Clinically Meaningful Outcomes Using Remestemcel-L In Patients With Chronic Graft Versus Host Disease
February 20 2020 - 6:00AM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in cellular
medicines for inflammatory diseases, today announced that the
investigator-initiated expanded access protocol using its
cryopreserved allogeneic cell therapy product candidate
remestemcel-L for steroid-refractory chronic graft versus host
disease (chronic GVHD) has resulted in clinically meaningful
outcomes in all three treated patients, two children and one adult,
within 28 days after two infusions. On the basis of these outcomes,
the investigator-initiated collaboration will be expanded to
evaluate remestemcel-L in a pivotal trial for chronic GVHD.
Lead investigator Dr Joanne Kurtzberg, Jerome Harris
Distinguished Professor of Pediatrics and Professor of Pathology,
and Director, Pediatric Blood and Marrow Transplant Program at Duke
University Medical Center, said: “We are delighted with these
initial efficacy outcomes using remestemcel-L as first-line therapy
in steroid-refractory chronic graft versus host disease, where
there is a major unmet medical need for a safe and effective
therapy.”
Chronic GVHD occurs in 30-70% of recipients of an allogeneic
bone marrow transplant.1,2 Over 30,000 patients worldwide undergo
an allogeneic bone marrow transplant annually, primarily during
treatment for blood cancers, and these numbers are increasing.3 In
both the chronic and acute forms of GVHD, the donated bone marrow
stem cells view the recipient’s body as foreign, and attack the
body causing significant morbidity and mortality. Acute GVHD
usually manifests within 100 days following a transplant while
chronic GVHD generally manifests later (>100 days), and the two
may occur separately or within the same patient. The prevalence of
chronic GVHD in the US is over 14,000 patients, with an estimated
annual patient medical cost of approximately US$300,000.4
About Remestemcel-LRemestemcel-L is an
investigational therapy being developed for a range of rare
diseases. The product candidate comprises culture-expanded
mesenchymal stem cells derived from the bone marrow of an unrelated
donor. Remestemcel-L is believed to have immunomodulatory
properties to counteract the inflammatory processes that are
implicated in steroid-refractory GVHD by down-regulating the
production of pro-inflammatory cytokines, increasing production of
anti-inflammatory cytokines, and enabling recruitment of naturally
occurring anti-inflammatory cells to involved tissues.
References1. Arai et al. Biol Blood Marrow
Transplant. 2015; 21(2): 266–274. 2. Grube et al. Biol Blood Marrow
Transplant: 2016; 22 (10): 1781-179.3. Niederwieser D, Baldomero H,
Szer J. (2016) Hematopoietic stem cell transplantation activity
worldwide in 2012 and a SWOT analysis of the Worldwide Network for
Blood and Marrow Transplantation Group including the global
survey.4. Bachier C, Aggarwal S, Hennegan K (2019) Epidemiology and
Real-World Treatment of Chronic Graft-Versus-Host Disease Post
Allogeneic Hematopoietic Cell Transplantation: A US Claims
Analysis. Blood 2019; 134:Supplement 1.
About MesoblastMesoblast Limited (Nasdaq: MESO;
ASX: MSB) is a world leader in developing allogeneic
(off-the-shelf) cellular medicines. The Company has leveraged its
proprietary mesenchymal lineage cell therapy technology platforms
to establish a broad portfolio of commercial products and
late-stage product candidates. Mesoblast’s proprietary
manufacturing process yields industrial-scale, cryopreserved,
off-the-shelf, cellular medicines. These cell therapies, with
defined pharmaceutical release criteria, are planned to be readily
available to patients worldwide.
Mesoblast has filed a Biologics License Application to the
United States Food and Drug Administration (FDA) to seek approval
of its product candidate Ryoncil™ (remestemcel-L) for
steroid-refractory acute graft versus host disease (acute GvHD).
Remestemcel-L is also being developed for other rare diseases.
Mesoblast is completing Phase 3 trials for its rexlemestrocel
product candidates for advanced heart failure and chronic low back
pain. If approved, RYONCIL is expected to be launched in the United
States in 2020 for pediatric steroid-refractory acute GVHD. Two
products have been commercialized in Japan and Europe by
Mesoblast’s licensees, and the Company has established commercial
partnerships in Europe and China for certain Phase 3 assets.
Mesoblast has locations in Australia, the United States and
Singapore and is listed on the Australian Securities Exchange (MSB)
and on the Nasdaq (MESO). For more information, please see
www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter:
@Mesoblast
Mesoblast’s Forward-Looking Statements This
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; 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.
Release authorized by the Chief Executive.
For further information, please contact:
Julie Meldrum |
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Schond Greenway |
Corporate Communications |
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Investor Relations |
T: +61 3 9639 6036 |
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T: +1 212 880 2060 |
E: julie.meldrum@mesoblast.com |
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E: schond.greenway@mesoblast.com |
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