Vertex Inks Deal Worth $250 Million for Concert Pharma's Cystic Fibrosis Drug
March 6, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Boston-based Vertex Pharmaceuticals said today that it is buying cystic fibrosis (CF) drug candidate CTP-656 from Concert Pharmaceuticals .
CTP-656 is an investigational CF transmembrane conductance regulator (CFTR) potentiator. It is being developed as part of a once-daily combination regimen of CFTR modulators.
Vertex will pay Concert $160 million in cash for global development and commercialization rights. If the compound is approved as part of a combination regimen for CF, Concert could receive an additional $90 million in milestone payments in the U.S. and reimbursement in the United Kingdom, Germany or France.
“Our vision is to develop the most effective and convenient medicines for people with CF,” said Jeffrey Chodakewitz, Vertex’s executive vice president and chief medical officer, in a statement. “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 is basically the same drug as Vertex’s Kalydeco. The difference is that hydrogen atoms are replaced by deuterium atoms. This makes it last longer. CF patients take Kalydeco twice daily. Concert’s data indicates that CTP-656 could be taken one a day. Deuterium, known as “heavy hydrogen” is a stable isotope of hydrogen.
Vertex plans to combine CTP-656 with tezacaftor so that patients would only take one per day. Currently, Vertex has a combination of tezacaftor and Kalydeco in a late-stage clinical trial for a single tablet in the morning, but would also require a second dose of Kalydeco at night.
Concert initiated a Phase II trial in late 2016 of CTP-656. Once the licensing deal closes, Vertex will take over the trial and handle all future drug development activities.
TheStreet writes, “Concert’s business model is based on using deuterium chemistry to develop improved versions of existing drugs. Concert’s chemical modifications are typically done to drugs that are otherwise under patent protection, so from a legal standpoint, it’s easier for the company to enter into partnerships, which it has done with Avanir Pharma , Celgene and Jazz Pharmaceuticals ."
In a note to investors, LeerinkGeoffrey Porges wrote, “CTP-656 was one of numerous CF competitors in early clinical trials that have been advanced in parallel with Vertex’s race to concoct a triple-therapy regimen for the majority of CF patients, and today’s news signals, in our view, the willingness of Vertex to defend its dominant and valuable position in the market category.”
CF is a rare, life-threatening genetic disease. It affects about 75,000 people in North America, Europe and Australia. It is caused by a defective or deleted CFTR protein from mutations in the CFTR gene. In order for children to have CF, they must inherit two defective CFTR genes, one from each parent. There are about 2,000 known CFTR gene mutations.
The defective or missing CFTR protein causes poor salt and water flow into and out of the cells in numerous organs, including the lungs. This causes a buildup of thick, sticky mucus that can lead to chronic lung infections and progressive lung damage that eventually leads to death. For a person born with CF, the median predicted age of survival is 41 years. The median age of death, however, is 27 years.