Positive results from GENESIS Phase III trial of motixafortide in stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma .BioLineRx Ltd

BioLineRx Ltd. announced positive top-line results from the Company's GENESIS Phase III trial evaluating its lead clinical candidate, motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this indication), for hematopoietic stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients. An analysis of data on all 122 enrolled patients (the intent to treat, or ITT, population) found highly statistically significant evidence across all primary and secondary endpoints favoring motixafortide in addition to G-CSF, as compared to placebo plus G-CSF. In addition, the combination was found to be safe and well tolerated. The primary endpoint of the study demonstrated a 4.9-fold increase (70.0% vs 14.3%; difference 54.6%; 95% CI 39.7-69.5%; p<0.0001) in the proportion of patients in the treatment arm, as compared to the control arm mobilizing greater than 6 million cd34+ cells kg in up to two apheresis sessions, and after only one administration of motixafortide. this translates to an odds-ratio of 12.9. the study also achieved its main secondary endpoint, demonstrating a 14.1-fold increase (67.5% vs 4.8%; difference 61.7%; 95% ci 49.5-73.8%; p><0.0001) in the proportion of patients in the treatment arm, as compared to the control arm, who mobilized greater tha n 6 million cd34+ cells kg in just one apheresis session. this translates to an odds-ratio of 56.0. other important data from the study include median number of cd34+ cells collected on the first day of apheresis (8.5 million in the treatment arm vs 1.5 million in the control arm) – a 5.6-fold increase. the addition of motixafortide to g-csf also allowed 88.3% of patients to undergo transplantation after only one apheresis session, compared to 10.8% in the g-csf arm – an 8.2-fold increase. engraftment endpoints, including the number of days needed for engraftment, success of engraftment and the durability of engraftment 100 days post-transplant, further support the study's success. "the results of the genesis study are extremely impressive, and all the more so when considering that almost 90% of the patients in the treatment arm proceeded to transplantation after only one apheresis session," stated john dipersio, md, washington university school of medicine, and lead investigator of the study. "this is a great achievement in alleviating the burden for the patients and reducing hospital resources. i believe these results make the combination of motixafortide and g-csf a very attractive candidate for use in all patients with multiple myeloma undergoing autologous stem-cell transplantation." about the genesis trial : the genesis trial (nct03246529) was initiated in december 2017. genesis was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of motixafortide and g-csf, compared to placebo and g-csf, for the mobilization of hematopoietic stem-cells for autologous transplantation in multiple myeloma patients. the primary objective of the study was to demonstrate that only one dose of motixafortide on top of g-csf is superior to g-csf alone in the ability to mobilize greater than 6 million cd34+ cells in up to two apheresis sessions. additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. local laboratories and a central laboratory were used to determine cd34+ cell yields. for regulatory purposes, efficacy endpoints were calculated using the percentage of cd34+ cells determined by the central laboratory. the local laboratory values were used for all clinical decisions, including the number of apheresis days and the decision to proceed to transplantation.>