Replies to post #126430 on Biotech Values

[DewDiligence]

ARIA—the median PFS improvement clearly understates the actual benefit given the median happened to be on a scallop on the K-M curves where the two curves were relatively close - the improvement is quite a bit better at say both 45% and 55%.

I’ve yet to see an NDA/BLA review where the FDA cares one iota about the median value of a probability distribution.

[JJM760]

Maven,

What the hell ever happened with the endometrial trial??

They announced positive results, did the financing THAT NIGHT, and that was the last we have heard of it.

[NP1986]

Further there is no quality of life data (for reasons I don't understand).

From what I recall, many oncology trials which try to measure quality of life end up with incomplete data and thus the investigators do not report it.

I also believe that many investigators will use incomplete data as an excuse not to report the results if there is a hint of a detrimental effect with the investigational treatment. It may be true that the results are not easily interpretable, but I think they should be reported regardless.

On it's own, I think PFS does not have much value. If it is accompanied by improved quality of life or a trend towards improve OS, then it is significant. As Dew says, if the HR for OS improves with more events, then it makes the case for ridaforolimus stronger.

In terms of commercial success, I really think this partly depends on whether they can find some way to break the AKT/mTOR inhibition feedback loop. If so, this could still be a big drug.

I agree that ridaforolimus could be a big drug if it can be successfully combined with other targeted agents, but it is still in mid-stage or early-stage development in other indications. On the other hand, Afinitor is way ahead in terms of clinical development and companies other than Novartis are studying their own compounds in combination with Afinitor. Based on just an indication for maintenance therapy for sarcomas, the commercial value is limited. But approval would make the drug more visible, and I'm hoping that as a result we will see more investigator-initiated trials with ridaforolimus.