Mesoblast Limited (ASX:MSB; Nasdaq: MESO), global leader in
cellular medicines for inflammatory diseases, announced today that
the United States Food and Drug Administration (FDA) has granted
the company’s product candidate, rexlemestrocel-L, Orphan Drug
Designation for prevention of post-implantation mucosal bleeding in
end-stage chronic heart failure (CHF) patients who require a left
ventricular assist device (LVAD).
Mesoblast Chief Executive Dr Silviu Itescu said: “We are very
pleased that the FDA has granted our heart failure cell therapy
product candidate, Revascor, Orphan Drug designation for use in
patients with LVADs to prevent mucosal bleeding, including from the
gastrointestinal (GI) tract. We look forward to our upcoming
meeting with the FDA to discuss a potential approval pathway under
the product’s existing Regenerative Medicine Advanced Therapy
(RMAT) designation for this life-threatening condition.”
About Orphan Drug Designation The FDA’s Orphan
Drug Designation Program provides orphan status to drugs and
biologics which are defined as those intended for the safe and
effective treatment, diagnosis or prevention of rare diseases and
disorders that affect fewer than 200,000 people in the United
States. Orphan designation qualifies the sponsor of the drug for
various development incentives, including eligibility for seven
years of market exclusivity upon regulatory approval, exemption
from FDA application fees, tax credits for qualified clinical
trials, and other potential assistance in the drug development
process.
About RMAT Designation The FDA’s RMAT
Designation Program 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. RMAT designation allows for multi-disciplinary,
comprehensive interactions with the FDA to support efficient
development of and potential accelerated approval pathway for cell
therapy candidates. 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.
About End-Stage Heart Failure, LVADs, And Major
Gastrointestinal BleedingIn the United States, over 60,000
patients annually suffer from end-stage heart failure, and despite
optimal medical therapy these patients have a one-year mortality
exceeding 50%.1 The only options to increase survival in these
patients are the use of left ventricular assist devices (LVADs) or
heart transplants.
Due to the decline in organ donations and limited availability
of healthy donor hearts, the use of LVADs in end-stage heart
failure patients is gaining momentum, with approximately 5,500
LVADs implanted annually in the United States.2,3,4 However,
high rates of re-hospitalization remain a major obstacle to greater
LVAD use, with up to 40% of patients being re-hospitalized as a
result of major GI bleeding.5,6
About Revascor Mesoblast’s investigational cell
therapy Revascor consists of 150 million allogeneic mesenchymal
precursor cells (MPCs) and is being developed for injection into
heart muscle of patients with moderate-advanced or end-stage
chronic heart failure.
Revascor For End-Stage Heart Failure
Patients With LVADsIn a 30-patient placebo-controlled
pilot trial in end-stage heart failure patients with LVADs,
conducted by the United States National Institutes of Health (NIH),
a single intra-myocardial dose of Revascor resulted in a 70%
reduction in GI bleeding and associated hospitalizations. These
results supported the RMAT designation granted in December 2017 by
the FDA for use of Revascor in LVAD patients.
In a subsequent meeting in 2018, the FDA advised Mesoblast that
the defined endpoint of reduction in major GI bleeding and
rehospitalization is an appropriate clinically meaningful endpoint
and could be the basis of an approved indication for use of
Revascor given the life-threatening nature of the condition, and
the RMAT designation under which Revascor is being regulated.
In November 2018, NIH investigators presented results of a
159-patient randomized placebo-controlled Phase 2b clinical trial
at the American Heart Association Scientific Sessions, showing that
a single intra-myocardial dose of Revascor resulted in a 76%
reduction in major GI bleeding events and 65% reduction in related
hospitalizations in end-stage heart failure patients implanted with
an LVAD.
Revascor In Patients With Moderate To Advanced Heart
FailureIn addition to evaluation in LVAD patients,
Revascor is also being evaluated in a placebo-controlled Phase 3
trial which has enrolled 566 patients with New York Heart
Association Class II/III moderate to advanced heart failure across
multiple sites in North America. The objectives of this Phase
3 events-driven trial are to evaluate the ability of Revascor to
reduce the primary endpoint of recurrent heart failure-related
major adverse cardiac events (HF-MACE) in patients with left
ventricular dysfunction, as well as delay or prevent disease
progression to terminal cardiac events, defined as death, left
ventricular assist device (LVAD) implantation, or cardiac
transplant. The trial has accrued approximately 85% of its
required target of primary endpoint events. In April 2017, the
Phase 3 trial was successful in a pre-specified futility analysis
of the primary efficacy endpoint in the first 270 patients.
About Mesoblast Mesoblast Limited (ASX: MSB;
Nasdaq: MESO) is a world leader in developing allogeneic
(off-the-shelf) cellular medicines. The Company has leveraged its
proprietary technology platform to establish a broad portfolio of
late-stage product candidates with three product candidates in
Phase 3 trials – acute graft versus host disease, chronic heart
failure and chronic low back pain due to degenerative disc disease.
Through a proprietary process, Mesoblast selects rare mesenchymal
lineage precursor and stem cells from the bone marrow of healthy
adults and creates master cell banks, which can be industrially
expanded to produce thousands of doses from each donor without the
need for tissue matching. Mesoblast has facilities in Melbourne,
New York, Singapore and Texas and is listed on the Australian
Securities Exchange (MSB) and on the Nasdaq (MESO).
www.mesoblast.com
References1 Gustafsson F, Rogers JG. Left
ventricular assist device therapy in advanced heart failure:
patient selection and outcomes. European Journal of Heart Failure
2017;19:595-602.2United Network for Organ Sharing.3Agency for
Healthcare Research and Quality – Healthcare Cost and Utilization
Project – Claims Analysis ICD- 37.6.4Data on file.5Chatterjee A,
Feldmann C, Hanke JS (2018) The momentum of HeartMate 3: a novel
active magnetically levitated centrifugal left ventricular assist
device (LVAD). J Thorac Dis 10 (Suppl 15): S1790-S1793.6Mehra, MR
Salerno C, Cleveland JC (2018) Health care resources use and cost
implications in the MOMENTUM 3 long-term outcome study: a
randomized controlled trial of a magnetically levitated cardiac
pump in advanced heart failure.
Forward-Looking Statements This 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 its product candidates
into, enroll and successfully complete, clinical studies; the
timing or likelihood of regulatory filings and approvals for use in
LVAD patients or other indications; 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
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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