Mesoblast Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for remestemcel-L in the Treatment ...
May 29 2019 - 8:49PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in cellular
medicines for inflammatory diseases, today announced that it has
filed the first component of a rolling submission for a Biologics
License Application (BLA) to the U.S. Food and Drug
Administration (FDA) for remestemcel-L in the treatment of
children with steroid-refractory acute graft versus host disease
(aGVHD), a life-threatening complication of an allogeneic bone
marrow transplant.
The FDA has agreed to a rolling review of the BLA which enables
individual components to be submitted and reviewed on an ongoing
basis rather than waiting for all sections to be completed. The
rolling process will provide opportunity for ongoing communication,
and during this process the Company expects it will be able to
adequately address any substantial matters raised by the FDA.
Remestemcel-L has received Fast Track designation for aGVHD and
under this designation Mesoblast intends to request a priority
review once its BLA filing is completed and accepted by the FDA.
Mesoblast Chief Executive Dr Silviu Itescu stated: “Initiation of
our rolling BLA submission to the FDA for potential United States
approval of our first product is a major corporate milestone for
the Company and an exciting moment in our history. We look forward
to making this therapy available as soon as possible to children
with this devastating disease.”
There are more than 30,000 allogeneic bone marrow transplants
performed annually worldwide1, primarily in patients being treated
for blood cancers, with up to 50% developing aGVHD. In the more
severe forms of the disease, Grades C/D or III/IV, six-month
mortality rates are as high as 90%.2,3
In Mesoblast’s Phase 3 trial of 55 children with aGVHD - 89% of
whom had Grade C/D disease - treatment with remestemcel-L resulted
in a six-month survival of 69%. In addition, achievement of an
Overall Response at Day 28, which occurred in 69% of patients,
predicted highest survival at Day 100 and Day 180, which was 85%
and 79%, respectively. The trial successfully met its primary
endpoint of increased Day 28 Overall Response compared with a
protocol-defined historical control rate of 45% (p=0.0003). These
data are consistent with 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.
About remestemcel-LMesoblast’s lead product
candidate, remestemcel-L, is an investigational therapy comprising
culture-expanded mesenchymal stromal cells derived from the bone
marrow of an unrelated donor. It is administered to patients in a
series of intravenous infusions. Remestemcel-L has demonstrated
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.
About Mesoblast Mesoblast
Limited (Nasdaq:MESO; ASX:MSB) is a world leader in developing
allogeneic (off-the-shelf) cellular medicines. The Company has
leveraged its proprietary technology platform to establish a broad
portfolio of late-stage product candidates with three product
candidates in Phase 3 trials – acute graft versus host disease,
chronic heart failure and chronic low back pain due to degenerative
disc disease. Through a proprietary process, Mesoblast selects rare
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 each donor
that meet stringent release criteria, have lot to lot consistency,
and can be used off-the-shelf without the need for tissue matching.
Mesoblast has facilities in Melbourne, New York, Singapore and
Texas and is listed on the Australian Securities Exchange (MSB) and
on the Nasdaq (MESO). www.mesoblast.com
- 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.
- Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory
acute GVHD: predictors and outcomes. Advances in
Hematology.
- 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
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 in aGVHD; Mesoblast’s ability to advance its aGVHD product
candidate into, enroll and successfully complete, clinical studies;
the timing or likelihood of regulatory filings and approvals for
aGVHD; 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
Corporate Communications
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
Schond GreenwayInvestor RelationsT: +1 212 880
2060E: schond.greenway@mesoblast.com
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