CAMBRIDGE, Mass., Oct.17, 2012 /PRNewswire/ --NeuroPhage Pharmaceuticals, Inc. announced today positive data with NPT001 in an alpha-synuclein pre-clinical model for Parkinson's disease (PD). The study was funded by The Michael J. Fox Foundation (MJFF). NPT001 is a first-in-class drug candidate with potential disease-modifying activity that disrupts and clears a variety of amyloid aggregates in the brain. In addition to reducing beta amyloid and tau aggregates in Alzheimer's disease (AD) preclinical studies, the new study demonstrates that NPT001 disrupts alpha-synuclein fibrils which are thought to play a critical role in PD.
The study was conducted in collaboration with Dr. Eliezer Masliah at the University of California San Diego (UCSD) and demonstrated that a single NPT001 treatment produced significant reductions in neuropathology along with improved motor performance in the PD model. Specifically, NPT001 significantly reduced alpha-synuclein deposits in the brain and restored dopamine-producing cells to normal function. Deficits in dopamine production are responsible for many of the behavioral dysfunctions in PD. In addition, NPT001 was well-tolerated and produced no observable adverse effects.
The data will be presented at the upcoming 2013 ADPD meeting in Florence, Italy. "The effects produced by NPT001 are robust and impressive, and the treatment improved the critical functions that are impaired in the brain of Parkinson patients," said Dr. Franz Hefti, PD expert and Chairman of NeuroPhage's Scientific Advisory Board.
"We are excited by the results of this study showing dose-dependent amelioration of neuropathology and functional improvement in a Parkinson's disease pre-clinical model following treatment with NPT001. These results, taken together with our biochemical and cell data for alpha-synuclein, support the development of NPT001 for PD in addition to the ongoing clinical development for Alzheimer's disease," said Dr. Kimberley S. Gannon, NeuroPhage's Senior Vice President of Preclinical Research & Development.
NeuroPhage's technology platform permits the development of therapeutics that target multiple misfolded proteins involved in neurodegeneration such as beta amyloid and tau (involved in AD), as well as alpha-synuclein (involved in PD). In February 2012, NeuroPhage announced that it had received a grant from MJFF for PD research on NPT001.
About Parkinson's Disease
Parkinson's disease is a chronic, progressive disorder of the central nervous system and results from the loss of cells in an area of the brain called the substantia nigra. These cells produce dopamine, a chemical messenger responsible for transmitting signals within the brain. Loss of dopamine causes critical nerve cells in the brain, or neurons, to fire out of control, leaving patients unable to direct or control their movement in a normal manner. The symptoms of Parkinson's may include tremors, difficulty maintaining balance and gait, rigidity or stiffness of the limbs and trunk, and general slowness of movement (also called bradykinesia). Patients may also eventually have difficulty walking, talking, or completing other simple tasks. Symptoms often appear gradually yet with increasing severity, and the progression of the disease may vary widely from patient to patient. There is no cure for Parkinson's disease. Drugs have been developed that can help patients manage many of the symptoms; however they do not prevent disease progression.
About The Michael J. Fox Foundation
The Michael J. Fox Foundation is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today. The Foundation has funded over $304 million in research to date.
About NeuroPhage
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