Magenta Therapeutics has announced that the company presented Phase 2 clinical data and preclinical research on its MGTA-456 program at the TCT annual meeting.
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.
MGTA-456 is a cell therapy derived from cord blood designed to provide a high dose of hematopoietic stem cells that are well-matched to the patient. The Company plans to enroll 12 patients in the ongoing Phase 2 study in inherited metabolic disorders, which include cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy and globoid cell leukodystrophy. The study previously enrolled patients with Hurler syndrome.
The primary endpoint of the study is engraftment after transplantation. Both short- and long-term disease specific outcomes are also being collected. Data from the first five evaluable patients treated in this study were highlighted in an oral presentation in the Pediatric Best Abstracts Session by Paul Orchard, M.D., Medical Director, Inherited Metabolic & Storage Disease Bone Marrow Transplantation Program, University of Minnesota.
In a separate oral presentation in the Best Pediatric Abstracts Session, Kevin Goncalves, Ph.D., Magenta Therapeutics, highlighted data demonstrating that the high stem cell doses in MGTA-456 accelerate and improve engraftment of human microglia in the brains of transplanted mice. Tony Boitano, Ph.D., Magenta Therapeutics, presented a third data set in the TCT Meeting Best Abstracts session demonstrating that MGTA-456 contains large doses of the stem cells responsible for engraftment, which are also correlated with rapid neutrophil recovery in patients following transplant.
“Inherited metabolic disorders are rare diseases that cause progressive damage to multiple organs, including the brain, and are often fatal. Stem cell transplant is the only disease-modifying treatment option but delivering sufficient doses of stem cells has been a persistent challenge.” … “MGTA-456 provides patients with a large number of stem cells to help overcome these challenges, and we are pleased that all five evaluable patients treated with MGTA-456 thus far have met the primary endpoints with robust and consistent engraftment. Further, in patients with Hurler syndrome, the production of normal levels of enzyme was associated with reduction in toxic disease-related metabolites. In patients with cALD, we have seen persistent decrease in brain inflammation as measured by imaging. These are early signs that MGTA-456 may provide disease-modifying clinical benefit to these patients.” John Davis, M.D., M.P.H., chief medical officer, Magenta Therapeutics