Oncology Programs (For Partnership)
Mavorixafor in Waldenström’s 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 June 2022 data cut of the trial are presented here
- Further clinical development of mavorixafor in WM will now be subject to completing a strategic partnership.
X4P-002 in Oncology
- IND-enabling toxicology studies are being finalized for the pre-clinical candidate, a novel, small-molecule CXCR4 antagonist that has demonstrated potential across a number of leukemias and lymphomas and has been shown to cross the blood-brain barrier.
- An IND filing and clinical development will be subject to completing a strategic partnership.