Mesoblast Clinical Program Update for MPC-150-IM in Patients With Chronic Heart Failure
18 April 2018 - 8:00PM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today provided an update
on the Phase 3 and Phase 2b clinical trials of the Company's
proprietary allogeneic mesenchymal precursor cell (MPC) product
candidate MPC-150-IM in patients with moderate-to-severe and
end-stage advanced chronic heart failure (CHF), respectively. These
indications represent major unmet medical needs due to the high
rates of morbidity and mortality despite existing therapies.
In the United States alone, there are more than 1.3 million
patients with New York Heart Association (NYHA) Class III CHF,
250,000 patients with NYHA Class IV CHF, and 50,000 patients with
end-stage CHF, representing a potential multi-billion dollar market
opportunity for Mesoblast.
Positive clinical signals have been previously published in
Phase 2 trials of Mesoblast’s MPC therapy in advanced and end-stage
heart failure1,2, supporting a common underlying mechanism of
action (MOA). The MOA by which MPC-150-IM is thought to exert its
effects in these patient populations, based on preclinical
evidence, is through reduction of damaging inflammation, maturation
of the vasculature, and cardiac repair.
Phase 3 DREAM-HF Trial Targeting NYHA Class
II/III Patients With Moderate-to-Advanced Chronic Heart
Failure
- The objectives of this Phase 3 events-driven trial are to
evaluate the ability of MPC-150-IM to reduce the primary endpoint
of recurrent non-fatal heart failure-related major adverse cardiac
events (HF-MACE) in patients with left ventricular dysfunction, as
well as delay or prevent disease progression to end-stage HF and
terminal cardiac events (TCE), defined as death, left ventricular
assist device (LVAD) implantation, or cardiac
transplant.
- As reported in April 2017, an interim futility analysis of the
recurrent non-fatal HF-MACE primary efficacy endpoint in the
DREAM-HF’s first 270 patients, as performed by the trial’s
independent Data Monitoring Committee (DMC), was
successful. According to the futility guidelines, the trial
met the pre-specified threshold for continuation.
- In April 2018, the DMC for the Phase 3 trial undertook a
scheduled review of available data from 465 randomized patients,
including the primary and secondary endpoints of HF-MACE and TCEs,
and all safety data. The DMC recommended continuation of the trial
without modification. Enrollment of this Phase 3 trial is expected
to be completed by the end of 2018.
Phase 2b Trial Targeting End-State Heart Failure
Patients with a Left Ventricular Assist Device (LVAD)
- End-stage heart failure is a major unmet medical need, with
mortality approaching 50% at one year despite existing medical
therapies. While LVAD implantation improves 12-month survival in
these patients, fewer than 5,000 patients annually are given
potentially life-saving LVADs due to the high risks of increased
morbidity, recurrent hospitalizations, and inflammatory
complications, including gastrointestinal bleeding, associated with
these devices.
- Based on prior Phase 2 trial results which suggested that
Mesoblast’s MPCs improved native heart function, prolonged time to
re-hospitalization and improved early survival in patients with an
LVAD, in December 2017 the United States Food and Drug
Administration (FDA) granted Mesoblast Regenerative Medicine
Advanced Therapy (RMAT) designation for its novel MPC therapy in
the treatment of heart failure patients with left ventricular
systolic dysfunction and a LVAD.
- 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. 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.
- A 159-patient trial, funded by the National Institutes of
Health (NIH) and the Canadian Institute of Health Research, and
evaluating Mesoblast’s investigational MPC cells as add-on therapy
to LVADs has completed enrollment with a 12-month data read-out
expected in the next quarter (Q3 CY18). The objectives
of this Phase 2b trial are to confirm and extend the prior Phase 2
results.
- Mesoblast is actively pursuing a potential accelerated entry
pathway for MPC-150-IM into the cardiovascular market that may be
provided by RMAT designation.
About Chronic Heart Failure (CHF)CHF is a
progressive disease and is classified in relation to the severity
of the symptoms experienced by the patient. The most commonly used
classification system was established by the NYHA and ranges from
Class I-II (mild to moderate) to Class III/IV (severe to
end-stage). In 2016, more than 15 million patients in the
seven major global pharmaceutical markets were estimated to have
been diagnosed with CHF3. Prevalence is expected to grow 46% by
2030 in the United States alone, affecting more than 8 million
Americans4. Approximately half of people who develop heart failure
die within 5 years of diagnosis5,6 . Patients with advanced CHF
(NYHA Class III or Class IV) have the highest burden of disease,
recurrent hospitalizations and mortality. In the United States
alone, the NYHA Class III patient population is estimated at 1.3
million patients and the NYHA class IV population at 250,000
patients. There are approximately 50,000 patients with
end-stage class IV heart failure who, despite optimal medical
therapy, have a one-year mortality exceeding 50%7. The only options
to increase survival in these patients are the use of LVADs or of
heart transplants, the latter limited by donor availability to less
than 3000 patients annually8. In the United States and worldwide,
CHF results in severe economic, social, and personal costs to
patients and their respective healthcare systems6.
References:
- Perin EC, et al. Circ Res 2015;117:576-584
- Ascheim DD, et al. Circulation. 2014;129:2287-2296
- PharmaPoint: Heart Failure – Global Drug Forecast and Market
Analysis to 2025.
- GlobalData-PharmaPoint (2016): Heart Failure-Global Drug
Forecast and Market Analysis to 2025.
- AHA Statistical Update – Heart Disease and Stroke
Statistics-(2017). Circulation. 2017;131.
- Voight J, et al. Clinl.Cardiol. 2014;37:312-321
- Gustafsson G, Rogers JG. Eur J Heart Failure
2017;19,595-602
- Agency for Healthcare Research and Quality: HCUPnet: ICD-9
principal procedure code 27.51 2014.
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 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
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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 Greenway Investor Relations T: +1 212 880 2060
E: schond.greenway@mesoblast.com
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