Replies to post #189663 on Biotech Values

[biotech jim]

Great post, PGS, and I thank you for your blog discussions on these trials.

In Cupid-1, the joint frailty model gave statistical significance for the high dose group, but after 3 years of follow-up, mortality did not. The FDA may require additional data.

Do you think the basis for this could be endothelial expression of SERCA, or simply cardiomyocyte expression of SERCA that simply loses expression over time? I do understand at least partly the potential issues with AAV and potential carbohydrate recognition mismatch between species.

As for Cupid-1's high dose arm being a false positive, I can't make a meaningful quantitative argument. It's more a gut feeling based on the preclinical data, and the fact that I've sat through many presentations describing trials with large effects in tiny CV cohorts.

To me, the pig model data is quite telling though I have not seen a peer-reviewed presentation of the data. I agree with your thoughts here. Having said that, this SERCA isoform is one of the best ways (if not THE best) to deal with CHF if expression can be directed and exist for the long haul.

In a similar vein, do you have a view on QURE?

[mr_o]

PSG, could you post the url to your blog, and your twitter handle?

[lgonber]

Thanks for your reply. I understand your concers, and obviously is a clinical trial and until data is unblinded, we dont know what would happen. Nevertheless I am quite positive in the future outcome and my gut feeling, says they will hit primary endpoint. By the way I read you have referred to an old article to discuss about the vector, published in 2008. Bear in mind CLDN uses AAV1, which is different to AAV2, it is not the same.