Amarantus BioSciences Explores Orphan Drug Strategy Based on MANF
Protein Folding
SUNNYVALE, Calif., Nov. 1, 2012 /PRNewswire/ -- Amarantus
BioSciences, Inc. (OTCQB: AMBS), a biotechnology company developing
new treatments and diagnostics for Parkinson's disease and
Traumatic Brain Injury centred on its proprietary anti-apoptosis
therapeutic protein MANF, today announced that it intends to pursue
the identification of one or multiple therapeutic indications for
its lead therapeutic candidate MANF that could lead to the therapy
receiving orphan drug designation(s) with the Food and Drug
Administration (FDA). The strategy is centred on exploiting MANF's
unique mechanism of action related to facilitating proper protein
folding and processing in the endoplasmic reticulum in order to
identify rare and/or ultra-rare diseases where MANF treatment may
play a significant role in improving patient outcomes in disease
states where no other treatment options are currently
available.
"Protein misfolding and aggregation plays a significant role in
a wide range of human diseases, including very common diseases such
as Parkinson's and Alzheimer's, as well as many rare and ultra-rare
diseases," said Gerald E.
Commissiong, President & CEO of Amarantus. "MANF has a
unique ability to mediate protein folding in an extracellular
fashion, making it potentially an ideal biologic drug candidate for
a wide range of human conditions. Parkinson's continues to remain
the Company's primary focus, especially in light of recently
announced data where MANF demonstrated superiority over GDNF in a
neurorestoration animal model of Parkinson's disease. However, the
recent grant the Company was awarded by the Center of Excellence in
Apoptosis Research will allow our scientific team to explore MANF's
utility in diseases that have much smaller patient populations, and
we will make a concerted effort to focus on rare and ultra-rare
diseases when reviewing the results. This strategy may afford the
Company an accelerated pathway to commercialization by identifying
therapeutic applications for MANF that would require few small
clinical trials. Data already pooled from publicly available
databases suggests this orphan designation strategy may bear fruit
for the Company in the near-term."
The Orphan Drug Act (ODA) provides for granting special status
to a product to treat a rare disease or condition upon request of a
sponsor. The combination of the product to treat the rare disease
or condition must meet certain criteria. This status is referred to
as orphan designation. Orphan designation qualifies the sponsor of
the product for the tax credit and marketing incentives of the ODA.
A marketing application for a prescription drug product that has
been designated as a drug for a rare disease or condition is not
subject to a prescription drug user fee unless the application
includes an indication for other than a rare disease or
condition.[1]
A prime example of a successful strategy is Genzyme, who was
successful in turning its orphan drug strategy into a $20.1B buyout by Sanofi Aventis in 2011. Another
example of a successful orphan strategy is FerroKin Biosciences,
who was successfully acquired by Shire for $325M in early 2012 with only $27M in paid-in-capital and a virtual staff of 7
employees. In 2011, Alexion Pharmaceuticals reported $783M in revenue based on sales of its only
product Soliris, a drug that treats a population of approximately
10,000 patients in the US and Western
Europe.
About Amarantus BioSciences, Inc.
Amarantus BioSciences, Inc. is a development-stage biotechnology
company founded in January 2008. The Company has a focus on
developing certain biologics surrounding the intellectual property
and proprietary technologies it owns to treat and/or diagnose
Parkinson's disease, Traumatic Brain Injury and other human
diseases. The Company owns the intellectual property rights
to a therapeutic protein known as Mesencephalic-Astrocyte-derived
Neurotrophic Factor ("MANF") and is developing MANF-based products
as treatments for brain disorders. The Company also is a Founding
Member of the Coalition for Concussion Treatment (#C4CT), a
movement initiated in collaboration with Brewer Sports
International seeking to raise awareness of new treatments in
development for concussions and nervous-system disorders. For
further information please visit www.Amarantus.com.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Such statements include, but are not limited to, statements
about the possible benefits of MANF therapeutic applications and/or
advantages presented by Amarantus' PhenoGuard technology, as well
as statements about expectations, plans and prospects of the
development of Amarantus' new product candidates. These
forward-looking statements are subject to a number of risks,
uncertainties and assumptions, including the risks that the
anticipated benefits of the therapeutic drug candidates or
discovery platforms, as well as the risks, uncertainties and
assumptions relating to the development of Amarantus' new product
candidates, including those identified under "Risk Factors" in
Amarantus' most recently filed Annual Report on Form 10-K and
Quarterly Report on Form 10-Q and in other filings Amarantus
periodically makes with the SEC. Actual results may differ
materially from those contemplated by these forward-looking
statements Amarantus does not undertake to update any of these
forward-looking statements to reflect a change in its views or
events or circumstances that occur after the date of this
presentation.
[1]
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm
MEDIA CONTACTS
Amarantus BioSciences, Inc.
Gerald E. Commissiong
408-737-2734
pr@amarantus.com
SOURCE Amarantus BioSciences, Inc.