NEW YORK and MELBOURNE, Australia, Sept. 25, 2018 (GLOBE NEWSWIRE) -- Mesoblast Limited (ASX:MSB, Nasdaq:MESO) today
announced that Mesoblast's proprietary allogeneic mesenchymal precursor cell (MPC) heart failure product candidate MPC-150-IM for
use in children with hypoplastic left heart syndrome (HLHS) was featured at the First Cardiac Regenerative Symposium for Congenital
Heart Disease in Baltimore, Maryland on the weekend. The symposium focused on the potential for using cellular therapies in the
treatment of complex congenital heart conditions.
A randomized, placebo-controlled 24-patient trial is ongoing at Boston Children’s Hospital and combines an injection of
MPC-150-IM into the left ventricle with corrective heart surgery in children under the age of five with HLHS. To date there have
not been any cell-related safety concerns in the trial. Consequently, this trial has the potential to extend the safety profile
of MPC-150-IM beyond adults, where it is being studied in two complementary late-stage clinical trials in patients with
advanced and end-stage chronic heart failure, to children with congenital heart disease.
Dr Sitaram Emani, Associate Professor of Surgery at Harvard Medical School and Director of the Complex Biventricular Repair
Program at Boston Children’s Hospital, and the trial’s Principal Investigator, said: “We believe that a direct injection of
Mesoblast’s cellular medicine into the hypoplastic left ventricle as an adjunct to surgical rehabilitation has the potential
to promote growth and regeneration of that ventricle and recruit it back into the circulation. This provides a chance for the
patient with this congenital disease to regain normal two-ventricle circulation commensurate with improved quality of life and
longevity.”
The underlying mechanism of action by which MPC-150-IM is thought to exert its therapeutic effects in both pediatric and
adult patient populations, based on preclinical evidence, is through reduction of damaging inflammation, maturation of the
vasculature, reduction in fibrosis, and cardiac repair.
A Phase 2b trial comparing an injection of MPC-150-IM or placebo into the left ventricle in 159 adult patients with end-stage
heart failure receiving a left ventricular assist device (LVAD) implant completed enrollment in September 2017, with all patients
having a planned follow-up of at least one year. The United States Food and Drug Administration (FDA) has granted Mesoblast a
Regenerative Medicine Advanced Therapy (RMAT) designation for use of MPC-150-IM in these patients based on prior Phase 2
trial results showing improved heart function, prolonged time to re-hospitalization and improved early survival after a single
intra-myocardial injection of Mesoblast’s MPCs at the time of an LVAD implant. Full results of this Phase 2b trial will be
presented by the trial’s independent investigators at an upcoming conference.
A Phase 3 trial in approximately 600 patients with New York Heart Association (NYHA) Class II/III chronic heart failure is also
being actively conducted in the United States, with over 85% of patients enrolled. The objectives of this Phase 3
events-driven trial are to evaluate the ability of a single catheter-based injection of MPC-150-IM to reduce 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 heart failure and terminal cardiac events. In a prior Phase 2 trial, HF-MACE events were
significantly reduced by a single intra-myocardial injection of MPC-150-IM. This trial has previously been successful in a
futility analysis of the primary endpoint.
About Hypoplastic Left Heart Syndrome
The normal heart contains left and right ventricles. Children with hypoplastic left heart syndrome (HLHS) have a functioning right
ventricle, but have a small left ventricle that is incapable of supporting the systemic circulation. If left untreated, HLHS is
uniformly fatal. Current treatment, known as single-ventricle palliation, uses the right ventricle to support the entire
circulation through a series of surgeries. However, the right ventricle eventually tires out, leading to nearly 50% mortality by
adolescence. When successful, biventricular conversion gives the patient a normal circulation and can prevent complications
associated with single ventricle circulation including renal failure, arrhythmias, and the need for a heart transplant. With
surgical rehabilitation alone, only one third of patients are able to undergo biventricular conversion. The key to successful
ventricular recruitment and biventricular conversion is cardiac muscle growth and regeneration.
The trial using Mesoblast’s product candidate MPC-150-IM in combination with surgery (NCT03079401) is sponsored and funded by
the Boston Children’s Hospital with support from Bulens and Capozzi Foundation and the Ethan Lindberg Foundation.
About Boston Children’s Hospital
Boston Children’s Hospital, the primary pediatric teaching affiliate of Harvard Medical School,
is home to the world’s largest research enterprise based at a pediatric medical center. Its discoveries have benefited both
children and adults since 1869. Today, more than 3,000 scientists, including nine members of the National Academy of
Sciences, 17 members of the National Academy of Medicine and 11 Howard Hughes Medical Investigators comprise
Boston Children’s research community. Founded as a 20-bed hospital for children, Boston Children’s is now a 415-bed comprehensive
center for pediatric and adolescent health care. For more, visit our Vector and Thriving blogs and follow us on social media @BostonChildrens, @BCH_Innovation, Facebook and YouTube.
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 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
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 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 Meldrum
Corporate Communications
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
Schond Greenway
Investor Relations
T: +1 212 880 2060
E: schond.greenway@mesoblast.com
