Vertex and Parion Sciences to Co-Develop Epithelial Sodium Channel (ENaC) Inhibitors in Cystic Fibrosis

Vertex Pharmaceuticals Incorporated  and Parion Sciences recently announced a joint collaboration for the development of investigational epithelial sodium channel (ENaC) inhibitors to treat cystic fibrosis (CF) and other pulmonary conditions. Based on the agreement rights, Vertex will develop and commercialize Parion’s ENaC inhibitors, named P-1037 and P-1055, for the treatment of CF and other pulmonary conditions.

The efficacy of P-1037 is being assessed in a Phase 2a trial in CF patients, regardless of genotype. The companies are planning to launch another Phase 2a trial that adds P-1037 to an experimental combination treatment of lumacaftor and ivacaftor (also known by its brand name Orkambi) in CF patients with two copies of the F508del mutation.

Under the agreement, Parion will receive $80 million from Vertex, with the potential to receive other payments and royalties from P-1037 and P-1055 for treatment of CF and other pulmonary conditions.

“This collaboration with Parion complements our ongoing work in CF and supports our two key goals in this disease — to increase the number of people eligible for new CF medicines and to enhance the benefit of treatment,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex in a recent press release.  “The goal of these planned studies of P-1037 is to determine whether ENaC inhibition can improve lung function in people with CF, including those with mutations unlikely to respond to treatment with the investigational combination of lumacaftor and ivacaftor.  Beyond CF, this agreement helps to diversify our pipeline by providing opportunities to evaluate P-1037 as part of Phase 2a studies in multiple other diseases that impact the lungs.”

“ENaC inhibition represents a promising opportunity to potentially enhance the benefit of existing treatments for people with CF, and we have worked diligently to bring P-1037 from our research labs and into Phase 2 development,” said Paul Boucher, President and Chief Executive Officer of Parion.  “Vertex is the leader in developing new medicines that treat the underlying cause of CF.  We are pleased to enter into this collaboration to unify the scientific expertise of both companies to advance P-1037 in CF and other pulmonary diseases.”

Cystic fibrosis is a genetic disease caused by defect or absence of cystic fibrosis transmembrane conductance regulatory (CFTR) proteins due to CFTR gene mutation, which causes poor flow of salt and water into and out of the cell in a number of organs, including the lungs.