Expanded Access Protocol Initiated for Compassionate Use of Remestemcel-L in Children With Multisystem Inflammatory Syndrome ...
July 06 2020 - 6:00AM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that an
expanded access protocol (EAP) has been initiated in the United
States for compassionate use of its allogeneic mesenchymal stem
cell (MSC) product candidate remestemcel-L in the treatment of
COVID-19 infected children with cardiovascular and other
complications of multisystem inflammatory syndrome (MIS-C).
Patients aged between two months and 17 years may receive one or
two doses of remestemcel-L within five days of referral under the
EAP.
The protocol was filed with the United States Food and Drug
Administration (FDA) and provides physicians with access to
remestemcel-L for an intermediate-size patient population1 under
Mesoblast’s existing Investigational New Drug (IND) application.
According to the FDA, expanded access is a potential pathway for a
patient with an immediately life-threatening condition or serious
disease or condition to gain access to an investigational medical
product for treatment outside of clinical trials when no comparable
or satisfactory alternative therapy options are available.
MIS-C is a life-threatening complication of COVID-19 in
otherwise healthy children and adolescents that includes massive
simultaneous inflammation of multiple critical organs and their
vasculature. In approximately 50% of cases this inflammation
is associated with significant cardiovascular complications that
directly involve heart muscle and may result in decreased cardiac
function. In addition, the virus can result in dilation of coronary
arteries with unknown future consequences. Recent articles from
Europe and the United States have described this disease in
detail.2-5
Mesoblast Chief Medical Officer Dr Fred Grossman said: “The
extensive body of safety and efficacy data generated to date using
remestemcel-L in children with graft versus host disease suggest
that our cellular therapy could provide a clinically important
therapeutic benefit in MIS-C patients, especially if the heart is
involved as a target organ for inflammation. Use of
remestemcel-L in children with COVID-19 builds on and extends the
potential application of this cell therapy in COVID-19 cytokine
storm beyond the most severe adults with acute respiratory distress
syndrome.”
Remestemcel-L Remestemcel-L is an
investigational therapy comprising culture-expanded mesenchymal
stem cells derived from the bone marrow of an unrelated donor and
is administered in a series of intravenous infusions. Remestemcel-L
is believed to have immunomodulatory properties to counteract the
inflammatory processes that are implicated in several diseases 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.www.clinicaltrials.gov; NCT04456439 2.Lancet2020; May 7. DOI:
https://doi.org/10.1016/S0140-6736(20)31094-13.Lancet. 2020; (May
13)
https://doi.org/10.1016/S0140-6736(20)31103-X4.https://www.nejm.org/doi/full/10.1056/NEJMoa20217565.https://www.nejm.org/doi/full/10.1056/NEJMoa2021680
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
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; 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. 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 |
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InvestorsSchond GreenwayT: +212 880
2060E: schond.greenway@mesoblast.com |
Paul Hughes T: +61 3 9639
6036E: paul.hughes@mesoblast.com |
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