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Amarantus Bioscience Holdings, Inc. (OTCQX: AMBS) -  February 10, 2016

 



Amarantus Receives Orphan Drug Designation from the US FDA for Eltoprazine in the Treatment of Parkinson’s disease Levodopa-Induced Dyskinesia

 

SAN FRANCISCO, CA, - February 10, 2016 - Amarantus BioScience Holdings, Inc. (OTCQX: AMBS), a biotechnology company focused on developing products for Regenerative Medicine, Neurology and Orphan Diseases, today announced that it has received orphan drug designation from the US FDA for Eltoprazine in the treatment of Parkinson’s disease levodopa-induced dyskinesia (PD-LID). Amarantus published positive results from a Phase 2 initial proof-of-concept clinical study in February of 2015 in the journal Brain, and highlighted the publication of two independent peer-reviewed scientific publications describing the mechanism of action of eltoprazine for the treatment of PD LID in August of 2015 and December 2016.

“The grant of this orphan drug designation for eltoprazine in PD-LID squarely positions Amarantus as an orphan drug company, as each of our pipeline candidates in our therapeutics division has received such designations from the FDA for one or more indications,” said Gerald E. Commissiong, President & CEO of Amarantus. “PD-LID is a tremendously debilitating disorder, and we will now begin evaluating expedited pathways to market for eltoprazine that may now be afforded by the orphan drug designation.”

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 Parkinson's disease Levodopa-induced dyskinesia (PD LID)

Parkinson's disease is a chronic, progressive motor disorder that causes tremors, rigidity, slowed movements and postural instability. The Parkinson's Disease Foundation estimates that there were approximately one million people living with Parkinson's disease in the United States in 2011. The most commonly-prescribed treatments for Parkinson's disease are levodopa-based therapies. In the body, levodopa is converted to dopamine to replace the dopamine loss caused by the disease. The therapeutic efficacy of levodopa is gradually lost over time, and abnormal involuntary movements, dyskinesias, gradually emerge as a prominent side-effect in response to previously beneficial doses of the drug. Levodopa-induced dyskinesia can be severely disabling, rendering patients unable to perform routine daily tasks.

About Eltoprazine
Eltoprazine is a small molecule 5HT1A/1B partial agonist in clinical development for the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID), adult attention deficit hyperactivity disorder (ADHD) and Alzheimer’s aggression. Eltoprazine has been evaluated in over 680 human subjects to date, and has a well-established safety profile. Eltoprazine was originally developed by Solvay Pharmaceuticals for the treatment of aggression. Upon Solvay's merger with Abbott Pharmaceuticals, the eltoprazine program was out-licensed to PsychoGenics. PsychoGenics licensed eltoprazine to Amarantus following successful proof-of-concept trials in PD-LID and adult ADHD.

About Amarantus BioScience Holdings, Inc.
Amarantus BioScience Holdings (AMBS) is a biotechnology company developing treatments and diagnostics for diseases in the areas of neurology, regenerative medicine and orphan diseases. AMBS’ Therapeutics division has development rights to eltoprazine, a Phase 2b-ready small molecule indicated for Parkinson's disease levodopa-induced dyskinesia, adult ADHD and Alzheimer’s aggression, 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. More recently, AMBS acquired the rights to the Engineered Skin Substitute program (ESS), a regenerative medicine-based approach for treating severe burns with full thickness autologous skin grown in tissue culture. ESS is entering Phase 2 clinical studies under a CRADA agreement with the US Army. 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) at first clinical presentation, 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 intellectual property for the diagnosis of Parkinson's disease (NuroPro). AMBS also owns the discovery of neurotrophic factors (PhenoGuard™) that led to MANF’s discovery.

For further information please visit www.Amarantus.com, or connect with the Company on Facebook, LinkedIn, Twitter and Google+.

Forward-Looking Statements
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.

Investor and Media Contact:
Ascendant Partners, LLC
Fred Sommer
732-410-9810
fred@ascendantpartnersllc.com

Source: Amarantus Bioscience Holdings, Inc.

