Mesoblast Phase 3 Trial Results for Acute Graft Versus Host Disease Presented at 2018 International Society for Cell and Gen...
May 08 2018 - 6:00AM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that
results of the Phase 3 trial evaluating its product candidate
MSC-100-IV (remestemcel-L) in children with steroid-refractory
acute Graft versus Host Disease (aGVHD) were presented at the 2018
International Society for Cell and Gene Therapy (ISCT) plenary
breakout session held in Boston last week. These results were
presented by the trial’s lead investigator Dr Joanne Kurtzberg,
Jerome Harris Distinguished Professor of Pediatrics and Director of
the Pediatric Blood and Marrow Transplant Program at Duke
University Medical Center.
Dr Kurtzberg described the properties of remestemcel-L that are
relevant to understanding proposed mechanisms of action (MOA) in
aGVHD, including sensing of damaging inflammatory mediators and
consequent reduction in activation of immune cells. These proposed
MOAs provide the rationale for the potency assays developed for
this product candidate. During the presentation, Dr Kurtzberg
stressed the importance of Mesoblast’s proprietary manufacturing
processes for remestemcel-L, which have been shown to deliver a
product with consistent batch-to-batch characterization and
activity, critical to providing a reproducible clinical
outcome. As previously reported, the
open label, single arm Phase 3 trial successfully met its
pre-specified primary endpoint of Day 28 overall response rate,
which is significantly increased (69%, p=0.0003) in children
treated with remestemcel-L compared to the protocol’s defined
hypothesized control rate of 45%. Remestemcel-L was well tolerated
in the Phase 3 trial with a safety profile consistent with prior
controlled studies in greater than 1,000 patient exposures.
This Phase 3 trial is being conducted under a United States Food
and Drug Administration (FDA) Investigational New Drug Application
(NCT#02336230). Key secondary endpoints include survival at
100 days, the results of which are expected to be read out shortly,
and safety and survival through 180 days.
There are currently no products approved in the United States
for treatment of steroid-refractory aGVHD. Given the serious nature
of this condition, in 2017 the United States Food and Drug
Administration (FDA) granted Mesoblast Fast Track designation for
the use of remestemcel-L to achieve improved overall response rate
in children with aGVHD. If successful, remestemcel-L will be the
first approved therapy for this devastating condition in the United
States. Japan is the only jurisdiction where this therapy is
available, through Mesoblast’s licensee JCR Pharmaceuticals Co.
Ltd.
About Graft Versus Host Disease Mesoblast is
developing MSC-100-IV for the treatment of aGVHD following an
allogeneic bone marrow transplant (BMT). In patients who have
received a BMT, donor cells may attack the recipient (the person
receiving the transplant), causing aGVHD, resulting in activation
of pro-inflammatory T-cells and tissue damage in the skin, gut and
liver. This condition, when severe and unresponsive to initial
steroid therapy, is often fatal. According to the Center for
International Blood and Marrow Transplant Research, there are
approximately 30,000 allogeneic BMTs globally per year for diseases
including hematological cancers, with approximately 20%1 of all
cases in the pediatric population. Approximately 50%6 of all
allogeneic BMT patients develop aGVHD. Liver or gastrointestinal
involvement occur in up to 60%2 of all patients with aGVHD and are
associated with the greatest risk of death, with mortality rates of
up to 85%.
1. CIBMTR 2015 Volume Data Set 2. Jagasia, M., Arora, M.,
Flowers, M. (2012) Risk Factors for acute GVHD and Survival after
Hematopoietic Cell Transplantation. Blood, 5 January
(119):296-307
About MesoblastMesoblast Limited (Nasdaq:MESO)
(ASX:MSB) is a global leader in developing innovative cell-based
medicines. Through a proprietary process, Mesoblast acquires highly
purified mesenchymal lineage precursor and stem cells from the bone
marrow of healthy adults, and creates master cell banks which can
be industrially expanded to produce thousands of doses from a
single donor that can be used off the shelf without the need for
tissue matching.
The Company has leveraged its proprietary technology platform to
establish a broad portfolio of late-stage product candidates.
Mesoblast’s allogeneic, ‘off-the-shelf’ cell product candidates
target advanced stages of diseases with high, unmet medical needs
including cardiovascular conditions, orthopedic disorders,
immunologic and inflammatory disorders and oncologic/hematologic
conditions.
Forward-Looking StatementsThis announcement
includes forward-looking statements that relate to future events or
our future financial performance and involve known and unknown
risks, uncertainties and other factors that may cause our actual
results, levels of activity, performance or achievements to differ
materially from any future results, levels of activity, performance
or achievements expressed or implied by these forward-looking
statements. We make such forward-looking statements pursuant to the
safe harbor provisions of the Private Securities Litigation Reform
Act of 1995 and other federal securities laws. Forward-looking
statements should not be read as a guarantee of future performance
or results, and actual results may differ from the results
anticipated in these forward-looking statements, and the
differences may be material and adverse. Forward-looking statements
include, but are not limited to, statements about the timing,
progress and results of Mesoblast’s preclinical and clinical
studies; Mesoblast’s ability to advance product candidates into,
enroll and successfully complete, clinical studies; the timing or
likelihood of regulatory filings and approvals; ability to
successfully produce and distribute product sufficient to support
market demand; and the pricing and reimbursement of Mesoblast’s
product candidates, if approved. You should read this press release
together with our risk factors, in our most recently filed reports
with the SEC or on our website. Uncertainties and risks that may
cause Mesoblast’s actual results, performance or achievements to be
materially different from those which may be expressed or implied
by such statements, and accordingly, you should not place undue
reliance on these forward-looking statements. We do not undertake
any obligations to publicly update or revise any forward-looking
statements, whether as a result of new information, future
developments or otherwise.
For further information, please contact:
Julie
Meldrum |
Schond Greenway |
Corporate
Communications |
Investor Relations |
T: +61 3
9639 6036 |
T: +1 212 880 2060 |
E: julie.meldrum@mesoblast.com
|
E: schond.greenway@mesoblast.com |
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