Vertex recently announced a collaboration with Parion Sciences with the goal of developing investigational epithelial sodium channel (ENaC) inhibitors as potential therapies for cystic fibrosis (CF) and other pulmonary disorders.
CF is a life-threatening genetic disease in which a defective gene (cystic fibrosis transmembrane conductance regulatory, CFTR) induces a salt imbalance, causing the body to form unusually thick, sticky mucus that can obstruct the airways and promote dangerous lung infections, resulting in serious respiratory and also gastrointestinal manifestations. The majority of the CF patients die due to respiratory failure. There is no cure for the disease, and it is estimated that almost 70,000 individuals worldwide suffer from CF.
The defective CFTR protein in patients with CF is thought to increase the function of ENaC, an ion channel important for active sodium reabsorption, which is essential for the maintenance of the body’s salt and water homeostasis. This increase may lead to dehydration of the lung cell surface in CF patients.
Parion is at the forefront of the development of investigational ENaC inhibitors, with their experimental P-1037 and P-1055 therapies, for CF and other pulmonary disorders such as non-CF bronchiectasis (NCFB) and chronic obstructive pulmonary disease (COPD). These inhibitors are expected to restore or improve the hydration of the cell surface in the lung, ultimately leading to an improved lung function.
“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 the President and Chief Executive Officer of Parion Paul Boucher in the press release. “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.”
P-1037 is currently being tested in a Phase 2a trial in CF patients, and Vertex and Parion plan to initiate in early 2016 an additional Phase 2a trial to assess the addition of P-1037 to treatment based on Vertex’s investigational combination of lumacaftor and ivacaftor for CF patients who have two copies of the F508del mutation (a specific CFTR mutation present in approximately 85% of the CF patients). Preclinical data in human bronchial epithelial cells from CF patients suggested that the addition of the investigational P-1037 to the combination lumacaftor/ivacaftor led to an increase in the hydration of the cell surface of the airways, which could potentially result in an improvement in lung function in patients.
“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 the Executive Vice President and Chief Medical Officer at Vertex Dr. Jeffrey Chodakewitz. “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.”
Based on the agreement, Vertex obtained worldwide development and commercial rights over Parion’s ENaC investigational inhibitors (including P-1037 and P-1055) for the potential treatment of CF and other pulmonary disorders. In turn, Parion received an $80 million up-front payment, with the possibility of receiving up to an additional $490 million depending on the achievement of specific milestones in the development of the ENaC inhibitors.
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