Mesoblast Submits Clinical Efficacy And Safety Data To FDA In Rolling Biologics License Application For Remestemcel-L
January 02 2020 - 6:00AM
Mesoblast Limited (ASX:MSB; Nasdaq: MESO), global leader in
cellular medicines for inflammatory diseases, announced that the
United States Food and Drug Administration (US FDA) has confirmed
receipt of Mesoblast’s filing of clinical efficacy
and safety data for remestemcel-L in its
rolling Biologics License Application (BLA)
for the treatment of children with steroid-refractory
acute graft versus host disease (SR-aGVHD). The final module will
be filed during January, and Mesoblast will request an
expedited FDA review of the BLA under the product candidate’s
existing Fast Track designation. If approved, remestemcel-L is
planned to be launched in the US in 2020.The clinical submission
included analyses of 309 children with SR-aGVHD who have
received remestemcel-L across three separate studies. In
addition, Mesoblast provided new data in control pediatric
subjects from the contemporaneous database of the Mount Sinai Acute
GVHD International Consortium (MAGIC) to provide an unbiased and
independent estimate of response rates and outcomes in matched
pediatric control patients treated with institutional standard of
care. The results of the comparative
analysis between Mesoblast’s open-label Phase 3 study and
contemporaneous controls receiving institutional standard of care
demonstrate the effectiveness of remestemcel-L in this patient
population, with particular efficacy and survival benefit in
patients with the most severe forms of aGVHD. These conclusions are
supported by prior results from an Expanded Access Program in 241
children where remestemcel-L was used as salvage therapy after
failure of steroids and other agents. Acute GVHD is a
potentially life-threatening condition which occurs in about 50% of
patients who receive an allogeneic bone marrow transplant.
Over 30,000 patients worldwide receive an allogeneic bone
marrow transplant, primarily during treatment for blood cancers,
and 20-25% are children1. Mortality at 12 months is as
high as 90% in those with aGVHD and Grade C/D or III/IV
disease severity2,3, and there are no approved treatments in the US
for children under 12.In Mesoblast’s open-label Phase 3 trial of
remestemcel-L in 55 children with SR-aGVHD, 89% of whom had Grade
C/D disease, the primary endpoint of Day 28 Overall Response in
those exposed to remestemcel-L was achieved in 70% and Day 100
survival was 75%. These outcomes were superior to those from
a cohort of 30 pediatric patients with SR-aGVHD from the MAGIC
consortium matched for inclusion criteria and disease
severity (80% Grade C/D). In the MAGIC controls, Day 28
Overall Response was 43% and Day 100 survival was
57%. Mesoblast Chief Medical Officer Dr Fred Grossman said:
“We are pleased to have submitted to the FDA clinical efficacy and
safety data for remestemcel-L, as well as comparative
clinical outcome data from contemporaneous controls. We are working
closely with the FDA to make our cellular medicine available
and improve outcomes in children with this devastating
condition.”
About
Remestemcel-LMesoblast’s lead product candidate,
remestemcel-L, is an investigational therapy comprising
culture-expanded mesenchymal stem cells derived from the bone
marrow of an unrelated donor. It is administered to patients in a
series of intravenous infusions. Remestemcel-L is believed to have
immunomodulatory properties to counteract the inflammatory
processes that are implicated in aGVHD 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.
References 1. 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. 2. Westin, J., Saliba, RM., Lima, M. (2011)
Steroid-refractory acute GVHD: predictors and outcomes. Advances in
Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective
single center analysis of outcome, risk factors and therapy in
steroid refractory graft-versus-host disease after allogeneic
hematopoietic cell transplantation. Bone Marrow
Transplantation.
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 cell therapy technology platform to establish a broad
portfolio of commercial products and late-stage product candidates.
Two products have been commercialized in Japan and Europe by its
licensees, and it has established commercial partnerships in Europe
and China for certain Phase 3 assets. In the United States,
Mesoblast has initiated submission of a rolling Biologics License
Application to the FDA to seek approval of its product candidate
for acute graft versus host disease following a successful Phase 3
trial, and is completing Phase 3 trials for its advanced heart
failure and chronic low back pain product
candidates. Mesoblast’s proprietary manufacturing process
yields industrial-scale, frozen, off-the-shelf, cellular medicines
based on its mesenchymal lineage cell platform technology. These
cell therapies, with defined pharmaceutical release criteria, are
planned to be readily available to patients
worldwide. Mesoblast has locations in Melbourne, New York,
Singapore and Texas 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
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; 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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