Consistent Outcomes Using Ryoncil™ as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Ac...
February 24 2020 - 6:00AM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that
aggregated results from 309 children treated with Ryoncil™
(remestemcel-L) were presented at the American Society for
Transplantation Cellular Therapy and the Center for International
Blood & Bone Marrow Transplant Research (TCT) meeting in
Orlando, Florida on February 22. The data showed that treatment
with RYONCIL across three separate trials resulted
in consistent treatment responses and survival
outcomes in children with steroid-refractory acute graft
versus host disease (SR-aGVHD).
Key findings and conclusions were:
- Consistent safety and efficacy were observed across the
continuum from first-line treatment after steroid failure through
the most challenging patients who received RYONCIL as salvage after
exhausting all other options.
- In the aggregated dataset, 204 of the 309 (66%) patients
achieved an overall response at Day 28 following a four-week course
of RYONCIL.
- Results were consistent across all grades of disease, including
most severe (IBMTR Grade C/D or Glucksberg Grade 3/4).
- In the most severe patients (Grade C/D), who accounted for 82%
of all treated patients, Day 28 overall response was
65%.
- Overall response at Day 28 was strongly predictive of survival
at Day 100 and Day 180.
- Day 28 responders were more than twice as likely to survive as
non-responders (84% vs 39% at Day 100, and 83% vs 38% at Day
180).
- RYONCIL was well tolerated with no infusion-related toxicity
and no identified safety concerns.
Mesoblast Chief Medical Officer Dr Fred Grossman said: “These
aggregated data from three studies demonstrate consistent efficacy
and safety of RYONCIL in children suffering from steroid refractory
acute graft versus host disease. If approved, RYONCIL has the
potential to be an effective and safe therapy to improve survival
outcomes in the most vulnerable population of children with severe
forms of this disease who can have mortality rates as high as 90
percent.”
In January, Mesoblast filed a Biologics License Application
(BLA) to the United States Food and Drug Administration (FDA) for
RYONCIL for the treatment of children with steroid-refractory
aGVHD. The Company has requested Priority Review of the BLA by the
FDA under the product candidate’s existing Fast Track designation.
If approved, RYONCIL is expected to be launched in the US in
2020.
About Acute GVHDAcute 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, and these numbers are increasing.1 In patients with the
most severe form of acute GVHD (Grade C/D or III/IV) mortality is
as high as 90% despite optimal institutional standard of care.2,3.
There are currently no FDA-approved treatments in the US for
children under 12 with SR-aGVHD.
About Ryoncil™ Mesoblast’s lead product
candidate, RYONCIL, is an investigational therapy comprising
culture- expanded mesenchymal stem cells derived from the bone
marrow of an unrelated donor. It is administered to patients in a
series of intravenous infusions. RYONCIL is believed to have
immunomodulatory properties to counteract the inflammatory
processes that are implicated in SR- 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.
References1. 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.2. Westin, J., Saliba, RM., Lima, M. (2011)
Steroid-refractory acute GVHD: predictors and outcomes. Advances in
Hematology.3. 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.
About MesoblastMesoblast 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 platforms
to establish a broad portfolio of commercial products and
late-stage product candidates. Mesoblast’s proprietary
manufacturing process yields 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 has filed a Biologics License Application to the
United States Food and Drug Administration (FDA) to seek approval
of its product candidate Ryoncil™ (remestemcel-L) for
steroid-refractory acute graft versus host disease (acute GvHD).
Remestemcel-L is also being developed for other rare diseases.
Mesoblast is completing Phase 3 trials for its rexlemestrocel
product candidates for advanced heart failure and chronic low back
pain. If approved, RYONCIL is expected to be launched in the United
States in 2020 for pediatric steroid-refractory acute GVHD. 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
Mesoblast’s 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.
Release authorized by the Chief Executive.
For further information, please contact: |
Julie Meldrum
Corporate Communications T: +61 3 9639 6036 E:
julie.meldrum@mesoblast.com |
Schond
GreenwayInvestor RelationsT: +1 212 880 2060E:
schond.greenway@mesoblast.com |
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