“We are excited to be working with Roche to develop PRX002 as a disease-modifying treatment for Parkinson’s disease and potentially other synucleinopathies,” Dale Schenk, Ph.D., president and CEO of Prothena, commented in a statement. “Roche is a global leader in drug development with significant experience in developing drugs to treat neurological diseases. 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.”

 

The two companies will collaborate to develop the compound for Parkinson’s disease, and possibly other diseases characterized by synuclein proteins.  In addition, Roche and Prothena will kick off a research collaboration that will seek to optimize early-stage antibodies that target alpha-synuclein and incorporate Roche’s proprietary Brain Shuttle technology, for increased delivery of antibodies to the brain.

 

Per the terms of the agreement, Roche will pay Prothena $45 million in upfront and near-term clinical milestone payments, with the potential for Prothena to receive up to an additional $380 million if certain development, regulatory and first commercial sales milestones are met, as well as up to $175 million in ex-United States commercial milestone payments. Prothena will hold an option to co-promote PRX002 in the United States, with Roche and Prothena sharing U.S. development and commercialization costs and profits on a 70/30 basis, respectively. Outside of the United States, Roche will be solely responsible for PRX002’s development and commercialization, and Prothena will stand to receive up to double-digit royalties on net sales. All told, the deal could be worth $600 million.

 

“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,” Luca Santarelli, head of Neuroscience and Small Molecules Research at Roche, noted in a press release. “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. 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 slowdown or halt the progression of disease.”

 

The synuclein family of proteins are found throughout the body, and of them, alpha-synuclein, is found extensively in neurons. As such, it plays a large role in pathological inclusions typical of a variety of neurodegenerative disorders, including Parkinson’ disease, dementia with Lewy bodies, neurodegeneration with brain iron accumulation type 1 and multiple system atrophy.

 

Mouse models of Parkinson’s disease have shown that passive immunization with 9E4 (the mouse version of PRX002), the appearance of synuclein pathology was reduced, synaptic connections were protected and behavioral testing resulted in improved performance by the mice. It is thought that the compound might be capable of slowing or reducing neurodegeneration caused by synuclein misfolding and/or cell-to-cell transmission of pathogenic forms of the protein.

 

 

SOURCE: Prothena press release