Re: AGM
in response to
by
posted on
Sep 12, 2018 05:58PM
I listened to the webcast. A few more tid bits I didn't pick up on in the slide deck:
- Don expects one more DSMB report to occur before the trial is over
- The trial protocol was originally 104 weeks dosing; however, all patients interviewed want to stay on longer and therefore all patients will stay on drug until end of study. Longest patient now close to 3 years on drug.
- If they did SSRA, they wouldn't get SSRA answer until December 2018. By that point, enrollment would have been completed for ~8 months and if they had to start enrollment again, keeping in mind that patients have to be on drug for at least 6 months, this would push BETonMACE trial end to late 2019. Based on this potential delay, and the statistical penalty associated with the SSRA as well, they see no good reason to do the SSRA at this point. So I will now join the SSRA will not be done camp and this previous potential inflection point is no longer relevant. This puts TREMENDOUS emphasis on the top-line data read out inflection point.
-So w/o SSRA, they now anticipate 250 events to be reached by end of year and at this time they will stop dosing. They anticipate ~2 months to complete the adjudication process after 250 events are "unofficially" reached, during which time there may be some additional events that occur that will bring the final total closer to ~270. When adjudication is complete, we will get top-line read out. Although they could stop dosing early in a couple of months at around 230-235 events to speed up getting to 250, Don prefers to stay conservative and continue dosing until 250 events occur (~ end of year).
-They will be launching the kidney program first in China; Hepalink will be responsible for that portion of the trial; Don travels there tomorrow. They will soon be adding a 7th kidney disease program clinical adisory board member. She will be from Bejing University.
-Waiting to launch next kidney and cognition trials until BETonMACE sub-studies read out so that they can learn from this and make improvements to the design of the future trials. For the PAH trial, a group collaborating with Resverlogix is applying for funding for this trial.