New Collaboration on Unfolded Protein Response Modulators Could Benefit Cystic Fibrosis Drug Development

Ana Pamplona, PhD avatar

by Ana Pamplona, PhD |

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shutterstock_227880307Proteostasis Therapeutics, a company developing novel therapeutics for Cystic Fibrosis and other diseases to address defects in protein folding, trafficking and clearance, and Astellas Pharma Inc. (“Astellas”), a company dedicated to improving people’s health by providing innovative and reliable pharmaceuticals, announced a worldwide joint collaboration to research and develop therapeutic candidates that modulate the Unfolded Protein Response (UPR) through the use of Proteostasis Therapeutics’ “Disease Relevant Translation” (DRT™) and “Proteostasis Network” platform. The “Disease Relevant Translation” (DRT™) consists of a screening approach with state-of-the-art medicinal chemistry to synthesize highly selective drug candidates, and the  DRT™ platform features functional assays and important disease models to identify therapies associated with the modulation of protein homeostasis pathways in the cell.

The unfolded protein response (UPR) is a signaling pathway that is highly conserved in eukaryotic cells. This pathway is activated when proteins in the endoplasmic reticulum (ER), an organelle in the cell where synthesized secretory and transmembrane proteins, accumulate and are folded into their correct structures. Malfunction of the UPR causes numerous conformational diseases, including neurodegenerative disease, metabolic disease, inflammatory disease, diabetes mellitus, cancer, and cardiovascular disease. Thus, unfolded protein response (UPR) is a signaling pathway may be as potential therapeutic target for ER stress-related diseases.

This collaborative research will focus on one genetic disease, and will explore additional indications that may be targeted through modulation of the UPR pathway. In previous non-clinical investigations, the selective modulation of the UPR pathway improved the stress response and restored function, suggesting that this pathway is a potential target for disease-modifying therapies for various diseases with high unmet medical needs, such as CF.

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“We are very pleased to be collaborating with Astellas, a worldwide leader in the development of innovative therapeutics,” said Meenu Chhabra, President and CEO at Proteostasis Therapeutics, in the press release. “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.”

“Proteostasis Therapeutics has a novel platform that offers a differentiated approach to discovering drugs for unmet medical needs,” commented Kenji Yasukawa, Ph.D., Senior Vice President and Chief Strategy Officer at Astellas, “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. We look forward to working closely with Proteostasis Therapeutics to identify lead candidates for clinical development and potential commercialization,” added Dr. Kenji Yasukawa.

As part of the new research agreement, both companies will perform the identification, screening, and preclinical research of novel compounds for clinical development. After candidate selection, Proteostasis Therapeutics will have the rights to choose for worldwide co-development and co-promotion in United States. This collaboration is being conducted by Innovation Management (“AIM”) of Astellas Pharma Inc., a new Astellas’s division to improve and accelerate the process of screening and performing external collaborations to reinforce innovation at the preclinical development stage. The results from this joint collaboration are expected to be released in the end of March 2015.