"This isn't a rare disease we're talking about,..."
You're correct. It is, however, a first-time, DNA altering treatment that can't be "stopped" like traditional oral meds. Once delivered, it will continue to work its treatment for the life of the cell. We are talking about permanently changing someone's DNA, not just ingesting a pill. As such, safety is top priority, not efficacy. If Benitec were to screw up by treating someone that had the potential for an adverse reaction, Benitec, and all its shareholders would be screwed. ddRNAi is their platform, not just their "drug". All other treatments in their pipeline rest on how this trial works. If this one fails in any small area, you can kiss their whole pipeline goodbye for many years. I doubt anyone who is a shareholder wants to take that risk.
If safety, and eventually efficacy, are demonstrated, I'm certain there will be a relaxation of the inclusion/exclusion criteria. But this is the first time such a treatment has been done, and it needs to be done carefully. Share price isn't an issue to me. We're barely out of the starting gate on this therapy, and people are already whining about the SP when we haven't even gotten close to efficacy.
This is basically a start-up Biotech, with what I personally consider a kick-@$$ platform. It needs all the space necessary to watch it bloom. If we get safety and efficacy, I doubt shareholders will be disappointed. But to think the SP needs to reflect something now when we haven't even demonstrated its safety and efficacy in humans yet is a bit demanding to say the least. The market only operates so much on the "potential" of something. The real numbers come when it is demonstrated.
Shareholders need to ask themselves:
"Am I going to whine about the current SP despite the fact that we have zero therapies approved by the FDA, and not having even reached safety and efficacy on our first trial of anything, or can I wait until safety and efficacy are at least demonstrated by the end of this trial before I have a final say on this platform technology?"
Additionally, many people suffer with Hep C, and it still has not been fully eradicated yet considering Gilead's high price treatment, and the fact that it is narrow in its genotype target. Additionally, with there only being 2 sites open, and the aav8 antibodies they've encountered, its just taking a little longer. Nothing about the vector, platform, money in the bank, ....basically anything substantial has changed. Nothing. Until something significant does, patients will be dosed, and we will see this to the end... and more than likely in my opinion, for the better.
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