Astellas, Proteostasis Forge $1.2 Billion Genetic Disease Drug Pact

Astellas Pharma, Proteostasis Therapeutics Forge $1.2 Billion Genetic Disease Drug Development Pact

November 4, 2014

By Mark Terry, Breaking News Staff

Proteostasis Therapeutics, Inc., located in Cambridge, Mass., announced today an R&D and commercialization agreement with Tokyo-based Astellas Pharma Inc . The focus of the agreement will be to research and develop compounds that modulate the Unfolded Protein Response (UPR) through the use of Proteostasis’s proprietary “Disease Relevant Translation” and “Proteostasis Network” platform.

The research will focus on a single genetic disease, then spread out to additional conditions that are affected by modulation of the UPR pathway. A number of genetic diseases, neurodegenerative and retinal degenerative diseases are related to unfolded proteins in the endoplasmic reticulum (ER). In non-clinical studies, selective modulation of the UPR pathway has improved stressresponse and restored function.

Proteostasis Therapeutics has a novel platform that offers a differentiated approach to discovering drugs for unmet medical needs,” said Astellas Senior Vice President and Chief Strategy Officer Kenji Yasukawa in a press release. “It complements our existing internal and externalized R&D initiatives and we continue to invest in innovative technologies that can provide new therapeutic options to patients.”

Astellas will make an initial upfront payment to Proteostasis as part of the agreement, along with securities investments. Proteostasis will also be eligible for research funding, future development and commercial milestones that could total more than $400 million in addition to tiered royalties. Astellas also may exercise two additional projects under the same terms for a potential collaboration value of $1.2 billion.

“We are very pleased to be collaborating with Astellas, a worldwide leader in the development of innovative therapeutics,” said Proteostasis President and CEO Meenu Chhabra in a statement. “Our novel approach to drug discovery, coupled with Astellas’ track record in drug development, will enable rapid discovery and development of therapies for important unmet medical needs.”

Astellas also announced today a three-year collaboration agreement with the Dana-Farber Cancer Institute to develop small molecule inhibitors of oncogenic K-Ras for cancer treatment, including lung cancer. About 30 percent of all cancers have mutations linked to K-Ras, which are typically difficult to treat with poor clinical outcomes. The research will be led by Nathanael Gray of the Dana-Farber Cancer Biology Department.

“We are pursuing open innovation in drug discovery and are looking for new drug discovery opportunities that satisfy unmet medical needs through collaboration with external partners,” said Yasukawa in a press release. “This collaboration with Dana-Farber perfectly illustrates this strategy and it is our hope that this collaboration to advance drug discovery will bring innovative cancer treatments to patients around the world.”

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