NeuroPhage Awarded Second Grant from Michael J. Fox Foundation for Parkinson’s Disease Research
Novel Approach to Target Disease-driving Misfolded Proteins
Cambridge MA, December 2013 – NeuroPhage Pharmaceuticals, Inc. today announced that it has been awarded an $822,000 grant by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) for its second-generation drug candidate, NPT088, based on NeuroPhage’s GAIM (general amyloid interaction motif) platform, which simultaneously targets multiple misfolded proteins central to many neurodegenerative diseases. In particular, the MJFF grant will be applied to research the effect of NPT088 on alpha-synuclein deposits in the brain, a pathological hallmark of Parkinson’s disease (PD). This is NeuroPhage’s second grant from MJFF applied to the GAIM platform. “NeuroPhage is honored to receive its second grant from the MJFF, which supports our GAIM platform as a unique approach to the treatment of PD and other neurodegenerative diseases characterized by deposits of misfolded proteins. This award will allow us to make continued progress on our program in PD with our second-generation candidate, NPT088, continuing to build on our strong preclinical data,” said Kimberley S. Gannon, PhD, Senior Vice President of Preclinical R&D at NeuroPhage. The proceeds of the new MJFF grant will be used to test the effect of NPT088 on alpha-synuclein accumulation in the brain in a preclinical model of PD. The preclinical study will be conducted in collaboration with Dr. Eliezer Masliah at the University of California, San Diego. NeuroPhage previously demonstrated that a single treatment of NPT001, a first-generation GAIM molecule, produced significant reductions in neuropathology along with improved motor performance in preclinical PD models. “We are pleased with the progress that NeuroPhage has made since our original collaboration began in June 2011 under a Therapeutics Development Initiative Award,” said Kuldip Dave, PhD, Senior Associate Director of Research Programs at MJFF. “NeuroPhage’s unique approach has been successful in reducing alpha-synuclein levels in the brain and improving motor function preclinically. Alpha-synuclein is a high priority target for our Foundation, and this is a promising approach for the treatment of PD.” NPT088 is in development for the treatment of conditions characterized by multiple misfolded proteins, with initial focus on PD and Alzheimer’s disease (AD). Multiple misfolded proteins are often present in such diseases and can increase disease severity and accelerate disease progression.
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 for Parkinson’s Research
As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $375 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
NeuroPhage Pharmaceuticals has fusion-protein drug candidates in development for neurodegenerative diseases, many of which cause progressive mental deterioration and dementia. NeuroPhage’s technology is based on a General Amyloid Interaction Motif (GAIM) that simultaneously targets multiple misfolded proteins or amyloids central to many neurodegenerative diseases such as Alzheimer’s and Parkinson’s diseases. In addition to targeting multiple misfolded pathological proteins, this unique disease-modifying approach recognizes early amyloid assemblies and existing protein aggregates to maximize potential therapeutic effect. NeuroPhage is initially developing candidates to treat Alzheimer’s and Parkinson’s disease, where a number of different misfolded proteins are present and their interactions can exacerbate disease progression.