Proteostasis Therapeutics Announces Presentation of Data For Its CFTR Amplifier for Cystic Fibrosis

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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Proteostasis Therapeutics, Inc. (PTI), a company that develops disease-modifying therapeutics for diseases involving protein processing, recently announced at a presentation during the 29th Annual North American Cystic Fibrosis Conference data on its transmembrane conductance regulator (CFTR) amplifier program for use in combined treatments for patients with cystic fibrosis (CF).

In Proteostasis’ presentation entitled “CFTR Amplifiers Are a New Class of CFTR Modulators,” the company demonstrated an increase in CFTR modulating activity in human bronchial epithelial (hBE) cells when its composites were used as single therapies, as well as results that these composites are able to further enhance CFTR when utilized as a combined treatment with the currently available clinical-stage correctors.

Since genetic mutations in CTFR are the underlying cause of cystic fibrosis, amplifiers of CFTR represent a new class of CFTR modulating drugs that can be used as treatments for CF. These compounds augment currently available CFTR modulators, including correctors and potentiators. In pre-clinical studies, CFTR amplifiers have shown to have an effect across CFTR mutation classes. This data forms the basis for the company’s strategy to build broad-acting combined treatment for patients with CF.

“We are very pleased with the clinical potential of the CFTR amplifier compounds that double the activity of the most effective combination of clinical-stage correctors in the gold-standard HBE cell assay, not only for the most common mutation, F508del/F508del, but in other mutations found in the cystic fibrosis population as well,” said Meenu Chhabra, President and Chief Executive Officer of PTI. “We are confident that we will continue to build on our promising preclinical results to advance our products toward clinical trials.”

PTI is working on the development of PTI-428 as a treatment for CF patients. The drug agent has exhibited pharmacologic properties amenable to oral dosing.

Results from a 28-day, non-GLP preclinical toxicology trial assessing multiple dose groups of PTI-428 in non-human primates revealed that the drug agent was safe and tolerable to be further developed. The company will advance PTI-428 as its drug candidate for patients with CF and aims to file an Investigational New Drug Application (IND) with the FDA by the end of this year.

The company has also started a preclinical program of studies in chronic obstructive pulmonary disease (COPD) using PTI-130 (CFTR amplifier), PTI-130.

COPD causes coughing, shortness of breath, and boosts the formation of mucus. In this regard, PTI-130 may be a new treatment strategy as it increases CFTR-mediated ion transport in non-CF hBE cells.

Pointing to the function of the CFTR gene may help to recover hydration as well as to restore the formation of mucus to normal airway levels.

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