Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
provided an update on upcoming milestones for its lead product
candidate remestemcel-L, and an activity report for the fourth
quarter ended June 30, 2020.
Mesoblast Chief Executive Dr Silviu Itescu stated:
“Remestemcel-L has two imminent major milestones, the interim
analysis in the ongoing Phase 3 trial of remestemcel-L in COVID-19
patients with acute respiratory distress syndrome and the FDA
advisory committee panel review of our submission for potential
approval of RYONCIL™ (remestemcel-L) in children with
steroid-refractory acute graft versus host disease. Together with
the upcoming Phase 3 read-outs in chronic heart failure and back
pain, these key milestones will take the Company into the most
significant period in its history.”
Remestemcel-L Phase 3 Trial for COVID-19 Acute
Respiratory Distress Syndrome
The independent Data Safety Monitoring Board (DSMB) has set
a date for early September to complete the first interim
analysis of the Phase 3 trial of remestemcel-L in
ventilator-dependent COVID-19 patients with moderate to
severe acute respiratory distress syndrome (ARDS). The trial’s
first 90 patients will have completed 30 days of follow up during
August, after which the DSMB will perform an interim analysis
review of the safety and efficacy data. The DSMB will then inform
Mesoblast on whether the trial should proceed as planned, or should
stop early.
There are currently no approved treatments for COVID-19 ARDS,
the primary cause of death in patients infected with
COVID-19.
The clinical protocol evaluating remestemcel-L in patients in
the Phase 3 trial was based on results from a pilot study using
remestemcel-L under emergency compassionate care at Mt Sinai
Hospital in New York, with 75% (nine of 12) of patients with
moderate to severe ARDS successfully taken off a ventilator and
discharged from hospital within a median of 10 days.
The placebo-controlled, double-blinded trial commenced
enrollment in the reporting quarter of ventilator-dependent
patients in intensive care units with moderate to severe COVID-19
ARDS randomized (1:1) to receive either two intravenous infusions
of remestemcel-L three to five days apart or placebo on top of
maximal care. The primary endpoint is all-cause mortality within 30
days of randomization, with the key secondary endpoint being the
number of days off mechanical ventilator support.
Up to 30 leading medical centers across the United States are
expected to participate in the trial, which is expected to complete
recruitment in September 2020.
Remestemcel-L for Steroid-Refractory Acute Graft Versus
Host Disease
The Oncologic Drugs Advisory Committee (ODAC) of the United
States Food and Drug Administration (FDA) has scheduled a meeting
for August 13, 2020 to review data supporting the Company’s
Biologics License Application (BLA) for approval of RYONCIL in the
treatment of steroid-refractory acute graft versus host disease
(SR-aGVHD) in children.
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.
Although the FDA will consider the recommendation of the panel,
the final decision regarding the approval of the product is made by
the FDA solely, 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, RYONCIL is planned for launch in
the United States in 2020 with product inventory in place.
Cash Flow Report for the Fourth Quarter
FY2020
Cash on hand at the end of the quarter was US$129.3 million
(A$188.4 million). Mesoblast completed a US$90 million (A$138
million) capital raise from global institutional investors in May
2020. Over the next 12 months, Mesoblast may have access to an
additional US$67.5 million through existing financing facilities
and strategic partnerships.
Total Operating Activities resulted in net cash usage of US$19.6
million in the quarter ended June 30, 2020, as the Company
continues to prepare for the potential approval and commercial
launch of RYONCIL in the United States.
- Research and Development payments were US$6.7 million,
primarily for our Phase 3 programs.
- Manufacturing payments were US$7.4 million for commercial
manufacturing investment to support potential launch of
RYONCIL.
- Royalty receipts received from JCR Pharmaceuticals Co. Ltd for
the sales of TEMCELL HS Inj.(R)1 in Japan for the
treatment of aGVHD were US$2.1 million.
- Payments to Related Parties, detailed in Item 6 of the Appendix
4C cash flow report for the quarter, comprise approximately
US$361,000 in Non-executive Director fees and Executive Director’s
salary.
A copy of the Appendix 4C – Quarterly Cash Flow Report for the
fourth quarter FY2020 is available on the investor page of
www.mesoblast.com.
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 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 (ARDS).
Following positive pilot trial results under compassionate care
use, remestemcel-L is being evaluated in a Phase 3 randomized
controlled trial in 300 patients with COVID-19 ARDS in the United
States. 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
1.TEMCELL®1 HS Inj. is a registered trademark of JCR
Pharmaceuticals Co. Ltd.
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, 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; estimates of
Mesoblast’s expenses, future revenues, capital requirements and its
needs for additional financing; Mesoblast’s financial performance;
developments relating to Mesoblast’s competitors and industry; 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 Hughes T: +61 3 9639
6036E: paul.hughes@mesoblast.com |
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