Magenta Therapeutics Presents Updated Phase 2 Clinical Data on MGTA-456 Cell Therapy at American Academy of Neurology Annual Meeting
-- Six-month follow-up on patients with cerebral adrenoleukodystrophy (cALD) shows stable neurological function scores and early and persistent resolution of brain inflammation on MRI --
MGTA-456 is a cell therapy 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 cALD, Hurler syndrome,
metachromatic leukodystrophy and globoid cell leukodystrophy. The
primary endpoint of the study is neutrophil engraftment after
transplantation. The study is also collecting both short- and long-term
disease-specific outcomes. Data from the first five evaluable patients
treated in this study were highlighted in a poster presented by
In a separate oral presentation today,
“We are very pleased to see signs of durable disease benefit in patients
with cALD,” said
Patients with Inherited Metabolic Disorders (IMDs) transplanted with MGTA-456, a CD34+ Expanded Cell Therapy Product, Show Rapid Engraftment in Preliminary Phase 2 Trial Results
Key results in patients with cALD:
- Both patients had stable neurological function scores, which remained unchanged between baseline and six months post-transplant, suggesting progress of the disease has been arrested.
- The Loes score, a method for quantifying the severity of brain abnormalities and atrophy found on MRI, also remained stable in both patients after six months.
Both patients showed resolution of gadolinium enhancement on MRI, an
indicator of brain inflammation, by one month post-transplant, and the
resolution persisted at six months.
- Durable resolution of gadolinium enhancement is correlated with long-term disease benefit in patients with cALD.
Key results in patients with Hurler Syndrome:
As previously reported, all three patients with Hurler syndrome
achieved normal levels of blood leukocyte IDUA enzyme, the enzyme that
is deficient in untreated patients with Hurler syndrome, by Day 42
post-transplant. This suggests that transplant with MGTA-456 is
affecting the disease process in these patients.
- Normalization of blood leukocyte IDUA enzyme after transplant has been significantly associated with improvement in disease.
- Patients showed a marked decline in urine total glycosaminoglycan (GAG), the toxic metabolites implicated in disease, after transplant.
- Both of these findings are correlated with improved long-term disease outcomes.
- All five patients met the primary endpoint of neutrophil engraftment
- MGTA-456 was well tolerated
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