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Company Announces New Class of Agents for Cystic Fibrosis

CFTR amplifier set for phase I trials

Proteostasis Therapeutics, Inc., based in Cambridge, Mass., has announced a new class of agents, cystic fibrosis transmembrane conductance regulator (CFTR) amplifiers, for the treatment of cystic fibrosis (CF).

CFTR amplifiers represent a new drug class that is able to enhance the effect of known CFTR-modulating agents, such as potentiators and correctors. According to Proteostasis, the amplifiers are effective across CFTR mutation classes.

The company also announced that it has nominated PTI130 as a clinical development candidate for the treatment of CF. PTI130, an amplifier, was found to have pharmacologic properties amenable for oral dosing. Preclinical toxicology studies testing multiple doses of PTI130 in animals demonstrated favorable safety and tolerability profiles for clinical development.

PTI130 was derived from medicinal chemistry optimization of hits from the company’s internal compound library based on its ability to double the efficacy of CFTR-modulating agents, such as correctors and potentiators, in human bronchial epithelial cells. These data provided a basis for the clinical exploration of adding PTI130 to the emerging standard of care (corrector/potentiator combinations). The discovery of PTI130 was partially funded by research grants from Cystic Fibrosis Foundation Therapeutics Inc., a nonprofit affiliate of the Cystic Fibrosis Foundation.

Proteostasis Therapeutics is expected to file an investigational new drug (IND) application for PTI130 with the FDA, and the molecule will enter phase I clinical trials by the end of 2015.

CF is the most common genetic disorder, affecting approximately 70,000 people worldwide. Improvement in disease management protocols and the approval of new drugs to treat the symptoms of the disease have extended the life expectancy for CF patients, which is now approaching 40 years of age. However, CF remains an incurable disease that leads to death. Recently, CFTR mutation-specific disease modulators, such as correctors and potentiators, have shown the ability to restore CFTR function in selected genotypes.

Source: Proteostasis Therapeutics; February 4, 2015.

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