Replies to post #184227 on Biotech Values

[iwfal]

RNA

GSK published details as PDF files on their website many downloaded, they were examined and discussed in details on Twitter over and over at the time, RNA presented in conferences over and over in the past year.

You'll be 'happy' to know I refound the poster I was referencing that has much more detail on significant adverse events. And it just re-emphasizes that RNA and GSK have not been exactly clear about their significant adverse events.

Of all the patients who reported AEs, only seven (4%) had to withdraw or permanently discontinue the study drug: five (3%) had thrombocytopenia, one (<1%) each had asthenia and proteinurea.

One subject had elevated alanine aminotransferase 43 days after the first dose of drisapersen but continued treatment with drisapersen in the continuous dosing arm.

While it isn't clear exactly what population base and time span was used for this, the most common numerical base used in the safety data is 186. But it is completely unclear what the time base is since there aren't any times that have anywhere near 186 treated patients. Also note that they report 6 percent with thrombocytopenia despite reporting no thrombocytopenia at the end of the phase 3. Finally note that they report that the thrombo (presumably the severe thrombo) resolved in all cases after discontinuation.

All told I think I was and am pretty safe in saying that the safety data, as available to the public, is still fairly unclear. But certainly greater than the ERT data in Fabry that I referenced earlier and it is likely to be a topic in the AdCom. But we'll probably have to wait for the AdCom data to understand it. E.g. It is possible that all these significant AE occurred only in the extension and thus caused the quotes from GSK I referenced earlier, but none occurred during the ph3 itself. Seems improbable, but possible. But then that raises the question of whether more time on drug leads to ever more significant AE.

BTW - an interesting point is that I suspect the extension study efficacy data for the ph 3 is showing stat sig at 96 weeks using the methodology that Sarepta likes to tout. (They don't give stats, and well they don't since the methodology is suspect, but still interesting).