Press Release Details
Magenta Therapeutics Announces Updated Phase 2 Data on MGTA-456 Cell Therapy, Demonstrating Continued Durability in Inherited Metabolic Disorders
-- Additional data from Phase 2 study show that MGTA-456 demonstrates clinically meaningful durable benefits for patients with inherited metabolic disorders one year following treatment –
-- Magenta intends to complete enrollment in Phase 2 in 2020 and continue dialogue with the
MGTA-456 is a cell therapy designed to provide a high dose of hematopoietic stem cells (HSCs) that are well matched to the patient to enable safe immune and blood system rebuild in IMD patients and remission in patients with blood cancers. Magenta is currently developing MGTA-456 in an ongoing Phase 2 study in patients with inherited metabolic disorders (IMD), including cerebral adrenoleukodystrophy (cALD), mucopolysaccharidosis type IH (MPS I, or Hurler syndrome), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease). These are rare, rapidly progressive neurologic disorders that are fatal when left untreated. Investigators at the
“The clinical demonstration of rapid and durable resolution of disease in patients with inherited metabolic disorders is very compelling; it’s particularly encouraging as these results are not seen with currently available treatments, nor with gene therapies under investigation,” said
Magenta intends to complete enrollment in the Phase 2 in 2020 and continue dialogue with the
Updated Results from Ongoing MGTA-456 Phase 2 Study in Inherited Metabolic Disease
Title: MGTA-456 Cell Therapy in Inherited Metabolic Disease Yields Rapid and Durable Long-Term Improvement of Disease-Specific Outcomes in a Phase 2 Trial (Abstract #20)
- Treatment with MGTA-456 in patients with inherited metabolic disorders showed early, robust engraftment and immune reconstitution.
- Patients received a median CD34+ cell dose of 110 x 106 cells/kg and TNC dose of 26 x 107 cells/kg with a median duration of neutropenia of one day (range 0-9), compared to a median of eight days for historical controls.
- Myeloid chimerism was ≥98% donor in evaluable patients by day +14 and immune reconstitution of CD4 and CD8 T-cell subsets were comparable or better than historical controls.
- Two steroid-responsive episodes of skin-only aGvHD were observed and no cGvHD has been reported.
Key results in patients with cALD:
- Patients showed resolution of gadolinium enhancement on MRI, an indicator of brain inflammation, by one month post-treatment, and the resolution persisted throughout the follow-up period. Sustained resolution of gadolinium enhancement is correlated with long-term disease benefit in patients with cALD.
- The Loes score, a method for quantifying the severity of brain abnormalities and atrophy found on MRI, remained stable over the follow-up period, consistent with a halt in disease progression.
- The NFS remained stable in both patients over one year, suggesting a durable halt in disease progression.
Key results in patients with MPS I / Hurler Syndrome:
- Patients with MPS I / Hurler syndrome showed normalized levels of blood a-L-iduronidase by Day +42 and had decreased levels of Hurler-specific urine. glycosaminoglycans, the toxic metabolites implicated in disease at a >42-day timepoint.
In a separate presentation today,
This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption “Risk Factors” in Magenta’s Registration Statement on Form S-1, as updated by Magenta’s most recent Quarterly Report on Form 10-Q and its other filings with the
Manisha Pai, Vice President, Communications & Investor Relations
Lyndsey Scull, Director, Corporate Communications