Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, announced
today that the US Food and Drug Administration (FDA) has issued a
Complete Response Letter to its Biologics License Application (BLA)
for remestemcel-L for the treatment of pediatric steroid-refractory
acute graft versus host disease (SR-aGVHD). While the Oncologic
Drugs Advisory Committee (ODAC)1 of the FDA voted 9:1 that the
available data support the efficacy of remestemcel-L in pediatric
patients with SR-aGVHD, the FDA recommended that Mesoblast conduct
at least one additional randomized, controlled study in adults
and/or children to provide further evidence of the effectiveness of
remestemcel-L for SR-aGVHD. As there are currently no approved
treatments for this life-threatening condition in children under
12, Mesoblast will urgently request a Type A meeting with the FDA,
expected within 30 days, to discuss a potential accelerated
approval with a post-approval condition for an additional study.
Joanne Kurtzberg, MD, Jerome Harris Distinguished
Professor of Pediatrics, Director, Pediatric Blood and Marrow
Transplant Program, and Co-Director, Stem Cell Transplant
Laboratory Duke University Medical Center, said: “The Phase 3 trial
results showed that remestemcel-L provides a meaningful treatment
for children with SR-aGVHD who have a very dismal prognosis. I look
forward to having this much-needed therapy available to our
patients.”
Mesoblast is currently conducting a randomized,
controlled Phase 3 trial evaluating remestemcel-L in up to 300
ventilator-dependent adults with moderate to severe acute
respiratory distress syndrome (ARDS) due to COVID-19. A second
interim analysis by the trial’s independent Data Safety Monitoring
Board is expected in early November, with completion of patient
enrollment expected in December. COVID-19 ARDS is an inflammatory
disease with a similar profile of damaging inflammatory cytokines
as is seen in children with SR-aGVHD, and is the primary cause of
death in COVID-19 infection. The trial’s primary endpoint is
reduction of all-cause mortality within 30 days of
randomization.
The FDA also identified a need for further
scientific rationale to demonstrate the relationship of potency
measurements to the product’s biologic activity. Assays measuring
the potency of remestemcel-L will continue to be refined to provide
further scientific rationale for its use in severe inflammatory
diseases with high mortality risk, such as SR-aGVHD and COVID-19
ARDS.
Mesoblast Chief Executive Dr Silviu Itescu stated:
“We are working tirelessly to bring remestemcel-L to patients with
life threatening inflammatory conditions, including SR-aGVHD and
COVID-19 ARDS.”
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.2 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.3,4 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 Remestemcel-L Mesoblast’s
lead allogeneic cell therapy product candidate, remestemcel-L, is
an investigational therapy comprising culture-expanded mesenchymal
stem cells derived from the bone marrow of an unrelated donor.
Remestemcel-L is thought to have immunomodulatory properties to
counteract the cytokine storms that are implicated in various
inflammatory conditions 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. This vote includes
a change to the original vote by one of the ODAC panel members
after electronic voting closed. 2. 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. 3. Westin, J., Saliba, RM.,
Lima, M. (2011) Steroid-refractory acute GVHD: predictors and
outcomes. Advances in Hematology 2011;2011:601953. 4. 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
Conference Call An audio webcast
will begin at 9.15am Friday, October 2 AEST / 7.15pm Thursday,
October 1, 2020 EDT. The audio webcast can be accessed
via https://webcast.boardroom.media/mesoblast-limited/20200930/NaN5f7147e5581a8100190f7687 or Webcast
link
The archived webcast will be available on the
Investor page of the Company’s website www.mesoblast.com
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 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.
Remestemcel-L is being developed for inflammatory
diseases in children and adults including steroid-refractory acute
graft versus host disease and 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 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. All statements other than statements of
historical fact are forward-looking statements, which are often
indicated by terms such as “anticipate,” “believe,” “could,”
“estimate,” “expect,” “goal,” “intend,” “likely,” “look forward
to,” “may,” “plan,” “potential,” “predict,” “project,” “should,”
“will,” “would” and similar expressions and variations thereof. 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. The risks, uncertainties and other factors that may impact
our forward-looking statements include, but are not limited to: 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 (including
our request to have a Type A meeting with the FDA, the outcome of
such a meeting, and any future decision that the FDA may make on
the BLA for remestemcel-L for pediatric patients with SR-aGVHD);
and the pricing and reimbursement of Mesoblast’s product
candidates, if approved; 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. 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. Unless required by
law, 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 Board.
For further information, please contact:
Media Julie Meldrum T: +61 3 9639 6036
E:julie.meldrum@mesoblast.com |
Kristen Bothwell T: +1 917 613 5434 E:kbothwell@rubenstein.com |
|
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Investors Schond Greenway T: +212 880 2060
E: schond.greenway@mesoblast.com |
Paul Hughes T: +61 3 9639 6036
E: paul.hughes@mesoblast.com |
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