Replies to post #27869 on 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.

[mskatiescarletohara]

PGS

++++Is it the FDA acting as the bottleneck, or are companies skimping on the patient numbers that they bring into these trials?

I think both apply and it depends on the pre-clinical/Phase I data package presented. The viral market is so tremendous, it's quite easy to get a viral protocol approved. Prove safety dosing in a small patient pop and move on to combo studies provided you have animal models to support MOA. Some company's can move right into repeat dosing after initial data, and some cannot because flags are raised about PK/PC values. VRTX has a good data package on healthy volunteers, and they moved directly to repeat dosing, yielded impressive log reduction, took advantage of it, tested a few combo variations to set up for Phase II and VOILA...here they are moving aggressively into a large Phase II protocol. This same type of clinical development can be noted with other various viral compounds on the market today, especially in companies focused on HIV...GILD and TRMS.

katie....