Vertex Pharma Cystic Fibrosis Combo Therapy Hits Key Endpoints in Two Pivotal Trials

BOSTON (TheStreet) -- Combination therapy of two Vertex Pharmaceuticals (VRTX) drugs, VX-809 and Kalydeco, produced a statistically significant improvement in lung function for patients with the most common form of cystic fibrosis. The two phase III studies dubbed "TRAFFIC" and "TRANSPORT" both met their primary endpoints separately. When the studies were pooled together, cystic fibrosis patients treated with the higher of two doses of VX-809 (also known as lumacafator) and Kalydeco saw their lung function improve by 3.3% on an absolute basis compared to placebo.

Vertex's combination regimen also achieved statistical significance versus placebo on two key secondary endpoints of the phase III studies -- a 30% and 39% reduction in pulmonary exacerbations and improvements in weight gain (body mass index.) [Vertex shared the study results with me on Monday night under embargo.]

OMG! This sounds like the home run scenario Vertex bulls were dreaming about for the TRAFFIC and TRANSPORT studies, right? 

Maybe not a home run, per se, but sticking with the baseball analogy, I'd say the study results are a base-clearing triple. Vertex skeptics are going to furrow their brow and question whether the 3.3% absolute improvement in lung function is clinically meaningful. Sure, a 5% absolute improvement in lung function would have been preferred, but the pooled, 3.3% lung function improvement (at the highest VX-809 dose) was highly statistically significant. Couple that with statistically significant reductions in pulmonary exacerbations and the weight gain -- both of which are really important clinical benefits for cystic fibrosis patients -- and you have a clear winner here.

By the way, the lower dose of VX-809 plus Kalydeco also produced a statistically significant, absolute improvement in lung function of 2.8% across the pooled studies. 

So, Vertex shares do what this morning?

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