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Clinicians Report Updated Phase 2 Data On Experimental Cell Therapy For cALD.

CAMBRIDGE, Mass., May 9, 2019 -- Magenta Therapeutics (NASDAQ: MGTA) announced that the company presented Phase 2 clinical data on its cell therapy, MGTA-456, at the annual meeting of the American Academy of Neurology in Philadelphia, Pa.

MGTA-456 is a cell therapy designed to provide a high dose of hematopoietic SCs 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 longterm disease-specific outcomes.

Data from the first five evaluable patients treated in this study were highlighted in a poster presented by Ashish Gupta of the University of Minnesota.

In a separate oral presentation, Kevin Goncalves of Magenta highlighted preclinical data demonstrating that the high stem cell dose in MGTA-456 accelerates and improves engraftment of human microglia in transplanted mice.

"We are very pleased to see signs of durable disease benefit in patients with cALD," said Magenta CMO John Davis.

cALD is a rapidly progressive disease, and patients whose disease progresses quickly typically have poor longterm outcomes.

The stable neurological function score and persistent decrease in brain inflammation in these two patients suggest that the therapy halted the inflammatory process associated with the disease and may provide long-term benefits.

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 pa ents had stable neurological func on scores, which remained unchanged between baseline and six months post-transplant, sugges ng progress of the disease has been arrested.

* The Loes score, a way to quantify the severity of brain abnormali es and atrophy found on MRI, also were stable in both pa ents a er six months.

* Both pa ents showed resolu on of gadolinium enhancement on MRI, an indicator of brain inflamma on, by one month post-transplant, and the resolu on persisted at six months.

* Durable resolu on of gadolinium enhancement is correlated with long-term benefit in cALDpa ents.

Key results in patients with Hurler Syndrome:

* As previously reported, all three pa ents with Hurler syndrome achieved normal levels of blood leukocyte IDUA enzyme, the enzyme that is deficient in untreated pa ents with Hurler syndrome, by Day 42 post-transplant. This suggests that transplant with MGTA-456 is affec ng the disease process in these pa ents.

* Normaliza on of blood leukocyte IDUA enzyme a er transplant has been significantly associated with improvement in disease.

* Pa ents showed a marked decline in urine total glycosaminoglycan (GAG), the toxic metabolites implicated in disease, a er transplant.

* Both of these findings are correlated with improved long-term disease outcomes.

Overall results: all patients met the primary endpoint of neutrophil engraftment; MGTA-456 was well tolerated


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Title Annotation:Clinical Research
Publication:Stem Cell Research News
Date:May 20, 2019
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