Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
provided an update on the potential pathway to marketing approval
for its lead product candidate remestemcel-L. It also provided a
financial report for the first quarter ended September 30, 2020.
Mesoblast Chief Executive Dr Silviu Itescu stated:
“We believe the immunomodulatory properties of remestemcel-L
position this potential therapy at the forefront of treatment for
severe and life-threatening inflammatory conditions, including
COVID-19 acute respiratory distress syndrome (ARDS) and
steroid-refractory acute graft versus host disease (SR-aGVHD). We
are pursuing an accelerated approval pathway for remestemcel-L in
the treatment of children with SR-aGVHD, and a parallel approval
pathway for COVID-19 ARDS if the randomized controlled Phase 3
trial is positive.”
Children with Steroid-Refractory Acute
Graft Versus Host DiseaseIn August, the Oncologic Drugs
Advisory Committee (ODAC)1 of the United States Food and Drug
Administration (FDA) voted 9:1 that the available data from a
single-arm Phase 3 trial and evidence from additional studies
support the efficacy of remestemcel-L in pediatric patients with
SR-aGVHD. Despite the overwhelming ODAC vote, in September 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.
Mesoblast believes that remestemcel-L meets the
criteria for accelerated approval as there are currently no
approved treatments for this life-threatening condition in children
under 12. Mesoblast has formally requested a Type A meeting
with the FDA to discuss a potential accelerated approval of the
Biologics License Application (BLA) for remestemcel-L for the
treatment of SR-aGVHD in children, with an additional randomized
controlled study in patients 12 years and older as a post-approval
requirement. Mesoblast expects this meeting will occur in
November.
Adults with COVID-19 ARDSAs cases
of COVID-19 surge in the United States and
globally, deaths continue to increase from ARDS
in ventilator-dependent patients as a result of an overactive
immune response in the lungs to COVID-19. It is now evident
that in both adults and children COVID-19 causes severe
inflammation of other organ systems in addition to the lungs,
including the heart, brain and kidneys. The immunomodulatory
mechanism of action of remestemcel-L may be beneficial in the
treatment of ARDS as well as in involvement of other organ
systems.
Indeed, nine of 12 ventilator-dependent adult
patients with COVID-19 ARDS who received two doses of remestemcel-L
under emergency compassionate use at New York’s Mt Sinai Hospital
were successfully discharged within a median of 10
days. Additionally, two COVID-19 infected children with
multisystem inflammatory syndrome (MIS-C) who received
remestemcel-L for severe heart failure fully recovered heart
function and were discharged within 30 hours of the second
dose.
To confirm these pilot data, remestemcel-L is
being evaluated for its potential to reduce mortality in a Phase 3
randomized controlled trial of up to 300 ventilator-dependent
adults with moderate or severe COVID-19 ARDS. The dosing
regimen in the Phase 3 is the same as in the pilot
trial. Trial enrollment across more than 20 hospitals in the
United States has surpassed 50% of the total 300 patient target.
The trial’s first 135 patients will complete 30 days of follow up
during October, after which the independent Data Safety Monitoring
Board (DSMB) will perform an interim analysis of the trial’s
primary endpoint of all-cause mortality within 30 days of
randomization. The DSMB is expected to inform Mesoblast in early
November on whether the trial should proceed as planned, or should
stop early. A further interim analysis is planned after 60% of
the trial has been enrolled.
Crohn’s DiseaseAccording to
recent estimates, more than three million people (1.3%) in the
United States alone have inflammatory bowel disease, with more than
33,000 new cases of Crohn’s disease and 38,000 new cases of
ulcerative colitis diagnosed every year.2-4 Despite recent
advances, approximately 30% of patients are primarily unresponsive
to anti-TNFα agents and even among responders, up to 10% will lose
their response to the drug every year. Up to 80% of patients with
medically-refractory Crohn’s disease eventually require surgical
treatment of their disease,5 which can have a devastating impact on
quality of life.
