Magenta Therapeutics, a clinical-stage biotechnology company presented initial Phase 2 clinical data and preclinical research on its cord blood derived MGTA-456 program at the 60th annual meeting of the American Society of Hematology (ASH) this December in San Diego, USA.
Founded in 2015, headquartered in Cambridge, Massachusetts, USA, Magenta Therapeutics is a clinical-stage biotechnology company developing novel medicines for patients with autoimmune diseases, blood cancers and genetic diseases.
By creating a platform focused on critical areas of unmet need, Magenta Therapeutics is pioneering an integrated approach to allow more patients to receive one-time, curative therapies by making the process more effective, safer and easier.
MGTA-456 is a cell therapy derived from cord blood providing a high dose of hematopoietic stem cells that are well-matched to the patient, administered through a transplant procedure.
The ongoing Phase 2 study in inherited metabolic disorders aims to enroll 12 patients with cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy or globoid cell leukodystrophy, and previously also enrolled patients with Hurler’s syndrome.
The primary endpoint of the clinical study is engraftment after transplantation and the secondary endpoint is transplant-related safety and tolerability. Early data from this Phase 2 study were highlighted in a poster presentation by John Wagner, M.D., Director, Blood and Marrow Transplantation Division, University of Minnesota.
In a separate oral presentation, Kevin Goncalves, Ph.D., Magenta Therapeutics, highlighted data showing that MGTA-456 significantly improved engraftment and the number of human microglia in the brains of transplanted mice.
Tony Boitano, Ph.D., Magenta Therapeutics, presented a third data set showing that MGTA-456 contains large doses of the cells responsible for engraftment, which are also correlated with rapid neutrophil recovery in patients following transplant.
“Inherited metabolic disorders are rare and often fatal diseases. The only disease-modifying treatment option is bone marrow transplant, which can be challenging for patients without a matched sibling donor. MGTA-456 is a cell therapy with a high dose of stem cells that are well matched to the patient and may represent a promising treatment option for these patients.”
“We are pleased to see robust and consistent engraftment, the primary endpoint of the study, in all five of the evaluable patients treated thus far. We are also very encouraged by the changes in early disease biomarkers and brain imaging evidence that are correlated with positive long-term disease outcomes.”
“In addition to leukodystrophies, we are developing MGTA-456 for patients with a broad range of diseases where we believe it has the potential to deliver transformative benefit. A Phase 2 investigator-initiated study of MGTA-456 in blood cancers will begin in late 2018, and we will start a Phase 2 study of MGTA-456 in sickle cell disease in the first half of 2019.” – John Davis, M.D., M.P.H., chief medical officer, Magenta Therapeutics.