Diseases that strike the body’s neural systems are particularly difficult to treat. Once the damage is done, it can’t be reversed. Intervening early to slow or halt its progression offers the best hope to stave off a neurodegenerative disease’s debilitating effects.

Roche hopes to do just that for patients suffering from Parkinson’s disease. The Swiss drugmaker entered into a global collaboration with Irish biotech Prothena, worth up to $600 million, to develop and commercialize a monoclonal antibody to treat Parkinson’s disease, which is expected to enter the clinic in 2014. The experimental drug, PRX002, targets alpha-synuclein, part of a family of charged proteins found throughout the body. Alpha-synuclein is especially prevalent in neurons and plays a role in several neurodegenerative disorders including Parkinson’s disease.

“Parkinson's is a severely debilitating and progressive neurodegenerative disease that leads to both a gradual worsening of motor function and cognitive and behavioral alterations,” says Luca Santarelli, head of Neuroscience and Small Molecules Research at Roche. “Currently, there is no treatment that modifies its course, and by targeting one of Parkinson’s key molecular determinants, PRX002 has the potential to slowdown or reduce its progression.”

Roche’s approach to tackling neurodegenerative diseases, such as Parkinson’s, Alzheimer’s, Huntington, multiple sclerosis, or spinal muscular dystrophy, is to target its molecular pathophysiology and intervene early.

Roche and Prothena will collaborate on the development of PRX002 for Parkinson’s disease and potentially other diseases affected by synucleins. In the United States, the companies will share all development and commercialization costs in a ratio of 70 percent Roche and 30 percent Prothena. Prothena also has an option to co-promote PRX002 in the United States.Outside the United States, Roche will have sole responsibility and Prothena will be eligible for royalties on net sales.

Under the terms of their agreement, Roche will pay Prothena $45 million in an upfront and near-term clinical milestone payment. Prothena is also eligible to receive up to $380 million in development, regulatory and first commercial sales milestones, plus an additional $175 million in ex-U.S. commercial milestones.

As part of their agreement, the partners will also begin a research collaboration focused on optimizing early-stage antibodies targeting alpha-synuclein that includes using Roche’s proprietary delivery technology to increase delivery of therapeutic antibodies to the brain.
Parkinson’s disease affects about seven to ten million people worldwide. Current treatments are effective at managing the early motor symptoms of the disease but become less effective as the disease progresses.