World's First Approved Stem Cell Drug; Osiris Receives Marketing Clearance from Health Canada for Prochymal
Historic decision offers hope to children suffering from
life-threatening GvHD
COLUMBIA, Md.--(BUSINESS WIRE)--
Osiris Therapeutics Inc. (NASDAQ: OSIR) announced today it has received
market authorization from Health Canada to market its stem cell therapy
Prochymal® (remestemcel-L), for the treatment of acute
graft-vs-host disease (GvHD) in children. The historic decision marks
the world's first regulatory approval of a manufactured stem cell
product and the first therapy approved for GvHD — a devastating
complication of bone marrow transplantation that kills up to 80 percent
of children affected, many within just weeks of diagnosis.
"I am very proud of the leadership role Canada has taken in advancing
stem cell therapy and particularly gratified that this historic decision
benefits children who would otherwise have little hope," said Andrew
Daly, M.D., Clinical Associate Professor, Department of Medicine and
Oncology at the University of Calgary, Canada and Principal Investigator
in the phase 3 clinical program for Prochymal. "As a result of Health
Canada's comprehensive review, physicians now have an off-the-shelf stem
cell therapy in their arsenal to fight GvHD. Much like the introduction
of antibiotics in the late 1920's, with stem cells we have now
officially taken the first step into this new paradigm of medicine."
Prochymal was authorized under Health Canada's Notice of Compliance with
conditions (NOC/c) pathway, which provides access to therapeutic
products that address unmet medical conditions and which have
demonstrated a favorable risk/benefit profile in clinical trials. Under
the NOC/c pathway, the sponsor must agree to carry out confirmatory
clinical testing.
"Today is not only a great day for Osiris, but for everyone involved in
the responsible development of stem cell therapies," said C. Randal
Mills, Ph.D., President and Chief Executive Officer of Osiris. "Most
importantly, today is a great day for children and their families who
bravely face this horrific disease. While today marks the first approval
of a stem cell drug, now that the door has been opened, it will surely
not be the last."
Health Canada's authorization was made following the recommendation of
an independent expert advisory panel, commissioned to evaluate
Prochymal's safety and efficacy. In Canada, Prochymal is now authorized
for the management of acute GvHD in children who fail to respond to
steroids. The approval was based on the results from clinical studies
evaluating Prochymal in patients with severe refractory acute GvHD.
Prochymal demonstrated a clinically meaningful response at 28 days post
initiation of therapy in 61-64 percent of patients treated. Furthermore,
treatment with Prochymal resulted in a statistically significant
improvement in survival when compared to a historical control population
of pediatric patients with refractory GvHD (p=0.028). The survival
benefit was most pronounced in patients with the most severe forms of
GvHD. As a condition of approval, the clinical benefit of Prochymal will
be further evaluated in a case matched confirmatory trial and all
patients receiving Prochymal will be encouraged to participate in a
registry that will monitor the long-term effects of the therapy.
"Refractory GvHD is not just deadly to the patients it afflicts, but is
devastating for the family, friends, and caregivers who watch helplessly
as the disease progresses," said Joanne Kurtzberg, MD, Head of the
Pediatric Bone Marrow Transplant Program at Duke University and Lead
Investigator for Prochymal. "I have personally seen Prochymal reverse
the debilitating effects of severe GvHD in many of my patients and now,
after nearly two decades of research, the data demonstrating
consistently high response rates, a strong safety profile and improved
survival clearly support the use of Prochymal in the management of
refractory GvHD."
Prochymal is currently available in several countries, including the
United States, under an Expanded Access Program (EAP). Prochymal will be
commercially available in Canada later this year.
"Today Osiris turns the promise of stem cell research into reality,
delivering on decades of medical and scientific research," said Peter
Friedli, Chairman and Co-founder of Osiris. "It took 20 years of hard
work and perseverance and I want to personally thank everyone involved
for their dedication to this important mission."
In addition to the extensive intellectual property protection Osiris has
around Prochymal, which includes 48 issued patents, Health Canada's
decision will also provide Prochymal with regulatory exclusivity within
the territory. Canada affords eight years of exclusivity to Innovative
Drugs such as Prochymal, and an additional six-month extension is
available since it addresses a pediatric population.
Webcast and Conference Call
A webcast and conference call is scheduled for tomorrow, May 18, 2012 at
9:00 a.m. ET. To access the webcast, visit the Investor Relations
section of the company's website at http://investor.osiris.com/events.cfm.
Alternatively, callers may participate in the conference call by dialing
(877) 303-6133 (U.S. participants) or (970) 315-0493 (international
participants).
A replay of the conference call will be available approximately two
hours after the completion of the call through May 24, 2012. Callers can
access the replay by dialing (855) 859-2056 (U.S. participants) or (404)
537-3406 (international participants). The audio replay confirmation
code is 82905846. To access a replay of the webcast, visit the Investor
Relations section of the company's website at http://investor.osiris.com/events.cfm.
