Amarantus Announces Positive Orphan Data for MANF in Retinitis Pigmentosa
First Orphan Indication for MANF Identified in Ophthalmology
SUNNYVALE, Calif., Aug. 12, 2013 (GLOBE NEWSWIRE) -- Amarantus BioScience Holdings, Inc. (OTCQB:AMBS), a biotechnology company discovering and developing treatments and diagnostics for diseases associated with neurodegeneration and apoptosis, today announced positive data for MANF in the degenerative disease known as Retinitis Pigmentosa ("RP"). The study concluded that Intravitreal injection of recombinant human MANF protein protects both rods and cones from retinal degeneration in an animal model of RP. RP is a degenerative disorder of the eye that affects roughly 100,000 people in the United States, meaning that it qualifies as an orphan disease under FDA guidelines.
RP refers to a group of inherited diseases causing 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 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.
"The data announced today is tremendously important to our MANF Orphan program," said Gerald E. Commissiong, CEO of Amarantus. "As we continue to move our internal MANF programs forward related to mediating endoplasmic reticulum (ER) stress, we can now expand the MANF therapeutic development franchise into Ophthalmology. By identifying Retinitis Pigmentosa as an indication for MANF, we have identified a potentially reduced timeline to get MANF to commercialization with a very substantial market opportunity."
Methods: Recombinant human MANF was expressed in E. coli and purified.
To examine the neuroprotective effect of MANF on rod photoreceptors, recombinant human MANF (6 µg) was intravitreally injected to the left eyes of transgenic rat S334ter rats (line 3) at PD9, and the right eyes were injected with PBS as controls. Eyes were collected at PD21 and examined by light microscopy.
To examine effect of MANF on cone photoreceptors, MANF (6 µg) was injected intravitreally to the left eyes of the transgenic rats at PD20, and the right eyes were injected with PBS as controls. Retinas were collected at PD30 and cone outer segments were stained with Alexa 488 conjugated peanut agglutinin (PNA). Flat-mounted retinas were examined by confocal microscopy.
Results: MANF significantly protected rod photoreceptors. In MANF-treated retinas, three to four rows of nuclei remained in the outer nuclear layer (ONL), as compare to only one row of nuclei in PBS-treated fellow eyes. The thickness of ONL in MANF-treated retinas (superior region) (17.47±3.96 µm, mean±SD, n=5) is significantly greater than that in PBS-treated retinas (7.07±1.12, n=5) (P < 0.001, student t-test). Loss of cone outer segments was characterized by many PNA-negative areas distributed across the retina in PBS treated retinas, as reported previously. Quantitative analysis showed that PNA-positive cells are significantly more in MANF treated retinas (569.5±46.5/0.1 mm2, mean±SD, n=6) than in PBS-treated retinas (398.7±25.4, n=6) (P< 0.001, student t-test).
- Conclusions: Intravitreal injection of recombinant human MANF protein protects both rods and cones from degeneration.
"The identification of an orphan indication for MANF is a key milestone towards the initial commercialization for MANF," said Dr. Joseph Rubinfeld, Amgen co-founder and current member of the Company's Board of Advisors. "The delivery of MANF directly to the eye in this indication will be local, potentially significantly reducing timelines to get this into the marketplace versus systemic applications. We are continuing to look at MANF in other orphan indications and expect to have data in other areas in the coming weeks."
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.
Amarantus 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, Alzheimer's disease, Traumatic Brain Injury, and other human diseases. The Company 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 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. The Company also owns intellectual property for the diagnosis of Parkinson's disease ("NuroPro") and the discovery of neurotrophic factors ("PhenoGuard"). For further information please visit www.Amarantus.com, or connect with the Company on Facebook, LinkedIn, Twitter and Google+.
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