Vertex Pharmaceuticals and Parion Sciences, a developmental-stage biopharmaceutical company, have formed a collaboration to develop investigational epithelial sodium channel (ENaC) inhibitors for the potential treatment of cystic fibrosis (CF) and other pulmonary diseases. Under the agreement, 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. Vertex and Parion plan to begin an additional Phase IIa study 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.

Parion received an $80 million up-front payment and has the potential to receive up to an additional $490 million in development and regulatory milestone payments for development of ENaC inhibitors in CF, including $360 million related to global filing and approval milestones. Parion has the potential to receive up to $370 million in additional development and regulatory milestones for P-1037 and P-1055 in non-CF pulmonary indications. Parion may also receive an additional $230 million in development and regulatory milestones should Vertex elect to develop an additional ENaC inhibitor from Parion’s research program. Parion will receive tiered royalties on potential sales of P-1037 and P-1055 in CF and other pulmonary diseases that range from the low double digits to mid-teens as a percentage of sales. Vertex will lead future development activities for P-1037 and P-1055 in CF and other pulmonary diseases.

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, non-CF cronchiectasis, and primary ciliary dyskinesia. Parion will conduct Phase IIa development in these diseases and retains an option to participate in co-development and cocommercialization activities related to one of these non-CF pulmonary diseases.

Source: Vertex Pharmaceuticals