Vertex Pharmaceuticals Shares Explode on Phase 2 Cystic Fibrosis Study Results

After wrapping Thursday down by more than 2 percent, Vertex Pharmaceuticals (VRTX) rifled upward in extended trading after announcing that data from its phase II of VX-661 and ivacaftor showed a statistical improvement in lung function among patients with cystic fibrosis who have two copies of the most common mutation in the cystic fibrosis transmembrane conductance regulator gene. The mutation of the gene, also called F508del, leads to the body’s inability to regulate sweat, digestive fluids and mucus, among other things.

On the whole, cystic fibrosis, an autosomal recessive genetic disorder, attacks the lungs, pancreas, liver and intestine of its victims. There is no known cure for the disease

The study tested four dose levels of VX-661 dosed one time per day for 28 days in combination with ivacaftor dosed twice per day. Separately, the study evaluated patients treated with VX-661 independently for 28 days.

Patients at the higher dosages (100 and 150 mg combination treatment) showed a 9 percent improvement in lung function compared to the placebo group improvement of 7.5 percent at day 28.

“This first study of VX-661 and ivacaftor provides further validation of the strategy of combining a corrector and potentiator to improve lung function in people with the most common type of cystic fibrosis,” Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex.

Vertex added that VX-661 was well tolerated both alone and in combination with with ivacaftor.

The news is a breath of fresh air to Vertex after posting a loss in the fourth quarter on declining revenue for its hepatitis C drug Incivek. First quarter results are supposed to come on April 30.

Earlier this week, research firm Zacks boosting its rating of Vertex from “neutral” to “outperform” while keeping its price target of $56.80 on the stock. Chardan Capital also gave the Cambridge, Massachusetts-based biotech a “buy” rating earlier this month as it initiated coverage with an $80 price target.

Adding to the excitement around Vertex was news in April was news that it entered a non-exclusive agreement with Bristol-Myers Squibb (BMY) for phase II trials evaluating once-daily all-oral treatment regimens of Vertex’s hepatitis C drug and Bristol’s NS5A replication complex inhibitor daclatasvir for hep C patients.

With the downward close on Thursday, shares of VRTX were still ahead by more than 25 percent this year. The after-market news, however, sent shares sailing in excess of $80, marking gains of greater than 51 percent on the day.