Amarantus Announces Positive Preclinical Data on the Effects of MANF on Vision in a Model of Retinitis Pigmentosa
SAN FRANCISCO and GENEVA, Nov. 25, 2014 (GLOBE NEWSWIRE) -- Amarantus BioScience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on the development of diagnostics and therapeutic products in the areas of neurology, psychiatry, ophthalmology and regenerative medicine, announced positive effects of Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF) for the protection from vision loss in an animal model of retinitis pigmentosa (RP). MANF was discovered utilizing Amarantus' proprietary PhenoGuard™ Protein Discovery Engine.
The aim of the study was to evaluate the efficacy of protecting visual acuity by a single intravitreal injection of MANF in the rd10/rd10 genetic mouse model of RP. These animals carry a spontaneous missense point mutation in Pde6b (cGMP phosphodiesterase 6B, rod receptor, beta polypeptide). Mutations in this gene are found in human autosomal recessive RP. Visual acuity was determined by measuring the spatial frequency threshold by optokinetic tracking. This method allows for a non-invasive assessment of visual acuity in rodents and higher species, including humans.
A single intravitreal administration of MANF after the onset of retinal degeneration on Day 31 resulted in a statistically significant protective effect on visual acuity on Day 38 and Day 45, as compared to vehicle treated animals. This is the first observation of MANF providing a functional benefit on vision in a model of RP. These data complement previously announced effects of MANF on preserving retinal morphology in RP models. The experiments were conducted at a leading ophthalmology contract research laboratory.
"Our results are the first observation of protection of vision in an RP model by MANF, and add a seminal functional visual acuity readout to the initial morphological protection findings reported from the University of Miami's Bascom Palmer Eye Institute," said Dr. David A. Lowe, President & CEO of NeuroAssets and member of the Amarantus Board of Directors. "These data further support our focus on ophthalmic orphan indications, as exemplified by our recent application to the FDA for orphan designation in RP."
In October 2014, the Company applied to the U.S. Food & Drug Administration for Orphan Drug Designation for MANF in RP. The Company expect to receive a response in the near future.
"These promising data provide a compelling basis to continue the further development of MANF in RP towards first-in-man studies," said Gerald E. Commissiong, President & CEO of Amarantus. "We will continue moving forward with a strategic development plan focused initially on orphan ocular diseases, where we see tremendous potential for MANF. We are hopeful to develop better treatments for patients suffering from diseases that lead to blindness due to an array of medical conditions. Ultimately, we believe proof of concept for MANF in human studies in ocular diseases will support MANF development in other therapeutic areas where significant compelling pre-clinical efficacy data has been published and confirmed by independent groups, as well as the Company's own datasets. We firmly believe MANF has blockbuster potential in ophthalmology, neurology, cardiology and metabolic disorders, including diabetes."
About Retinitis Pigmentosa
Retinitis Pigmentosa (RP) refers to a group of inherited diseases involving retinal degeneration. The cell-rich retina lines the back inside 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 patient to patient, with most people with RP becoming legally blind by age 40. There are approximately 100,000 patients in the United States, 100,000 patients in Europe and 50,000 patients in Japan diagnosed with RP, qualifying it as an orphan indication. There are currently no approved treatments in the market. It is estimated that RP is a multi-billion dollar market opportunity.
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 current lead indication is Retinitis Pigmentosa, and other applications including Parkinson's disease, Diabetes and Wolfram's Syndrome. Additional 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 (AMBS) is a biotechnology company developing treatments and diagnostics for diseases associated with neurodegeneration and protein misfolding-related apoptosis. AMBS has licensed Eltoprazine ("Eltoprazine"), a phase 2b ready small molecule indicated for Parkinson's disease 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 ophthalmic disorders. AMBS also owns intellectual property for the diagnosis of Parkinson's disease ("NuroPro") and the discovery of neurotrophic factors ("PhenoGuard™"). In November 2014, AMBS entered into an exclusive option agreement with Lonza Walkersville, Inc., a subsidiary of Lonza Group Ltd., to acquire Cutanogen Corporation, a subsidiary of Lonza Walkersville, to develop Engineered Skin Substitute (ESS-W), an autologous skin replacement product for the treatment of Stage 3 and Stage 4 intractable severe burns. For further information please visit www.Amarantus.com, or connect with the Company on Facebook, LinkedIn, Twitter and Google+.
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