Proteostasis Therapeutics, Inc. just announced a new class of drugs – CFTR AMPLIFIERS – to be used in the treatment of Cystic Fibrosis. The company is a leading developer of new therapeutics for diseases characterized by defects in protein folding, trafficking and clearance.
Cystic fibrosis is a life-threatening disease caused by mutations in the Cftr gene (short for cystic fibrosis transmembrane conductance regulator) that leads to a progressive decline in lung function, affecting other organs as well, including the pancreas, liver, kidneys and intestines. Specifically, the CFTR protein regulates chloride and sodium ions membrane transport in cells. Thus, cystic fibrosis patients accumulate thick mucus that impairs cells’ normal function rendering cystic fibrosis patients more susceptible to infections. Cystic fibrosis is a deadly disease and the most common cause of death in cystic fibrosis patients is respiratory failure.
CFTR AMPLIFIERS are a new class of experimental drugs that work by enhancing the action of CFTR-modulating agents, a class of small molecules targeting specific defects caused by mutations in the CFTR gene, that include both Potentiators (increase the activity of defective CFTR at the cell surface) and Correctors (overcome defective protein processing that leads to misfolded CFTR).
Proteostasis Therapeutics also announced its selection of a specific candidate from its CFTR AMPLIFIERS – the PTI130 – to be developed for future clinical use in treating cystic fibrosis patients, mainly due to its excellent pharmacologic profile as an oral drug. PTI130 was already tested in preclinical toxicology studies where it was assessed as a safe and tolerable agent for clinical use. PTI130 was chosen due to its action of enhancing by two-fold the efficacy of CFTR correctors and potentiators in functional assays performed with human bronchial epithelial cells. Thus, PTI130 is a potential candidate to be introduced in current therapeutics and magnify patients’ benefits.
Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics commented, “We are proud of our drug discovery platform that has allowed us to identify molecules with novel mechanisms of action such as CFTR amplifiers. We believe that PTI130 places amplifiers on the vanguard of the rapidly evolving CF treatment paradigm and may prove to be the lynchpin for maximizing benefit for all CF patients regardless of the underlying CFTR mutation class.”