Vertex and Parion Sciences Sign Up to $1.17 Billion Cystic Fibrosis Deal

Vertex Pharmaceuticals and Parion Sciences have teamed up to develop new drugs for treatment of cystic fibrosis (CF) and other pulmonary diseases.

The companies have initiated a collaboration worth up to $1.17 billion, under which Vertex and Parion will work together to develop investigational epithelial sodium channel (ENaC) inhibitors for the potential treatment of CF and other pulmonary diseases, including Chronic Obstructive Pulmonary Disease (COPD), Non-CF Bronchiectasis (NCFB) and Primary Ciliary Dyskinesia (PCD). Vertex will pay privately-owned Parion $80 million upfront and nearly $1.1 billion in potential milestone payments.

Under the deal, Vertex gains worldwide development and commercial rights to Parion’s investigational ENaC inhibitors, including P-1037 and P-1055, for CF and other pulmonary diseases. P-1037 is currently being evaluated in an exploratory Phase IIa study in people with CF, regardless of genotype. The companies plan to start an additional Phase IIa study that adds P-1037 to treatment with the investigational combination of Vertex’s lumacaftor and ivacaftor for people with CF who have two copies of the F508del mutation.

“This collaboration with Parion complements our ongoing work in CF and supports our two key goals in this diease – to increase the number of people eligible for new CF medicines and to enhance the benefit of treatment,” said Jeffrey CHodakewitz, MD, Executive Vice President and Chief Medical Officer at Vertex. “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 diversify our pipeline by providing opportunities to evaluate P-1037 as part of Phase 2a studies in multiple other diseases that impact the lungs.”

CF is a rare genetic disease that is caused by defective or missing CF transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing CFTR proteins result in poor flow of salt and water in and out of the cell in several organs, including the lungs. The defective CFTR protein that causes CF is also believed to increase the function of ENaC, which may contribute to dehydration of the cell surface of lungs in CF patients. Patients with CF buildup abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage, eventually leading to death. The disease affects approximately 75,000 people in North America, Europe and Australia.

“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.”

Source: Vertex Pharmaceuticals Incorporated

Last updated: 6/8/15; 12:00pm EST

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