Replies to post #187478 on Biotech Values

[iwfal]

2. I also believe the efficacy of both driseapersen and eteplirsen is small

Agreed. But with the further caveat that my belief in that efficacy is almost entirely based upon the Dris data. The Etep data is essentially useless (as you'd expect from a weakly efficacious drug in a historical compare). In as close as we can get to case matched comparison against a post hoc mITT they get 14m to 21m difference and that difference isn't stat sig even in much larger RCTs. (RCTs are wildly more perceptive than historical compares as everyone on this board undoubtedly knows - think of all those wonderful single arm ph2s that were a complete bust in ph3)

As jq and BMRN have pointed out there are cases where a drug has failed Phase 3 (granted the data was more robust) been approved anyway and today its fairly obvious of the benefit especially when given earlier/over a much longer period then the trial.

First I'll point out your caveat ("granted the data was more robust") is more than a little important. Second, my point about the risk of approving a drug with a failed ph 3 even after some reasonably successful small ph 2s is that the ph 3, as the confirmatory trial with a larger group of treatment centers, provides a much better indication of how it works in the real world. Not having that does cause risk for patients. Third, the sine qua non for science is, in fact, generate hypothesis, then do confirmation. Separate activities. Dris didn't do the second step and that is inherently not good science by somewhat pedantic definition. And thus risky and poor precedent.

All that said, I was uncomfortable being quite as black and white in the quote you excerpted.

I actually think the street would get scared by a PTC failure as it would cast broad doubts on the ability to show a treatment benefit in a more robust DMD study and likely further question the understanding of the disease.

Disagree. As best I can tell PTCT, for a variety of reasons, gets very little respect. Much less than SRPT. This is just plain odd in my view, but ... . It makes for an interesting drama.