X4 Pharmaceuticals (Cambridge, MA) is planning to start a Phase 3 clinical study within the next few months, entitled “A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial of mavorixafor (X4P-001) in Patients with WHIM Syndrome with Open-Label Extension”
WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) is a rare autosomal dominant primary immunodeficiency disease caused by gain-of-function mutations in the chemokine receptor CXCR4. CXCR4 mutations induce increased leukocyte homing and retention in the bone marrow resulting in severe chronic neutropenia and lymphopenia.
X4 Pharmaceuticals is planning to sponsor a 52-week global Phase 3 study to evaluate the safety and efficacy of mavorixafor, a once-daily oral CXCR4 antagonist, in genetically confirmed WHIM patients who are aged 12 years or older. Mavorixafor is a first-in-class, oral, small molecule antagonist of the CXCR4 chemokine receptor that has demonstrated a favorable safety profile and dose-dependent increases in ANC and ALC in a Phase 2 study. Trial subjects will participate in a one-year trial with an opportunity to continue in the subsequent open label extension. Travel support to study sites will be provided.
To support confirming the diagnosis of WHIM, X4 Pharmaceuticals will offer genetic testing at no charge to patients meeting eligibility criteria suggestive for the disease. For information and support for genetic confirmation of your patients with suspected WHIM, please contact firstname.lastname@example.org.