Proteostasis Therapeutics, a Cambridge, Mass.-based company developing novel therapeutics to address diseases caused by defects in protein folding, trafficking and clearance, and Astellas Pharma have entered into a worldwide R&D collaboration for therapeutic candidates that modulate the unfolded protein response (UPR) through the use of Proteostasis Therapeutics’ proprietary Disease Relevant Translation (DRT) and Proteostasis Network platform.
Proteostasis Therapeutics will receive an initial upfront payment from Astellas, along with a securities investment. Proteostasis Therapeutics also is eligible for research funding support, future development and commercial milestones that could result in total payments of over $400 million, as well as tiered royalties. Further, Astellas has the right to begin two additional projects under the same terms, which, if it fully exercised this right, would bring the total potential value of the collaboration to $1.2 billion.
The collaboration will focus on one genetic disease and further explore additional indications that can be affected through modulation of the UPR pathway. Stress induced by accumulation of unfolded proteins in the endoplasmic reticulum (ER) is observed in many diseases which now are recognized as protein conformational diseases, including genetic diseases, neurodegenerative diseases and retinal degenerative diseases. Selective modulation of the UPR pathway in non-clinical investigations improved the stress response and restored function, suggesting that it can be beneficial as potential novel disease-modifying therapies for multiple diseases with high unmet medical needs.
The companies will conduct discovery, screening and preclinical research to identify lead compounds for clinical development. Upon candidate selection, Proteostasis Therapeutics will have the rights to opt in for global co-development and U.S. co-promotion.