In March 2015, we reported successful top-line safety and efficacy results from a Phase 1 safety and efficacy trial for the use of motixafortide as a novel hematopoietic stem-cell mobilization treatment for allogeneic bone marrow transplantation.
In March 2016, we initiated a Phase 2 trial using motixafortide for hematopoietic stem cell mobilization for allogeneic and haploidentical stem-cell transplantation in various hematological malignancies, conducted in collaboration with the Washington University School of Medicine, Division of Oncology and Hematology. In May 2018, we announced positive top-line results of this study showing, among other things, that a single injection of motixafortide mobilized sufficient amounts of CD34+ cells required for transplantation at a level of efficacy similar to that achieved by using 4-6 injections of G-CSF, the current standard of care.
In December 2017, we presented data supporting motixafortide as robust mobilizer of hematopoietic stem cells associated with long-term engraftment at the 59th American Society of Hematology (ASH) Annual Meeting and Exhibition in Atlanta, GA.
In December 2017, we commenced a randomized, controlled Phase 3 registrational trial for motixafortide, known as the GENESIS trial, for the mobilization of HSCs for autologous transplantation in patients with multiple myeloma. The trial began with a lead-in period for dose confirmation, which was to include 10-30 patients and progress to the placebo-controlled main part, which is designed to include 177 patients in more than 25 centers. Following review of the positive results from treatment of the first 11 patients, the Data Monitoring Committee (DMC) recommended that the lead-in part of the study should be stopped and that we should move immediately to the second part.
In October 2020, we announced positive results from a planned interim analysis of the ongoing GENESIS Phase 3 trial of motixafortide for stem cell mobilization in multiple myeloma patients. At a meeting of the study's independent DMC, a planned interim analysis of the study's primary endpoint was conducted independently by the DMC. Based on the statistically significant evidence favoring treatment with motixafortide, the DMC issued a recommendation that patient enrollment may be ceased immediately, without the need to recruit all 177 patients originally planned for the study. In accordance with the DMC's recommendation, study enrollment is now complete at 122 patients.
Topline results were announced in May-2021. The study met all primary and secondary endpoints with exceptionally high level of statistical significance (p<0.0001).
The outstanding results include:
- ~90% of patients in treatment arm underwent transplantation following one administration and one apheresis
- Treatment effects and fold-increases compare favorably to plerixafor phase 3 results
- Results support goal of Motixafortide becoming standard-of-care mobilization agent for autologous bone-marrow transplantation and provides strong clinical and pharmaco-economic advantage for its use, on top of G-CSF, in all transplant procedures
- 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 combination is safe and tolerable
We are working aggressively to gain regulatory approval and plan to submit and NDA to the FDA in H1/22.