Roche, Prothena take aim at Parkinson’s
Collaboration could total $600 million if all milestones are met
BASEL, Switzerland—Roche and Prothena Corp. plc, a spin out from Elan plc, have announced the establishment of a worldwide collaboration for the development and commercialization of antibodies targeting alpha-synuclein. The collaboration will include PRX002, Prothena’s monoclonal antibody for the treatment of Parkinson’s disease. The compound is in preclinical development and is slated to begin Phase 1 clinical trials in Parkinson’s disease patients this year.
Per the terms of the agreement, Roche will pay Prothena $45 million in an upfront payment and near-term clinical milestone, with the potential for additional payments of as much as $380 million if certain development, regulatory and first commercial sales milestones are met, plus an added $175 million in ex-U.S. commercial milestone payments. The companies will collaborate to develop PRX002 for the treatment of Parkinson’s disease and potentially other synucleinopathies as well. Prothena will have an option to co-promote the compound in the U.S., where Roche and Prothena will share all development and commercialization costs and profits on a 70/30 basis, respectively. Outside of the United States, Roche will be solely responsible for developing and commercializing PRX002, and Prothena will be eligible for up to double-digit royalties on net sales.
“We are excited to be working with Roche to develop PRX002 as a disease-modifying treatment for Parkinson’s disease and potentially other synucleinopathies. Roche is a global leader in drug development with significant experience in developing drugs to treat neurological diseases,” Dr. Dale Schenk, president and CEO of Prothena, said in a news release. “By combining Roche’s expertise with our own, this collaboration will greatly enhance our development efforts with PRX002 and allow us to move forward in a more comprehensive manner. This collaboration also represents an important milestone in our growth as we continue to execute on our corporate strategy to be a leading fully-integrated biotechnology company.”
PRX002 has been shown in mouse models of Parkinson’s to reduce the appearance of synuclein pathology, protect synapse connections and improve mouse performance in behavioral testing. The compound is thought to slow or reduce the neurodegeneration associated with synuclein misfolding and/or cell-to-cell transmission of pathogenic forms of synuclein.
“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,” said 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 slow down or reduce its progression. This approach is consistent with our strategy in other neurodegenerative diseases, such as Alzheimer’s, Huntington, multiple sclerosis or spinal muscular atrophy, where we target the molecular pathophysiology and intervene early with the objective to slow down or halt the progression of disease.”
Alpha synuclein is a charged protein found extensively in neurons, and is a key factor of pathological inclusions in neurodegenerative disorders such as Parkinson’s disease, dementia with Lewy bodies, neurodegeneration with brain iron accumulation type 1 and multiple system atrophy. In conjunction with this agreement, Roche and Prothena will also kick off a research collaboration focused on the optimization of early-stage antibodies targeting alpha-synuclein. That work will incorporate Roche’s proprietary Brain Shuttle technology, which has demonstrated promising potential in increasing the transport of antibodies through the blood-brain barrier to the brain, a persistent challenge with large molecules.
Some 7 million to 10 million people are thought to suffer from Parkinson’s disease worldwide, making it the second-most-common neurodegenerative disorder after Alzheimer’s disease. While treatments exist that are capable of managing early symptoms of the disease, they lose effectiveness as the disease progresses.