Vertex Pharmaceuticals has signed a $160 million purchase agreement to acquire CTP-656, a potential treatment for cystic fibrosis (CF), from Concert Pharmaceuticals. The agreement covers development and marketing rights worldwide.
CTP-656 is a potentiator of the protein defective in CF, known as CFTR (cystic fibrosis transmembrane conductance regulator). Concert developed CTP-656 as a potential part of a CF combination treatment, by applying deuterium chemistry to Kalydeco (ivacaftor), a Vertex therapy for CF.
Kalydeco is approved by the U.S. Food and Drug Administration, and by similar regulatory agencies in Europe, Australia and Canada, to treat CF patients with certain gating mutations in the CFTR gene. It is first approved therapy that addresses the underlying causes of CF, rather than disease symptoms.
Concert is currently conducting a Phase 2 study (NCT02971839) of oral CTP-656 in about 40 CF patients who have at least one copy of certain CFTR gating mutations. Treatment will be given once daily for 28 days at three doses (20 mg, 100 mg and 150 mg), and its efficacy will be compared to patients treated with Kalydeco (open-label, 150 mg twice daily) or given placebo.
The trial, which is expected to end in December, is now enrolling patients in the U.S.; more information is available here.
“Our vision is to develop the most effective and convenient medicines for people with CF,” Jeffrey Chodakewitz, MD, Vertex’s executive vice president and chief medical officer, said in a press release.
“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,” he added.
CTP-656 is designed to be a new version of ivacaftor — deuterium-modified ivacaftor — using a platform that modifies drugs to create new products with greater metabolic stability, lower toxic by-products, and increased half-life.
If CTP-656 is approved in the U.S. as a combination therapy for CF, Concert under the agreement may receive up to an additional $90 million in milestones. It may also be eligible for reimbursement in the U.K., Germany and France. Vertex, in turn, will also acquire rights to all other CF research and preclinical programs from Concert.
CF is caused by a defective or missing CFTR protein, resulting from mutations in the CFTR gene.
About 2,000 mutations in the CFTR gene are known. Some of these lead to CF by creating defective or too few CFTR proteins at the cell surface, which results in poor salt and water flow in and out of cells in organs that include the lungs, resulting in abnormally thick, sticky mucus build-up that can lead to chronic lung infections and lung damage.