Biogen Idec and Proteostasis forge $200M+ neurodegenerative pact

CAMBRIDGE, Mass.—Alzheimer’s disease, and some of its neurodegenerative cousins, came under fire from a new direction as Proteostasis Therapeutics Inc. recently brought in the R&D and clinical firepower of fellow Cambridge-based company Biogen Idec in a worldwide collaboration agreement.

The deal combines Proteostasis Therapeutics’ proprietary scientific platform and preclinical work on protein degradation with Biogen Idec’s neurodegenerative disease research and clinical development capabilities, with a plan to research and develop therapeutic candidates based on the inhibition of Usp14.

Proteostasis will receive an initial upfront payment from Biogen Idec, along with an equity investment, and is eligible for research funding support and future development and commercial milestones. This could mean total payments of as much as $200 million, plus tiered royalties if one or more products enters the market. Under the agreement, the companies will conduct preclinical research to identify lead compounds for clinical development. At specified points in development, Proteostasis will have the option to receive potential milestones or opt in for global co-development and U.S. co-commercialization rights.

“We look forward to advancing our disease-modifying approach to develop therapeutic candidates that can address several protein aggregation disorders, including Parkinson’s and Alzheimer’s disease,” said Dr. Markus Haeberlein, senior vice president and head of research at Proteostasis Therapeutics, in a news release.

Proteostasis develops novel therapeutics that regulate protein homeostasis, with an eye toward helping to improve outcomes for patients with orphan and neurodegenerative diseases. According to the company, preclinical research has shown that the inhibition of Usp14, a deubiquitinating enzyme, modulates proteasome activity and increases the degradation rate of aggregation-prone proteins, including a-synuclein in Parkinson’s disease and tau in Alzheimer’s disease. The collaboration is intended to develop Usp14 inhibitors as a disease-modifying approach for a wide range of disorders involving toxic protein aggregation.

“Proteostasis is a leader in this field, and we are pleased to be working with them to develop meaningful new treatments for patients with neurodegenerative and other diseases,” said Dr. Spyros Artavanis-Tsakonas, chief scientific officer at Biogen Idec.

Much of the news lately on the Biogen Idec website has been related to hemophilia therapies; the most recent major news from the company on the neurological front was in Sept. 2013, when Biogen Idec announced a deal with Isis Pharmaceuticals to advance treatment of neurological disorders based on antisense approaches—a deal potentially worth more than $320 million with combined upfront and milestone payments.