Amarantus Applies to FDA for MANF Orphan Drug Designation in Retinitis Pigmentosa
SAN FRANCISCO and GENEVA, Oct. 16, 2014 (GLOBE NEWSWIRE) -- Amarantus Bioscience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the development of diagnostics and therapeutics for Alzheimer's disease, Parkinson's disease and ophthalmological disorders, announced that it has applied to the US Food & Drug Administration for Orphan Drug Designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP). MANF was discovered utilizing Amarantus' proprietary PhenoGuard Protein Discovery Engine; all intellectual property is owned by the company.
MANF, a naturally-occurring protein that reduces and prevents apoptosis (programmed cell death) in response to injury or disease, has demonstrated efficacy in three animal models of retinitis pigmentosa. Amarantus is collaborating with the University of Miami's Bascom Palmer Eye Institute and the Buck Institute for Research on Aging in its retinitis pigmentosa program. Additionally, the company has completed toxicology experiments, concluding that MANF was well-tolerated following intravitreal administration at therapeutically relevant doses.
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 Retinitis Pigmentosa
Retinitis pigmentosa (RP) refers to a group of inherited diseases causing retinal degeneration often leading to blindness. The cell-rich retina lines the inside back wall of the eye and is responsible for capturing images from the visual field. People with RP experience a gradual decline in their vision because photoreceptor cells (rods and cones) die. Symptoms include a progressive degeneration of peripheral and night vision, as well as the degeneration in color perception and central vision. Night blindness is one of the earliest and most frequent symptoms of RP.
RP is typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies from person to person. Most people with RP are legally blind by age 40. It is estimated that the market opportunity for Retinitis Pigmentosa exceeds $10B annually.
About Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF)
MANF (mesencephalic-astrocyte-derived neurotrophic factor) is a naturally-occurring protein, produced by the body in response to injury or disease, to reduce or prevent apoptosis (cell death), with potentially broad application in conditions that lead to cell death. Amarantus' MANF-based therapeutic platform is a regenerative medicine approach designed 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. The MANF platform's current lead indication is retinitis pigmentosa (RP). Other indications include glaucoma, Parkinson's disease, Alzheimer's disease and Wolfram's Syndrome. Additional applications for MANF may include traumatic brain injury (TBI), myocardial infarction, diabetes, antibiotic-induced ototoxicity and certain other rare orphan diseases currently under evaluation.
About Amarantus BioScience Holdings, Inc.
Amarantus BioScience Holdings (AMBS) is a biotechnology company developing treatments and diagnostics for diseases associated with neurodegeneration and protein misfolding-related apoptosis. AMBS has licensed Eltoprazine, a phase 2b- ready small molecule indicated for Parkinson's levodopa-induced dyskinesia and Adult ADHD. AMBS has an exclusive worldwide license to the Lymphocyte Proliferation test (LymPro Test®) for Alzheimer's disease and 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 and ocular disorders. AMBS also owns intellectual property for the diagnosis of Parkinson's disease (NuroPro) and the discovery of neurotrophic factors (PhenoGuard).
Amarantus operations are located at Janssen Labs @QB3 in San Francisco, CA. For further information please visit www.Amarantus.com, or connect with the Company on Facebook, LinkedIn, Twitter and Google+.
Certain statements, other than purely historical information, including estimates, projections, statements relating to our business plans, objectives, and expected operating results, and the assumptions upon which those statements are based, are forward-looking statements. These forward-looking statements generally are identified by the words "believes," "project," "expects," "anticipates," "estimates," "intends," "strategy," "plan," "may," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Forward-looking statements are based on current expectations and assumptions that are subject to risks and uncertainties which may cause actual results to differ materially from the forward-looking statements. Our ability to predict results or the actual effect of future plans or strategies is inherently uncertain. Factors which could have a material adverse effect on our operations and future prospects on a consolidated basis include, but are not limited to: changes in economic conditions, legislative/regulatory changes, availability of capital, interest rates, competition, and generally accepted accounting principles. These risks and uncertainties should also be considered in evaluating forward-looking statements and undue reliance should not be placed on such statements.
CONTACT: Amarantus Bioscience Holdings, Inc. Aimee Boutcher, Investor Relations 408.737.2734 x 101 firstname.lastname@example.org Planet Communications Deanne Eagle, Media Contact (917) 837-5866