X4 Pharmaceuticals

Boston,  MA 
United States


X4 Pharmaceuticals is a late-stage biopharmaceutical company. The company is a leader in the discovery and development of novel therapies for the treatment of rare diseases resulting from dysfunction of the CXCR4 receptor pathway. The lead candidate, mavorixafor, is a first-in-class, oral, small molecule antagonist of CXCR4. X4 believes that inhibition of CXCR4 creates the potential for mavorixafor to provide therapeutic benefit across a wide variety of diseases. The efficacy and safety of mavorixafor is currently being evaluated in a global Phase 3 clinical trial in WHIM syndrome, and in two Phase 1b clinical trials – in combination with ibrutinib in Waldenström’s macroglobulinemia, and as monotherapy in Severe Congenital Neutropenia.