Vertex shares rocketed yesterday after the company reported stellar results for three separate triple-drug treatments for cystic fibrosis, significantly improving lung function in patients with the genetic disease.
The new phase I and II data with the combinations suggests that Vertex could offer therapy that would benefit up to 90% of patients with CF. At the moment its two approved therapies – Kalydeco (ivacaftor) and Orkambi (lumacaftor and ivacaftor) – together account for less than half of all CF mutations.
In terms of the number of eligible patients, Vertex notes that around 30,000 people with CF are currently eligible for treatment with its drugs, but the triples could expand that to somewhere between 44,000 and 68,000. The prospects of a substantially bigger market for Vertex’ CF franchise spurred a 20%-plus rise in the firm’s share price – and prompted speculation that it may become a takeover target.
The trials involved people with CF caused by the F508del mutation – considered the toughest to treat in CF – plus another ‘minimal function mutation’ (F508del/Min). The studies tested three drugs VX-152, VX-440 and VX-659, given in triple combinations with tezacaftor and ivacaftor – a two-drug combination that has been submitted for approval in the US with an EU filing due in the coming weeks.
The three new drugs all achieved improvements in lung function (measured by forced expiratory volume) of around 10-12% over 15 days, and reduced the amount of chloride produced in the sweat glands – a biomarker for activity against the mutations in the CFTR gene that cause the disease. The improvements are roughly in line with those seen with Kalydeco and Orkambi in registration trials.
“Vertex plans to initiate pivotal development of any one or more of these triple regimens in the first half of 2018,” said the company’s chief medical officer Jeff Chodakewitz on a conference call to highlight the data.
That would give the US company a healthy lead over nearest rival – Belgian biotech Galapagos and partner AbbVie – which are also working on a triple regimen but look to be at least a year behind Vertex in clinical development.
Analysts at Jefferies said that the new data are a big boost to the sales projections for Vertex’ CF portfolio, which brought in $481m collectively in the first quarter of the year, a rise of 22% on the same period of 2016. They have raised the franchise’s peak sales potential from around $3bn to almost $7bn.