Key Day 100 Survival Outcomes of Phase 3 Trial for Acute Graft Versus Host Disease Presented at 2018 ISSCR Annual Meeting
21 June 2018 - 8:00PM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced key Day
100 survival outcomes of its Phase 3 trial for remestemcel-L, an
allogeneic mesenchymal stem cell product candidate, in children
with steroid refractory acute Graft Versus Host Disease (aGVHD).
The results are being presented today at the 2018 annual meeting of
the International Society for Stem Cell Research (ISSCR), being
held in Melbourne from June 20-23.
The open-label Phase 3 trial enrolled 55 children with
steroid-refractory aGVHD (aged between two months and 17
years) in 32 sites across the United States, with 89% of
patients suffering from the most severe form, grade C/D
aGVHD. The trial was performed under an United States
Food and Drug Administration (FDA) Investigational New Drug
Application (NCT#02336230).
This trial previously met the primary endpoint of Day 28 overall
response rate (69% versus 45% historical control rate, p=0.0003).
Top line Day 100 results demonstrated 87% survival rate for Day 28
responders to remestemcel-L treatment (33/38), and an
overall survival rate of 75% (41/55). The multi-infusion regimen of
remestemcel-L was well tolerated.
Acute GVHD is associated with significant morbidity and is a
leading cause of mortality after allogeneic hematopoietic stem cell
transplantation for blood cancers or other conditions. Severe aGVHD
(determined by grade C/D, liver or gut involvement, or high risk
stratification) has the highest risk of failure to first-line
corticosteroids and high transplant-related
mortality1. Day 100 mortality can reach 70% in patients
who fail to respond to initial steroid therapy2-4, and 12-month
mortality approaches 90%5.
Mesenchymal stem cells have anti-inflammatory and
immunomodulatory biological activity that supports their
investigational use in
aGVHD6. The immunomodulatory actions of these
cells are triggered through receptor activation
by inflammation, resulting in regulation of multiple cellular
arms of the immune system that are central to aGVHD
pathogenesis.
Based on interactions with the FDA, Mesoblast believes that
successful results from the completed Phase 3 trial, together with
Day 180 safety, survival and quality of life parameters in these
patients, may provide sufficient clinical evidence to file for
accelerated approval of remestemcel-L in the United States, where
there are currently no approved products for steroid-refractory
aGVHD.
Mesoblast Chief Executive Dr Silviu Itescu said, “It is
wonderful to see that our unique cellular therapy has shown such
promising survival rates at Day 100 in children suffering from this
devastating disease. Our objective is to bring this new therapy to
market, and make it available to patients who are in desperate need
with this life-threatening complication of an allogeneic bone
marrow transplant.”
1. Jaqasia M, Arora M, Flowers ME, et al. Risk factors for
acute GVHD and survival after hematopoietic cell transplantation.
Blood. 2012; 119 (1): 296-307.2. MacMillan ML, DeFor TE,
Weisdorf DJ. The best endpoint for acute GVHD treatment trials.
Blood. 2010; 115 (26): 5412-5417.3. MacMillan ML, Couriel D,
Weisdorf DJ, et al. A phase 2/3 multicenter randomized clinical
trial of ABX-CBL versus ATG as secondary therapy for
steroid-resistant acute graft-versus-host disease. Blood. 2007; 109
(6): 2657-2662.4. Pidala J, Kim J, Field T, et al. Infliximab
for managing steroid-refractory acute graft-versus-host disease.
Biol Blood Marrow Transplant. 2009; 15 (9): 1116-1121.5. Arai
S et al, Poor outcome in steroid refractory graft versus host
disease with anti-thymocyte globulin treatment. Biol Blood
Marrow Transplant. 2002; 8: 155-160.6. Aggarwal S, Pittenger
MF. Human mesenchymal stem cells modulate allogeneic immune
responses. Blood. 2005; 105:1815-22.
About MesoblastMesoblast Limited (Nasdaq:MESO;
ASX:MSB) is a global leader in developing innovative cell-based
medicines. Through a proprietary process, Mesoblast selects highly
purified 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 that meet stringent release criteria, have lot to lot
consistency, and can be used off the shelf without the need for
tissue matching.
The Company has leveraged its proprietary technology platform to
establish a broad portfolio of late-stage product candidates.
Mesoblast’s allogeneic, ‘off-the-shelf’ cell product candidates are
being evaluated in their ability to target advanced stages of
diseases with high, unmet medical needs including cardiovascular
conditions, orthopedic disorders, immunologic and inflammatory
disorders and oncologic/hematologic conditions.
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 timing,
progress and results of Mesoblast’s preclinical and clinical
studies; Mesoblast’s ability to advance product candidates into,
enroll and successfully complete, clinical studies; the timing or
likelihood of regulatory filings and approvals; 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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