Last updated on April 23, 2014 at 20:10 EDT

Positive Results from Phase 2 Trial of Mesoblast’s Adult Stem Cell Therapy Presented at the American Heart Association Annual Meeting

November 14, 2011

ORLANDO, Florida and MELBOURNE, Australia, November 14, 2011 /PRNewswire/ –

        - Revascor(TM), Mesoblast's proprietary Mesenchymal Precursor
          Cell (MPC) product for cardiovascular diseases, was safe and
          well-tolerated at all doses, with no clinically relevant immune
          responses to donor cells
        - Revascor(TM) therapy significantly reduced cardiac mortality and
          major adverse cardiac events (MACE) in patients with congestive heart
          failure over a mean follow-up of 22 months
        - Highest dose of Revascor(TM) completely prevented any episodes
          of heart failure hospitalization over 18 months of follow-up
        - Clinical improvement was associated with evidence of remodeling
          (reduction in heart volumes) and improvement in functional capacity
          (increased walking distance), which are key parameters in congestive
          heart failure
        - Revascor(TM) anticipated to progress to Phase 3 trial in first
          half of 2012

Regenerative medicine company Mesoblast Limited (ASX:MSB; OTC ADR:MBLTY)
today announced positive Phase 2 heart failure trial results of its
allogeneic, or off-the-shelf, adult stem cell product Revascor(TM) after all
patients had completed a minimum follow-up of 12 months, and a mean
follow-up of 22 months. The Phase 2 trial results were presented at the
American Heart Association annual meeting in Orlando, Florida, by the
trial’s independent principal investigator Dr Emerson C. Perin, Director of
Research in Cardiovascular Medicine and Medical Director, Stem Cell Center,
Texas Heart Institute in Houston.

MPC treatment pooled across all doses resulted in significant reduction
in cardiac mortality, and at the highest dose resulted in complete
prevention of heart failure hospitalization events. Mesoblast expects that
these outcomes will be central to the primary endpoint of a Revascor(TM)
Phase 3 trial for product regulatory approval by the United States Food and
Drug Administration (FDA).

The randomized, placebo-controlled 60-patient Phase 2 trial compared the
safety and efficacy of three doses of Revascor(TM) on top of maximal
approved therapies versus maximal therapies alone in patients with
moderate-to-severe congestive heart failure (CHF) defined by New York Heart
Association (NYHA) class II or III status and ejection fraction below 40%.
The trial enrolled both ischemic and non-ischemic heart failure patients.
Heart failure patients with this degree of severity are known to have a high
cardiac mortality over a 12-24 month period despite being on maximal
approved drug and device therapies.

Treatment with MPCs was well-tolerated, with 13% of patients developing
transient or persistent anti-donor antibodies which were without clinical
consequence. Over a 22-month mean follow-up period, only 1/45 (2%) patients
who received a single injection of Revascor(TM) died of cardiac causes
compared with 3/15 (20%) controls (p=0.02). In addition, MPC treatment
significantly delayed the time to a first Major Adverse Cardiac Event, MACE,
a composite of cardiac death, heart attack or revascularization procedures
(p=0.036), and reduced the overall risk for MACE by 78% (p=0.011).

Over a mean follow-up of 18 months, 0/15 patients who received the
highest dose of MPC (150M) had been hospitalized for heart failure or had
died. In contrast, 3/15 (20%) controls and 6/30 (20%) patients who received
low (25M) or mid (75M) doses of MPC had either been hospitalized with heart
failure or had died. This clinical improvement associated with the 150M dose
was accompanied by evidence of cardiac remodelling (reduction in left
ventricular end systolic volumes compared with controls at 6 months,
p=0.015) and improved functional capacity (gain of 52.6 meters over 6
minutes’ walk compared with controls at 12 months, p=0.06).

After 12 months, 40% of all treated patients had reverted to class I
NYHA status compared with 14% of all controls, and this effect remained when
patients were matched for the presence of class II status at baseline. The
group who received the 25M MPC dose showed a significant 8.9 point
improvement in ejection fraction over controls at 3 months (p=0.008), with a
sustained but less pronounced effect over 12 months. In contrast, the group
who received 150M MPC did not show improved ejection fraction, suggesting
that the positive effects of this dose on clinical outcomes, remodeling, and
functional capacity may be due to other mechanisms such as anti-fibrosis.

“These clinical findings are the first using any cell therapy in heart
failure patients to show a concordant positive effect on clinical outcomes,
cardiac remodelling, and functional capacity, the three key parameters in
congestive heart failure. Together, they indicate that a single 150 million
dose of Revascor(TM) may significantly reduce both heart failure
hospitalizations and death in these very sick patients who have such a poor
prognosis despite maximal existing therapies,” Dr Perin said.

“Based on their defined product characterization, batch to batch
consistency, immediate availability, and lack of clinically relevant
immunogenicity, MPCs appear to be an ideal cell type to provide a new level
of patient care in congestive heart failure. We look forward to progressing
the Revascor(TM) clinical program into Phase 3,” Dr Perin added.

Revascor(TM) is being jointly developed by Mesoblast and its strategic
alliance partner, Teva Pharmaceutical Industries Ltd.

Teva’s Corporate Vice President Global Branded Products, Kevin Buchi,
said: “These independently-reviewed results serve to reinforce Teva’s
commitment to its strategic investment in Mesoblast’s adult stem cell
technology and to our continued support for the clinical development of

Mesoblast Chief Executive, Professor Silviu Itescu, said, “Together with
our partners at Teva, we are deeply committed to bringing to market an
effective cell therapy product to reduce recurrent hospitalization episodes
and risk of death in patients with progressive heart failure. The exciting
results presented at the American Heart Association meeting reinforce the
strength of our technology and emphasize the need to maintain a rapid
development path in order to make this product available for the many
patients suffering with heart failure.”

SOURCE Mesoblast

Source: PR Newswire