Proteostasis, Biogen Team up on Protein Homeostasis Drugs

12/9/13Follow @benthefidler

Proteostasis Therapeutics has just gotten some big-name help to try to tackle neurodegenerative diseases like Alzheimer’s and Parkinson’s.

Cambridge-based Proteostasis has inked a deal with Biogen Idec (NASDAQ: BIIB) to develop drugs that could treat certain neurodegenerative disorders by blocking an enzyme known as Usp14 . Proteostasis is getting an unspecified upfront payment and equity investment from Biogen as part of the deal, and also stands to receive up to $200 million in research funding support, and development and commercial milestone payments—and eventually a royalty stream—if things break right.

Proteostasis will have the chance to either receive some of these milestone payments, or “opt in” for co-worldwide or co-U.S. rights to the drugs that come out of the partnership, the companies said.

The partnership is based on Proteostasis’s work in the emerging field of protein homeostasis, or the ability of cells to properly manufacture or deactivate proteins. When this homeostasis is upset, proteins do things they aren’t supposed to do, and this can lead to diseases. The idea, then, is to find tools that manipulate protein levels in a cell and use them to bring those cell processes back into balance. Proteostasis is one of a few companies, including Cleave Biosciences, Forma Therapeutics, and Millennium: The Takeda Oncology Company, that are interested in this type of approach.

What Biogen and Proteostasis will specifically work together on are drugs that inhibit Usp14, a deubiquitinating enzyme, or DUB. Researchers believe these enzymes play a key role in protein homeostasis. Proteostasis’ plan is to make a drug that blocks Usp14, and eradicate “aggregation-prone proteins” like tau (in Alzheimer’s) and alpha-synuclein (Parkinson’s) from building up and causing damage. The two companies intend to apply this approach to a “wide range of disorders,” though it didn’t specify which ones. Proteostasis licensed the technology behind the Usp14 blocking drug candidates from Harvard University. It was originally developed by Harvard professors Daniel Finley and Randall King, who are both on Proteostasis’ scientific advisory board.

The Biogen Idec deal adds another big name to Proteostasis’ investor group. Proteostasis made big news in 2008 when it raised $45 million in venture dollars from HealthCare Ventures, Fidelity Biosciences, New Enterprise Associates, Novartis Option Fund, and Genzyme. It handed out some more equity as part of a licensing with Elan in 2011, but it’s unclear where that partnership stands—Perrigo acquired Elan earlier this year, and while Elan is listed as an investor on Proteostasis’ website, it doesn’t make any mention of the deal.

This is the second Parkinson’s-related partnership for Biogen, meanwhile, in the past three months. In September, it partnered up with Amicus Therapeutics (NASDAQ: FOLD) to advance small molecule drugs for the disorder that target an enzyme known as glucocerobrosidase (GCase).

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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