Replies to post #195510 on Biotech Values

[iwfal]

SRPT

But I wish they had a better steward of this drug.

Agree wholeheartedly. But 2 comments and a question:

1) in regards to data quality and valid analysis the data presented today was easily the best so far (e.g. ITT and if you are going to use historical compares you want to get as close as possible to a case matched (which still leaves huge problems) and they moved strongly in that direction). Sadly the data still doesn't look great. E.g. The most reliable statistics are loss of ambulation (for reasons similar to why OS is a more reliable endpoint in cancer) and they had 2 of 12 vs 6 of 13. Via fishers exact that is a one sided p=0.12. And that is before adjustments for post hoc decisions etc.

2) SRPT parents have some culpability in this - by believing everything CG said, no matter how outrageous, and the subsequent politics, they probably abetted the delays. (that SRPT Parent effect seems to be ameliorating, but it is a good lesson that the modern FDA might need some tweaking, but if the company is telling you that the FDA is incompetent then you, the patient, should be very wary.)

QUESTION:

On twitter you made reference to the Western Blot still being fairly incompetent. Could you provide more detail on that?

[masterlongevity]

agree $srpt has totally messed up development. I think FDA will approve based on pressure, but its still not clear from the actual data if the drug works. Disclosure- I own $srpt shares based on the former