Parion Sciences, a biopharmaceutical company developing treatments to improve and extend the lives of patients with innate mucosal surface defense deficiencies of the eye or lungs, recently announced that it has begun enrollment of patients with Cystic Fibrosis (CF) in a phase 2 clinical trial of its experimental therapy P-1037.
The trial, which is called “CLEAN-CF” and refers to “Clearing Lungs with ENaC inhibition in Cystic Fibrosis,” is a randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety and efficacy of P-1037 solution for inhalation in patients with CF, regardless of their genetic mutations.
Epithelial sodium channel (ENaC) inhibitors combined with a compound like P-1037 enables the blocking of sodium channels on airway surfaces. In pulmonary diseases where there is a build-up of excessively concentrated mucus, such as cystic fibrosis and chronic obstructive pulmonary disease, preclinical studies have found that blocking the ENaC channel reduces fluid absorption, allowing the mucus layers to be hydrated for a longer period of time. Hydration of mucosal surfaces can restore airway clearance, reduce infection and improve function. P-1037 is a novel, long acting ENaC inhibitor that demonstrated a superior safety profile versus other known ENaC inhibitors in both pre-clinical and Phase 1 studies. Parion believes that it can facilitate longer periods of hydration in the lungs’ mucus layers, improving the health of those with Cystic Fibrosis.
A grant from the Cystic Fibrosis Foundation Therapeutics Inc. (CFFT) funded the start of the Phase 2 clinical trial.
The company is expecting to enroll about 120 patients across 30 research sites. “With P-1037 we look forward to the potential of having a novel therapy that is effective for people with cystic fibrosis regardless of their specific genotype,” said Dr. Scott Donaldson, Associate Professor of Medicine at the University of North Carolina, Chapel Hill and Co-Primary Investigator for the CLEAN-CF Trial in a recent news release.
Cystic fibrosis is a life-threatening disorder that causes severe damage to the lungs and digestive system. An inherited condition, cystic fibrosis affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery. In Cystic Fibrosis, however, a defective gene causes the secretions to become thick and sticky. Instead of acting as a lubricant, the secretions plug up tubes, ducts and passageways, especially in the lungs and pancreas.
“Initiating this phase 2 study in CF represents a major milestone for Parion as we continue on our mission to advance our first-in-class therapy to treat all CF patients,” said Paul Boucher, President of Parion. “The Parion team, our partners and the participating clinical sites, combined with Cystic Fibrosis Foundation Therapeutics’ support, have worked expeditiously to achieve this milestone as we now focus towards enrolling patients.”
More information about the trial can be found at: https://clinicaltrials.gov/ct2/show/NCT02343445?term=p-1037