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August 03, 2015 12:43 AM ET


Company Overview of Proteostasis Therapeutics, Inc.

Company Overview

Proteostasis Therapeutics, Inc. engages in discovering and developing small molecule drugs that restore protein function to treat neurodegenerative, metabolic, genetic, and inflammatory disorders. Its products include therapies or Proteostasis regulators to treat various genetic and degenerative disorders associated with deficiencies of the Proteostasis Network, such as Alzheimer's disease, emphysema, Huntington's disease, and type II diabetes. The company develops its small molecule therapeutics to control the body's protein homeostasis or Proteostasis Network, which is cellular machinery for protein folding, trafficking, and clearance to maintain the body's balance of proteins. Proteostasi...

200 Technology Square

Suite 402

Cambridge, MA 02139

United States

Founded in 2007





Key Executives for Proteostasis Therapeutics, Inc.

Chief Executive Officer, President and Director
Age: 41
Chief Scientific Officer and Senior Vice President
Head of External Innovation
Head of Chemistry
Head of Systems Biology
Compensation as of Fiscal Year 2015.

Proteostasis Therapeutics, Inc. Key Developments

]Proteostasis Therapeutics, Inc. Unveils a New Class of Agents for Cystic Fibrosis Called CFTR Amplifiers

]Proteostasis Therapeutics, Inc. unveiled a new class of agents, CFTR AMPLIFIERS, for the treatment of cystic fibrosis (CF). CFTR amplifiers represent a new drug class able to enhance the effect of known cystic fibrosis transmembrane conductance regulator (CFTR) modulating agents, such as potentiators and correctors. The amplifiers are effective across CFTR mutation classes and form the basis for Proteostasis' strategy to develop a broad acting combination therapy able to serve CF patients with most mutations. Proteostasis Therapeutics also announced that it has nominated PTI130 as a clinical development candidate for the treatment of CF. PTI130, an amplifier, was found to have excellent pharmacologic properties amenable for oral dosing. Fourteen-day, non-GLP preclinical toxicology studies testing multiple dose groups of PTI130 in two different animal species demonstrated a favorable safety and tolerability profile for clinical development. PTI130 is fully owned by Proteostasis Therapeutics and was internally discovered through the Company's proprietary Disease-Relevant Translation, DRT™ platform. DRT™ utilizes functionally pertinent phenotypic assays and disease relevant models to identify highly translatable therapies associated with the modulation of protein homeostasis pathways within the cell. PTI130 was derived from medicinal chemistry optimization of hits from the Company's internal compound library based on its impressive ability to double the efficacy of CFTR modulating agents such as correctors and potentiators, in the HBE (human bronchial epithelial) cell Ussing functional assay. These data provide a basis for the clinical exploration of adding PTI130 on to the emerging standard of care (corrector/potentiator combination) to deliver even greater benefit to CF patients. The discovery of PTI130 was partially funded by research grants from Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation. Proteostasis Therapeutics is expected to file an IND for PTI130 with the US FDA and the molecule will enter the clinic in phase I trials by the end of 2015.

Proteostasis Therapeutics, Inc Appoints Dr. Po-Shun Lee as Vice President of Clinical Development

Proteostasis Therapeutics, Inc. announced appointment of Dr. Po-Shun Lee, MD, as Vice President of Clinical Development. His initial focus will be on leading the strategic selection process of which of the Company's proprietary PTI CFTR compounds should proceed to clinical development, as part of its leading program in cystic fibrosis. Prior to joining Proteostasis, Dr. Lee was the Translational Medicine Expert that led the cystic fibrosis and asthma programs from early development to proof of concept at the Novartis Institute for Biomedical Research.

Proteostasis Therapeutics and Astellas Pharma Enter Worldwide Collaboration Agreement

Proteostasis Therapeutics and Astellas Pharma have entered into a worldwide collaboration agreement for the discovery, screening, and clinical development of potential therapeutic candidates that target the unfolded protein response (UPR) cellular mechanism. By leveraging Proteostasis's proprietary disease relevant translation and Proteostasis network platform, candidates will be identified as potential therapeutic agents to treat one undisclosed genetic disease, as well as the potential to explore additional indications known to be modulated by the UPR pathway. Once a candidate is selected, Proteostasis will have the option for co-development with Astellas globally, and will co-promote the potential therapy in the United States. Based on the terms of the agreement, Proteostasis will receive an upfront payment, as well as a securities investment from Astellas, and will be eligible for additional payments for financing research and achieving certain development and commercial milestones that total USD 400 million, in addition to tiered royalties on sales. Furthermore, Astellas will have the option to initiate two other programmes under the same agreement conditions, which could raise the cumulative potential value of the deal to USD 1.2 billion. The partnership will be managed by Astellas's Innovation Management arm. Proteostasis is focused on the development of novel therapeutics that treat disorders caused by defects in protein folding, trafficking, and clearance. The misfolding and accumulation of unfolded proteins is implicated in a variety of diseases that are referred to as protein conformational diseases, including genetic diseases, neurodegenerative conditions such as Alzheimer's and Parkinson's diseases, as well as retinal degenerative diseases. Modulation of the UPR system that typically responds to this type of protein-misfolding situation in the endoplasmic reticulum could improve the stress response and restore function within the cell. UPR modulators could offer a novel disease-modifying treatment option for disorders and conditions that currently have no effective treatment or cure. Meanwhile, the partnership will benefit from Astellas's extensive experience in clinical development that will probably expedite the process of potentially bringing new UPR modulators to market.

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