Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment

According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This drug is being developed as a treatment for WHIM syndrome, a rare disease. X4 is committed to the development of innovative new treatments for rare diseases. The drug has produced encouraging results in previous rounds of clinical testing.

About WHIM Syndrome

WHIM syndrome, which stands for Warts, Hypogammaglobulinemia, Immunodeficiency, and Myelokathexis, is a rare, congenital syndrome that causes immunodeficiency that is characterized by chronic neutropenia, or low levels of neutrophils in the blood. This syndrome is caused by mutations that affect the CXCR4 gene. The activity of the GRK3 gene has been associated with the syndrome as well. Signs and symptoms of WHIM syndrome include the retention of neutrophils in the bone marrow, increased vulnerability to bacterial and viral infections (especially HPV), deficiencies in IgG antibodies and lymphocytes, and warts on the hands and feet. Treatment is mostly symptomatic and includes therapies to reduce bacterial infections and improve counts of neutrophils in the blood. There is currently no known cure. Research studies have suggested that CXCR4 antagonists could be an effective approach. To learn more about WHIM syndrome, click here.

About The Trial

This phase 3 trial is expected to last for 52 weeks. The study is intended to include as many as 28 patients from as many as 20 different countries. The primary endpoint of this trial will be the measurement of circulating neutrophils ( a type of white blood cell) in the blood, comparing a placebo and mavorixafor side by side. This will be measured in several different 24 hour sections during the treatment period. The secondary endpoints in the trial will involve changes in a number of other symptoms, such as quality of life and immune system assessments, changes in warts, and changes in rates of infection. 

About Mavorixafor

Mavorixafor has earned Orphan Drug designation from the US Food and Drug Administration (FDA) and is also in development for several other indications, including Waldenstrom’s macroglobulinema, severe congenital neutropenia, and clear cell renal cell carcinoma.


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