Proteostasis Therapeutics, Inc. (PTI), a biotechnology company focused on discovering and developing novel small molecule therapeutics designed to control the body’s protein homeostasis, has just revealed a novel class of therapeutics called, CFTR Amplifiers, indicated for cystic fibrosis (CF). These agents work to enhance other cystic fibrosis transmembrane conductance regulator (CFTR) modulating agents, such as potentiators and correctors, and have demonstrated efficacy in various CFTR mutation classes. This new class strengthens the company’s plans to discover and advance a broad-acting combination therapy, effective in CF patients carrying most mutations.
The company also announced it has begun developing PTI130, also a CFTR amplifier for CF. This compound has demonstrated superior pharmacologic properties that make it ideal for oral administration. Several 14-day, non-GLP preclinical toxicology tests on several dose groups showed PTI130’s favorable safety and tolerability profile in two distinct animal models. PTI130 was discovered by Proteostasis thanks to its proprietary Disease-Relevant Translation (DRT™) platform, which makes use of phenotypic assays and disease relevant models to identify highly translatable therapies associated with the modulation of protein homeostasis pathways within the cell; and through the generous support of the Cystic Fibrosis Foundation Therapeutics, Inc. – the not-for-profit subsidiary of the CF Foundation.
The company plans to file for an Investigational New Drug application with the US Food and Drug Administration for PTI130, and expects to launch the agent into Phase I clinical trials by the end of 2015.
“We are proud of our drug discovery platform that has allowed us to identify molecules with novel mechanisms of action such as CFTR amplifiers,” said Meenu Chhabra, President and Chief Executive Officer. “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.”
In other news on cystic fibrosis, a recent study from Israel found that three or more patients with Cystic Fibrosis in one family may be a risk for more severe disease and poor prognosis. This was clearly noticed in lower PFTs, a faster decline rate of PFTs, more bacterial airway colonization, and increased frequency of lung transplant. The authors concluded that healthcare professionals should strongly consider this risk factor, and manage the families’ burdens with CF accordingly.