Amarantus seeks US FDA orphan drug status for eltoprazine to treat levodopa-induced dyskinesia Saturday, October 24, 2015, Amarantus Bioscience Holdings, Inc., a biotechnology company, has submitted a request to the US FDA for orphan drug designation (ODD) for eltoprazine in the treatment of levodopa-induced dyskinesia (PD-LID)."A successful application to the US FDA for ODD for eltoprazine in PD LID would complete the transition of our therapeutics portfolio into the orphan drug arena, thereby squarely positioning Amarantus as an orphan drug company," said Gerald E. Commissiong, President & CEO of Amarantus. The FDA Orphan Drug designation programme 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 US. 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. 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. 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 programme was out-licensed to PsychoGenics. PsychoGenics licensed eltoprazine to Amarantus following successful proof-of-concept trials in PD-LID and adult ADHD.
GEORGETOWN ASSAY The Georgetown Assay is a blood-based approach to diagnosing Alzheimer's disease (AD). Its validated 10 lipid panel provides up to 90% specificity with a 90% sensitivity in predicting those at risk of developing prodromal or manifest AD. According to the Alzheimer's Association, it is estimated that over 5.4 million people in the United States suffer from Alzheimer's disease. Over 500,000 patients are diagnosed annually, with nearly one-in-eight older Americans affected by the disease. Alzheimer's disease is the third leading cause of death in the United States. The cost of unpaid care in the United States is estimated at over $210 billion annually. Total payments for care are estimated at over $200 billion annually, including $140 billion in cost to Medicare and Medicaid. Alzheimer's expenditures in the United States are expected to exceed $1.2 trillion by 2050. There is no cure or effective treatment for Alzheimer's disease. Worldwide, about 35.6 million individuals have the disease and, according to the World Health Organization, the number will double every 20 years to 115.4 million people with Alzheimer's by 2050. On January 15, 2015 Amarantus executed a one-year, exclusive option agreement with Georgetown University to enter into a license for the patent rights related to certain blood based biomarkers for memory loss that Georgetown University and University of Rochester jointly own. Amarantus will be required to achieve timely milestones including providing Georgetown with development and commercialization plans for the biomarkers, share information related to Amarantus' diagnostic assets, CLIA validation of biomarkers, recruitment of a senior executive to lead Amarantus' diagnostics division and other requirements as defined in the agreement.
The Lymphocyte Proliferation Test (LymPro Test®) is a diagnostic blood test that determines the ability of peripheral blood lymphocytes to withstand an exogenous mitogenic stimulation that induces them to enter the cell cycle. It is believed that certain diseases, most notably Alzheimer's disease, are the result of compromised cellular machinery that leads to aberrant cell cycle re-entry by neurons. LymPro is unique in the use of peripheral blood lymphocytes (PBLs) as a surrogate for neuronal cell function, suggesting a common immune-based relationship between PBLs and neurons in the brain.
TRAUMATIC BRAIN INJURY / CHRONIC TRAUMATIC ENCEPHALOPATHY Traumatic brain injury (TBI) occurs when an external force traumatically injures the brain. TBI can be classified based on severity, mechanism (closed or penetrating head injury), or other features). Head injury and concussions usually refers to TBI, but is a broader category because it can involve damage to structures other than the brain, such as the scalp and skull. TBI is a major cause of death and disability worldwide, especially in children and young adults. Causes include falls, vehicle accidents, and violence. Prevention measures include use of technology to protect those suffering from automobile accidents, such as seat belts and sports or motorcycle helmets, as well as efforts to reduce the number of automobile accidents, such as safety education programs and enforcement of traffic laws. In addition to the damage caused at the moment of injury, brain trauma causes secondary injury, a variety of events that take place in the minutes and days following the injury. These processes, which include alterations in cerebral blood flow and the pressure within the skull, contribute substantially to the damage from the initial injury. Chronic Traumatic Encephalopathy (CTE) is a brain disorder caused by multiple Traumatic Brain Injuries. CTE is a neurological degenerative disease found in individuals who have been subjected to repetitive traumatic brain injuries by way of the acceleration of the head on impact and the subsequent damage to axons. While repetitive brain trauma is thought to be necessary to cause CTE, it is not sufficient, meaning that not everyone exposed to repetitive brain trauma will get the disease. Professional level athletes are the largest demographic to suffer from CTE due to frequent concussions from play in contact-sport. These contact-sports include American football, ice hockey, rugby, boxing, and wrestling. Other individuals that have been diagnosed with CTE were involved in military service, had a previous history of chronic seizures, victims of domestic abuse, and or were involved in activities resulting in repetitive head collisions. Reports of CTE have steadily increased in younger athletes, most likely due to increased awareness of the issue and perhaps due in part to athletes becoming bigger and stronger producing greater magnitudes of force in collision. The primary physical manifestations of CTE include a reduction in brain weight, associated with atrophy of the frontal and temporal cortices and medial temporal lobe. The lateral ventricles and the third ventricle are often enlarged, with rare instances of dilation of the fourth ventricle. Other physical manifestations of CTE include anterior cavum septi pellucidi and posterior fenestrations, pallor of the substantia nigra and locus ceruleus, and atrophy of the olfactory bulbs, thalamus, mammillary bodies, brainstem and cerebellum. As CTE progresses, there may be marked atrophy of the hippocampus, entorhinal cortex, and amygdala.
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