About GvHD
GvHD represents a major unmet medical need with no approved treatment
until Prochymal. GvHD is the leading cause of transplant related
mortality, in which immune cells contained within the transplanted
marrow recognize the recipient as foreign and mount an immunologic
attack. Severe GvHD can cause blistering of the skin, intestinal
hemorrhage and liver failure. Severe GvHD is extremely painful and fatal
in up to 80 percent of cases. Currently, steroids are used as first-line
therapy with a success rate of only 30-50 percent. When steroids fail,
treatment options are limited to immunosuppressive agents used off-label
with little benefit and significant toxicities.
About Prochymal (remestemcel-L)
Prochymal is the world's first approved drug which has a stem cell as
its active ingredient. Developed by Osiris Therapeutics, Prochymal is an
intravenous formulation of mesenchymal stem cells (MSCs), which are
derived from the bone marrow of healthy adult donors between the ages of
18 and 30 years. The MSCs are selected from the bone marrow and grown in
culture so that up to 10,000 doses of Prochymal can be produced from a
single donor. Prochymal is truly an off-the-shelf stem cell product that
is stored frozen at the point-of-care and infused through a simple
intravenous line without the need to type or immunosuppress the
recipient. Prochymal is currently approved in Canada for the management
of acute graft-versus-host disease (GvHD) in children and is available
for adults and children in eight countries including the United States,
under an Expanded Access Program. Prochymal is currently in Phase 3
trials for refractory Crohn's disease and also being evaluated in
clinical trials for the treatment of myocardial infarction (heart
attack) and type 1 diabetes.
About Notice of Compliance with Conditions
An NOC/c is a form of market approval granted to a product on the basis
of promising evidence of clinical effectiveness following review of the
submission by Health Canada. Products approved under Health Canada's
NOC/c policy are intended for the treatment, prevention or diagnosis of
a serious, life-threatening or severely debilitating illness. Such
products have demonstrated promising benefit, are of high quality and
possess an acceptable safety profile based on a benefit/risk assessment.
In addition, they either respond to a serious unmet medical need in
Canada or have demonstrated a significant improvement in the
benefit/risk profile over existing therapies. Health Canada has provided
access to such products on the condition that sponsors carry out
additional clinical trials to verify the anticipated benefit within an
agreed upon time frame.
About Osiris Therapeutics
Osiris Therapeutics, Inc. is the leading stem cell company, having
developed the world's first approved stem cell drug, Prochymal. The
company is focused on developing and marketing products to treat medical
conditions in inflammatory, cardiovascular, orthopedic areas and wound
healing areas. Osiris currently markets Prochymal for refractory GvHD,
Grafix® for burns and chronic wounds, and Ovation® for orthopedic
applications. The company's pipeline of internally developed biologic
drug candidates under evaluation includes Prochymal for inflammatory,
autoimmune and cardiovascular indications, as well as Chondrogen for
arthritis in the knee. Osiris is a fully integrated company with
capabilities in research, development, manufacturing and distribution of
stem cell products. Osiris has developed an extensive intellectual
property portfolio to protect the company's technology, including 48
U.S. and 144 foreign patents.
Osiris, Prochymal, Grafix and Ovation are registered trademarks of
Osiris Therapeutics, Inc. More information can be found on the company's
website, www.Osiris.com.
(OSIR-G)
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking
statements include statements about our expectations, beliefs, plans,
objectives, intentions, assumptions and other statements that are not
historical facts. Words or phrases such as "anticipate," "believe,"
"continue," "ongoing," "estimate," "expect," "intend," "may," "plan,"
"potential," "predict," "project" or similar words or phrases, or the
negatives of those words or phrases, may identify forward-looking
statements, but the absence of these words does not necessarily mean
that a statement is not forward-looking. Examples of forward-looking
statements include, but are not limited to, statements regarding the
following: our product development efforts; our clinical trials and
anticipated regulatory requirements and the ability to successfully
navigate these requirements; the success of our product candidates in
development; status of the regulatory process for our biologic drug
candidates; implementation of our corporate strategy; our financial
performance; our product research and development activities and
projected expenditures, including our anticipated timeline and clinical
strategy for Prochymal, Chondrogen and our other MSC and biologic drug
candidates; our cash needs; patents and proprietary rights; the safety
and ability of our potential products to treat disease and the results
of our scientific research; our plans for sales and marketing; our plans
regarding our facilities; types of regulatory frameworks we expect will
be applicable to our potential products; and results of our scientific
research. Forward-looking statements are subject to known and unknown
risks and uncertainties and are based on potentially inaccurate
assumptions that could cause actual results to differ materially from
those expected or implied by the forward-looking statements. Our actual
results could differ materially from those anticipated in
forward-looking statements for many reasons, including the factors
described in the section entitled "Risk Factors" in our Annual Report on
Form 10-K and other Periodic Reports filed on Form 10-Q, with the United
States Securities and Exchange Commission. Accordingly, you should not
unduly rely on these forward-looking statements. We undertake no
obligation to publicly revise any forward-looking statement to reflect
circumstances or events after the date of this press release or to
reflect the occurrence of unanticipated events.
Osiris Therapeutics, Inc.
Erica Elchin, 443-545-1834
OsirisPR@Osiris.com
Source: Osiris Therapeutics, Inc.
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