Mesoblast Provides Update Following Meeting With FDA’s OTAT on Remestemcel-L for Children With Acute Graft Versus Host Disease
December 30 2021 - 6:38PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
provided a regulatory update on remestemcel-L for
steroid-refractory acute graft versus host disease in children
following its recent meeting with the FDA’s OTAT. Mesoblast
requested the meeting to address the appropriateness of a potency
assay related to remestemcel-L’s proposed immunomodulatory
mechanism of action as well as the approach to outstanding CMC
items identified in the CRL.
OTAT indicated that Mesoblast’s approach to address outstanding
CMC items is reasonable, that the in vitro immunomodulatory
activity of remestemcel-L proposed by Mesoblast as a measure of its
potency is a reasonable CQA for the product in the treatment of
children with SR-aGVHD, and the relevance of this immunomodulatory
activity to clinical outcomes should be established.
Mesoblast has now generated substantial new data which it
believes establish the relevance of the proposed in vitro
immunomodulatory activity of remestemcel-L to the clinical effect
of the product in the completed Phase 3 trial in pediatric
SR-aGVHD, including to survival outcomes and biomarkers of the
product’s in vivo activity. Mesoblast will provide these new data
to OTAT and address other remaining CRL items as required for the
BLA resubmission.
By demonstrating the relevance of the in vitro potency assay to
clinical outcomes, Mesoblast believes it will be able to show that
the remestemcel-L product used in the Phase 3 trial in pediatric
SR-aGVHD was standardized as to identity, strength, quality,
purity, and dosage form, and that this will address OTAT’s
recommendation for an additional adequate and well-controlled
study.
Mesoblast continues to be in a well-established process with
FDA’s Center for Biologics Evaluation and Research (CBER), and if
the resubmission is accepted, CBER will consider the adequacy of
the clinical data in the context of the related CMC issues noted
above.
About Steroid-Refractory Acute Graft Versus Host Disease
(SR-aGVHD) GVHD is a severe inflammation in the
bloodstream caused by complications of bone marrow transplants. The
disease occurs in up to 50% of the 30,000 patients who receive an
allogeneic bone marrow transplant each year, primarily during
treatment for blood cancers. In patients with the most severe form
of GVHD, mortality can be as high as 90%. There are no therapies
approved for treating SR-aGVHD in children under the age of 12.
About Mesoblast Mesoblast is a world leader in
developing allogeneic (off-the-shelf) cellular medicines for the
treatment of severe and life-threatening inflammatory conditions.
The Company has leveraged its proprietary mesenchymal lineage cell
therapy technology platform to establish a broad portfolio of
late-stage product candidates which respond to severe inflammation
by releasing anti-inflammatory factors that counter and modulate
multiple effector arms of the immune system, resulting in
significant reduction of the damaging inflammatory process.
Mesoblast has a strong and extensive global intellectual
property portfolio with protection extending through to at least
2041 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 is developing product candidates for distinct
indications based on its remestemcel-L and rexlemestrocel-L stromal
cell technology platforms. 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. Rexlemestrocel-L is in
development for advanced chronic 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 press release
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
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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
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and accordingly, you should not place undue reliance on these
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Release authorized by the Chief Executive.
For more information, please contact:
Corporate Communications / Investors |
Media |
Paul Hughes |
Sumit Media |
T: +61 3 9639 6036 |
Grant Titmus |
E: investors@mesoblast.com |
T: +61 419 388 161 |
|
E: grant@sumitmedia.com.au |
|
|
|
Rubenstein |
|
Nadine Woloshin |
|
T: +1 917-699-9456 |
|
E: nwoloshin@rubenstein.com |
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