Proteostasis, Astellas Partner On Unfolded Protein Response Therapeutics
By Cyndi Root
Proteostasis Therapeutics and Astellas Pharma have begun a partnership to develop therapeutics modulating the Unfolded Protein Response (UPR). The two companies announced the collaboration in a press release, stating that studies will utilize Proteostasis’ proprietary Disease Relevant Translation (DRT) platform to focus on one genetic disease initially, with further explorations in additional indications possible. Kenji Yasukawa, Ph.D., Senior VP at Astellas, said, “Proteostasis Therapeutics has a novel platform that offers a differentiated approach to discovering drugs for unmet medical needs.”
Proteostasis and Astellas Agreement
Astellas intends to work closely with Proteostasis to identify lead candidates for clinical development and commercialization. Astellas will tender an upfront payment, a securities investments, development funding, and milestones worth up to $400 million, as well as provide royalties. If Astellas opts to continue the arrangement and development of two other projects, the deal is valued at $1.2 billion. Proteostasis retains the right to opt in for global co-development and co-promotion in the United States.
Unfolded Proteins
According to a review titled “Targeting the unfolded protein response in disease” published in Nature Reviews Drug Discovery Journal, unfolded proteins are evident in many diseases such as cancer, diabetes, autoimmune conditions, liver disorders, obesity, and neurodegenerative disorders. When unfolded proteins accumulate, stress accrues in the endoplasmic reticulum (ER) causing cell death and protein conformational diseases. By selectively modulating the UPR pathway, non-clinical investigations have shown improved stress response and restored function.
Proteostasis’ DRT platform
Proteostasis’ DRT platform identifies translatable therapies that show promise in modulating protein homeostasis pathways. The therapeutic candidates modify the cell’s quality control system also known as the Proteostasis Network (PN). The investigational products correct imbalances in the PN and may be able to correct genetic factors, disease states, and aging.
About Proteostasis
Proteostasis Therapeutics is headquartered in Cambridge, MA. The company is partnered with Biogen Idec on protein therapeutics. In July 2014, the two companies announced the successful completion of a preclinical milestone, with Proteostasis receiving a multi-million dollar milestone payment as a result. The study achieved protein reduction through inhibition of Usp14 in a whole animal system. The result was a biological validation and demonstration of the disease-modifying potential of the protein inhibitor against neurodegenerative disorders. Biogen Idec and Proteostasis began their collaboration in 2013 based on the inhibition of Usp14.