Mesoblast’s Phase 3 Trial in Children With Acute Graft Versus Host Disease Completes Enrollment
20 December 2017 - 10:00PM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that the
Phase 3 trial of its proprietary allogeneic mesenchymal stem
cell (MSC) product candidate MSC-100-IV in children with
steroid-refractory acute graft versus host disease (aGVHD) has
completed enrollment. Top-line results are expected in Q1
2018. In November 2016, the Phase 3 trial was successful in a
pre-specified interim futility analysis of the primary
endpoint.
There are currently no therapeutic products
approved in the United States to treat this life-threatening
complication of allogeneic bone marrow transplants. Based on
discussions with the United States Food and Drug Administration
(FDA), Mesoblast intends to use the results of this single, open
label trial to support a Biologics License Application (BLA) filing
for accelerated product approval. Mesoblast has received Fast Track
designation for MSC-100-IV for treatment of steroid-refractory
acute GVHD in children.
In 2016, Mesoblast’s licensee in Japan, JCR Pharmaceuticals Co.
Ltd, launched TEMCELL® HS. Inj.1, an allogeneic MSC product, after
receiving marketing approval from the Japanese Ministry of Health,
Labour and Welfare for the treatment of aGVHD in children and
adults.
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 25% of all cases in the
pediatric population. Nearly 50% of all allogeneic BMT patients
develop aGVHD. Liver or gastrointestinal involvement occur in up to
40% of all patients with aGVHD and are associated with the greatest
risk of death, with mortality rates of up to 85%.
1TEMCELL® HS. Inj. is the registered trademark of JCR
Pharmaceuticals Co. Ltd.
About MesoblastMesoblast Limited (Nasdaq:MESO)
(ASX:MSB) is a global leader in developing innovative cell-based
medicines. The Company has leveraged its proprietary technology
platform, which is based on specialized cells known as mesenchymal
lineage adult stem cells, 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
initiation, timing, progress and results of Mesoblast’s preclinical
and clinical studies, and Mesoblast’s research and development
programs; Mesoblast’s ability to advance product candidates into,
enroll and successfully complete, clinical studies, including
multi-national clinical trials; Mesoblast’s ability to advance its
manufacturing capabilities; the timing or likelihood of regulatory
filings and approvals, manufacturing activities and product
marketing activities, if any; the commercialization of Mesoblast’s
product candidates, if approved; regulatory or public perceptions
and market acceptance surrounding the use of stem-cell based
therapies; the potential for Mesoblast’s product candidates, if any
are approved, to be withdrawn from the market due to patient
adverse events or deaths; the potential benefits of strategic
collaboration agreements and Mesoblast’s ability to enter into and
maintain established strategic collaborations; Mesoblast’s ability
to establish and maintain intellectual property on its product
candidates and Mesoblast’s ability to successfully defend these in
cases of alleged infringement; the scope of protection Mesoblast is
able to establish and maintain for intellectual property rights
covering its product candidates and technology; estimates of
Mesoblast’s expenses, future revenues, capital requirements and its
needs for additional financing; Mesoblast’s financial performance;
developments relating to Mesoblast’s competitors and industry; 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 MeldrumCorporate CommunicationsT: +61 3 9639
6036E: julie.meldrum@mesoblast.com
Schond GreenwayInvestor RelationsT: +1 212 880
2060E: schond.greenway@mesoblast.com
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