Magenta Therapeutics (NASDAQ:MGTA) reported preliminary data from its Phase 2 trial testing MGTA-456, a hematopoietic stem cell (HSC) therapy, in patients with inherited metabolic disorders.
The Phase 2 study is assessing neutrophil engraftment after HSC transplantation in patients with cerebral adrenoleukodystrophy (cALD), Hurler syndrome, metachromatic leukodystrophy and globoid cell leukodystrophy.
“cALD is a rapidly progressive disease, and patients whose disease progresses quickly typically have poor long-term outcomes,” Dr. John Davis, Magenta’s CMO, said in a statement.
All of the first five evaluable patients treated with MGTA-456 met the primary endpoint of neutrophil engraftment. In addition, patients with cALD had stable neurological function scores and early and persistent resolution of brain inflammation on a six-month follow-up.
“The stable neurological function score and persistent decrease in brain inflammation in these two patients suggest that we have halted the inflammatory process associated with the disease, which may provide long-term benefits,” he added.
Magenta expects to provide additional data from this study by the end of 2019.
