Vertex says cystic fibrosis drugs boost lung function

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Vertex Pharmaceuticals Inc Thursday said a combination of its cystic fibrosis Kalydeco and an experimental compound was shown to improve lung function in a mid-stage trial, sending its shares up nearly 8 percent.

The study found that treatment with Kalydeco and the experimental drug VX-661 for 28 days resulted in a 4.6 percentage point improvement in mean lung function for patients with two specific genetic mutations.

If eventually approved by regulators, VX-661 would be the second drug from Vertex that works by treating the underlying cause of cystic fibrosis, a rare genetic disease that impairs the lungs and digestive .

The disease is caused by defective or missing CF transmembrane conductane regulator (CFTR) proteins. Kalydeco is designed for patients with certain CFTR mutation. VX-661 is being tested in combination with Kalydeco, as in this Phase 2 trial, and in patients with different genetic mutations.

“It is good data and some believe it does have an incremental positive read-through to the all-important Phase 3 data later this summer,” said RBC Capital Markets analyst Michael Yee.

That trial will answer the question of whether a combination of Vertex drugs is effective in patients with two copies of the gene mutation not currently addressed by Kalydeco – a market representing about half of the 30,000 cystic fibrosis patients in the States.

A positive result “would be transformative to the cystic fibrosis community,” Yee said.

The most common side effects seen during the Phase 2 trial included cough, headache and upper respiratory tract infection.

But ISI Group analyst Mark Schoenebaum cautioned that Vertex has not disclosed results from the mid-stage trial for patients treated with a placebo.

Vertex also announced that first quarter sales of Kalydeco totaled $100 million, which fell short of the average analyst estimate of $107 million, according to Wells Fargo Securities.

Separately, The company reported an adjusted first quarter loss of $1.00 per share, also below the average analyst per-share loss estimate of 67 per cent.

Vertex said it would discontinue research into treatments for hepatitis C, a market dominated by sales of Gilead Sciences Inc’s Sovaldi. Vertex late last year sold off its non-North America royalty rights to hepatitis C drug Incivek. (Reuters)