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October 09, 2015 1:53 PM ET


Company Overview of Proteostasis Therapeutics, Inc.

Company Overview

Proteostasis Therapeutics, Inc. develops therapeutics to treat diseases of protein folding, trafficking, and clearance. The company offers disease-modifying therapeutics for cystic fibrosis, genetic diseases, and neurodegenerative diseases; and therapeutic strategies for unfolded protein response. It also develops therapeutics to pharmacologically control the Proteostasis Network (PN) by restoring its normal state or enhancing the capacity of the compromised PN to create a therapeutic state sufficient to control or delay progression of disease. In addition, the company offers products to treat gain-of-toxic-function disorders, which include Alzheimer’s, Huntington’s, and Parkinson’s diseases...

200 Technology Square

Suite 402

Cambridge, MA 02139

United States

Founded in 2006





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. Presents at Leerink Partners 4th Annual Rare Disease Roundtable, Sep-30-2015 11:55 AM

Proteostasis Therapeutics, Inc. Presents at Leerink Partners 4th Annual Rare Disease Roundtable, Sep-30-2015 11:55 AM. Venue: Le Parker Meridian, New York, New York, United States. Speakers: Markus Haeberlein, Chief Scientific Officer and Senior Vice President.

Proteostasis Therapeutics, Inc. Presents at Baird 2015 Healthcare Conference, Sep-09-2015 01:25 PM

Proteostasis Therapeutics, Inc. Presents at Baird 2015 Healthcare Conference, Sep-09-2015 01:25 PM. Venue: The New York Palace, 455 Madison Avenue, New York, New York, United States.

]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.

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