A randomized, controlled study of remestemcel-L
delivered by an endoscope directly to the areas of inflammation and
tissue injury in up to 48 patients with medically refractory
Crohn’s disease and ulcerative colitis has commenced at Cleveland
Clinic. Mesoblast’s objective is to confirm the potential for
remestemcel-L to induce luminal healing and early remission in a
wider spectrum of diseases with severe inflammation of the gut, in
addition to SR-aGVHD. The investigator-initiated study at
Cleveland Clinic is the first in humans using local cell delivery
in the gut, and will enable Mesoblast to compare clinical outcomes
using this delivery method with results from an ongoing randomized,
placebo-controlled trial in patients with biologic-refractory
Crohn’s disease where remestemcel-L was administered
intravenously.
Chronic Heart FailureIn the
United States alone, of more than 6.5 million patients with chronic
heart failure, there are more than 1.3 million patients with
advanced stage of the disease.6 The objective of treatment with
Mesoblast’s allogeneic cellular product candidate REVASCOR® is to
reduce or reverse the severe inflammatory process in the damaged
heart of these patients, and thereby prevent or delay further
progression of heart failure or death. In an earlier randomized
placebo-controlled 60-patient Phase 2 trial, a single
intra-myocardial injection of REVASCOR at the dose administered in
the subsequent Phase 3 trial prevented any hospitalizations or
deaths over three years of follow-up in patients with advanced
chronic heart failure. Results from Mesoblast’s randomized
placebo-controlled Phase 3 trial in patients with advanced forms of
New York Heart Association Class II or Class III disease are
expected during Q4 CY2020.
Chronic Low Back PainThere is a
significant need for a safe, efficacious and durable treatment in
patients with chronic low back pain due to severely inflamed
degenerative disc disease, affecting over 3.2 million patients in
the United States and approximately 4 million in the EU5.7 Results
from a Phase 3 randomized placebo-controlled trial evaluating
Mesoblast’s product candidate in patients with chronic low back
pain due to degenerative disc disease are expected during Q4
CY2020. In parallel, Mesoblast and its partner Grünenthal GmbH
are collaborating on the clinical protocol for a confirmatory Phase
3 trial in Europe.
Cash Flow Report for the First Quarter
FY2021Cash on hand at the end of the quarter
was US$108.1 million. 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 excluding product
inventory resulted in net cash usage of US$23.0 million in the
quarter ended September 30, 2020. In addition, $5.2 million
was invested in RYONCIL® (remestemcel-L) commercial inventory in
anticipation of product launch for either children with SR-aGVHD or
COVID-19 patients with moderate to severe ARDS.
- Research and Development payments were US$9.9 million for the
current quarter compared to US$7.8 million for quarter ended
September 30, 2019. The increase was primarily due to
recruitment activities for our Phase 3 trial of remestemcel-L in
COVID-19 patients with moderate to severe ARDS.
- Product manufacturing and operating costs were US$5.2 million
for the current quarter compared to US$3.5 million for quarter
ended September 30, 2019. The increase was primarily due to
investment in scale-up and increased capacity requirements of
remestemcel-L in parallel to development for COVID-19 ARDS.
- Royalty receipts received from JCR Pharmaceuticals Co. Ltd for
the sales of TEMCELL HS Inj.(R)8 in Japan for the
treatment of aGVHD were US$0.7 million for the current quarter
compared to US$1.7 million for the quarter ended September 30,
2019. The decrease was due to a temporary shutdown in
production as JCR expands its facility capacity to meet increasing
demand far in excess of its initial forecast (as announced by JCR
Pharmaceuticals on July 31, 2020).
- Payments to Related Parties, detailed in Item 6 of the Appendix
4C cash flow report for the quarter, comprise approximately
US$387,000 in Non-executive Director fees and Executive Director’s
salary.
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
1.This vote includes a change to the original vote
by one of the ODAC panel members after electronic voting closed.
2.CDC Facts and Figures 2015. 3.Globaldata Pharmapoint 2018.
4.Dahlhamer JM, MMWR Morb Mortal Wkly Rep. 2016;65(42):1166–1169.
5.Crohn’s and Colitis Foundation 6.AHA’s 2017 Heart Disease and
Stroke Statistics 7.Decision Resources: Chronic Pain December 2015
8.TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals
Co. Ltd.
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. 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:
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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