Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced top-line long-term survival results for remestemcel-L
from its pivotal Phase 3 trial (GVHD-001) in children with
steroid-refractory acute graft-versus-host disease (SR-aGVHD). The
results showed durable survival through 4 years of follow-up. These
new long-term survival data are a key component of the company’s
BLA resubmission to the FDA for remestemcel-L in the treatment of
children with SR-aGVHD, a life-threatening condition with no
approved treatments for children under 12 years.
“These exciting long-term results provide
further evidence of remestemcel-L’s potential as a highly effective
treatment for SR-aGVHD in children,” said Dr. Joanne Kurtzberg,
Jerome Harris Distinguished Professor of Pediatrics and Professor
of Pathology, Duke University Medical Center, the Phase 3 trial’s
principal investigator. “Responses are durable, reducing mortality
of this often lethal complication of hematopoietic stem cell
transplantation.”
A four-year observational cohort survival study
was performed by the Center for International Blood and Marrow
Transplant Research (CIBMTR) on 51 evaluable children with SR-aGVHD
who were enrolled in Mesoblast’s phase 3 clinical trial of
remestemcel-L across 20 centers in the US.
"CIBMTR is proud that the high-quality
comprehensive data included in our database supports critical
clinical advances such as this to improve outcomes for cellular
therapy patients," said Patricia Steinert, PhD, MBA, Executive
Scientific Director, CIBMTR MCW Associate Professor, Department of
Medicine Center for International Blood & Marrow.
Overall survival in the remestemcel-L cohort was
63% at 1 year, 51% at 2 years, and 49% at 4 years, with median
survival of 2 to 3 years. In recently published studies of children
or adults with SR-aGVHD who received best available therapy (BAT)
or the only FDA-approved agent for adults, ruxolitinib, 1 year
survival was 40-49% and 2 year survival was 25%-38%,1-4 with median
survival between 6.5 months and 11.1 months.3
Moreover, in the observational cohort study 88%
of children treated with remestemcel-L had severe disease with
highest mortality risk, defined by either IBMTR Grade C/D or
Glucksberg Grade III/IV, whereas only 22% to 68% of patients in the
other studies were considered to be severe.1-4 These results
reaffirm the potential significance of remestemcel-L as a
life-saving therapy for children with SR-aGVHD.
Mesoblast Chief Executive, Dr Silviu Itescu
said: “These substantial and durable long-term survival outcomes
seen in our Phase 3 trial with remestemcel-L are a cornerstone to
our BLA resubmission.”
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.5,6 SR-aGVHD is associated with mortality as
high as 90% and significant extended hospital stay costs.7,8 There
are currently no FDA-approved treatments in the US for children
under 12 with SR-aGVHD.
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 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
About the CIBMTR The Center for
International Blood and Marrow Transplant Research is a nonprofit
research collaboration between the National Marrow Donor Program
(NMDP)/Be The Match, in Minneapolis, and the Medical College of
Wisconsin, in Milwaukee. The CIBMTR collaborates with the global
scientific community to increase survival and enrich quality of
life for patients. The CIBMTR facilitates critical observational
and interventional research through scientific and statistical
expertise, a large network of centers, and a unique database of
long-term clinical data for more than 600,000 people who have
received hematopoietic cell transplantation and other cellular
therapies. Learn more at cibmtr.org or follow the CIBMTR on
Facebook, LinkedIn, or Twitter @CIBMTR.
References / Footnotes
- Rashidi A et al. Outcomes and
predictors of response in steroid-refractory acute
graft-versus-host disease: single-center results from a cohort of
203 patients. Biol Blood Bone Marrow Transplant 2019;
25(11):2297-2302.
- MacMillan ML et al. Pediatric acute
GVHD: clinical phenotype and response to upfront steroids. Bone
Marrow Transplant 2020; 55(1): 165-171
- Zeiser R et al. Ruxolitinib for
Glucocorticoid-Refractory Acute Graft-versus-Host Disease. N Engl J
Med 2020;382:1800-10.
- 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 BLA resubmission), 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 |
|
|
|
Rubenstein |
|
Tali Mackay |
|
E: tmackay@rubenstein.com |
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