Successful Interim Analysis of Efficacy Endpoint in Mesoblast’s Phase 3 Trial for Chronic Heart Failure
April 10 2017 - 6:00AM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that
the Phase 3 trial of its allogeneic mesenchymal precursor
cell (MPC) product candidate MPC-150-IM in patients with moderate
to advanced chronic heart failure (CHF) was successful in
the pre-specified interim futility analysis of the
efficacy endpoint in the trial's first 270 patients. It is expected
that the trial will enroll in total approximately 600 patients.
After notifying the Company of the interim analysis results,
the trial’s Independent Data Monitoring Committee (IDMC)
additionally stated that they had no safety concerns relating to
MPC-150-IM and formally recommended that the trial should continue
as planned.
Dr. Emerson C. Perin, Director, Research in
Cardiovascular Medicine and Medical Director, Stem Cell Center at
the Texas Heart Institute, and a lead investigator on the ongoing
Phase 3 trial said: "It is very pleasing to see that
this large and rigorously conducted Phase 3 trial of
Mesoblast's cell therapy was successful in the pre-specified
interim futility analysis for the trial's efficacy endpoint in the
first 270 patients. Advanced heart failure is a very serious
and life-threatening disease, and there is an urgent need to
develop a safe and effective new therapy for these patients that
may halt or reverse disease progression and prevent the high
associated mortality.”
Mesoblast Chief Executive Silviu Itescu commented: “Passing
this interim futility analysis for MPC-150-IM is an important
milestone for Mesoblast and our cardiovascular disease program.
This validates our strategy and our prioritization of this valuable
program.”
This ongoing double-blinded randomized (1:1) trial is currently
being conducted across multiple study sites in the United States
and Canada. It is evaluating MPC-150-IM in adult patients with
moderate to advanced New York Heart Association (NYHA) Class II/III
chronic heart failure with left ventricular systolic dysfunction.
The trial’s primary efficacy endpoint is a comparison of recurrent
non-fatal heart failure-related major adverse cardiac events
(HF-MACE) in moderate to advanced CHF patients receiving either
MPC-150-IM by catheter injection into the damaged left ventricular
heart muscle or sham control. A Joint Frailty Model is the
statistical method that evaluates multiple non-fatal heart
failure-related events per patient (such as repeated
hospitalizations for decompensated heart failure) while accounting
for increased likelihood of a terminal cardiac event (such as
death, implantation of a mechanical heart assist device or a heart
transplant) for patients with multiple non-fatal heart failure
events. In line with best practice for blinded Phase 3 clinical
trials, the interim analysis data are only reviewed by the IDMC.
Mesoblast, the United States Food and Drug Administration (FDA),
and trial investigators are blinded to grouped safety and efficacy
data for the ongoing trial as well as the numerical results of this
interim analysis.
About Mesoblast’s MPC-150-IM Cardiovascular
Program MPC-150-IM is Mesoblast's lead allogeneic,
cell-based product candidate for the treatment of moderate to
advanced chronic heart failure (CHF) due to left ventricular
systolic dysfunction.
In Phase 2 results, a single injection of MPC-150-IM into the
myocardium of patients with moderate to advanced chronic heart
failure prevented any HF related hospitalizations or cardiac deaths
over three years of follow-up.1 Nonclinical studies showed that
intramyocardial administration of MPCs in animal models of heart
failure improved cardiac function and attenuated pathological
ventricular remodelling. These effects were attributable, at least
in part, to MPC secretion of biomolecules that stimulate reparative
processes in the failing heart including new blood vessel
formation, cardiac muscle cell survival, and reduction in tissue
fibrosis.
MPC-150-IM is also being studied in a Phase 2b trial in 159
patients with NYHA Class IV end-stage heart failure patients in
conjunction with implantation of a left ventricular assist device
(LVAD). A major objective of this trial, which is being
sponsored by the United States National Institutes of Health (NIH),
is to assess the ability of MPC-150-IM to help wean patients from a
LVAD dependent existence for survival (so-called “bridge to
recovery”).
Additionally, the FDA recently cleared the commencement of a
24-patient trial which is being sponsored by Boston’s Children’s
Hospital. This study combines Mesoblast's proprietary allogeneic
MPC-150-IM product with corrective heart surgery in children under
the age of 5 with hypoplastic left heart syndrome.
About Chronic Heart Failure In 2016, more than
15 million patients in the seven major global pharmaceutical
markets are estimated to have been diagnosed with CHF.2 Prevalence
is expected to grow 46% by 2030 in the United States alone,
affecting more than 8 million Americans.3 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 (mild) to Class IV or end stage (severe). Approximately
half of people who develop heart failure die within 5 years of
diagnosis.4 Patients with late NYHA Class II or Class III CHF
continue to represent a significant unmet medical need despite
recent advances in new therapies. CHF causes severe economic,
social, and personal costs. In the United States, it is estimated
that CHF results in direct costs of $60.2 billion annually when
identified as a primary diagnosis and $115 billion as part of a
disease milieu.5
1.Perin EC, Borow KM, Silva GV, et al. A phase II
dose-escalation study of allogeneic mesenchymal precursor cells in
patients with ischemic or nonischemic heart failure. Circ Res.
2015; 117:576-84
2.GlobalData-PharmaPoint (2016): Heart Failure-Global Drug
Forecast and Market Analysis to 2025
3.AHA Statistical Update – Heart Disease and Stroke
Statistics-(2017). Circulation. 2017;131:00-00. DOI:
10.1161/CIR.0000000000000485
4.Mozzafarian D, Benjamin EJ, Go AS, et al. on behalf of the
American Heart Association Statistics Committee and Stroke
Statistics Subcommittee. Heart disease and stroke statistics—2016
update: a report from the American Heart Association. Circulation.
2016;133:e38-e360
5.A Re-Evaluation of the Costs of Heart Failure and its
Implications for Allocation of Health Resources in the United
States. Voigt J. Clinl.Cardiol. 37, 5, 312-321 (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.
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Forward-looking statements should not be read as a guarantee of
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For further information, please contact:
CORPORATE COMMUNICATIONS
Julie Meldrum
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
INVESTOR RELATIONS
Schond Greenway
T: +1 212 880 2060
E: schond.greenway@mesoblast.com
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