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Vertex Pharma unveiled the first phase 3 data of its long-awaited combination of three drugs for cystic fibrosis (CF), establishing a potential regulatory filing next year.
The interim results come from two studies of his two-drug drug Symdeko (ivacaftor / tezacaftor) – which has just been approved as Symkevi in Europe – with the experimental candidate VX-659, and reveal that the treatment has led to an improvement significant lung function in CF patients compared to Symdeko plus placebo.
One study was performed in CF patients with F508del mutation and minimal function mutation, with a 14% improvement over placebo, while the other involved patients with two F508del mutations and a 10%.
William Blair's badysts said the results were "solid and replenished the Phase 2 data set," while Jefferies said Vertex "reached or exceeded that high bar and met the expectations of 39, highest efficiency ".
Cystic fibrosis is a rare genetic disease that shortens life and is caused by mutations in the CFTR gene that result in the formation of abnormally thick sticky mucus in the lungs and digestive tract. This in turn can cause chronic lung infections and progressive lung injury in many patients, eventually leading to death.
So far, the data only concern the first four weeks of treatment, but 12 data should be available early next year, but Vertex is already convinced that the triple is on the right track for them regulatory filings. The company will expect this, however, until the results reach us for a second triple combining Symdeko and another experimental candidate, called VX-445, because it intends to choose the best performing regimen to to follow.
"These data mark a major step forward in our efforts to develop new cystic fibrosis medications, as they underscore the important clinical benefit that a triple combination regimen can provide to the vast majority of CF patients with at least one form of cystic fibrosis. background treatment. F508del mutation, "said
"We plan to evaluate the triple combination regimens VX-445 and VX-659 in the first quarter of next year and to select the best treatment regimen for potential approval for the purpose of offer a new treatment option to F508del mutation and a minimal function mutation and to those with two F508del mutations as quickly as possible. "
The triple VX-445 studies are fully enrolled and the data must be reported in the first quarter of next year. Once these results are obtained and the selection made, Vertex indicates that it will initially file in the United States, followed by submissions in Europe and other markets by the end of 2019.
The Vertex Vertex Vertex and Vertex Vertex drugs are already generating sales of blockbusters and are expected to hit $ 3 billion this year, but William Blair believes the triple will be even more successful. Analysts forecast record sales of $ 8.9 billion on the basis of an estimated launch in the first half of 2020.
They also believe that Vertex's data looks better than those of a combination of three drugs from Galapagos and AbbVie, which generated leading data last month, which they describe as "disappointing". However, stronger competition for Vertex could come from Proteostasis, which has already released impressive data with a two-drug regimen and has a triple study to read in the coming weeks.
The size of a triple Vertex product will depend on the outcome of the competition studies as well as the price it can charge for the treatment regimen.
England is already in decline – a major market with the second largest number of cystic fibrosis patients in the world – in terms of pricing and reimbursement for two Orkambi (lumacaftor / ivacaftor) medications , older.
Vertex recently rejected an offer from NHS England that gave it the "potential" to generate revenues of GBP 1 billion over the next 10 years, but required a reduction of almost 90% in the price paid by the company. Germany for one of Vertex's drugs. In an increasingly acrimonious dispute, MPs have threatened to make Vertex's confidential bid for Orkambi public.
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