of seeing those results, Vertex expects to pull the blinds off a couple of small studies that could be equally important for the company’s long-term hepatitis C franchise. Those results will look at the safety and antiviral activity of a couple of nucleotide polymerase inhibitors that Vertex licensed last June from Alios Biopharma. These are the key compounds to watch. Vertex licensed them just a few months before the bidding war started over rival drugs in the same class from Pharmasset and Inhibitex. (Leiden notes that the Alios drugs were “a very smart use of Vertex resources” because the company only paid $60 million upfront, plus $1.5 billion in future milestones, for the right to those compounds, before the Pharmasset/Inhibitex frenzy started.)
Vertex has said little publicly about the Alios compounds, but Leiden said during an interview earlier this month at the JP Morgan Healthcare Conference that he’s confident they will stack up well. Vertex has long has its eye on putting together a portfolio of antiviral combo meds, and settled on the Alios compounds after a careful review of all the nucleotide polymerase inhibitors in development. Vertex knew the Alios drugs were potent, that Alios had unquestioned intellectual property ownership of them, and the drugs could easily be combined with telaprevir and other kinds of hepatitis C medicines in the works, Leiden says.
“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).”
One analyst told me on background yesterday that analysts are curious to see what the Alios compounds can do, but nobody on the Street knows how to value them because Vertex hasn’t published any scientific papers on them yet.
Without getting into too many specifics, Leiden says the Alios drugs can kill many of the different genetic mutant forms of the hepatitis C virus, which can emerge when the virus is pressured by a new therapy. “We know they are potent, and they are pan-genotypic. Each could be anchor in all-oral regimen,” Leiden says. Vertex also could combine one of them with telaprevir, or its non-nucleotide inhibitor, VX-222. “We believe our pipeline could allow us to sustain our leadership for many years to come.”
Whatever Vertex learns from its trials this year, it has got to be hoping for black-or-white results. There won’t be much time to ruminate over the data, or to fuss over how to design the next clinical trials, as it can hear the stampede of competitive footsteps. “Our goal is to go to pivotal studies as quickly as possible,” Leiden says.
