Amarantus Announces Preclinical Development Timeline for MANF Therapeutic

  Amarantus Announces Preclinical Development Timeline for MANF Therapeutic

Business Wire

SUNNYVALE, Calif. -- April 11, 2013

Amarantus BioScience Holdings, Inc. (OTCQB: AMBS), a biotechnology company
discovering and developing treatments and diagnostics for diseases associated
with neurodegeneration and apoptosis, today provided a timeline of key
activities and milestones related to the preclinical development of MANF
(Mesencephalic-Astrocyte-derived Neurotrophic Factor). The Company is
currently focused on developing MANF across three broad categories:
Parkinson's disease and Traumatic Brain Injury; large systemic applications
such as Ischemic Heart Disease and Diabetes; and orphan indications.

“We plan to move forward on a number of fronts this year with MANF, leading
potentially to one or more Investigational New Drug (IND) filings in 2014,”
said Gerald E. Commissiong, President and Chief Executive Officer of
Amarantus. “The pace and scope of activities outlined today reflect the unique
properties of MANF, the positive results reported earlier this year in
neuroprotective and neurorestorative animal models of Parkinson’s disease and
supportive data from the published literature.”

The upcoming activities related to Amarantus’ Parkinson’s disease program
include:

  *Create mammalian GMP production processes for MANF; the Company has
    sourced the expertise for these activities, and has started development of
    the supporting analytical and bioanalytical assays;
  *Form a partnership agreement in the third quarter of 2013 with a firm
    specializing in Convection-enhanced delivery (CED) of drugs to the brain;
  *Initiate non-human primate pharmacology studiesin Parkinson's disease
    models in the second half of 2013 in order to establish an appropriate
    dosing regimen for human clinical studies;
  *Potentially evaluate MANF gene therapy based upon upcoming developments in
    the field of neurotrophic factors for Parkinson’s disease, and the
    Company’s current patent position whereby the Company owns exclusive
    rights to MANF gene therapy applications in all vector systems.

Additional planned activities are expected to include:

  *Conduct pharmacokinetic and pharmacodynamic (PK/PD) studies, with data
    available in the third quarter of 2013, to evaluate the
    biologicalproperties of MANF when administered systemically, with data to
    be released as it becomes available;
  *Conduct pharmacology studies in traumatic brain injury, ischemic heart
    disease, diabetes and certain other animal models in the third quarter of
    2013, with data to be released as it becomes available;
  *Evaluate MANF ina variety of animal models of orphan diseases in the
    third quarter of 2013 that represent significant market opportunities, and
    where there is limited or very limited competition;
  *Initiate MANF toxicology studies upon development of a master cell bank of
    MANF protein material as one of the final pre-IND steps;
  *File an IND in 2014.

“Based on our progress with these preclinical activities and the interest and
level of data in specific indications, we will seek topartnerwith
biopharmaceutical companies or appropriate not-for-profit disease foundations
in order to accelerate our development program and assist in recruiting
patients for future clinical studies,” said Mr. Commissiong. “We will
prioritize our clinical development programs based upon the outcome of the
pharmacology studies that will begin in the third quarter of 2013 for the
indications outlined here today. We intend to give highest priority to those
indications where we have a shorter path to market, and where non-dilutive
financing is available to support further development. Fortunately for the
Company, it is likely that many of the pre-clinical studies will have
applications across indications, meaning we will get the advantage of a
particular set of data enabling multiple indications.”

The Company will seek to gain ‘Breakthrough,’ ‘Fast Track’ and/or ‘Orphan Drug
Designation’ status with the FDA where the Company believes the data generated
justifies such a designation, potentially significantly reducing the time to
market for MANF and/or improving the potential economic outcome for the
Company.

The Company believes MANF could address the following very significant market
opportunities:

  *Parkinson’s disease: $1 billion
  *Traumatic Brain Injury: $200 million
  *Ischemic Heart Disease: $2 billion
  *Diabetes: $2 billion
  *Orphan diseases: $2 billion

Large pharmaceutical companies have shown significant interest in neurotrophic
factors for Parkinson's disease for over two decades. In 1993, Amgen acquired
Synergen for $262 million shortly after Synergen successfully completed
studies using GDNF as a protein therapeutic in animal models of Parkinson's
disease. In 2007, Genzyme entered into a $150 million partnership with
Ceregene to gain access to ex-U.S. marketing rights to Neurturin after
Ceregene successfully completed a Phase 1 clinical study demonstrating safety
for Neurturin as a gene therapy product. Ceregene is expected to announce data
on a recently completed Phase 2 study this quarter.

The acute ischemic heart disease market represents an $8 billion market
worldwide. The chronic ischemic heart disease market represents a $30 billion
market. One of the key unmet medical needs in the ischemic heart disease
market is limiting ischemia/reperfusion associated with myocardial infarction,
the area where MANF has shown significant promise in the published literature.

Diabetes represents a $28 billion market worldwide. One of the key unmet needs
in the diabetes market is improving beta cell function, an area where MANF is
believed to hold promise.

A prime example of a successful orphan strategy is Genzyme, who was successful
in turning its orphan drug strategy into a $20.1 billion buyout by Sanofi
Aventis in 2011. Another example of a successful orphan strategy is FerroKin
Biosciences, which was successfully acquired by Shire for $325 million in
early 2012 with only $27 million in paid-in-capital and a virtual staff of
seven employees. In 2011, Alexion Pharmaceuticals reported $783 million in
revenue based on sales of its only product Soliris, a drug that treats a
population of approximately 10,000 patients in the U.S. and Western Europe.

About Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF)

MANF (Mesencephalic-Astrocyte-derived Neurotrophic Factor) is a protein that
corrects protein misfolding, one of the major causes of apoptosis (Programmed
Cell Death). Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF) is
believed to have broad potential because it is a naturally-occurring protein
produced by the body for the purpose of reducing and preventing apoptosis (in
response to injury or disease), via the unfolded protein response. By
manufacturing MANF and administering it to the body, Amarantus is seeking to
use a regenerative medicine approach to assist the body with higher quantities
of MANF when needed. Amarantus is the front-runner and primary holder of
intellectual property (IP) around MANF, and is initially focusing on the
development of MANF-based protein therapeutics. MANF's current lead indication
is Parkinson's disease with additional focus on Traumatic Brain Injury (TBI).
Future indications may include myocardial infarction and certain rare and
ultra-rare orphan diseases where MANF is currently being evaluated.

About Amarantus

Amarantus is a development-stage biotechnology company founded in January
2008. The Company is focused on developing unique products and proprietary
technologies for the potential treatment and/or diagnosis of Parkinson's
disease, Traumatic Brain Injury, Ischemic Heart Disease and other human
diseases. The Company owns the intellectual property rights to
Mesencephalic-Astrocyte-derived Neurotrophic Factor ("MANF") and is developing
MANF-based products as treatments for neurological disorders where there is a
significant unmet medical need. 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 related 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 progress of the MANF
technology in testing for Parkinson’s disease, statements about expectations,
plans and prospects of the development of Amarantus' diagnostic product
candidates for Alzheimer’s and Parkinson’s disease, traumatic brain injury,
ischemic heart disease, diabetes, and unspecified Orphan indications; and the
potential market size for the LymPro test for Alzheimer’s disease. These
forward-looking statements are subject to a number of risks, uncertainties and
assumptions, including the risks associated with development of therapeutic
drug candidates, 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.

Contact:

Investor/Media Contact
LHA
Don Markley, Senior Vice President
310-691-7100
dmarkley@lhai.com
 
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