Prothena Corporation plc (NASDAQ:PRTA), a clinical-stage biotechnology company focused on the discovery and development of novel therapies in the neuroscience category, today announced that results from the Phase 1b multiple ascending dose study of PRX002/RG7935, an investigational monoclonal antibody for the potential treatment of Parkinson’s disease, has been published in JAMA Neurology. PRX002/RG7935 is the focus of a worldwide collaboration between Prothena and Roche.
The data, which were previously presented as part of a late-breaking oral session at the 13th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in Vienna, Austria in April 2017, demonstrated that PRX002/RG7935 was found to have an acceptable safety and tolerability profile in patients with Parkinson’s disease, meeting the primary objective of this study. CNS penetration was demonstrated by a dose-dependent increase in PRX002/RG7935 levels in CSF, and a mean concentration of PRX002/RG7935 in CSF of 0.3 percent relative to serum across all dose levels. Additional results showed a rapid, dose- and time dependent mean reduction of free serum alpha-synuclein levels of up to 97 percent after a single dose, which were statistically significant (p<0.0001), and maintained following two additional monthly doses. The study results supported advancing PRX002/RG7935 into the PASADENA Phase 2 clinical study in patients with early Parkinson’s disease that is currently ongoing.
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.