U.S. FDA Advisory Committee Votes Nine to One in Favor of Remestemcel-L (Ryoncil™) for Efficacy in Children With Steroid-Re...
August 14 2020 - 6:00AM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in cellular
medicines for inflammatory diseases, today announced that the
Oncologic Drugs Advisory Committee (ODAC) of the United States Food
and Drug Administration (FDA) voted overwhelmingly in favor that
the available data support the efficacy of remestemcel-L (RYONCIL™)
in pediatric patients with steroid-refractory acute graft versus
host disease (SR-aGVHD).
Mesoblast Chief Medical Officer Dr Fred Grossman said:
“Steroid-refractory acute graft versus host disease is an area of
extreme need, especially in vulnerable children under 12 years old
where there is no approved therapy. We are very encouraged by
today’s outcome and are committed to working closely with the FDA
as they complete their review of our submission regarding approval
of RYONCIL for this life-threatening complication of an allogeneic
bone marrow transplant.”
The ODAC is an independent panel of experts that evaluates
efficacy and safety of data and makes appropriate recommendations
to the FDA. Although the FDA will consider the recommendation
of the panel, the final decision regarding the approval of the
product is made solely by the FDA, and the recommendations by the
panel are non-binding. RYONCIL has been accepted for Priority
Review by the FDA with an action date of September 30, 2020, under
the Prescription Drug User Fee Act (PDUFA). If approved by the
PDUFA date, Mesoblast plans to launch RYONCIL in the United States
in 2020.
Pediatric transplant physician Dr Joanne Kurtzberg, the Jerome
Harris Distinguished Professor of Pediatrics and Professor of
Pathology, and Director, Pediatric Blood and Marrow Transplant
Program at Duke University Medical Center, said: “This devastating
condition has an extremely poor prognosis and there are no
FDA-approved options for children under the age of 12. The clinical
studies I have directed have demonstrated the potential for this
treatment to fill a significant unmet medical need.”
Conference CallAn audio webcast can be accessed
via
https://webcast.boardroom.media/mesoblast-limited/20200813/NaN5f3237e85300840019de909d
The archived webcast is also available on the Investor page of
the Company’s website www.mesoblast.com
About Acute Graft Versus Host Disease Acute
GVHD occurs in approximately 50% of patients who receive an
allogeneic bone marrow transplant (BMT). Over 30,000 patients
worldwide undergo an allogeneic BMT annually, primarily during
treatment for blood cancers, and these numbers are increasing.1 In
patients with the most severe form of acute GVHD (Grade C/D or
III/IV) mortality is as high as 90% despite optimal institutional
standard of care.2,3 There are currently no FDA-approved
treatments in the United States for children under 12 with
SR-aGVHD, a potentially life-threatening complication of an
allogeneic bone marrow transplant for blood cancer.
About RYONCILTM Mesoblast’s lead product
candidate, RYONCIL (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. RYONCIL is believed to have
immunomodulatory properties to counteract the inflammatory
processes that are implicated in steroid-refractory acute graft
versus host disease 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.
1. Niederwieser D, Baldomero H, Szer J. 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. Bone Marrow Transplant 2016;
51(6):778-85. 2. Westin, J., Saliba, RM., Lima, M. (2011)
Steroid-refractory acute GVHD: predictors and outcomes. Advances in
Hematology 2011;2011:601953. 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 2019;54(11):1805-1814
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 platform to establish a
broad portfolio of commercial products and late-stage product
candidates. Mesoblast has a strong and extensive global
intellectual property (IP) portfolio with protection extending
through to at least 2040 in all major markets. The Company’s
proprietary manufacturing processes yield 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’s Biologics License Application to seek approval of
its product candidate RYONCIL™ (remestemcel-L) for pediatric
steroid-refractory acute graft versus host disease (acute GVHD) has
been accepted for priority review by the United States Food and
Drug Administration (FDA), and if approved, product launch in the
United States is expected in 2020. Remestemcel-L is also being
developed for other inflammatory diseases in children and adults
including moderate to severe acute respiratory distress syndrome.
Mesoblast is completing Phase 3 trials for its product candidates
for advanced heart failure and chronic low back pain. 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
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 (including any decision that the FDA may make
based upon the recommendation of the ODAC in relation to the
efficacy of remestemcel-L), 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; 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:
MediaJulie
MeldrumT: +61 3 9639 6036E:julie.meldrum@mesoblast.com |
Kristen BothwellT: +1 917 613
5434E:kbothwell@rubenstein.com |
InvestorsSchond
GreenwayT: +212 880 2060E: schond.greenway@mesoblast.com |
Paul HughesT: +61 3 9639
6036E: paul.hughes@mesoblast.com |
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