 

ABOUT AMARANTUS BIOSCIENCE HOLDINGS

Amarantus BioScience Holdings, Inc. is a publicly traded biotechnology company focused on developing therapeutic products with the potential for orphan drug designation in the areas of neurology, psychiatry, ophthalmology and regenerative medicine, and diagnostics in neurology.

The Company’s lead therapeutic program, eltoprazine is a small molecule indicated for the treatment of Levodopa-induced dyskinesia, one of the most difficult problems facing patients with Parkinson’s disease. Eltoprazine is currently in a Phase 2b clinical trial with results from the study expected in 2016. Eltoprazine is also being evaluated for the treatment of adult attention deficit hyperactivity disorder (ADHD) and Alzheimer’s aggression.

Amarantus recently completed the acquisition of Cutanogen Corporation (Cutanogen) from Lonza Walkersville, Inc., a subsidiary of Lonza Group Ltd. Cutanogen has an exclusive worldwide license to intellectual property rights associated with Engineered Skin Substitute (ESS). ESS is a full thickness skin replacement product prepared from autologous (patient's own) tissue-engineered skin cells in development for the treatment of severe burns. ESS received orphan drug designation from the FDA for the treatment of hospitalized patients with deep partial and full thickness burns requiring grafting. Amarantus expects to commence a Phase 2 study of ESS in the U.S. in the third quarter of 2015. Amarantus is also developing MANF (mesencephalic-astrocyte-derived neurotrophic factor), which was discovered by the company’s own discovery platform, PhenoGuard. MANF is being developed for the treatment of brain and ophthalmic disorders and has received Orphan Drug Designation from the FDA and the European Medicines Agency (EMA) for the treatment of Retinitis Pigmentosa.

The company’s diagnostics division, Amarantus Diagnostics is dedicated to the development and commercialization of neurology diagnostic products. The company’s lead diagnostic product, LymPro Test® is a blood based assay to diagnose Alzheimer’s disease and is approved for investigational use only to be used in biotech and pharmaceutical clinical trials.

Amarantus Diagnostics is also developing MSPrecise®, a proprietary, next-generation DNA sequencing (NGS) assay for the identification of patients with relapsing-remitting multiple sclerosis (RRMS) at first clinical presentation. In January 2015, the company entered into an exclusive option agreement with Georgetown University to enter into a license for rights related to certain blood based biomarkers for memory loss and Alzheimer’s disease.

The company is active exploring a number of strategic options for Amarantus Diagnostics, including a potential spin-off, to derive the full value of its premier neuro-diagnostics business.

ENGINEERED SKIN SUBSTITUTE (ESS)

Engineered Skin Substitute (ESS) is a full thickness skin replacement product prepared from autologous (patient's own) tissue-engineered skin cells in development for the treatment of severe burns. It is a combination of cultured epithelium with a collagen-fibroblast implant that produces a skin substitute that contains both epidermal and dermal components. This model has been shown in preclinical studies to generate a functional skin barrier. Most importantly, self-to-self skin grafts for autologous skin tissue are less likely to be rejected by the immune system of the patient, unlike with porcine or cadaver grafts in which immune system rejection is a possibility. ESS has been used in an investigator initiated clinical setting in over 130 human subjects, primarily pediatric patients, for the treatment of severe burns up to 95% total body surface area. ESS received orphan drug designation from the U.S. Food and Drug Administration for the treatment of hospitalized patients with deep partial and full thickness burns requiring grafting.

Amarantus recently completed the acquisition of Cutanogen Corporation (Cutanogen) from Lonza Walkersville, Inc., a subsidiary of Lonza Group Ltd. Cutanogen has an exclusive worldwide license to intellectual property rights associated with Engineered Skin Substitute.

Amarantus expects to commence a Phase 2 study of ESS in the U.S. in the third quarter of 2015.


ELTOPRAZINE

Eltoprazine is a small molecule 5HT1A/1B partial agonist in clinical development for the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID) and adult attention deficit hyperactivity disorder (ADHD). Eltoprazine has been evaluated in over 680 human subjects to date, and has a well-established safety profile. Eltoprazine was originally developed by Solvay Pharmaceuticals for the treatment of aggression. Upon Solvay's merger with Abbott Pharmaceuticals, the eltoprazine program was out-licensed to PsychoGenics. PsychoGenics licensed eltoprazine to Amarantus following successful proof-of-concept trials in PD-LID and adult ADHD.

