Mavorixafor in Waldenstrom Macroglobulinemia

  • Waldenström’s macroglobulinemia (WM)is a rare blood cancer and a type of B-cell non-Hodgkin’s lymphoma characterized by the accumulation of malignant B-cells in the bone marrow, leading to the overproduction of immunoglobulin M (IgM). Typical WM symptoms include increased blood viscosity due to high IgM levels, brushing or skin lesions, anemia and enlarged lymph nodes and spleen.
  • About 90% of WM patients present with mutations in the MYD88 gene, while 30-40% of patients present with additional mutations in the CXCR4 gene, leading to overstimulation of the CXCR4 pathway; WM patients with CXCR4 mutations tend to have increased bone marrow disease burden, higher serum IgM levels, and a higher risk of developing symptomatic blood hyperviscosity. WM patients whose cancer harbors CXCR4 mutations often progress more quickly, even while on treatment, and have poorer outcomes.
  • No current treatments show a complete response for WM patients.
  • We are currently evaluating the safety and efficacy of mavorixafor in combination with the oral BTK inhibitor ibrutinib in a Phase 1b trial in WM patients with MYD88 and CXCR4 mutations. Since mavorixafor targets the CXCR4 pathway directly, we are testing mavorixafor’s ability to mobilize white blood cells out of the bone marrow and improve treatment response.
  • This trial is fully enrolled; results from the trial are expected in the third quarter of 2022.
  • Further clinical development of mavorixafor in WM will now be subject to completing a strategic partnership.

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