Biopharmaceutical company Parion Sciences recently announced the start of the enrollment process for a Phase 2 clinical trial of its product P-1037, a therapy for cystic fibrosis (CF). The trial is called “CLEAN-CF,” referring to “Clearing Lungs with ENaC inhibition in Cystic Fibrosis.”
CF is a life-threatening genetic disease in which a defective gene, called cystic fibrosis transmembrane conductance regulator (CFTR), causes the body to form unusually thick, sticky mucus that can result in serious respiratory and gastrointestinal manifestations. This thick, difficult to clear mucus is the result of the incapacity of the lung’s epithelial cells to provide sufficient fluid to the mucus layer, so it accumulates in the lungs, creating an environment prone to bacteria growth that can potentially lead to severe infections. It is estimated that approximately 70,000 individuals worldwide suffer from this disease, including 30,000 individuals in the United States.
It has been reported that in respiratory disorders like CF and chronic obstructive pulmonary disease (COPD), where excessively concentrated mucus accumulates in the lungs, the blockade of epithelial sodium channels (ENaC) on airway surfaces results in a reduction in fluid absorption, allowing hydration of the mucosal surfaces which in turn restores airway clearance, improving the lung function and potentially reducing the risk for infections.
Parion’s P-1037 is a new, long acting ENaC inhibitor that has been proven safe in both pre-clinical and Phase 1 trials. P-1037 exerts its action by rehydrating the mucus layers and consequently restoring airway clearance, improving lung function and reducing exacerbations.
The company’s goal is to enroll 120 patients at 30 different sites, especially within the clinical trials national network of Cystic Fibrosis Foundation Therapeutics Inc. CF patients can be enrolled in this Phase 2 trial irrespective of their genetic mutation.
“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 the Co-Primary Investigator for the CLEAN-CF Trial and Associate Professor at the University of North Carolina, Chapel Hill, Dr. Scott Donaldson in the press release.
“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 the President of Parion, Paul Boucher. “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.”
If you are interested in accessing a list of the clinical sites that will be enrolling participants for the CLEAN-CF trial, please visit this link.