Amarantus Receives Orphan Drug Designation From the U.S. Food and Drug Administration for MANF for the Treatment of Retinal A...
September 14 2015 - 10:30AM
Amarantus BioScience Holdings, Inc. (OTCQX:AMBS), a biotechnology
company developing therapeutic and diagnostic product candidates in
orphan indications and neurology, announced that the U.S. Food and
Drug Administration (FDA) has granted the company's investigational
drug mesencephalic-astrocyte-derived neurotrophic factor (MANF)
orphan drug designation for the treatment of retinal artery
occlusion (RAO). RAO is a blockage of the blood supply to the
retina which causes severe and sudden loss of vision.
MANF, a naturally-occurring protein that reduces and prevents
apoptosis (programmed cell death) in response to injury or disease,
was discovered utilizing Amarantus' proprietary PhenoGuard™ Protein
Discovery Engine. Pre-clinical data demonstrated that MANF provided
protective functional effects in an animal model of RAO. Moreover,
toxicology studies have demonstrated that MANF was well-tolerated
following a single intravitreal administration of a therapeutically
relevant dose.
"We are extremely pleased to have received the second orphan
drug designation for MANF for use in ophthalmologic disorders. At
the end of 2014 we received orphan drug designation for MANF to
treat retinitis pigmentosa (RP), and this additional designation
for treatment of RAO is an important step in our regulatory
strategy for what we believe has the potential to become a broad
ophthalmic therapeutic franchise," said Gerald E. Commissiong,
President & CEO of Amarantus BioSciences Holdings, Inc. "RAO is
an acute condition that can potentially lead to blindness and the
toxicology data generated thus far directly supports the
translational potential for MANF in this indication. We believe
MANF has promise as a safe and effective therapeutic option to
treat RAO and RP."
The FDA Orphan Drug Designation program provides a special
status to drugs and biologics intended to treat, diagnose or
prevent so-called orphan diseases and disorders that affect fewer
than 200,000 people in the U.S. This designation provides for a
seven-year marketing exclusivity period against competition, as
well as certain incentives, including federal grants, tax credits
and a waiver of PDUFA filing fees.
About Retinal Artery Occlusion
Retinal artery occlusion (RAO) is a rare eye condition caused by
a loss of blood supply to the inner layer of the retina resulting
in acute and often severe vision loss. The currently-available
treatments are aimed at opening the occluded artery before
irreversible damage occurs and most often do not improve visual
acuity above natural history. There are no effective
neuroprotective agents for the treatment of acute retinal ischemia
available. The prevalence for RAO is approximately 10,450 patients
in the United States.
About Mesencephalic-Astrocyte-derived Neurotrophic
Factor (MANF)
MANF (mesencephalic-astrocyte-derived neurotrophic factor) 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 (cell death) 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 lead indication is retinitis pigmentosa, and additional
indications including Parkinson's disease, diabetes and Wolfram's
syndrome are currently pursued. Further applications for MANF may
include Alzheimer's disease, traumatic brain injury (TBI),
myocardial infarction, antibiotic-induced ototoxicity and certain
other rare orphan diseases currently under evaluation.
About Amarantus BioScience Holdings, Inc.
Amarantus BioScience Holdings (OTCQX:AMBS) is a biotechnology
company developing treatments and diagnostics for diseases in the
areas of neurology and orphan diseases. AMBS' Therapeutics division
has development rights to eltoprazine, a small molecule currently
in a Phase 2b clinical program for Parkinson's disease
levodopa-induced dyskinesia with the potential to expand into adult
ADHD and Alzheimer's aggression. The Company has an exclusive
worldwide license to intellectual property rights associated to
Engineered Skin Substitute (ESS), an orphan drug designated
autologous full thickness skin replacement product in development
for the treatment of severe burns currently preparing to enter
Phase 2 clinical studies. AMBS owns the intellectual property
rights to a therapeutic protein known as
mesencephalic-astrocyte-derived neurotrophic factor (MANF) and is
developing MANF as a treatment for orphan ophthalmic disorders,
initially in retinitis pigmentosa (RP). AMBS also owns the
discovery of neurotrophic factors (PhenoGuard™) that led to MANF's
discovery.
AMBS' Diagnostics division owns the rights to MSPrecise®, a
proprietary next-generation DNA sequencing (NGS) assay for the
identification of patients with relapsing-remitting multiple
sclerosis (RRMS), and has an exclusive worldwide license to the
Lymphocyte Proliferation test (LymPro Test®) for Alzheimer's
disease, which was developed by Prof. Thomas Arendt, Ph.D., from
the University of Leipzig, and owns further intellectual property
for the diagnosis of Parkinson's disease (NuroPro®).
For further information please visit www.Amarantus.com, or
connect with the Company on Facebook, LinkedIn, Twitter and
Google+.
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CONTACT: Investor and Media Contact:
Jenene Thomas
Jenene Thomas Communications, LLC
Investor Relations and Corporate Communications Advisor
T: (US) 908.938.1475
E: jenene@jenenethomascommunications.com
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