Magenta Therapeutics Announces Acceptance of Nine Abstracts Covering Clinical and Preclinical Data at ASH, Representing Progress across the Portfolio of Conditioning, Stem Cell Mobilization and Expansion Programs

From Startup Magenta Therapeutics

Link to Full Article: https://investor.magentatx.com/news-releases/news-release-details/magenta-therapeutics-announces-acceptance-nine-abstracts

Non-genotoxic antibody drug conjugates (ADCs) potently depleted
human stem cells in five abstracts on targeted conditioning programs
and demonstrated survival benefit in patient-derived leukemia models
MGTA-145 robustly mobilized cells that block graft-versus-host
disease in preclinical models in addition to large numbers of
hematopoietic stem cells that engrafted in primates following apheresis
All five patients with inherited metabolic disorders (IMDs) treated
with MGTA-456 in ongoing Phase 2 study met the primary endpoint of
successful engraftment by day 42, with median of one day of neutropenia
Company intends to launch a Phase 2 study of MGTA-456 in patients
with severe sickle cell disease in the first half of 2019, and
investigator-initiated Phase 2 study in blood cancers on track to
begin in late 2018
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 1, 2018–
Magenta
Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company
developing novel medicines to bring the curative power of bone marrow
transplant to more patients, today announced that the Company will
present clinical data and preclinical research at the 60th annual
meeting of the American Society of Hematology (ASH), taking place
December 1st through 4th in San Diego, Calif. Preliminary data in these
abstracts became available on the ASH conference website at 9:00 a.m.
ET today. The Company also provided an update today on its ongoing Phase
2 study of MGTA-456 in inherited metabolic disorders, including recent
data from the study.

ASH Abstracts“Magenta is developing potentially
transformative drugs to broaden the reach of one-time, curative cell
therapies – including bone marrow transplant, haploidentical transplant,
cell and gene therapy and genome editing – to more patients with
devastating diseases, including blood cancers, genetic diseases and
autoimmune diseases,” said Jason Gardner, D.Phil., president, chief
executive officer and co-founder, Magenta Therapeutics. “This year’s
nine data presentations at ASH highlight our patient conditioning,
mobilization and stem cell expansion programs, giving further insight
into our progress in addressing the major unmet needs of the transplant
process, including toxic conditioning regimens, inadequate stem cell
mobilization and low cell doses. We anticipate building on this progress
over the coming year as we move our mobilization program into the clinic
and advance our clinical development plan for stem cell expansion in
multiple diseases.”

Conditioning Programs
CD117-Amanitin Antibody Drug Conjugates Effectively Deplete Human
and Non-Human Primate HSCs: Proof of Concept as a Targeted Strategy for
Conditioning Patients for Bone Marrow Transplant, Abstract #3314

Presenter: Brad Pearse, Ph.D., Magenta TherapeuticsSession Name:
701. Experimental Transplantation: Basic Biology, Pre-Clinical Models:
Poster IISession Date: Sunday, December 2, 2018Session Time:
6:00 PM – 8:00 PMRoom: San Diego Convention Center, Hall GH

Targeting CD45 with an Amanitin Antibody-Drug Conjugate
Effectively Depletes Human HSCs and Immune Cells for Transplant
Conditioning, Abstract #4526

Presenter: Rahul Palchaudhuri, Ph.D., Magenta TherapeuticsSession
Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical
Models: Poster IIISession Date: Monday, December 3, 2018Session
Time: 6:00 PM – 8:00 PMRoom: San Diego Convention Center, Hall GH

Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117
or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit
in Patient Derived AML Models, Abstract #3316

Presenter: Jennifer Proctor, Magenta TherapeuticsSession Name:
701. Experimental Transplantation: Basic Biology, Pre-Clinical Models:
Poster IISession Date: Sunday, December 2, 2018Session Time:
6:00 PM – 8:00 PMRoom: San Diego Convention Center, Hall GH

Antibody Drug Conjugates Targeted to CD45 or CD117 Enable
Allogeneic Hematopoietic Stem Cell Transplantation in Animal Models,
Abstract #3324

