Mesoblast Receives FDA Regenerative Medicine Advanced Therapy Designation for Its Cell Therapy in Heart Failure Patients With...
21 December 2017 - 10:00PM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that
the United States Food and Drug Administration (FDA) has granted
Regenerative Medicine Advanced Therapy (RMAT) designation for its
novel mesenchymal precursor cell (MPC) therapy in the treatment of
heart failure patients with left ventricular systolic dysfunction
and left ventricular assist devices (LVADs). The RMAT
designation under the 21st Century Cures Act aims to expedite
the development of regenerative medicine therapies intended for the
treatment of serious diseases and life-threatening conditions.
This RMAT designation allows for multi-disciplinary,
comprehensive interactions with the FDA to support the efficient
development of and potential accelerated approval pathway for
Mesoblast’s allogeneic MPCs in the treatment of heart failure
patients with LVADs. The RMAT designation also offers eligibility
for priority review. Once the biologics license application (BLA)
for a product is approved, the FDA can require various
post-approval confirmatory commitments.
Mesoblast Chief Executive Silviu Itescu stated, “The RMAT
designation speaks to the strength of the clinical data generated
to date using our cell-based therapy in these heart failure
patients with LVADs who are at risk of high mortality and have
extremely limited treatment options. We are looking forward to
working closely with the FDA in advancing this program with the aim
of providing a new therapeutic option for these patients with
exceptionally high unmet clinical need.”
The basis of this RMAT designation grant came from the completed
study data set of a 30-patient randomized, blinded,
placebo-controlled pilot trial of Mesoblast’s MPCs at a dose of 25
million cells in heart failure patients with LVADs, and related
analyses. These preliminary clinical data suggest that Mesoblast’s
MPC product:
- improved native heart function,
- prolonged the time post LVAD implantation of a first
hospitalization for a non-surgical major gastrointestinal (GI)
bleeding event, and
- improved early survival rates in these LVAD recipients.
The results of the pilot study were published in the American
Heart Association Journal Circulation and can be found here.
A Phase 2b trial of MPCs at a dose of 150 million cells is
currently being conducted in 159 patients with heart failure and
LVADs and is funded by the NIH and the Canadian Institute of Health
Research. This trial has completed enrollment and the primary
endpoint will be reached in Q1 CY 2018.
FDA has invited Mesoblast to have a multidisciplinary
comprehensive discussion as soon as possible regarding the
development strategy and the evidence needed to achieve an approval
in an efficient manner.
For more information on RMAT designation, please see
https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm.
About Heart Failure Requiring LVADNew York
Heart Association Class IV heart failure affects more than 250,000
patients in the United States alone, with over 50,000 having
end-stage disease. The number of end-stage heart failure patients
is expected to rise in line with the 25% projected increase in
total heart failure patients between 2010 and 20301,2. There are
currently very few medical options for end-stage heart failure
patients, as only around 2,000 heart transplants can be performed
in the U.S. every year due to limited donor availability3. LVADs
have significantly improved survival for end-stage heart failure
patients, and are increasingly being used as a destination
therapy4,5. However, the 12-month mortality rates remain high, and
repeated hospitalizations are very common. The complications
arising from LVAD implantation have severely restricted their use
by the vast majority of the end-stage heart failure population, as
well as reducing its cost-effectiveness as a treatment.
About Mesoblast’s Allogeneic MPC Product Candidate for
the Treatment of Heart Failure:Mesoblast’s MPC-150-IM
product candidate at a dose of 150 million cells is currently being
evaluated in the following two ongoing randomized
placebo-controlled trials in patients with either advanced or
end-stage heart failure.
- A Phase 2b trial of MPC-150-IM at a dose of 150 million cells
in 159 patients with end-stage heart failure and LVADs. This
trial, which is funded by the United States National Institutes of
Health (NIH) and the Canadian Institute of Health Research and
sponsored by the Icahn School of Medicine at Mount Sinai Hospital,
New York, has completed enrollment. The primary endpoint will
be reached in Q1 CY 2018.
- A Phase 3 trial of MPC-150-IM at the same 150 million cell dose
in up to 600 patients with advanced heart failure. This trial
is expected to complete enrolment at the end of 2018.
The mechanism of action (MOA) by which MPC-150-IM is thought to
exert its effects in these patient populations is through secretion
of potent biomolecules which reduce damaging inflammation and
strengthen the native heart by induction of a mature blood vessel
network.
1 Heidenreich PA, et al. Forecasting the future of
cardiovascular disease in the United States: A policy statement
form the American Heart Association. Circulation 2011;123:933-9442
Heidenreich PA, et al. Forecasting the impact of heart failure in
the United States: a policy statement from the American Heart
Association. Circ Heart Fail 2013;6:606–6193
http://healthresearchfunding.org/24-heart-transplant-waiting-list-statistics4
Miller LW, Guglin M. Patient selection for ventricular assist
devices: a moving target. J Am Coll Cardiol 2013;61:1209–12215
Gustafsson G, Rogers JG. Left ventricular assist device therapy in
advanced heart failure: patient selection and outcomes. European
Journal of Heart Failure 2017;19,595-602
About MesoblastMesoblast
Limited (Nasdaq:MESO) (ASX:MSB) is a global leader in
developing innovative cell-based medicines. The Company has
leveraged its proprietary technology platform, which is based on
specialized cells known as mesenchymal lineage adult stem cells, to
establish a broad portfolio of late-stage product candidates.
Mesoblast’s allogeneic, ‘off-the-shelf’ cell product candidates
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
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, 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.
For further information, please contact:
Julie MeldrumCorporate CommunicationsT: +61 3 9639
6036E: julie.meldrum@mesoblast.com
Schond GreenwayInvestor RelationsT: +1 212 880
2060E: schond.greenway@mesoblast.com
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