Biotech Values

[DewDiligence]

>One question about companies in this space: why are the majority of these trials in such few patient numbers?<

Many of the trials reporting results at EASL this weekend are phase-1 and phase-1b studies. IDIX’s NM283 trial in treatment-naïve patients, which is a phase-2b, has approximately 150 patients in five arms, so it is not exactly tiny.

You are correct, however, that early-stage HCV trials tend to be smaller than trials in other medical indications where there is a large patient pool to draw on. Part of the reason for this is that the animal models for HCV are poor and few. Chimpanzees are probably the best animal model for HCV, but they are expensive to work with and in short supply compared to rodents.

Thus, a phase-1 trial in HCV typically begins with less confidence about dosing and PK/PD than in your typical phase-1 drug trial. It behooves the trial designers to aim for relatively small numbers of patients until they get a better handle on things.