Vertex and Parion Sciences Join Efforts to Develop Epithelial Sodium Channel (ENaC) Inhibitors for Cystic Fibrosis and Other Pulmonary Diseases

Vertex and Parion Sciences Join Efforts to Develop Epithelial Sodium Channel (ENaC) Inhibitors for Cystic Fibrosis and Other Pulmonary Diseases

Vertex Pharmaceuticals Incorporated and Parion Sciences will begin co-development of experimental epithelial sodium channel (ENaC) inhibitors as a therapy for treating cystic fibrosis (CF) and other pulmonary diseases such as Non-CF Bronchiectasis. The collaborative effort will provide Vertex with global development and commercial rights to ENaC inhibitors P-1037 and P-1055 from Parion.

Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex said in the news release that the collaboration with Parion adds to their current work in CF and “supports our two main objectives concerning this condition — to augment the number of individuals able to receive CF drugs and to improve the benefit of therapy.”

Presently, Parion is evaluating P-1037 by inhalation in individuals with CF, irrespective of genotype, in an exploratory Phase 2a study. The study is analyzing the use of P-1037, with and without hypertonic saline, compared to placebo. The study participants will continue to receive standard CF drugs.

Preclinical studies using human bronchial epithelial cells from people with CF with two copies of the F508del mutation demonstrated that the addition of experimental P-1037 to the combination of lumacaftor and ivacaftor led to an extra increase in both airway surface liquid and cilia beat frequency, indicative of increased hydration of the cell surface, when compared to baseline and to the use of P-1037 or lumacaftor/ivacaftor independently.

Based on these previous preclinical findings, both companies are planning to start in the beginning of 2016 an additional Phase 2a study that combines P-1037 with the experimental combination of lumacaftor and ivacaftor for individuals with CF who have two copies of the F508del mutation. The combination lumacaftor and ivacaftor therapy facilitates the proper functioning of salt and water transport in two phases. Lumacaftor seeks to supply the CFTR protein to the cell membrane and then ivacaftor enables the protein to interact more effectively with the cell membrane.

Dr. Chodakewitz stated the main objective of this collaborative work is to evaluate if ENaC inhibition can ameliorate lung function in individuals with CF, including the ones with mutations non-responsive to therapy using combined lumacaftor and ivacaftor. He added that this agreement will broaden their pipeline by providing opportunities to evaluate P-1037 as part of Phase 2a studies in several other conditions that affect 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.

Beyond CF, Vertex and Parion plan to conduct additional Phase 2a studies of P-1037 across multiple other pulmonary diseases where the disease results in defective hydration of the cell surfaces in the lung. These diseases include Chronic Obstructive Pulmonary Disease (COPD), Non-CF Bronchiectasis (NCFB) and Primary Ciliary Dyskinesia (PCD). Parion will conduct Phase 2a development in these diseases and retains an option to participate in co-development and co-commercialization activities related to one of these non-CF pulmonary diseases.