Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, announced
that the United States Food and Drug Administration (FDA) has
accepted its Biologics License Application (BLA) resubmission for
Ryoncil® (remestemcel-L) in the treatment of children with
steroid-refractory acute graft versus host disease (SR-aGVHD). FDA
considers the resubmission to be a complete response and Mesoblast
anticipates a decision on or before the FDA’s Prescription Drug
User Fee Act (PDUFA) goal date of January 7, 2025.
Mesoblast’s resubmission on July 8, 2024
addressed remaining CMC (Chemistry, Manufacturing, and Control)
items after being informed by FDA at the end of March 2024 that,
following additional consideration, the available clinical data
from the Phase 3 study MSB-GVHD001 appears sufficient to support
submission of the proposed BLA for remestemcel-L for treatment of
pediatric patients with SR-aGVHD. In May 2023, FDA conducted the
Pre-License Inspection (PLI) of the manufacturing process for
remestemcel-L, and this did not result in the issuance of any Form
483.
“We are pleased that FDA has accepted our BLA
resubmission for review, and look forward to the potential approval
of RYONCIL for children with SR-aGVHD,” said Mesoblast CEO Dr.
Silviu Itescu.
About Ryoncil®
(remestemcel-L)Mesoblast’s lead product candidate,
Ryoncil® (remestemcel-L), is an investigational therapy comprising
culture expanded mesenchymal stromal cells derived from the bone
marrow of an unrelated donor. It is administered to patients in a
series of intravenous infusions. RYONCIL has immunomodulatory
properties which counteract the inflammatory processes that are
implicated in SR-aGVHD by inhibiting activation and proliferation
of effector T cells, down-regulating the production of
pro-inflammatory cytokines, and enabling recruitment of
anti-inflammatory cells to involved tissues.
FDA granted remestemcel-L Fast Track
designation, a process to facilitate the development and expedited
review of therapies for serious conditions that fill unmet medical
needs, and Priority Review designation, which is given to drugs
that treat a serious condition and provide a significant
improvement in safety or effectiveness over existing
treatments.
About the Phase 3 Trial of
Ryoncil® (remestemcel-L) in
Children with Steroid-Refractory Acute Graft Versus Host
DiseaseThe Phase 3 Study GVHD001/002 was conducted in 54
children (89% Grade C/D) across 20 centers in the US where RYONCIL
was used as the first line of treatment for children who failed to
respond to steroids for acute GVHD.1 The trial met its
pre-specified primary endpoint, Day 28 Overall Response (OR), 70.4%
versus 45%, p=0.0003. An overall response at day 28 was highly
predictive of improved survival through day 100 (87% compared to
47% in patients that did not achieve day 28 OR p= 0.0001).
Compared with a matched control group of
pediatric subjects from the contemporaneous database of the Mount
Sinai Acute GVHD International Consortium (MAGIC) treated with best
available therapy, treatment with Ryoncil achieved higher Day 28 OR
(70% vs 43%) and higher Day 100 survival (74% vs 57%). A
propensity-matched study of outcomes in 25 children from
Mesoblast’s Phase 3 trial and 27 control children who received best
available treatment, including ruxolitinib, from the MAGIC database
showed that 67% of high-risk children (MAP scores >0.29) who
received Ryoncil achieved a Day 28 overall response and were alive
after 180 days compared to just 10% in both categories in the MAGIC
group.
In addition, results of a 4-year survival study
performed by the Center for International Blood and Marrow
Transplant Research (CIBMTR) on 51 evaluable patients with SR-aGVHD
who were enrolled in the Phase 3 trial, demonstrated durability of
the survival benefits, with 67% survival at 6 months, 63% survival
at 1 year, 51% at 2 years, and 49% survival through 4 years in
children with expected 2 year survival of just 25-38% using best
available therapy.2-4
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.
Survival outcomes have not improved over the
past two decades for children or adults with the most severe forms
of SR-aGVHD.2,9-10 The lack of any approved treatments for children
under 12 means that there is an urgent need for a therapy that
improves the dismal survival outcomes in children.
About Mesoblast Mesoblast (the
Company) 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, and biologic-resistant inflammatory bowel disease.
Rexlemestrocel-L is being developed 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
References / Footnotes
- Kurtzberg J. et al. A Phase 3,
Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo
Culture-Expanded Adult Human Mesenchymal Stromal Cells for the
Treatment of Pediatric Patients Who Failed to Respond to Steroid
Treatment for Acute Graft-versus-Host Disease. Biol Blood Marrow
Transplant 26 (2020) 845-854
- 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.
- 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.
- Berger M, Pessolano R, Carraro F,
Saglio F, Vassallo E, Fagioli F. Steroid-refractory acute
graft-versus-host disease graded III-IV in pediatric patients. A
mono-institutional experience with a long-term follow-up. Pediatric
Transplantation. 2020; 24(7):e13806
- Biavasco F, Ihorst G, Wasch R, Wehr
C, Bertz H, Finke J, Zeiser R. Therapy response of
glucocorticoid-refractory acute GVHD of the lower intestinal tract.
Bone Marrow Transplantation. 2022
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 any future decision that the FDA
may make on the BLA for remestemcel-L for pediatric patients with
SR-aGVHD), 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 |
Mesoblast (NASDAQ:MESO)
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