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news.Vertex Pharmaceuticals and Parion Sciences have entered into collaboration for the development of investigational epithelial sodium channel (ENaC) inhibitors to treat cystic fibrosis (CF) and other pulmonary diseases.
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As part of the deal, Vertex will have worldwide development and commercial rights to Parion’s investigational ENaC inhibitors, including P-1037 and P-1055.
Parion will receive an upfront payment of $80m and will be eligible to receive up to an additional $490m in development and regulatory milestone payments for development of ENaC inhibitors in CF, including $360m related to global filing and approval milestones.
Vertex will be responsible for future development activities for P-1037 and P-1055, which are indicated for the treatment of CF and other pulmonary diseases.
Currently, P-1037 is being evaluated in an exploratory Phase IIa study in about 120 people with CF, regardless of genotype.
The two firms also plan to initiate an additional Phase IIa trial that adds P-1037 to treatment with the investigational combination of lumacaftor and ivacaftor for people with CF who have two copies of the F508del mutation.
Vertex executive vice-president and chief medical officer Jeffrey Chodakewitz said: "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.
"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."
CF is a rare genetic disease that is caused by defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene.
Parion president and CEO Paul Boucher said: "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 II development.
"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."
Image: Vertex will have development and commercial rights to Parion’s ENaC inhibitors P-1037 and P-1055. Photo: courtesy of Michelle Meiklejohn / freedigitalphotos.net.