Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4 targeting WHIM syndrome. Proof of concept in WHIM patients has been observed with 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 has been well tolerated in prior early phase clinical studies. X4 Pharmaceuticalsrecently initiated 4WHIM, a pivotal Phase 3 global clinical trial of mavorixafor for the treatment of WHIM syndrome.

    “We are very pleased that the European Commission has granted orphan drug designation for mavorixafor, which we believe has the potential to become a transformative treatment for patients living with WHIM syndrome,” said Paula Ragan, PhD, President and Chief Executive Officer of X4 Pharmaceuticals, “We look forward to advancing mavorixafor through our recently commenced Phase 3 pivotal trial and bringing this drug to patients.”

    ODD in the European Union provides regulatory and financial incentives for companies to develop medicinal therapies to treat serious disorders affecting no more than five in 10,000 people in the European Union. Incentives include eligibility for protocol assistance, access to the European Union’s centralized marketing authorization procedure, and marketing exclusivity in the European Union for a period of ten years.

    About WHIM Syndrome

    WHIM syndrome is a rare, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene and is named for the characteristic clinical symptoms of the syndrome – Warts, Hypogammaglobulinemia, Infections, and Myelokathexis.1 Patients with WHIM may experience significant morbidity beginning in early childhood and continuing throughout life with an increased likelihood of various recurrent, potentially life-threatening infections, and may also be susceptible to malignancies such as HPV-related cervical cancer and lymphomas.1,2,3 The overall cancer risk in patients with WHIM is estimated to be 30 percent by 40 years of age.4 There are no approved therapies for WHIM, and current standards of care are limited to treatment of acute infections with antibiotics or prevention of infections mainly through immunoglobulin substitution or G-CSF.5 The exact prevalence of WHIM is unknown, however, in the U.S. alone there are between 15,000 and 100,000 patients classified as having a primary immunodeficiency disease of unknown origin – of which WHIM is one.6,7,8

    About Mavorixafor

    X4 Pharmaceutical’s lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in Phase 3 development 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 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 designated orphan drug status by the U.S. Food and Drug Administration in 2018 and by the European Commission in 2019 for the treatment of WHIM syndrome, and is also in development for Severe Congenital Neutropenia (SCN), Waldenström’s macroglobulinemia (WM), and clear cell renal cell carcinoma (ccRCC).