Presenter: Rahul Palchaudhuri, Ph.D., Magenta TherapeuticsSession
Name: 701. Experimental Transplantation: Basic Biology, Pre-Clinical
Models: Poster IIDate: Sunday, December 2, 2018Presentation
Time: 6:00 PM – 8:00 PMLocation: San Diego Convention Center, Hall
GH

Non-genotoxic conditioning facilitates robust HSPC engraftment and
multi-lineage development in a dose dependent manner in Fanconi anemia

Presenter: Meera Srikanthan, Ph.D., Fred Hutchinson Cancer Research
CenterSession Name: TBADate: TBAPresentation Time: TBALocation:
TBA

Preclinical Data, Stem Cell Mobilization Program
MGTA-145 in Combination with Plerixafor Rapidly Mobilizes High
Numbers of Hematopoietic Stem Cells and Graft-Versus-Host Disease
Inhibiting Myeloid-Derived Suppressor Cells in Non-Human Primates,
Abstract #116

Presenter: Kevin Goncalves, Ph.D., Magenta TherapeuticsSession
Name: 711. Cell Collection and Processing ISession Date: Saturday,
December 1, 2018Session Time: 9:30 AM – 11:00 AMPresentation
Time: 9:45 AMRoom: Manchester Grand Hyatt San Diego, Grand Hall A

Clinical Data, MGTA-456 Stem Cell Expansion
Program
Preliminary Phase 2 Results Demonstrate Engraftment with Minimal
Neutropenia with MGTA-456, a CD34+ Expanded Cord Blood Product in
Patients Transplanted for Inherited Metabolic Disorders, Abstract #3467

Presenter: John Wagner, M.D., Director, Blood and Marrow Transplantation
Division, University of MinnesotaSession Name: 732. Clinical
Allogeneic Transplantation: Results: Poster IISession Date:
Sunday, December 2, 2018Session Time: 6:00-8:00 p.m. PTRoom:
San Diego Convention Center, Hall GH

MGTA-456 Contains Large Numbers of Expanded Cord Blood (CB)
CD34+CD90+ Hematopoietic Stem Cells (HSC) Which Confer All Engraftment
Activity and Correlate with Accelerated Neutrophil Recovery after
Myeloablative Conditioning in Patients with Hematologic Malignancy,
Abstract #2083

Presenter: Tony Boitano, Ph.D., Magenta TherapeuticsSession Name:
721 Clinical Allogeneic Transplantation: Conditioning Regimens,
Engraftment, and Acute Transplant ToxicitiesSession Date:
Saturday, December 1, 2018Session Time: 6:15-8:15 p.m. PTRoom: San
Diego Convention Center, Hall GH

Preclinical Data, MGTA-456 Stem Cell Expansion
Program
MGTA-456, A First-in-Class Cell Therapy Produced from a Single
Cord Blood Unit, Enables A Reduced Intensity Conditioning Regimen and
Enhances Speed and Level of Human Microglia Engraftment in the Brains of
NSG Mice, Abstract #115

Presenter: Kevin Goncalves, Ph.D., Magenta TherapeuticsSession
Name: 711. Cell Collection and ProcessingSession Date: Saturday,
December 1, 2018Presentation Time: 9:30 a.m. PTRoom: San
Diego Convention Center, Grand Hall A

MGTA-456 Clinical Development Update

MGTA-456 is a cell therapy providing a high dose of hematopoietic stem
cells that are well-matched to the patient, administered through a
transplant procedure. The ongoing Phase 2 study in inherited metabolic
disorders aims to enroll 12 patients with Hurler’s syndrome, cerebral
adrenoleukodystrophy (cALD), metachromatic leukodystrophy or globoid
cell leukodystrophy. The primary endpoint is engraftment after
transplantation and the secondary endpoint is transplant-related safety
and tolerability.

The data as of October 22, 2018 show:

Five patients treated and evaluable: three with Hurler’s syndrome, two
with cALD

Five of five patients treated with MGTA-456 met the primary endpoint
of successful engraftment by day 42 following the transplant procedure.

In recent historical cohorts of patients undergoing regular cord
blood transplantation with identical pre-transplant conditioning,
up to 32% did not engraft at comparable time points.

The patients treated with MGTA-456 had minimal neutropenia, lasting
for a median of 1 day.

In the historical cohort, neutropenia lasted for a median of 8
days.

