Re: My Thoughts On Webcast
in response to
by
posted on
Mar 04, 2016 02:12PM
San Fran,
I too was left wanting more.
We already knew that they were working towards the PNH and Kidney trial(s), so we didn't learn anything new other than both trials (PNH, Kidney Dialysis) were on the 2016 launch milestone slide, will have ~20 patients (kidney dialysis) and 30-40 patients (PNH) and cost 1-2 MM (kidney dialysis) and 2-3 MM (PNH). They also mentioned that the only Alzheimer's related trial is the one embedded into BETonMACE that will have 800 of the 2400 patients being 70 or older and undergo cognitive testing with the hope that it gives future data to support a follow up trial.
DM did mentioned that they are completing some FDA required animal work with RVX-208 that is required in the process of FDA approving US sites in BETonMACE. However, even with these animals studies, DM said that FDA inclusion is no guarantee.
Regarding BETonMACE study sites, sounds like Taiwan will be included. No mention of other China sites. Austrailia was added as a back up in case they need to add more patients later in the trial. Recruiting is still early in the process. Only 60 out of 177 study sites activated, but Sweeney commented that half of the total should be active by end of March 2016. So recruiting is obviously slow now but will pick up "exponentially" as we get closer to activation of most study sites.
In BETonMACE, the futility analysis will occur at 125 events (Sweeney estimates this will be around mid 2017) and then a sample size estimate analysis will occur after 175 events. At this stage, there will be an option to expand to 375 events. The original 250 events are still estimated to be reached mid 2018.
Very nice presentation by Mike Sweeney. Very empty presentation by DM, with the exception of the 2nd licensing deal comment and the 3 new analyst comment.
BearDownAZ