Magenta Therapeutics Presents Updated Phase 2 Clinical Data on MGTA-456 Cell Therapy at American Academy of Neurology Annual ...
May 09 2019 - 7:30AM
Business Wire
-- 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 --
Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage
biotechnology company developing novel medicines to bring the
curative power of stem cell transplant to more patients, today
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, Pennsylvania.
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 Ashish Gupta, MBBS, M.P.H., Assistant
Professor, Department of Pediatrics and Division of Blood and
Marrow Transplantation, University of Minnesota.
In a separate oral presentation today, Kevin Goncalves, Ph.D.,
Magenta Therapeutics, will highlight preclinical data demonstrating
that the high stem cell dose in MGTA-456 accelerates and improves
engraftment of human microglia in the brains of transplanted
mice.
“We are very pleased to see signs of durable disease benefit in
patients with cALD,” said John Davis, M.D., M.P.H., Chief Medical
Officer, Magenta Therapeutics. “cALD is a rapidly progressive
disease, and patients whose disease progresses quickly typically
have poor long-term outcomes. 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.
We look forward to providing an additional update from the study
before the end of the year.”
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.
Overall results:
- All five patients met the primary
endpoint of neutrophil engraftment
- MGTA-456 was well tolerated
Forward-Looking Statement
This press release contains 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: uncertainties inherent in clinical studies and in the
availability and timing of data from ongoing clinical studies;
whether interim results from a clinical trial will be predictive of
the final results of the trial; whether results from preclinical
studies or earlier clinical studies will be predictive of the
results of future trials; the expected timing of submissions for
regulatory approval or review by governmental authorities,
including review under accelerated approval processes; orphan drug
designation eligibility; regulatory approvals to conduct trials or
to market products; whether Magenta's cash resources will be
sufficient to fund Magenta's foreseeable and unforeseeable
operating expenses and capital expenditure requirements; and other
risks set forth under the caption “Risk Factors” in Magenta’s
Registration Statement on Form S-1, as updated by Magenta’s most
recent Annual Report on Form 10-K and its other filings with the
Securities and Exchange Commission. In light of these risks,
uncertainties and assumptions, the forward-looking events and
circumstances discussed in this press release may not occur and
actual results could differ materially and adversely from those
anticipated or implied in the forward-looking statements. You
should not rely upon forward-looking statements as predictions of
future events. Although Magenta believes that the expectations
reflected in the forward-looking statements are reasonable, it
cannot guarantee that the future results, levels of activity,
performance or events and circumstances reflected in the
forward-looking statements will be achieved or occur.
Moreover, except as required by law, neither Magenta nor any
other person assumes responsibility for the accuracy and
completeness of the forward-looking statements included in this
press release. Any forward-looking statement included in this press
release speaks only as of the date on which it was made. We
undertake no obligation to publicly update or revise any
forward-looking statement, whether as a result of new information,
future events or otherwise, except as required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20190509005203/en/
Magenta Therapeutics:Manisha Pai, Vice President, Communications
& Investor Relations617-510-9193mpai@magentatx.com
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