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 monotherapy as a novel hematopoietic stem-cell mobilization treatment for allogeneic bone marrow transplantation.
In March 2016, we initiated a Phase 2 trial using motixafortide as a monotherapy 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, 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.
GENESIS Phase 3 Trial:
In December 2017, we commenced a randomized, controlled Phase 3 registrational trial, at approximately 20 sites in the US and Europe, 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 was designed to include 177 patients. 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 be stopped and that we should move immediately to the second, randomized part – consisting of two arms: (1) motixafortide + G-CSF and (2) placebo + G-CSF.
In October 2020, we announced positive results from a planned interim analysis of the GENESIS trial. Based on analysis of the study's primary endpoint (conducted independently by the DMC) demonstrating 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 originally planned 177 patients . In accordance with the DMC's recommendation, study enrollment was completed at 122 patients.
In May 2021 we announced outstanding topline results of the GENESIS study:
- Study met all primary and secondary endpoints with exceptionally high level of statistical significance (p<0.0001)
- The combination is safe and tolerable
- ~90% of patients in treatment arm underwent transplantation following one administration of motixafortide and one apheresis session
- Results compare very favorably to plerixafor phase 3 results
In October 2021 we announced results of a pre-planned pharmacoeconomic study showing significant cost savings with motixafortide on top of G-CSF vs G-CSF alone in multiple myeloma patients. In addition, we conducted a study indirectly comparing motixafortide + G-CSF versus plerixafor + G-CSF which we announced in March 2022. Both analyses demonstrated substantial cost savings from using motixafortide and further strengthened the case for use of motixafortide as a primary mobilization agent for all multiple myeloma patients undergoing autologous stem cell transplantation:
- Versus plerixafor + G-CSF, the study found that the addition of motixafortide to G-CSF is associated with a net cost savings of ~$30,000 per patient (not including the cost of motixafortide).
- Versus G-CSF alone, the study found that the addition of motixafortide to G-CSF is associated with a net cost savings of ~$19,000 per patient (not including the cost of motixafortide).
In December 2021, we completed a successful pre-NDA meeting with the FDA, where we gained alignment with the FDA that motixafortide’s proposed data package is sufficient to support an NDA submission as a stem-cell mobilization agent for autologous bone marrow transplantation in multiple myeloma patients. We are working aggressively to submit the NDA application to the FDA in mid-2022. We are seeking a third-party collaborator to commercialize motixafortide, and in parallel, we are undertaking pre-commercialization activities necessary for a timely launch, if approved by the FDA, with a view to potentially obtaining FDA approval and launching sales in 2023.