BOSTON—Vertex Pharmaceuticals Inc. is looking to boost its cystic fibrosis pipeline with the signing of a definitive asset purchase agreement to acquire CTP-656 from Concert Pharmaceuticals. Under the terms of the agreement, Vertex will pay $160 million in cash for all worldwide development and commercialization rights to CTP-656, and should the drug be approved as part of a combination regimen to treat cystic fibrosis (CF), Vertex will pay up to an additional $90 million in milestones based on regulatory approval in the United States and reimbursement in the U.K., Germany or France. The transaction is subject to Concert shareholder approval and expiration of the waiting period under the Hart-Scott-Rodino Act. The deal has unanimous support from Concert's board of directors, which recommends that the company's shareholders vote in favor of the transaction.
"With Vertex's clinical and commercial expertise in CF, this agreement provides the optimal pathway to rapidly advance the development of CTP-656 for the benefit of cystic fibrosis patients," Dr. Roger Tung, president and CEO of Concert Pharmaceuticals, commented regarding the deal. "The financial strength provided to Concert by this agreement will allow us to advance CTP-543 into pivotal testing and broaden our proprietary development pipeline."
CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Concert developed CTP-656 by applying deuterium chemistry to modify Vertex's CFTR potentiator, ivacaftor, which was discovered by Vertex scientists and is approved in the United States, Canada, Europe and Australia for patients with CF who present with specific mutation in the CFTR gene. It's thought that the compound could be used as part of once-daily combination regimens of CFTR modulators that treat the underlying cause of CF. Concert received Orphan Drug Designation for CTP-656 for the treatment of CF in January. As part of this agreement, Vertex will acquire the rights to all of Concert's other CF research and preclinical programs.
Concert is currently conducting a Phase 2 study of CTP-656 in individuals with CF who have gating mutations. The company announced in December 2016 that it had initiated the Phase 2 trial, in which patients will receive either placebo or 20 mg, 100 mg or 150 mg of CTP-656 once-daily. There will also be an open-label Kalydeco comparator arm. The primary endpoint is a change from baseline in sweat chloride at Day 28, with secondary endpoints of change in percent predicted forced expiratory volume and change from baseline in CFQ-R Respiratory Domain. Top-line results from the study are expected by the end of 2017.
"Our vision is to develop the most effective and convenient medicines for people with CF," Dr. Jeffrey Chodakewitz, executive vice president and chief medical officer at Vertex, 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."
CTP-656 will join other compounds in Vertex's pipeline geared toward cystic fibrosis. At present, Vertex reports five compounds on its website that it is advancing in cystic fibrosis: VX-659, VX-440, VX-152, VX-371 and VX-661, which is in Phase 3 development.
The news was well received by the market, with George Budwell of Motley Fool reporting that Concert's shares rose as much as 91.5 percent during morning trading on March 6, when the deal was announced, and “settled to a 72-percent gain” as of 11:45 a.m.
As Budwell noted, “As CTP-656 is basically a long-acting version of Vertex's FDA-approved Kalydeco, Concert's ability to successfully commercialize this drug wasn't altogether clear. Vertex, after all, probably could have claimed patent infringement and blocked its launch altogether, or forced Concert to pay a hefty royalty stream. So this deal gives Concert a much-needed windfall of up to $250 million in total, and removes the significant risk of a costly legal battle.”