X4 Pharmaceuticals Announces Initiation of Phase 2/3 Clinical Study of X4P-001-LD in Patients with WHIM Syndrome
January 30, 2017 at 08:30 am
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X4 Pharmaceuticals announced the initiation of a phase 2/3 study with the first patient dosed with X4P-001-LD for the treatment of WHIM syndrome, a sub-type of a primary immunodeficiency disease characterized by warts, hypogammaglobulinemia, infections, and myelokathexis or WHIM. The company is developing X4P-001-LD, a low dose formulation of X4P-001 that is currently in clinical development for the treatment of certain cancers, for use as a chronic treatment for patients with WHIM Syndrome. WHIM is a genetic primary immunodeficiency disease caused by the abberant trafficking of specific immune cells that are critical for proper immune system functions. Mutations in the CXCR4 receptor gene have been identified as an underlying cause of WHIM, based on sequencing and linkage studies conducted over the past 10 years. These mutations result in suppression of the immune system and lead to increased rates of severe health complications, including infections such as pneumonias, cellulitis, meningitis, deep soft tissue abscesses, and skin infections. Infections begin in early childhood and recur throughout life. Patients with WHIM also characteristically have numerous warts on their hands, feet, trunk and various mucosal surfaces that in some cases progress to cancer. By blocking the binding of CXCL12 ligand to the CXCR4 receptor, X4P-001-LD is designed to normalize the signaling from the mutant CXCR4 receptor to promote normal release of neutrophils and leukocytes, thereby restoring immune surveillance and normal immune function.
X4 Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company. The Company is focused on discovering and developing novel therapeutics for the treatment of rare diseases and those with limited treatment options, with a focus on conditions resulting from dysfunction of the immune system. Its lead clinical candidate is mavorixafor, a small-molecule selective antagonist of chemokine receptor CXCR4, that is being developed as an oral, once-daily therapy. It has developed a pipeline of small-molecule, oral antagonists of the chemokine receptor C-X-C receptor type 4 (CXCR4). It has two pre-clinical candidates: X4P-003, a second-generation CXCR4 antagonist designed to have enhanced properties relative to mavorixafor, potentially enabling opportunities in CXCR4-dependent disorders and primary immunodeficiencies, and X4P-002, a CXCR4 antagonist with a distribution profile and a demonstrated ability to cross the blood-brain barrier.
X4 PHARMACEUTICALS, INC. 
