Our grit and science drive solutions for patients. Every day, we strive to advance our pipeline of innovative therapeutic candidates to treat rare diseases. We are targeting the CRCX4 pathway, one of the most important chemokine pathways, in an effort to treat people with rare immune system disorders.
Aberrant functioning of CXCR4 is associated with certain PI diseases. X4 has now completed enrollment in its pivotal Phase 3 clinical trial for its lead candidate, mavorixafor, in patients with WHIM syndrome. X4 is developing novel, oral medicines designed to treat a range of rare diseases, including primary immunodeficiency diseases and certain rare cancers, such as lymphomas.
Aberrant functioning of CXCR4 is associated with certain PI diseases. X4 has now completed enrollment in its pivotal Phase 3 clinical trial for its lead candidate, mavorixafor, in patients with WHIM syndrome. X4 is developing novel, oral medicines designed to treat a range of rare diseases, including primary immunodeficiency diseases and certain rare cancers, such as lymphomas.
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X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company and a leader in the discovery and development of novel therapies for the treatment of diseases of the immune system via antagonism of the CXCR4 pathway, with a focus on rare diseases and those with limited treatment options.
We believe that inhibition of the CXCR4 receptor creates the potential to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer.Our lead clinical candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy.
We believe that inhibition of the CXCR4 receptor creates the potential to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer.Our lead clinical candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy.
At X4 our mission is to develop treatments that have a clear and profound impact for patients suffering with rare diseases, including primary immunodeficiencies (such as WHIM syndrome) and cancer. We are developing potential new treatments through a rigorous R&D process for diseases where we believe we can dramatically improve the overall health of patients.
We are currently advancing a pipeline of small molecule, oral antagonists of the chemokine receptor CXCR4. We believe that inhibition of the CXCR4 receptor creates the potential to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer.
X4 is developing mavorixafor as an oral, once-daily treatment for patients with WHIM syndrome. WHIM syndrome is a rare genetic primary immunodeficiency that results from "gain-of-function" mutations in the single gene that encodes for the CXCR4 receptor.
These genetic mutations cause premature truncations in the CXCR4 protein, causing the receptor to remain in an "on" state longer than normal, resulting in compromised immune cell maturation and mobilization.We have achieved clinical proof of concept for mavorixafor in a Phase 2 trial of WHIM syndrome where we observed reduction in severe neutropenia and significant lymphopenia; overall, mavorixafor was well tolerated.
These genetic mutations cause premature truncations in the CXCR4 protein, causing the receptor to remain in an "on" state longer than normal, resulting in compromised immune cell maturation and mobilization.We have achieved clinical proof of concept for mavorixafor in a Phase 2 trial of WHIM syndrome where we observed reduction in severe neutropenia and significant lymphopenia; overall, mavorixafor was well tolerated.
Primary Immunodeficiencies (PIs) are a group of rare, chronic disorders in which deficiencies in the immune system cause increased susceptibility to infections and, in some cases, increased risk of cancers. Within this broad disease classification, WHIM syndrome is one of a number PIs that are caused by the improper trafficking of immune cells.
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