Mesoblast will enter 2019 with the most mature cell therapy product
pipeline and technology platform in the regenerative medicine
industry. Two commercial products have already been approved
and marketed by the Company’s licensees JCR Pharmaceuticals Co,
Ltd. in Japan and Takeda Pharmaceutical Company in Europe.
Mesoblast has one product candidate which has successfully
completed Phase 3 and with near-term commercial potential in the
United States (U.S.), another product candidate having achieved
clinical outcomes in line with the U.S. Food and Drug
Administration (FDA) guidance for a registrable clinical indication
for market authorization, and two additional Phase 3 assets with
blockbuster potential.
Mesoblast has recently entered into a strategic cardiovascular
partnership for China with Tasly Pharmaceutical Group, China’s
leading cardiovascular company, and is in advanced and active
discussions with a number of potential global commercialization
partners to maximize the value proposition of each of our
blockbuster cell therapy candidates.
Mesoblast’s royalty income and milestone payments from licensees
continues to grow, the Company has sufficient cash to achieve key
commercial milestones, and access to additional non-dilutive
sources of capital from strategic financial institutions whose
extensive due diligence provides further third party validation of
the strength of the product portfolio and patent estate.
Below is a summary of the current status of the product
portfolio and the developments expected to take place in 2019.
Substantial Commercialization Opportunities
Mesoblast’s proprietary immunoselected and culture-expanded
allogeneic mesenchymal precursor cells (MPCs) are a homogeneous,
well characterized, and highly reproducible cell population that
are manufactured to industrial scale for commercial
purposes. They express an array of surface receptors that bind
pro-inflammatory cytokines and, when placed into a pro-inflammatory
microenvironment, release factors that switch off production of
these cytokines. Their immunomodulatory mechanism of action makes
MPCs uniquely suited to target resistant diseases where
inflammation plays a central role.
Acute Graft Versus Host Disease, a
Life-threatening Inflammatory ConditionIn Q1 2019, the
Company plans to initiate the FDA process of filing a Biologics
License Application (BLA) for market authorization of remestemcel-L
in the U.S., where there are no approved therapies for
steroid-refractory acute Graft Versus Host Disease (aGVHD).
Underpinning Mesoblast’s confidence in the U.S. market access plan
is the Japan experience, where Mesoblast’s licensee, JCR
Pharmaceuticals, markets TEMCELL®1 HS Inj. for children and adults
with aGVHD.
Importantly, TEMCELL has achieved substantial adoption rates in
just over 2.5 years, helping inform the view of the product value
proposition potential in the U.S. market. With an experienced
commercial leadership in place, Mesoblast is establishing a focused
sales team that will target the principal U.S. transplant centers,
and will be in place on FDA approval to ensure a successful product
launch.
Inflammation Due to Left Ventricular Assist Device
Implants in End-stage Heart Failure Patients In the
first half of 2019, Mesoblast plans to meet with the FDA to discuss
a potential approval pathway following the clinically meaningful
outcomes of reduction in major gastrointestinal (GI) bleeding and
related hospitalizations seen in the U.S. National Institutes of
Health (NIH)-sponsored Phase 2 trial of MPC-150-IM (Revascor) in
patients with end-stage heart failure and a left ventricular assist
device (LVAD). This potentially life-threatening complication is
the most common non-surgical complication in LVAD recipients and
occurs in up to 40% of patients.
In the 159-patient trial, a single intra-cardiac injection of
Revascor resulted in a 76% reduction in major GI bleeding episodes
and in 65% reduction in associated hospitalization events.
Reduction in GI bleeding and associated hospitalizations were the
basis of the Regenerative Medicine Advanced Therapy (RMAT)
designation granted in December 2017 by the FDA for use of Revascor
in LVAD patients based on concordant data from the earlier
30-patient Pilot Trial. In a subsequent meeting in 2018, the FDA
advised Mesoblast that reduction in major GI bleeding in LVAD
patients is considered a clinically meaningful outcome by the FDA
and an acceptable endpoint for product approval.
In end-stage heart failure, where intra-cardiac inflammation is
greatest, putting a foreign object (the LVAD) in contact with the
failing left ventricle results in further activation of
intra-cardiac inflammation, which exacerbates pre-existing vascular
dysfunction in the peripheral organs. The GI blood vessels
respond to vascular dysfunction and reduced flow by generation of
abnormal thin-walled, leaky capillaries
(angiodysplasia). These pre-dispose LVAD patients to massive
and life-threatening GI bleeding.
