Vertex outlines its survival plan for next-gen hep C combo

At the beginning of 2011, Vertex ($VRTX) was basking in the limelight of blockbuster success. Its hepatitis C drug was headed for approval, offering a game-changing approach to the disease, and was the clear favorite in a brewing market showdown with Merck's ($MRK) rival therapy. Vertex went on to win that battle but lost the war for the affection of the industry analysts, who were seduced by the interferon-free promises of the relatively puny Pharmasset ($VRUS), Inhibitex ($INHX) and others. The big M&A deals that followed with Bristol-Myers Squibb ($BMY) and Gilead ($GILD) shunted Vertex off to the sidelines.

But as Vertex CEO Jeff Leiden makes clear in his interview with Xconomy, the upstart biotech has a few promising experimental treatments up its sleeve. Vertex is making the case that it is fully prepared to compete in the new race to develop cocktail therapies that will offer a "cure" without all the ugly side effects linked to interferon. But it doesn't have much time, as analysts are zeroing out telaprevir's revenue as new drugs muscle into the market.

Game plan #1: Grab solid proof of concept data on a combo of telaprevir, VX-222 and ribavirin in the first quarter and then hustle into a pivotal study straightaway. "If we can do that, it will be a very exciting result. If you take that regimen into pivotal trials, now we're talking about 2014 to finish those trials," Leiden tells Luke Timmerman.

Game plan #2: The jewels in the pipeline were licensed from Alios just before the bidding war broke out for the hepatitis C drug developers. And an initial trial judging both the safety as well as early efficacy of these early-stage compounds will be crucial to regaining some of the lost hype around the company's prospects.

"I really like the way we've positioned ourselves because we have the component parts," Leiden says. "We have the best protease inhibitor, we have a non-nuc polymerase inhibitor (VX-222), and two different nucleotide polymerase inhibitors (ALS-2200 and ALS-2158)."

But with potential competitors further ahead in the clinic, Vertex's R&D team will have to play its best game. One slip in this race could give competitors a big lead that Vertex may not recover from easily. Significantly, though, Vertex also has a promising new drug for cystic fibrosis in the pipeline which is an odds-on favorite for near-term approval.

- here's the story from Xconomy