Vertex to Acquire CTP-656 from Concert Pharmaceuticals for the Treatment of Cystic Fibrosis

-Vertex to develop CTP-656 for potential use in future combination regimens aimed at treating the underlying cause of CF-
-Concert to receive $160 million in cash with potential for $90 million in future regulatory approval milestone payments-

BOSTON, MA, USA I March 6, 2017 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it has signed a definitive asset purchase agreement to acquire CTP-656 from Concert Pharmaceuticals (Nasdaq: CNCE). CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination regimens of CFTR modulators that treat the underlying cause of cystic fibrosis (CF). As part of the agreement, Vertex will pay Concert $160 million in cash for all worldwide development and commercialization rights to CTP-656. If CTP-656 is approved as part of a combination regimen to treat CF, Concert could receive up to an additional $90 million in milestones based on regulatory approval in the U.S. and reimbursement in the UK, Germany or France. The agreement is subject to approval by Concert’s shareholders and the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Concert’s Board of Directors unanimously supports the transaction and recommends that Concert’s shareholders vote in favor of it.

“Our vision is to develop the most effective and convenient medicines for people with CF,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We look forward to exploring once-daily regimens that combine CTP-656 with other potential medicines from our broad CF pipeline that treat the underlying cause of the disease.”

CTP-656 was developed by Concert through the application of deuterium chemistry to modify Vertex’s CFTR potentiator, ivacaftor. Ivacaftor was discovered by Vertex scientists and is approved in the U.S., Europe, Canada and Australia for people with CF who have specific mutations in the CFTR gene. CTP-656 has the potential to play a key role in future once-daily combination regimens to treat CF. Concert is currently conducting a Phase 2 study of CTP-656 in people with CF who have gating mutations. As part of the agreement, Vertex will acquire rights to all of Concert’s other CF research and preclinical programs.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median predicted age of survival for a person born today with CF is 41 years, but the median age of death is 27 years.

About Vertex

Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.

Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia. For seven years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. For additional information and the latest updates from the company, please visit www.vrtx.com.

SOURCE: Vertex Pharmaceuticals

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