Revamping Stem Cell Mobilization and Collection
Magenta is developing MGTA-145 to be the first-line therapy for same-day mobilization and collection of hemopoietic stem cells (HSCs) to enable successful rebuilding of the blood and immune system for all patients. MGTA-145 has the potential to be used across a broad range of diseases, including blood cancers, autoimmune diseases and genetic diseases, such as sickle cell disease.
MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, to harness the physiological mechanism of stem cell mobilization to rapidly and reliably mobilize HSCs for collection and transplant.
MGTA-145 has the potential to become the first medicine approved for the mobilization of stem cells in patients with genetic and autoimmune diseases. MGTA-145 also has the potential to replace the current standard of care for patients and allogeneic donors who currently rely on the use of granulocyte-colony stimulating factor (G-CSF), which takes five or more days to mobilize stem cells, does not mobilize reliably, causes significant bone pain and results in other side effects.
MGTA-145 Latest Results & Next Steps
In the first half of 2020, Magenta completed its Phase 1 trial of MGTA-145 in healthy volunteers, demonstrating MGTA-145 was well tolerated and enables same-day dosing, mobilization and collection of sufficient functional stem cells for transplant.
In May 2020, the U.S. FDA’s Office of Orphan Products and Development granted Orphan Drug Designation to MGTA-145 for the mobilization of HSCs to the peripheral blood for collection and subsequent transplant.
In May 2020, data presented at the American Society of Gene & Cell Therapy’s annual meeting showed MGTA-145 enables same day mobilization of functional HSCs that can be gene modified with CRISPR/Cas9 and mediate durable engraftment in preclinical models.
Stanford University is currently conducting a Phase 2 trial studying MGTA-145, in combination with plerixafor, in the mobilization and engraftment of hematopoietic stem cells for autologous transplantation in multiple myeloma patients.
Magenta intends to initiate multiple Phase 2 trials of MGTA-145 to include both allogeneic and autologous transplant settings across multiple diseases. These trials will evaluate mobilization and collection of functional HSCs and engraftment of the cells after transplant.