Dive Brief:
- Despite falling short of forecasts, Vertex Pharmaceuticals is moving ahead with three next generation correctors, designed to treat the underlying cause of cystic fibrosis.
- Vertex said it is committed to expanding its portfolio with plans to advance its drugs under development in triple combination regimens, VX-440 and VX-152, in Phase 2 studies, and a Phase 1 additional next-generation corrector, VS-659. The studies should provide data by the end of this year.
- The Boston drugmaker’s revenues increased 35% compared to the third quarter a year ago, including its Orkambi drug sales increasing 79%. The company maintained its $950 billion to $990 billion annual revenue forecast updated earlier this month, but lower than a previous outlook that was pegged at $1 billion to $1.1 billion.
Dive Insight:
Jeffrey Leiden, chairman and CEO for Vertex, was blunt in the aims of the company: expand the number of eligible patients treated with its medications and expand the brand for both Orkambi and its other major approved drug Kalydeco (ivacaftor), while also developing next generation combination regimens.
Vertex intends to increase the patients on drug by garnering reimbursement approvals outside the U.S.
"Our strategy all along has been to develop a portfolio of next-generation correctors, really for two reasons," Leiden added. "One: they do have different properties, they come in different flavors and we really want to see those different flavors in people and understand how they perform, and two: there is a probability of success here, right?"
In the third quarter, sales in the U.S. declined compared to the second quarter, largely the result of the slowing of refills during the summer months, while the company approached peak penetration for patients aged 12 and over, according to Stuart Arbuckle, the company’s chief commercial officer. "We have now seen the vast majority of these patients refill their prescriptions," Arbuckle said.
Since Sept. 30, when the Food and Drug Administration expanded approval to treat younger children ages 6 to 11, at least 6,400 patients have started treatment in the U.S., although there are 2,000 more eligible.
The company initiated therapy for 9,000 patients out of 29,000 eligible patients worldwide, but has failed to reach its sought-after margins in Germany and is still seeking reimbursement approvals for Orkambi in Europe, Canada and Australia.
The company said that it would begin two Phase 2 studies of next-generation correctors, VX-440 and VX-152 in a triple combination regimen, from 2 to 4 weeks including drugs tezacaftor and ivacaftor for CF patients with an F508del mutation. The first data from those studies are expected in the first half of 2017. An additional Phase 1 study of a next-generation corrector, VX-659, is slated to begin by the end of this year.
"We believe we are in a strong position to define the clinical activity of three different next generation correctors in patients and have data in 2017 that enhance the probability of success for our next-generation corrector program,” said Jeff Chodakewitz, chief medical officer.
Chodakewitz said four-week dosing would be evaluated in the VX-440 study, with a specific contraception requirement for women of child bearing potential seeking to enroll, based on results from a pre-clinical toxicological study.
The test found some abnormality, suggesting a potentially deleterious effect on a fetus. "We don’t know whether that actually translated to people, but the prudent thing to do is clearly to ensure that women in the study are using highly effective contraceptive and that’s what we flagged," he said.