Magenta Therapeutics (NASDAQ:MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients, today announced that the Company presented initial Phase 2 clinical data and preclinical research on its MGTA-456 program at the 60th annual meeting of the American Society of Hematology (ASH).
MGTA-456 is a cell therapy 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 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 improves 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,” said John Davis, M.D., M.P.H., chief medical officer, Magenta Therapeutics. “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.”
Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34+ Expanded Cord Blood Product in Patients Transplanted for Inherited Metabolic Disorders, Abstract #3467
Key results, as of November 2, 2018:
Preclinical Data, MGTA-456 Stem Cell Expansion Program
MGTA-456, A First-in-Class Cell Therapy Produced from a Single Cord Blood Unit, Enables A Reduced Intensity Conditioning Regimen and Enhances Speed and Level of Human Microglia Engraftment in the Brains of NSG Mice, Abstract #115
Key results presented by Kevin Goncalves, Ph.D., Magenta Therapeutics:
“Inherited metabolic disorders are characterized by defective enzyme function in the brains of patients, and engraftment of microglial cells in the brain after transplant is crucial for successful correction of the disease,” said Michael Cooke, Ph.D., chief scientific officer, Magenta Therapeutics. “These preclinical data provide compelling proof of mechanism for potential transformative benefit with MGTA-456 in patients with inherited metabolic disorders.”
MGTA-456 Contains Large Numbers of Expanded Cord Blood (CB) CD34+CD90+ Hematopoietic Stem Cells (HSC) Which Confer All Engraftment Activity and Correlate with Accelerated Neutrophil Recovery after Myeloablative Conditioning in Patients with Hematologic Malignancy, Abstract #2083
Key results, presented by Tony Boitano, Ph.D., Magenta Therapeutics:
About Magenta Therapeutics
Headquartered in Cambridge,
Mass., 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.
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Magenta Therapeutics:
Manisha Pai, Vice President, Communications &
Investor Relations
617-510-9193
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