MELBOURNE, Australia,
July 7, 2011 /PRNewswire/ --
Key points:
- Mesoblast's first Phase 3 trial submission cleared by United
States Food and Drug Administration (FDA)
- Rapid clearance (within 30 days) further validation of
Mesoblast's clinical, regulatory, and manufacturing
capabilities
- Mesoblast's proprietary adult stem cells expand hematopoietic
precursor cells in umbilical cord blood 40-fold, enabling rapid
bone marrow reconstitution with lowered risk of life-threatening
Graft Versus Host Disease (GVHD)
- Mesoblast's novel clinical approach could make the use of bone
marrow transplantation available to all those in need of the
procedure but who currently cannot find a donor, without the need
for full matching
- Commercial opportunity based on potential to increase the total
number of unrelated donor transplants performed globally by three-
to four-fold
- This product may be the first of Mesoblast's revenue generating
biologic therapies in both the US and Europe
Global regenerative medicine company Mesoblast Limited (ASX:MSB;
OTC ADR: MBLTY) today announced that it has received clearance from
the United States Food and Drug Administration (FDA) to begin a
Phase 3 clinical trial for bone marrow regeneration in patients
with blood cancers. FDA clearance was obtained within the 30-day
minimum time period after Mesoblast filed its Phase 3
Investigational New Drug (IND) submission.
The Phase 3 trial will aim to reproduce the positive pilot trial
results seen at the University of Texas
MD Anderson Cancer, where accelerated neutrophil and platelet
recoveries, together with excellent 100-day patient survival and
low GVHD rates, occurred in patients receiving partially mismatched
hematopoietic cells from umbilical cord blood expanded by
Mesoblast's proprietary allogeneic or 'off-the-shelf' mesenchymal
precursor cells (MPCs).
Mesoblast's off-the-shelf MPCs are being developed under an
Orphan Drug Designation granted for the condition of insufficient
hematopoietic stem cell production in patients with hematologic
malignancies who have failed treatment with conventional
chemotherapy. Such patients are in need of bone marrow
transplantation using hematopoietic stem cells that are of either
autologous (patient's own) or allogeneic (unrelated donor, e.g.
blood from other adults or umbilical cord) origin. Mesoblast's MPCs
may potentially be used for expansion of both autologous and
allogeneic hematopoietic stem cells for subsequent
transplantation.
According to the Center for International Blood and Marrow
Transplant Research (CIBMTR), there are now over 60,000 autologous
and allogeneic bone marrow transplants performed annually
worldwide, a number projected to further increase due to the
anticipated growth in incidence of hematologic malignancies
associated with an aging population.
Of the total transplants performed annually worldwide,
approximately 25,000 are allogeneic. This number represents less
than 30 per cent of individuals who would otherwise be eligible to
receive an unrelated donor bone marrow transplant because for the
rest a fully matched donor cannot be found. Perfect matching
is required for adult marrow transplants because of the very high
risk of potentially life-threatening GVHD when unmatched
transplants are performed. GVHD still occurs in as many as 60 per
cent of patients who receive fully matched bone marrow transplants
from unrelated adult donors.
In contrast, cord blood causes significantly less GVHD, and can
be used as a partially mismatched donor source. However, the number
of hematopoietic precursor cells in unexpanded cord blood is too
few to enable sufficiently robust and predictable bone marrow
engraftment.
Mesoblast's objective is to make available a source of unrelated
donor hematopoietic precursor cells from cord blood which can be
used without full matching to effect rapid bone marrow
reconstitution with a low risk of GVHD. The Company believes that
this would expand the use of allogeneic bone marrow transplantation
to all those in need of the procedure but who currently cannot find
a donor, with the potential to expand the total number of unrelated
donor transplants performed by three- to four-fold.
Mesoblast's Phase 3 trial will be conducted across 50 centers in
the United States, Europe and Australia, and will enroll 240 patients with
hematologic malignancies undergoing unrelated donor bone marrow
transplantation using matched or partially mismatched umbilical
cord blood. Patients will be randomized to receive either
non-expanded cord blood or cord blood expanded by Mesoblast's MPCs
and containing 40-fold higher numbers of hematopoietic cells. The
primary endpoint is a shortened time to neutrophil and platelet
recovery in the treatment group.
Mesoblast Chief Executive, Professor Silviu Itescu, said the initiation of a Phase 3
trial was a landmark milestone for the company.
"This achievement again underscores the strength and robustness
of Mesoblast's clinical, regulatory, and manufacturing
capabilities. It is a significant step in bringing our broad range
of adult stem cell therapeutics closer to global licensure, and to
patients suffering from severe, debilitating diseases.
"We hope that this particular product will make bone marrow
transplantation a more widely used and safer option for critically
ill patients who undergo chemotherapy to potentially cure blood
cancer.
"This product has the potential to be the first of our revenue
generating biologic therapies in both the
United States and Europe,"
Professor Itescu added.
The Phase 3 trial will be conducted together with Mesoblast's
strategic alliance partner, Cephalon Inc., who will fund the
trial.
About Orphan Drug Designation
Orphan drug designation is reserved for therapies which are
being developed for conditions affecting up to 200,000 patients
annually in the United States, and
allows for an accelerated review process by the FDA, seven-year
market exclusivity in the United
States upon obtaining marketing authorization, tax benefits,
and exemption from user fees.
Mesoblast Limited
Mesoblast Limited (ASX: MSB; OTC ADR: MBLTY) is a world leader
in the development, manufacture, and commercialization of biologic
products for the broad field of regenerative medicine. Mesoblast
has the worldwide exclusive rights to a series of patents and
technologies developed over more than 10 years relating to the
identification, extraction, culture and uses of adult Mesenchymal
Precursor Cells (MPCs). More information -
www.mesoblast.com
For further information, please contact:
Julie Meldrum
Corporate Communications Director
T: + 61 (0) 3 9639 6036
E: julie.meldrum@mesoblast
SOURCE Mesoblast Limited
