Let me be crystal clear: if the Elan-Pfizer-Johnson & Johnson triumvirate had positive Phase III data in Alzheimer's disease, executives would be grabbing megaphones and singing "Hallelujah" from the rooftops. In midtown last week near Pfizer headquarters, all I heard was car horns and ordinary conversation, so my guess is the data aren't good. In lieu of megaphones, company scientists are likely reaching for statistical pickaxes. Let the data mining begin!
The world might have to wait until the third quarter for the official announcement, but I'm pretty sure I know the answer to the bapi question now.
A Phoenix Rises: Hep C Yields to Cystic Fibrosis
(VRTX - Get Report) had a hepatitis C franchise? Neither do I.
Last week, Vertex unveiled interim data from a trial of the company's marketed cystic fibrosis (CF) drug Kalydeco in combination with the experimental VX-809, a "corrector" of the dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein. The study focused on individuals with a particular genetic mutation known as F508del; nearly half of the roughly 70,000 CF patients globally are homozygous F508del mutation carriers, meaning these individuals have two copies of the bad gene.
Despite having data from only a small cohort, the combined Kalydeco and VX-809 dose groups unexpectedly showed a statistically significant improvement in FEV1, a well-accepted measure of lung function, compared to placebo. Vertex bears initially focused on the fact that the company didn’t reveal the mean FEV1 change. Although I think the "back calculated" 3-5% mean FEV1 improvement estimated by several Wall Street analysts seems reasonable, investors should focus instead on the 46% and 30% of treated patients that had an FEV1 gain of greater than 5% and 10%, respectively. No placebo recipient had even a greater 5% improvement in FEV1.
I'm usually a skeptic, but that's extremely clinically relevant.
I can’t quite explain the lack of a significant benefit in sweat chloride -- a surrogate marker of disease that drove initial excitement about the Kalydeco-VX-809 combination in earlier studies -- but Vertex noted that trends favored drug recipients. Given the FEV1 differences observed, that's good enough for me. I am curious about the durability of the FEV1 improvements, but those questions will likely remain unanswered until Vertex completes Phase III trials, which should start later this year.