Mesoblast Submits Completed Biologics License Application for Ryoncil™ to US FDA
February 03 2020 - 6:00AM
Mesoblast Limited (Nasdaq: MESO; ASX:MSB), global leader in
cellular medicines for inflammatory diseases, today announced that
it has submitted its completed Biologics License Application (BLA)
to the United States Food and Drug Administration (US FDA) for
Ryoncil™ (remestemcel-L), its lead allogeneic cell therapy for the
treatment of children with steroid-refractory acute graft versus
host disease (SR-aGVHD).
Mesoblast filed the final module of the rolling BLA submission,
covering quality control and manufacturing, with the FDA on January
31. The Company has requested Priority Review of the BLA by
the FDA under the product candidate’s existing Fast Track
designation for SR-aGVHD. If approved, RYONCIL is expected to be
launched in the US in 2020.
Mesoblast Chief Executive Dr Silviu Itescu stated: “This is a
major corporate milestone for Mesoblast. We look forward to working
closely with the FDA to potentially bring RYONCIL to market and
providing our innovative biologic therapy to the many children with
this life-threatening condition.”
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, and these numbers are
increasing1. 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 care2,3. There are currently no
FDA-approved treatments in the US for children under 12 with
SR-aGVHD.
Mesoblast’s product candidate RYONCIL has been used in 309
children with SR-aGVHD across three separate studies. RYONCIL
was used as salvage therapy in an expanded access program in 241
children with SR-aGVHD (80% Grade C/D) who failed institutional
standard of care. RYONCIL was also used as first-line therapy in
Mesoblast’s open-label Phase 3 trial in 55 children with SR-aGVHD,
89% of whom had Grade C/D disease.
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.
References
- 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.
- 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.
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 cell therapy technology platform to establish a broad
portfolio of commercial products and late-stage product candidates.
Mesoblast has filed a Biologics License Application to the United
States Food and Drug Administration (FDA) to seek approval of its
product candidate Ryoncil™ for steroid-refractory acute graft
versus host disease, and is completing Phase 3 trials for its
advanced heart failure and chronic low back pain product
candidates. Two products have been commercialized in Japan and
Europe by its licensees, and it has established commercial
partnerships in Europe and China for certain Phase 3 assets.
Mesoblast’s proprietary manufacturing process yields
industrial-scale, frozen, off-the-shelf, cellular medicines based
on its mesenchymal lineage cell platform technology. These cell
therapies, with defined pharmaceutical release criteria, are
planned to be readily available to patients
worldwide. Mesoblast has locations in Melbourne, New York,
Singapore and Texas 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 |
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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