Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced that the US Food and Drug Administration (FDA) has
provided a complete response to its Biologics License Application
(BLA) resubmission for remestemcel-L for the treatment of pediatric
steroid-refractory acute graft versus host disease (SR-aGVHD) and
requires more data to support marketing approval. To obtain the
data required, Mesoblast will conduct a targeted, controlled study
in the highest-risk adults with the greatest mortality. This adult
study is in line with our overall commercial strategy, which
envisioned a sequenced progression from pediatric to adult SR-aGVHD
indications. Adults comprise 80% of the SR-aGVHD market.
Mesoblast Chief Executive Silviu Itescu said:
“FDA’s inspection of our manufacturing process resulted in no
observed concerns, the Agency raised no safety issues across more
than 1300 patients who have received remestemcel-L to date, and
acknowledged improvements to our potency assay. We remain steadfast
in making remestemcel-L available to both children and adults
suffering from this devastating disease, and have received
substantial clarity in how to bring this much-needed product to
these patients”.
Mesoblast intends to enroll adult patients at
highest mortality risk with SR-aGVHD where existing therapy has not
improved outcomes and 90-day survival remains as low as 20-30%.1
Mesoblast has generated pilot data through its emergency IND
program in adults showing a survival benefit with remestemcel-L in
this target population. In line with our overall commercial
strategy to expand into the adult SR-aGVHD indication, Mesoblast
has already been working with leading investigators at various US
centers of excellence to establish the adult follow-on study
protocol, potentially utilizing established clinical trials
networks. The company will seek alignment with FDA on the trial
design for the adult study at a Type A meeting within 45 days.
Prior to the resubmission, FDA guided Mesoblast
to resolve outstanding chemistry, manufacturing and controls (CMC)
issues before initiating any additional clinical trial. FDA
completed the Pre-License Inspection (PLI) of the manufacturing
facility, did not issue any Form 483, and found no objectionable
conditions. In addition, FDA acknowledged in the resubmission
review that changes implemented appear to improve assay performance
relative to the original version of the assay used in the pediatric
Phase 3 trial.
Mesoblast has successfully met the pre-specified
primary endpoint, prospectively agreed with FDA, of a single-arm
Phase 3 trial in 54 children with SR-aGvHD. While the Oncologic
Drugs Advisory Committee of FDA in August 2020 voted 9:1 in favor
of remestemcel-L’s efficacy in a pediatric patient population, in
September 2020 FDA recommended further steps be undertaken to
obtain approval. The BLA resubmission of January 2023 included
long-term follow-up data from the Phase 3 trial by the Center for
International Blood and Marrow Transplant Research (CIBMTR) showing
50% survival through more than 4 years of follow-up for
remestemcel-L treated patients in the Phase 3 trial for whom less
than 20% survival at two years was expected based on disease
severity. The resubmission also included a post-hoc propensity
matched study showing 6 month survival was 67% with remestemcel-L
vs 10% with other unapproved therapies in highest-risk patients as
identified using the Mount Sinai Acute GVHD International
Consortium (MAGIC). These pediatric data provide further support
for use of remestemcel-L in the proposed study in high-risk adults
with SR-aGVHD.
Conference Call There will be a
webcast today, beginning at 8.00pm EDT (Thursday, August 3);
10.00am AEST (Friday, August 4);. It can be accessed via:
https://webcast.openbriefing.com/msb-mu-2023/
The archived webcast will be available on the
Investor page of the Company’s website: www.mesoblast.com
About Steroid-Refractory Acute Graft
Versus Host Disease Acute GVHD occurs in approximately 50%
of patients who receive an allogeneic bone marrow transplant (BMT).
Over 30,000 patients worldwide undergo an allogeneic BMT annually,
primarily during treatment for blood cancers, including about 20%
in pediatric patients.2,3
First-line treatment involves systemic
corticosteroids. A significant proportion of patients have severe
disease that is refractory to steroids. SR-aGVHD is associated with
mortality as high as 90% and significant extended hospital stay
costs.4,5
About Mesoblast Mesoblast (the
Company) 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 allogeneic stromal cell technology platforms.
Remestemcel-L is being developed for inflammatory diseases in
children and adults including steroid refractory acute graft versus
host disease, biologic-resistant inflammatory bowel disease, and
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
References / Footnotes
- Jagasia M et al. Ruxolitinib for
the treatment of steroid-refractory acute GVHD (REACH1): a
multicenter, open-label phase 2 trial. Blood. 2020 May 14; 135(20):
1739–1749
- Niederwieser D, Baldomero H, Szer
J. (2016) Hematopoietic stem cell transplantation activity
worldwide in 2012 and a SWOT analysis of the Worldwide Network for
Blood and Marrow Transplantation Group including the global
survey.
- HRSA Transplant Activity Report,
CIBMTR, 2019
- Westin, J., Saliba, RM., Lima, M.
(2011) Steroid-refractory acute GVHD: predictors and outcomes.
Advances in Hematology.
- Axt L, Naumann A, Toennies J (2019)
Retrospective single center analysis of outcome, risk factors and
therapy in steroid refractory graft-versus-host disease after
allogeneic hematopoietic cell transplantation. Bone Marrow
Transplantation.
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 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 (including our request to have a Type A
meeting with the FDA, the outcome of such a meeting, and any future
decision that the FDA may make on the BLA for remestemcel-L for
pediatric patients with SR-aGVHD), 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 |
BlueDot Media |
T: +61 3 9639 6036 |
Steve Dabkowski |
E: investors@mesoblast.com |
T: +61 419 880 486 |
|
E: steve@bluedot.net.au |
|
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|
Rubenstein |
|
Tali Mackay |
|
E: tmackay@rubenstein.com |
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