Two patients less than 2 years of age with Hurler’s syndrome treated
with MGTA-456 in the Magenta study developed autoimmune cytopenia, a
known and frequent complication of transplant in these younger
patients and patients with Hurler’s syndrome.

The first patient subsequently died from this complication. This
was determined to be not related to the MGTA-456 product.

The second patient is currently undergoing treatment for the
autoimmune cytopenia.

“Blood stem cell transplantation remains the standard of care for
inherited metabolic disorders, which if untreated are generally
progressive and lethal. Unfortunately, transplantation remains a
difficult and complex procedure,” said Paul Orchard, M.D., Medical
Director of the Inherited Metabolic & Storage Disease Bone Marrow
Transplantation Program, University of Minnesota and principal
investigator in the MGTA-456 study. “Many patients experience
significant and life-threatening complications, such as dysregulation of
the immune system following transplantation, including the emergence of
antibodies to components of the blood system. Patients with inherited
metabolic disorders, particularly patients with Hurler’s syndrome, are
at higher risk for these antibody-associated cytopenias. In a prior
transplant protocol using the identical combination of chemotherapy
agents for pre-transplant conditioning, evidence of cytopenias was
present in 9 of 15 patients under 2 years of age (53%). This has proven
less common in older patients.”

In light of the elevated incidence of autoimmune cytopenias in very
young patients and patients with Hurler’s syndrome, the Company
announced today that it has voluntarily focused future enrollment
towards pediatric patients older than 2 years of age diagnosed with
leukodystrophies. The Company plans to continue enrolling patients with
leukodystrophies in the study.

“MGTA-456 is a first-in-class cell therapy with high doses of stem
cells, and larger doses of stem cells are correlated with more
successful transplant outcomes and faster time to engraftment and immune
recovery. It has demonstrated engraftment and robust immune recovery in
all 41 evaluable patients that have been treated to date in our Phase 2
study in inherited metabolic disorders and our Phase 1/2 study in blood
cancers,” said John Davis, M.D., M.P.H., chief medical officer, Magenta
Therapeutics. “We are developing MGTA-456 for patients with a broad
range of diseases, including leukodystrophies, sickle cell disease and
blood cancers, where we believe it has the potential to deliver
transformative benefit.”

The Company is on track to initiate a Phase 2 study of MGTA-456 in
patients with sickle cell disease in the first half of 2019, and an
investigator-initiated Phase 2 study in blood cancers is on track to
start in 2018.

About Magenta TherapeuticsHeadquartered in Cambridge,
Mass., Magenta Therapeutics is a clinical-stage biotechnology company
developing novel medicines for patients
with autoimmune diseases, blood cancers and genetic diseases. By
creating a platform focused on critical areas of unmet need, Magenta
Therapeutics is pioneering an integrated approach to allow more patients
to receive one-time, curative therapies by making the process more
effective, safer and easier.

Forward-Looking StatementThis press release may contain
forward-looking statements, including express or implied statements
regarding Magenta’s future expectations, plans and prospects, including
projections regarding future revenues and financing performance, our
long-term growth, the anticipated timing of our clinical trials and
regulatory filings, the development of our product candidates and
advancement of our preclinical programs, as well as other statements
containing the words “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “intend,” “may,” might,” “plan,” “potential,”
“project,” “should,” target,” “will” or “would” and similar expressions
that constitute forward-looking statements under the Private Securities
Litigation Reform Act of 1995. Although Magenta’s forward-looking
statements reflect the good faith judgment of its management, these
statements are based only on facts and factors currently known by
Magenta. As a result, you are cautioned not to rely on these
forward-looking statements. These and other risks concerning Magenta’s
programs and operations are described in additional detail in its
registration statement on Form S-1, its Quarterly Report on Form 10-Q
and its other filings made with the Securities and Exchange Commission
from time to time. Any forward-looking statement made in this press
release speaks only as of the date on which it is made. Magenta
undertakes no obligation to publicly update or revise any
forward-looking statement, whether as a result of new information,
future developments or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20181101005583/en/
Source: Magenta Therapeutics

Magenta Therapeutics:Manisha Pai, 617-510-9193Vice
President, Communications & Investor Relationsmpai@magentatx.com

Please visit their site for more information: Magenta Therapeutics.com

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2018-11-01 20:09:34