A recent article in Cancer Network announced that motixafortide met its primary endpoint in the GENESIS trial for multiple myeloma patients who were scheduled for stem cell transplantation.
Often, the number of stem cells required for transplantation from donors or patients is not met. This is especially true when patients have been heavily pretreated. It is also a factor if there has been damage to the bone marrow. For years bone marrow has been the primary source of stem cells for transplantation.
Recently, however, the use of mobilized peripheral blood stem cells plus granulocyte colony-stimulating factor (G-CSF) has begun to replace bone marrow as a source of stem cells. This applies to both allogeneic (from a donor) or autologous (from the patient) stem cell transplantation. G-CSF continues to be used as an adjunct to motixafortide to stimulate the bone marrow and produce more infection-fighting white blood cells (neutrophils).
Motixafortide is the lead product of BioLineRX, a clinical-stage biopharmaceutical company based in Israel.
About the GENESIS Phase III Trial (NCT03246529)
The trial involved 122 multiple myeloma patients who were scheduled to receive an autologous bone marrow transplant. The purpose of the trial was to investigate G-CSF in combination with motixafortide (a synthetic peptide) against G-CSF plus a placebo.
The primary endpoint involved mobilized peripheral blood stem cell isolation called apheresis. Patients or donors are given a drug such as motixafortide which activates the collection of stem cells from the bone marrow into the peripheral bloodstream. It then passes through a machine that collects the stem cells. The remaining portion of the blood is reinfused into the patient or donor.
The apheresis procedure is generally completed in one session although the trial endpoint allows two sessions.
Genesis Trial Results
The primary and secondary endpoints, including improved stem cell mobilization, remained consistent throughout twelve sensitivity analyses.
The motixafortide and G-CSF combination, which was found to be well-tolerated and safe, resulted in 88.3 percent of patients proceeding to transplants. This compares with only 10.8 percent of patients qualifying for transplants in the placebo group.
It is noteworthy that on day one of apheresis, 8.5 million cells were collected from the motixafortide group with only 1.5 million cells collected in the placebo arm.
Lead investigator, Dr. John DiPersio of the University of Washington, commented that the success of motixafortide plus G-CSF indicates that the combination should be considered for all multiple myeloma patients planning autologous stem-cell transplants.
BioLineRx plans to apply for regulatory approval for motixafortide in early 2022. The company also hopes to expand its success into other stem cell indications.