X4P-001-LD in WHIM
X4P-001-LD is a unique low-dose formulation of our lead drug candidate, X4P-001, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4. It is being developed for use as a life-long treatment for patients with WHIM and other primary immunodeficiencies. X4P-001-LD is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome.
Mutations in the CXCR4 receptor gene have been identified as the underlying cause of WHIM syndrome. In WHIM patients, CXCR4 signaling is over active and results in abberant trafficking of specific immune cells that are critical for proper immune system functions. X4P-001-LD is designed to normalize the signaling from the mutant CXCR4 receptor to promote normal release of neutrophils and leukocytes, thereby restoring normal immune surveillance and normal immune function.
X4P-001-LD may provide a new treatment approach focused on the underlying disease process of WHIM, whereas current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intraveneous immunoglobulins.