Do you know how ODAC/FDA would look at this trial if, say, leiomyosarcoma and a couple of the other sub-types showed very positive efficacy, yet, many of the other sub-types showed none whatsoever? Can/would the FDA decline the entire trial, or can the FDA approve the trial for those indications only, or could they approve the whole trial based on a handful of subsets when, even if the majority of subtypes showed no efficacy?
It's a very heterogeneous disease and I think the fact that it is, "washed out" (in the median PFS numbers) a lot of the big responders and their subtypes due to the non-responders. It's going to be really interesting to see the breakdown of the population of this trial.
I would be really surprised if Merck didn't file an NDA. What do they really have to lose at this point? If one is to look at the data of old trials, new trials (the Japanese Study which was reported at ASCO), there is a clearly a big trend in favor of some of the STS subsets versus osteo. At the very least, I expect at least 2-3 of the most prevalent STS subtypes (as you have noted) to show very positive PFS which is almost more than likely to translate into positive OS. My biggest question is how ODAC and the FDA are going to "break out" this data considering the heterogeneous population of this trial.
NOTE: I'm sure someone is going to come on here screaming that I am flip-flopping etc., and I "bashed" Ridaforolimus for a year, etc., and now I like the drug. My whole problem with the trial was the fact that I felt there were so many different subtypes that would "wash out" the best responders which would make the trial fail. Obviously, the trial met its endpoints and I am very happy about that - the fact that IT DID meet its endpoints and there are trends toward favorable OS and PFS means that THOSE SUBTYPES that DO RESPOND responded SO OVERWHELMINGLY that they made the trial "succeed" which says a lot about the subtypes that Ridaforolimus works in. And, that's a good thing....
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