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Cambridge, Mass., June 5, 2013 -- Proteostasis Therapeutics, Inc., a company developing novel therapeutics that regulate protein homeostasis to improve outcomes for patients with neurodegenerative and orphan diseases, today announced it has received a significant grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to fund preclinical research for disease-modifying therapies that treat Parkinson’s disease (PD) by enhancing the clearance of a-synuclein.
“We are pleased to collaborate with The Michael J. Fox Foundation as we work to advance proteostasis regulators into the clinic and closer to the many Parkinson’s disease patients in need of more effective treatments,” said Dr. David Weiner, Chief Medical Officer of Proteostasis Therapeutics. “MJFF is the premier funder of patient-relevant Parkinson’s disease research, and we look forward to a very productive relationship while we transition our program to lead optimization later this year.”
Proteostasis Therapeutics has assembled an integrated platform to identify small molecules that modulate Proteostasis Network pathways, including those involving protein folding, trafficking, and clearance. The Company’s work in protein clearance focuses on enhancing proteasome-mediated degradation of protein aggregates in neurodegenerative diseases. Preclinical research by Proteostasis Therapeutics and scientific co-founders Drs. Daniel Finley and Randall King of Harvard Medical School has identified significant changes in a key cellular protein degradation pathway in PD patient brain samples.
Further research has shown that inhibiting Usp14, a deubiquitinating enzyme that modulates proteasome activity, can increase the degradation of aggregation-prone proteins such as a-synuclein, implicated as one of the potential causes of PD. The Company has used high-throughput screening and structure-based medicinal chemistry optimization to identify potent and selective Usp14 inhibitors as its leading therapeutic candidates, which it hopes to advance into the clinic in 2015.
“Of all the genetic targets discovered to date, alpha-synuclein has the strongest rationale for drug development toward a disease-modifying therapy for Parkinson’s disease,” said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation. “Partnering with teams like Proteostasis is a critical element of our strategy to speed the translation of synuclein research into practical treatments for patients.”
About Proteostasis Therapeutics
Proteostasis Therapeutics is developing disease-modifying therapeutics for orphan and neurodegenerative diseases. The Company’s lead programs in Cystic Fibrosis (CF) and protein aggregation diseases such as Parkinson’s disease modulate protein chaperone and proteasomal degradation pathways within the cell. These pathways are part of the cellular ‘quality control’ machinery, called the protein homeostasis network or Proteostasis Network (PN) that regulates protein folding, trafficking, and clearance. By enhancing the function and capacity of the PN, the Company’s product candidates correct for imbalances in the PN resulting from the cumulative effects of disease, genetic mutations, environmental factors, and aging. For more information, please visit www.proteostasis.com.
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
Proteostasis Therapeutics Contact:
Stern Investor Relations, Inc.
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