Appendix 4C Quarterly Activity Report
July 29 2021 - 8:06PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
provided an update on its pipeline of late-stage product
candidates, and an activity report for the fourth quarter ended
June 30, 2021.
“During the quarter we made significant progress in both
regulatory and clinical outcomes for our lead product candidate,
remestemcel-L. FDA’s CBER has recently recommended the next steps
in the potential approval pathway for remestemcel-L in the
treatment of steroid-refractory acute graft versus host disease in
children,” said Silviu Itescu, Chief Executive of Mesoblast.
“Additionally, as COVID infections continue to surge, the 90-day
survival outcomes from the remestemcel-L trial in adults with COVID
ARDS demonstrated the potential for durable benefit of this therapy
in certain segments experiencing the most extreme complication of
this disease.”
Key operational highlights:
- Mesoblast met with the United States
Food & Drug Administration (FDA) on potential pathways to
regulatory approval of its lead technology platform product
remestemcel-L for steroid refractory acute graft versus host
disease (SR-aGVHD) in children and acute respiratory distress
syndrome (ARDS) in adults with COVID-19
- Regarding SR-aGVHD, FDA’s Center for
Biologics Evaluation and Research (CBER) recommended that Mesoblast
as a next step discuss with CBER’s review team at the Office of
Tissue and Advanced Therapies (OTAT) our approach to address
certain outstanding chemistry, manufacturing and controls (CMC)
items, including potency assays, which could support a resubmission
of the current Biologic License Application (BLA) with a six-month
review period
- Regarding COVID ARDS, Mesoblast met
with the FDA this week to determine the registration pathway for
approval of remestemcel-L in this indication, with formal minutes
expected in coming weeks
- Results from the randomized
controlled trial of remestemcel-L in 222 ventilator-dependent
COVID-19 patients with moderate/severe acute respiratory distress
syndrome (ARDS) were recently highlighted at the biennial Stem
Cells, Cell Therapies, and Bioengineering in Lung Biology and
Diseases conference hosted by the University of Vermont,
Burlington, VT, and at the International Society for Cell &
Gene Therapy (ISCT) Scientific Signatures Series event on Cell and
Gene-Based Therapies in Lung Diseases and Critical Illnesses
- The presented data included improved
respiratory function in patients treated with remestemcel-L, as
well as 90-day survival outcomes showing remestemcel-L
significantly reduced mortality by 48% at 90 days compared to
controls in a pre-specified analysis of 123 treated patients under
65 years old
- Mesoblast has entered into a license
and collaboration agreement with Novartis for the development,
manufacture, and commercialization of remestemcel-L, with an
initial focus on the treatment of acute respiratory distress
syndrome (ARDS), including that associated with COVID-19. The
agreement remains subject to certain closing conditions, including
time to analyze the results from this COVID-19 ARDS trial
- Mesoblast filed requests and expects
to hold meetings with the FDA during the next two quarters to
discuss the pathways to US regulatory approvals for its second
technology platform rexlemestrocel-L following the recently
completed Phase 3 trials in patients with chronic heart failure and
chronic inflammatory back pain due to degenerative disc
disease
- Mesoblast and its partner in Europe
and Latin America, Grünenthal, amended their collaboration
agreement in line with a strategy to achieve regulatory
harmonization, cost efficiencies and streamlined timelines aiming
to leverage the results from a planned US trial in support of
potential product approvals in both US and EU
Cash Flow Report for the Fourth Quarter
FY2021
Cash on hand at the end of the quarter was US$136.4 million.
Total Operating Activities resulted in net cash usage of US$20.7
million in the quarter ended June 30, 2021. This included an
investment of US$10.8 million associated with remestemcel-L for
SR-aGVHD and COVID-19 ARDS. Specifically:
- Sales of TEMCELL® HS Inj.1 in Japan
for the treatment of aGVHD continue to recover from the effects of
the temporary shutdown in production during mid-2020 which was
undertaken in order to increase capacity to meet growing demand for
the product
- Revenues from TEMCELL® royalties for
the quarter ended June 30, 2021 were US$1.9 million compared to
US$0.7 million in the quarter ended June 30, 2020. Royalty receipts
for the quarter were US$1.9 million, reflecting revenues recognized
in the prior quarter
- Research and Development payments
were US$5.9 million for the current quarter. This comprises
payments for the recently completed trials in COVID-19 ARDS, CHF
and CLBP, as well as potency assay work in support of these
programs
- Product manufacturing &
operating costs and manufacturing commercialization payments were
US$8.1 million for the current quarter, the majority being for
commercial manufacturing and inventory build in anticipation for
product launch of remestemcel-L
- Payments to Related Parties,
detailed in Item 6 of the Appendix 4C cash flow report for the
quarter, comprise approximately US$643,000 in Non-Executive
Director fees and Executive Director’s salary
A copy of the Appendix 4C – Quarterly Cash Flow Report for the
fourth quarter FY2021 is available on the investor page of the
company’s website www.mesoblast.com.
About the Trial of Remestemcel-L in Acute Respiratory
Distress Syndrome (ARDS) due
to COVID-19The trial enrolled 222
mechanically ventilated COVID-19 patients with moderate/severe ARDS
across the US, of whom 217 were randomized 1:1 and received either
standard of care alone or standard of care plus 2 intravenous
infusions of remestemcel-L at a dose of 2 million cells/kg 3-5 days
apart. This was the same remestemcel-L dosing regimen used in the
earlier compassionate use program where 11 of 12 patients were
younger than 65 and 75% successfully came off ventilatory
support.
The trial was halted in December 2020 after the Data Safety
Monitoring Board (DSMB) performed a third interim analysis on the
trial’s first 180 patients, noting that the trial was not likely to
meet the 30-day mortality reduction endpoint at the planned 300
patient enrolment. The trial was powered to achieve a primary
endpoint of 43% reduction in mortality at 30 days for treatment
with remestemcel-L on top of maximal care. The DSMB recommended
that the trial complete with the enrolled 222 patients, and that
all be followed-up as planned.
At follow-up through day 60, remestemcel-L reduced overall
mortality by 14% across the entire population of treated patients
(n=217), and by 46% in the pre-specified population of patients
under age 65 (n=123). In an exploratory analysis through day 60,
remestemcel-L reduced mortality by 75% and increased days alive off
mechanical ventilation in patients under age 65 when combined with
dexamethasone, in comparison with controls on dexamethasone.
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
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 has completed Phase 3 trials of rexlemestrocel-L for
advanced chronic heart failure and chronic low back pain.
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. 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
References / Footnotes
- TEMCELL® 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 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 |
|
|
|
Kristen Bothwell |
|
T: +1 917 613 5434 |
|
E:
kbothwell@rubenstein.com |
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