High Economic Burden in Children With Steroid Refractory Acute Graft Versus Host Disease
June 18 2019 - 6:00AM
Mesoblast Limited (ASX: MSB; Nasdaq: MESO), global leader in
cellular medicines for inflammatory diseases, presented health
economics and outcomes research data for pediatric acute graft
versus host disease (aGVHD) at the 24th European Hematology
Association (EHA) Congress in Amsterdam, Netherlands. Key
findings indicated that a steroid refractory state in aGVHD may
result in significant deterioration in quality of life (QOL) and
additional direct healthcare costs of up to $500,000 on average per
patient.
Acute GVHD is a potentially life-threatening
complication of an allogeneic bone marrow transplant, with the most
severe forms of the disease, Grades C/D or III/IV, frequently being
refractory to steroid therapy and associated with mortality rates
as high as 90%.1,2
In studies from the United States (US), children
with aGVHD have longer hospitalizations (incremental 17.9 – 45.4
days) and increased costs (incremental $114,698 - $224,000)
compared to recipients of allogeneic bone marrow transplants who
did not develop aGVHD.3 In steroid-refractory patients, the
magnitude of the burden may be larger, with a preliminary US
pediatric claims analysis suggesting additional direct healthcare
costs of up to $500,000 on average in steroid-refractory patients
compared to those who responded to steroids.4
Mesoblast recently initiated a rolling Biologic
License Application (BLA) to the United States Food and Drug
Administration for its product candidate remestemcel-L in children
with steroid-refractory aGVHD.
The BLA follows completion of Mesoblast’s Phase
3 trial which recruited 55 children with aGVHD - 89% of whom had
Grade C/D disease. The trial successfully met its primary
endpoint of increased Day 28 Overall Response compared with a
protocol-defined historical control rate (69% vs 45%, p=0.0003).
Overall Response at Day 28 predicted highest survival at Day 100
and Day 180, 85% and 79%, respectively. These data are consistent
with prior results from an Expanded Access Program in 241 children
where remestemcel-L was used as salvage therapy after failure of
steroids and other agents.
About Remestemcel-LMesoblast’s
lead product candidate, remestemcel-L, is an investigational
therapy comprising culture-expanded mesenchymal stromal cells
derived from the bone marrow of an unrelated donor. Remestemcel-L
is administered to patients in a series of intravenous infusions of
60 minutes or less in duration over the course of 4-8 weeks.
Remestemcel-L has demonstrated immunomodulatory properties to
counteract the inflammatory processes that are implicated in aGVHD
by down-regulating the production of pro-inflammatory cytokines,
increasing production of anti-inflammatory cytokines, and enabling
recruitment of naturally occurring anti-inflammatory cells to
involved tissues.
About Mesoblast Mesoblast
Limited (ASX: MSB; Nasdaq: MESO) is a world leader in developing
allogeneic (off-the-shelf) cellular medicines. The Company has
leveraged its proprietary technology platform to establish a broad
portfolio of late-stage product candidates with three product
candidates in Phase 3 trials – acute graft versus host disease,
chronic heart failure and chronic low back pain due to degenerative
disc disease. Through a proprietary process, Mesoblast selects rare
mesenchymal lineage precursor and stem cells from the bone marrow
of healthy adults and creates master cell banks, which can be
industrially expanded to produce thousands of doses from each donor
without the need for tissue matching. Mesoblast has facilities in
Melbourne, New York, Singapore and Texas and is listed on the
Australian Securities Exchange (MSB) and on the Nasdaq (MESO).
www.mesoblast.com
- Jagasia M, Arora M, Flowers ME, et al. Risk factors for acute
GVHD and survival after hematopoietic cell transplantation. Blood.
2012;119(1):296.
- 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
- European Hematology Association 2019 Congress Meeting: Abstract
PF718, The economic and humanistic burden of
graft-versus-host disease (GVHD) in pediatric patients: A
systematic literature review (SLR)
- Data on file: claim analysis with HealthCore Q42015
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
timing, progress and results of Mesoblast’s preclinical and
clinical studies in aGVHD; Mesoblast’s ability to advance its aGVHD
product candidate into, enroll and successfully complete, clinical
studies; the timing or likelihood of regulatory filings and
approvals for aGVHD; 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.
For further information, please contact:
Julie
Meldrum |
Schond
Greenway |
Corporate Communications |
Investor Relations |
T: +61 3 9639 6036 |
T: +1 212 880 2060 |
E: julie.meldrum@mesoblast.com |
E: schond.greenway@mesoblast.com |
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