January 29, 2015 11:23 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
Age: 40
Chief Scientific Officer and Senior Vice President
Head of External Innovation
Head of Chemistry
Head of Systems Biology
Compensation as of Fiscal Year 2014.

Proteostasis Therapeutics, Inc. Key Developments

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.

Proteostasis Therapeutics, Inc. Announces Executive Appointments

Proteostasis Therapeutics, Inc. announced the appointment of six of the world's leading cystic fibrosis experts to form its clinical advisory board. The newly appointed board will serve as a strategic resource for the upcoming selection and study of the company's leading compounds that double the activity of the most effective combination, ivacaftor/lumacaftor in the gold-standard HBE cell assay, for the most common mutation, F508del/F508del, found in the cystic fibrosis population. Dr. Richard B. Moss, MD, a Professor Emeritus of Pediatrics at the Lucile Salter Packard Children's Hospital at Stanford University, will serve as Chairman of the Clinical Advisory Board. He is the former Chief of Pediatric Pulmonary and Allergy Divisions, and allergy-immunology and pulmonary fellowship director. Dr. Jane C. Davies, MD, FRCPCH, is Professor of Paediatric Respirology and Experimental Medicine at Imperial College, London and an Honorary Consultant in Paediatric Respiratory Medicine at the Royal Brompton & Harefield NHS Foundation Trust. Dr. Michael R. Knowles, MD, is Professor of Pulmonary and Critical Care Medicine at University of North Carolina (UNC), Chapel Hill. He is also the head of two multicenter studies, the first, Genetic modifiers of disease phenotype (severity) in cystic fibrosis lung and liver disease, which also includes a recently formed international consortium that is conducting a whole genome scan. Dr. Felix A. Ratjen, MD, PhD, is the Chief of Paediatric Respiratory Medicine at The Hospital for Sick Children, Professor of Paediatrics at The University of Toronto, and Senior Scientist at the Research Institute in the Department of Physiology and Experimental Medicine. Dr. Isabelle Sermet-Gaudelus, MD, PhD, is Professor of Pediatric Medicine at l'Hôpital Necker-Enfants Malades in Paris, France. She has developed and conducted several therapeutic trials for cystic fibrosis for both academic and company-initiated investigations. Her research focuses on modulation of the clinical severity of cystic fibrosis depending on fluid transfer and its therapeutic applications. Dr. Pamela L. Zeitlin, MD, PhD, is Professor and Director of Pediatric Pulmonary Medicine and the Co-Director of the Cystic Fibrosis Center at Johns Hopkins University.

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