The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs, today announced an agreement under which they will seek to accelerate the discovery of novel biomarkers and development of assays to measure Parkinson’s disease progression. The joint initiative will focus on biomarkers that may facilitate therapeutic approaches targeting alpha-synuclein, a protein potentially involved in the onset and progression of Parkinson's disease.
“As more potential therapies come closer to and cross the line to clinical testing — and, in parallel, the number of people with Parkinson’s grows as the population ages — the need for Parkinson’s biomarkers grows more urgent,” said Todd Sherer, PhD, CEO of MJFF. “Prothena is a leader in the development of potential treatments against our highest priority target, alpha-synuclein, and we are pleased to collaborate with them toward these tools to speed research.”
As part of the initiative, Prothena and MJFF will support scientific research studies directed toward biomarkers of disease progression and drug efficacy. In addition, Prothena and MJFF will work to identify opportunities where novel endpoints or assays may be incorporated into ongoing or future studies.
“We are committed to identifying new insights about the underlying cause and progression of Parkinson’s disease and believe that biomarkers clearly defining disease progression may both enhance this understanding and enable more effective, efficient clinical development of disease-modifying therapeutics for patients and families impacted by Parkinson’s disease,” said Dale Schenk, PhD, President and Chief Executive Officer of Prothena. “In addition, we believe this collaboration with MJFF will help inform the clinical development strategy for PRX002, a monoclonal antibody for the potential treatment of Parkinson’s disease. We look forward to reporting results from our ongoing Phase 1 multiple ascending dose study of PRX002 in patients with Parkinson’s disease during the first half of 2016.”
The Michael J. Fox Foundation has supported research in Parkinson’s biomarkers from its earliest days and leads the landmark observational Parkinson’s Progression Markers Initiative, a $60 million study taking place at 33 clinical sites around the world that aims to validate biomarkers of Parkinson’s disease. Biological markers that point to risk, onset or progression of Parkinson’s disease would allow researchers to more accurately diagnose and monitor Parkinson’s, stratify subjects for clinical studies, and efficiently evaluate impact of disease-modifying therapies, informing critical go/no-go decisions.
Alpha-synuclein is a protein found in neurons and is a major component of pathology that characterizes several neurodegenerative disorders including Parkinson’s disease, dementia with Lewy bodies, and multiple system atrophy, which collectively are termed synucleinopathies. While the normal function of synuclein is not well understood, the protein generally occurs in a soluble form. In synucleinopathies, the synuclein protein can misfold and aggregate to form soluble aggregates and insoluble fibrils that contribute to the pathology of the disease. There is also increasing evidence that this disease-causing synuclein can be propagated and transmitted from neuron to neuron, resulting in an infection-like spread of neuronal death. Recent studies in pre-clinical models suggest that the spread of synuclein-associated neurodegeneration can be disrupted by targeting aberrant forms of synuclein.
About Parkinson's Disease
Parkinson's disease is a degenerative disorder of the central nervous system that affects one in 100 people over age 60, and after Alzheimer's disease is the second most common neurodegenerative disorder. There are an estimated seven to ten million patients living with Parkinson's disease worldwide. Current treatments for Parkinson's disease are only effective in managing symptoms of the disease, mainly through the use of levodopa and dopamine agonists. As the disease progresses and dopaminergic neurons continue to be lost, these drugs eventually become less effective at treating the symptoms. In contrast, therapies that target alpha-synuclein may slow or reduce the neurodegeneration associated with aberrant forms of alpha-synuclein.