benitec delivery, page-55

The science behind BLT baffles me completely but the trial development process here is not rocket science. And I suspect that post isn’t from Dr Kss because it displays a lack of understanding of what is going on here.

If you go back to the Recombinant DNA Advisory Committee where the protocol for this study was reviewed it is pretty clear that safety, ethics and science were the drivers of the key decisions. And the practicalities around recruiting 14 patients took a back seat.

A key issue was the possibility that sub-therapeutic doses would be used. This meant that there was a trade off and balance needed between establishing safety and the potential for achieving meaningful biologic activity against HCV.

It is known the response rate to interferon-based anti-viral therapy for chronic hepatitis C is lower in patients who are obese. Either due to sub-optimal dosing or an altered response in obese people. Thats why many HCV trials stratify or exclude obese patients.

So in this context it makes perfect sense that in a study designed to establish dosing regimes you would exclude a group of patients that could very well confound the results of exactly what you are seeking to establish in the first place.

Its a bit silly to say this can be fixed at the stroke of pen. First you probably don’t actually want to fix anything. But second I doubt that it is a simple as writing two lines to the FDA requesting a change. You would think it would have to go back to the RAC. And there you might expect that slowness in recruiting would take second place to the other issues involved here. The ethical risks taken here can only be justified in good science is going to result.

The idea that a company spends years and millions of dollars to start a trial that it really doesn’t want to do as some sort of ruse to investors is pretty implausible. If it were true .. well a company would never the run risk of letting things go so far as they have – to actually screening patients for eligibility and relying on luck of the draw.

One might also wonder whether the Duke researchers are silly enough to start releasing sensitive information about recruitment, This is pretty problematic at many levels, no doubt breaching their contract with BLT but exposing them to potential problems with their IRB.

But having said all this after the QRX failure the trust has gone out of things. Dr Kss got QRX completely wrong like everyone else (including me). Everyone is rattled and the assumption now is that if there is a sniff of something not at all right with anything .. shoot first and ask questions later.

In this respect the bar is lifted on the onus on companies to provide much better levels of transparency and much more detailed information about trial rationales, processes and progress. Failure to do this in the context of investors in the US able to take short positions (which BLT seems to attract its fair share of) means that companies share prices are even more able to be easily manipulated through the vehicle of social media. Its not really a practical solution to tell people not to read blogs (in the 21st century).

All imo, GLTH, Southoz

http://osp.od.nih.gov/sites/default/files/RAC_Minutes_06_13_0.pdf