PARKINSON'S DISEASE LEVADOPA-INDUCED DYSKINESIA

Parkinson's disease is a chronic, progressive neurodegenerative disorder that causes motor symptoms such as tremors, rigidity and slowed movements as well as non-motor symptoms including cognitive impairment, mood disorders and autonomic dysfunction. The Parkinson's Disease Foundation estimates that there are approximately one million people living with Parkinson's disease in the United States and seven to 10 million PD patients worldwide. The most commonly prescribed treatments for Parkinson's disease are levodopa-based therapies. In the body, levodopa is converted to dopamine to replace the dopamine loss caused by the disease. As dopamine neurons in the brain are lost the therapeutic efficacy of levodopa attenuates, and increased use is associated with a side effect of dyskinesias. These are involuntary, uncontrollable and often exaggerated and jerky movements. They are distinct from the static, rhythmic tremor as a symptom of Parkinson's disease. Levodopa-induced dyskinesia can be severely disabling, rendering patients unable to perform routine daily tasks.

ATTENTION DEFICIT HYPERACTIVITY DISORDER

Attention deficit hyperactivity disorder (ADHD), is a psychiatric disorder of the neurodevelopmental type in which there are significant problems of attention, hyperactivity, or acting impulsively that are not appropriate for a person's age.

SUMMARY OF THE DATA WITH ELTOPRAZINE:

The data produced demonstrated that at both 5mg and 10 mg, the study met its Primary endpoint as measured by change from baseline in ADHD-RS-IV score in 5mg (p=0.003) and 10mg (p=0.037) doses which were statistically significantly superior to placebo with approximately 25% greater efficacy compared to placebo. Total ADHD-RS-IV scores improved by 13.6, 17.9 and 17.4 points from baseline for placebo, 5mg and 10mg of eltoprazine, respectively. Inattention, Hyperactivity, and Impulsivity ADHD-RS-IV subscales were also analyzed.
For the Inattention subscale, both 5mg and 10mg groups showed a statistically significant benefit over placebo (0.003 and 0.039, respectively).
For the Hyperactivity subscale, the 5mg dose showed a statistically significant benefit in favor of eltorprazine treatment compared to placebo (p=0.008); the 10mg dose was superior to placebo, however the difference was not statistically significant (p=0.130).
For the Impulsivity subscale, no significant benefit was observed for either drug dose compared to placebo.
Both 5mg and 10mg demonstrated significantly greater improvement over placebo for CGI-I scores (p=0.023 and 0.004, respectively).
The percentage of subjects who were considered improved by the investigator was 57.9% for placebo, 68.4% for 5mg, and 81.1% for 10mg. The percentage difference was significant between 10mg and placebo (0.029), but it was not between 5mg and placebo (p=0.342).
The results indicate the overall positive outcomes reported on the ADHD-RS-IV were largely driven by the Inattention and Hyperactivity subscales. This phenomenon was expected because most enrolled subjects had primary deficit in Inattention at baseline. Significant benefits of the active treatments were also observed for the following secondary efficacy variables:

Profile of Mood States (POMS):

The 5mg dose was statistically significantly better than placebo for POMS total score (p=0.006)
Both the 5mg and 10mg groups were statistically significantly better than placebo for anger-hostility score (p<0.001 and p=0.036, respectively)
The 5mg group was also statistically significantly better than placebo for tension anxiety score (p=0.046)
Barnes Akathisia Scale (BAS)

Both 5mg and 10mg groups showed a reduction in restlessness and were statistically significantly better than placebo for BAS (p=0.029 and p=0.007, respectively)
Both 5mg and 10mg groups were statistically significantly better than placebo for Awareness of Restlessness subscore (p=0.003 and p<0.001, respectively).
The 10mg group was also statistically significantly better than placebo for Distress Related Restlessness subscore (p=0.047)
Abnormal Involuntary Movement Scale (AIMS)

10mg showed a significantly greater reduction in abnormal movements than placebo (p<0.001)




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