Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced the United States Food and Drug Administration (FDA) has
granted its second generation allogeneic, STRO3-immunoselected, and
industrially manufactured stromal cell therapy Revascor®
(rexlemestrocel-L) Regenerative Medicine Advanced Therapy (RMAT)
designation following submission of results from the randomized
controlled trial in children with hypoplastic left heart syndrome
(HLHS), a potentially life threatening congenital heart condition.
Mesoblast Chief Executive Silviu Itescu said:
“We appreciate FDA’s support in designating REVASCOR both RMAT and
RPD status, a recognition of the potential impact of our therapy on
the long-term adverse outcomes of these desperately ill children.
Under the RMAT designation, we plan to meet with FDA to discuss a
potential approval pathway in this indication.”
Earlier this year, FDA granted REVASCOR both
Rare Pediatric Disease Designation (RPDD) and Orphan-Drug
Designation (ODD) for treatment of children with HLHS. RPDD
demonstrates that the disease is serious or life-threatening and
the manifestations primarily affect individuals aged from birth to
18 years, including age groups often called neonates, infants,
children, and adolescents, and that the disease is a rare disease
or condition. On FDA approval of a BLA for REVASCOR for the
treatment of HLHS, Mesoblast may be eligible to receive a Priority
Review Voucher (PRV) that can be redeemed for any subsequent
marketing application or may be sold or transferred to a third
party.
RMAT designations aim to expedite the
development of regenerative medicine therapies intended to treat,
modify, reverse, or cure a serious or life-threatening disease or
condition where preliminary clinical evidence indicates that the
drug has the potential to address unmet medical needs for the
disease or condition. An RMAT designation for rexlemestrocel-L
provides all the benefits of Breakthrough and Fast Track
designations, including rolling review and eligibility for priority
review on filing of a Biologics License Application (BLA).
Results from a blinded, randomized,
placebo-controlled prospective trial of REVASCOR conducted in the
United States in children with HLHS were published in the December
2023 issue of the peer reviewed The Journal of Thoracic and
Cardiovascular Surgery Open (JTCVS Open).1 In the HLHS trial
conducted in 19 children, a single intramyocardial administration
of REVASCOR at the time of staged surgery resulted in the desired
outcome of significantly larger increases in left ventricular (LV)
end-systolic and end-diastolic volumes over 12 months compared with
controls as measured by 3D echocardiography, (p=0.009 & p=0.020
respectively).
These changes are indicative of clinically
important growth of the small left ventricle, facilitating the
ability to have a successful surgical correction, known as full
biventricular (BiV) conversion, which allows for a normal two
ventricle circulation with the surgically repaired left ventricle
taking over circulatory support to the body. Without full BiV
conversion the right heart chamber is under excessive strain with
increased risk of heart failure, liver failure, and death.
About Hypoplastic Left Heart Syndrome
(HLHS)HLHS is a severe congenital heart disease in which
the left side of the heart does not fully develop and effective
pumping of oxygenated blood by the left ventricle to the rest of
the body is reduced. Without immediate surgery after birth, the
prognosis is dismal with HLHS overall being responsible for 25% to
40% of all neonatal cardiac mortality.2 In the longer term, surgery
that creates a two-ventricle series circulation with the left
ventricle (LV) pumping blood to the body and the right ventricle
pumping blood to the lungs is the ideal anatomic repair.
Unfortunately, achievement of this objective is limited by the
inability in most patients for the left ventricle to grow
sufficiently to support the circulation to the body.
About
Revascor® (rexlemestrocel-L) in
Heart DiseaseREVASCOR is an allogeneic preparation of
immunoselected and culture-expanded mesenchymal precursor cells
which have been shown previously to have multiple
mechanisms-of-action that may be beneficial to children with HLHS
including neovascularization, anti-fibrosis, anti-apoptosis,
immunomodulation, reduction in inflammation, and reversal of
endothelial dysfunction. In the DREAM-HF randomized sham-placebo
controlled prospective trial of REVASCOR in 565 randomized adult
patients with heart failure with low ejection fraction (HFrEF), a
single intramyocardial administration of REVASCOR into the left
ventricle resulted in significant improvement in LV ejection
fraction at 12 months,3 indicative of strengthened overall LV
systolic function. The greatest treatment benefit was on 2- and
3-Point Major Adverse Cardiovascular Events (MACE), particularly in
patients with HFrEF with ischemia and inflammation, who are at
highest risk of mortality.4
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
- Wittenberg RE, Gauvreau K, Leighton J, Moleon-Shea M, Borow KM,
Marx GR, Emani SM, Prospective randomized controlled trial of the
safety and feasibility of a novel mesenchymal precursor cell
therapy in hypoplastic left heart syndrome, JTCVS Open Volume 16,
Dec 2023, doi: https://doi.org/10.1016/j.xjon.2023.09.031
- Kritzmire, S. M, et al. (2022). Hypoplastic left heart
syndrome.
- Perin EC. Et al. Randomized Trial of Targeted Transendocardial
Mesenchymal Precursor Cell Therapy in Patients with Heart Failure.
JACC Vol. 81, No. 9, 2023.
https://doi.org/10.1016/j.jacc.2022.11.061
- Perin EC. Et al. Mesenchymal precursor cells reduce mortality
and major morbidity in ischaemic heart failure with inflammation:
DREAM-HF. Eur J Heart Fail 2024.
https://doi.org/10.1002/ejhf.3522
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 |
|
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