Mesoblast believes that reduction in major GI bleeding episodes
by Revascor is due to reduction in intra-cardiac inflammation and
the associated vascular dysfunction in peripheral organs, including
the GI vessels. If this is correct, this will have significant
read-through to the Phase 3 trial in patients with class II/III
heart failure where intra-cardiac inflammation and peripheral
vascular dysfunction are thought to directly result in recurrent
hospitalizations and terminal cardiac events.
Phase 3 Trial in Moderate to Advanced Chronic Heart
Failure, a Progressive Disease of Cardiac
InflammationMesoblast expects to complete patient
recruitment in the Phase 3 trial evaluating Revascor in patients
with moderate-to-severe advanced chronic heart failure very
shortly. In the U.S. alone, there are more than 1.3 million
patients with New York Heart Association (NYHA) class III chronic
heart failure who have high rates of morbidity and mortality
despite existing therapies. The major unmet medical need in
these patients represents a potential multi-billion dollar market
opportunity for Mesoblast.
The primary endpoint for this Phase 3 trial is the ability of
Revascor to reduce recurrent non-fatal heart failure-related major
adverse cardiac events (HF-MACE) in patients with left ventricular
dysfunction. The key secondary endpoint is to delay or prevent
terminal cardiac events (TCEs), defined as death, left ventricular
assist device implantation, or heart transplant.
It is important to note that in the Phase 2 LVAD trial patients
with ischemic cause of their heart failure showed the greatest
benefits after being treated with Revascor and these patients
closely resemble the majority of patients enrolled in the ongoing
Phase 3 trial of patients with moderate to advanced heart failure.
If the mechanism of action by which Revascor improved GI bleeding
is indeed reduction of intra-cardiac inflammation and reversal of
impaired functioning of blood vessels (endothelial dysfunction), a
known primary cause of morbidity, exercise intolerance, and
mortality in heart failure and a proven mechanism of action for
many drugs in early heart failure, one would expect to see a
reduction in HF-MACE and mortality in this Phase 3 trial.
Chronic Low Back Pain Due to Inflammatory Degenerative
Disc Disease In the U.S., the declared opioid public
health emergency and significant associated mortality has brought
additional attention to Mesoblast’s product candidate, MPC-06-ID,
with the Phase 3 trial completing enrollment of 404 patients in
2018. More than half of the prescriptions for opioids are for
people seeking relief from chronic low back pain. There is a
desperate need for a therapy that can offer both a durable
reduction in pain and improvement in function without the risk of
opioid addiction.
Underpinning Mesoblast’s confidence that MPC-06-ID may meet this
medical need are the Phase 2 data outcomes that supported the
ongoing Phase 3 trial which showed that a single intra-discal
injection of MPC-06-ID alleviated pain and improved function for up
to three years in patients whose symptoms were not adequately
treated with current standard of care therapies.
The patient population suffering from chronic low back pain due
to intervertebral disease is estimated at more than 3.2 million
patients in the U.S. alone. Mesoblast’s objective is to select and
secure the ideal strategic partner to maximize the value creation
potential inherent in MPC-06-ID.
Upcoming Milestones
Remestemcel-L for Acute Graft Versus Host
Disease - FDA meetings and BLA filing
(Early CY19)
Revascor for End-Stage Heart Failure
- Meet with FDA to discuss the clinically meaningful
GI bleeding Phase 2 trial data for potential BLA filing (1H
CY19)
Phase 3 Events-driven Trial in Advanced Heart
Failure - Complete recruitment (Q4
CY18/Q1 CY19) - Cardiovascular partner Tasly
Pharmaceuticals to meet with National Medical Products
Administration to discuss the regulatory approval pathway in China
(Q1 CY19) - Establish global
partnership
MPC-06-ID for Chronic Low Back Pain
- Establish global partnership
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
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 that meet stringent
release criteria, have lot to lot consistency, and can be used
off-the-shelf 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
1. TEMCELL® HS Inj. is the registered trademark of JCR
Pharmaceuticals Co. Ltd.
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.
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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