March 4, 2014
Proteostasis Therapeutics, Inc., a company developing novel therapeutics that regulate protein homeostasis to improve outcomes for patients with orphan and neurodegenerative diseases, today announced an extension of its funded collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, to research, develop and commercialize therapeutic candidates to treat people with cystic fibrosis (CF) who have the most common CF mutation, DF508del. This extension will focus on moving the Company’s lead compounds toward a development candidate this year and a goal of filing an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration in 2015.
The collaboration will continue to focus on the development of small molecule proteostasis regulators that modulate protein homeostasis pathways within the cell to correct the folding, trafficking and functional activity of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). Preclinical research by Proteostasis Therapeutics, including data presented at the North American Cystic Fibrosis Conference in October 2013, has shown that the lead corrector series identified by the Company is able to significantly increase functional activity in DF508 patient-derived human bronchial epithelial cells. Additionally, the Company’s lead candidates have shown significant synergistic properties with existing clinical-stage corrector candidates, more than doubling maximal activity and providing a strong foundation for the potential of combination therapies.
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