Vertex Gains $8 Billion Overnight as Cystic Fibrosis Combo Drug Wows in Trials

Published: Jul 21, 2017

Vertex Gains $8 Billion Overnight as Cystic Fibrosis Combo Drug Wows in Trials July 19, 2017
By Alex Keown, Breaking News Staff

BOSTON –Shares of Vertex Pharmaceuticals are skyrocketing in premarket trading after the company announced positive efficacy data from the company’s Phase I and Phase II studies of three different triple combination regimens for cystic fibrosis patients with strains that have been hard to treat using current medications on the market.

Late Tuesday Boston-based Vertex, the maker of CF drugs Kalydeco and Orkambi, said trial data demonstrate the potential to treat the underlying cause of cystic fibrosis in patients who have one F508del mutation and one minimal function mutation (F508del/Min) – one of the most difficult types of CF to treat. Trial data shows patients experienced a significant boost in lung function of 9.6 percent to 12 percent in the different trials using the experimental Vertex corrector drugs, VX-440, VX-152 and VX-659. Patients diagnosed with cystic fibrosis typically experience difficulty breathing due to a thickening of the mucus lining the lungs. Improved lung function was the primary endpoint of the trials.

Investors are clearly happy with the early-stage results and see a bright future for the company, as well as for the hopes of cystic fibrosis patients. If the drugs continue to show strong data through late stage trials, Vertex will be well on its way to providing treatment for thousands upon thousands more cystic fibrosis patients. Stat News’ Adam Feuerstein noted that Vertex, which has two approved CF drugs and another waiting regulatory approval, “can already successfully treat the underlying cause of cystic fibrosis in roughly half of the 75,000 patients worldwide.” If the three drug regimens from these trials make it to the market, Feuerstein said the company will be able to treat about 90 percent of the patient population. There are an estimated 24,000 patients with the F508del mutation, Feuerstein said.

Steven Rowe, director of the Gregory Fleming James Cystic Fibrosis Research Center and a member of Vertex’s steering committee, said patients with minimal function mutations have been waiting for a medicine to treat the underlying cause of their disease.

Vertex said the triple combination regimens were generally well tolerated across all three studies, and the majority of adverse events were mild to moderate in severity. Two patients who were in the VX-440 group did experience high levels of liver enzymes, which could have led to toxicity issues. However those symptoms went away when the treatment was discontinued for them, Vertex said. Share prices are up more than 26 percent, taking the stock to $166.80 in premarket trading.

Jeffrey Chodakewitz, Vertex’s chief medical officer, said the trial data are compelling and indicate “significant potential benefit for people with CF from each of these three different triple combination regimens.”

“We will be collecting and evaluating additional data from these and other studies and will make a decision on which regimen(s) to take forward into pivotal program(s), which we expect to begin in the first half of 2018,” Chodakewitz said in a statement.

Vertex has advanced four next-generation correctors in parallel with the goal of developing the best triple combination regimen or regimens for people living with CF, the company said late Tuesday. Vertex said it has accelerated the development programs for VX-445 and VX-659. A VX-445 Phase II study is underway and a VX-659 Phase II study will begin in early August. VX-445 and VX-659 will be evaluated in triple combination with tezacaftor and ivacaftor (Kalydeco) in people with one F508del mutation and one minimal function mutation and will be evaluated in people with two copies of the F508del mutation who are already receiving tezacaftor and ivacaftor.

Data from both of these Phase II studies are expected in early 2018. Pending additional data from these Phase II studies and the ongoing studies of VX-152 and VX-440, Vertex said it plans to initiate pivotal development of one or more triple combination regimens in the first half of 2018.

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