Prothena Announces Phase 1b Clinical Trial Results of PRX002/RG7935 for Parkinson's Disease Published in JAMA Neurology
The data, which were previously presented as part of a late-breaking oral session at the 13th
Alpha-synuclein, a protein found in neurons and other cells, 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 alpha-synuclein is not well understood, the protein normally occurs in a soluble form. In synucleinopathies, the alpha-synuclein protein can misfold and aggregate to form soluble aggregates and insoluble fibrils that contribute to disease pathology. There is increasing evidence that this disease-causing alpha-synuclein can be propagated and transmitted from neuron to neuron, resulting in a spreading of neuronal death. Recent studies in cellular and animal models suggest that the spread of alpha-synuclein-associated neurodegeneration can be disrupted by targeting aberrant forms of alpha-synuclein.
PRX002/RG7935 is a monoclonal antibody under development for the potential treatment of Parkinson's disease. PRX002/RG7935 targets alpha-synuclein and is designed to slow the progressive neurodegeneration associated with alpha-synuclein misfolding and/or the cell-to-cell transmission of the aggregated pathogenic forms of alpha-synuclein found in Parkinson's disease and other synucleinopathies. Prior to initiating clinical trials,
About Parkinson's Disease
Parkinson's disease is a progressive degenerative disorder of the central nervous system (CNS) that affects one in 100 people over age 60. With an estimated seven to 10 million patients living with Parkinson's disease worldwide, it is the second most common neurodegenerative disorder after Alzheimer's disease. The disease is characterized by the neuronal accumulation of aggregated alpha-synuclein in the CNS and peripheral nervous system that results in a wide spectrum of worsening progressive motor and non-motor symptoms. While diagnosis relies on motor symptoms classically associated with Parkinson's disease, non-motor symptoms may present many years earlier. Current treatments for Parkinson's disease are symptomatic and only address a subset of symptoms such as motor impairment, dementia, or psychosis. Symptomatic therapies do not target the underlying cause of the disease and lose effectiveness, often leading to debilitating side effects as the disease progresses.
This press release contains forward-looking statements. These statements relate to, among other things, the design of PRX002/RG7935, its proposed mechanism of action and its potential as a treatment for Parkinson's disease; how disease-causing alpha-synuclein might spread and how it might be disrupted; and amounts we might earn under our collaboration with
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Source: Prothena Corporation plc