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Vertex increases the data on cystic fibrosis triple therapy


Vertex Pharma is the first phase 3 data from its highly anticipated three combination combinations for cystic fibrosis (CF), with the potential for regulatory registration in the following year.

Intermediate results were derived from two studies of two-drug treatment symptom (ivacafe / tezacafto), which was just approved as Symkevi in ​​Europe, with the experimental candidate VX-659 and found that the regimen significantly improved lung function in patients with CF compared to Symdecco plus placebo.

One study was performed on KF patients with one F508del mutation and one minor function mutation with an improvement of 14% versus placebo, while on the other side, patients with two F508del mutations were involved and improved by 10%.

William Blair analysts said the results were "strong and again captured by the Phase 2 data set," while Jefferies said that Vertex has "reached or exceeded this high bar and reached the high level of efficiency expectations."

CF is a rare, life-threatening genetic disorder caused by mutations in the CFTR gene, resulting in the formation of an unusually thick, contagious mucus in the lungs and the digestive tract. This in turn can lead to chronic pulmonary infections and progressive lung damage to many patients, which ultimately leads to death.

So far, data has only been collected from the first four weeks of treatment, 12 weekly data are available early next year, but Vertex is already convinced that the triple preparation is regulated. The company will hold it up until the second triple Symdeko and one more experimental candidate, the VX-445, will be seized as it plans to choose the best mode of operation.


Vertex & # 39; s Rehs Kewalramani

"These data are an important milestone in our efforts to develop new CF medicines as they highlight the important clinical benefits that can be provided by the triple combined regimen for most CF patients with at least one F508del mutations, "said Rehs Kewalramani, M.D., Executive Vice President World drug development and a medical specialist and chief medical specialist at Vertex.

"In the first quarter of next year, we plan to evaluate the VX-445 and VX-659 Triple Combination Scheme data and choose the best scheme to submit for potential approval with a view to introducing a new treatment option for those who have one F508del mutation and one minimal function mutation and those with two F508del mutations as soon as possible. "

VX-445 triple research results are fully included in the first quarter of next year. After these results are available and selected, Vertex says it will be initially processed by the United States, followed by applications in Europe and other markets at the end of 2019.

Values ​​for one and two drugs CF drugs are already importing booklets and is estimated to reach $ 3 billion this year, but William Blair believes the triple will be even more successful. Its analysts predict a maximum sales volume of $ 8.9 billion, based on estimates expected to begin in the first half of 2020.

They also believe that Vertex looks better than the combination of three drugs from Galapagos and AbbVie, which last month generated top-line data that they described as "inadequate". However, stronger competition with Vertex could be from Proteostasis, which has already reported spectacular data with a two-drug use scheme and a triple study, as it will be read in the coming weeks.

Just how big a Vertex product is tripled will depend on the results of the competitor's research, as well as on the price it can charge for the scheme.

In England, which is an important market, since it is the world's second largest population of KF patients, it is already undergoing re-imprisonment – the pricing and remuneration of Orkambi (snow chalet / ivacaft) for the old two-drug regime.

Vertex recently rejected the NHS offer in England, which gave it "potential" in the next decade to generate revenue from £ 1bn from current drug stability, but needed a nearly 90% discount that Germany pays for one of the Vertex medicines. Members of Parliament were threatened with disclosure of Vertex's confidential quotation to Orkambi with an increasingly dangerous dispute.

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