Paula Ragan, PhD, CEO and President, X4 Pharmaceuticals, gives an overview of Waldenström’s Macroglobulinemia (WM).

As Dr. Ragan explains, WM is a rare blood cancer and a type of non-Hodgkin’s lymphoma which causes an overproduction of immunoglobulin M (IgM). Common symptoms of this overproduction include anemia, fatigue, and bleeding issues. There is currently no cure for WM and only one approved treatment option, ibrutinib. 

While about 90% of WM patients present with mutations in the MYD88 gene, 30-40% of patients present with mutations in the CXCR4 receptor gene as well. These double-mutation patients fare worse than patients with only the one mutation even when receiving ibrutinib, the current standard of care. Other treatment options include rituximab or rituximab plus chemotherapy; however, according to Dr. Ragan, these treatments tend to have a number of toxic side effects and are thus unfavorable compared to more recent and innovative treatment options.

Positive interim data on X4 Pharmaceuticals’ investigational drug, mavorixafor, in combination with ibrutinib, was recently published. The details of these results can be found here.

To learn more about WM and other rare cancers, visit