SanFrancisco99 wrote: "Enrollment seems ahead of what I expected.   That might explain next point: Regarding Timing, Don basically said yesterday top line results q4 of 2018.    That was huge info -ahead of what I expected.   I was very happy about that."

There were ~2000 patients enrolled as of early November 2017, so why are you surprised by the current enrollment of ~2100? Am I missing something? It doesn't appear that anything in the BETonMACE trial design slide changed, so as of right now there appears to be room for 300-400 US patients at most to reach the original enrollment of 2400-2500 patients. I was somewhat surprised by the top-line estimate of Q4 2018 since just a month ago on slide 18 of the BIO Europe presentation they had BETonMACE timeline extending to mid-2019. Perhaps, as Tada has previously suggested, that top-line data Q4 2018 and full trial report mid-2019 is how to best interpret this. Of course, all of this is subject to change pending the results of the pending futility/sample size estimate analysis.

My notes from the AGM webcast (I wasn't there):

New molecules and indications: I was a little disappointed in the lack of information shared about the new molecules yesterday. Anyone hear anymore from the Q&A about additional molecules beyond apabetalone? Lots of exciting stuff going on with the additional indications (pre-clinical stuff). I'm excited to hear more about the Pulmonary Arterial Hypertension program. DM mentioned that 3 universities are collaborating on this PAH project right now and working on a paper together. Also I look forward to hearing more about their efforts to develop an alternative delivery method (topical) for potential treatment of Muscular Dystrophy/Facio Scapulo Humeral Dystrophy.

Pending trials: We learned that the first patient will be randomized in the Phase 2a Dialysis study in Q2 2018, with a primary endpoint of Alkaline Phosphatase. It is frustrating that this dialysis trial has been delayed from starting for so long now. It will sure be nice when it finally starts. The first patient will be enrolled in Phase 2 Fabry trial in Q2 2018. The launch of the complement/PNH trial is being delayed. DM mentioned there is much competition in getting a principal investigator for this, so hopefully they can get this worked out. 

BETonMACE: Glad to hear that first US patients will be randomized in Phase 3 BETonMACE in Q1 2018 and BETonMACE full enrollment Q2 2018. But if top-line data is expected Q4 2018 then those last patients to be enrolled won't have been in the trial very long. DM also mentioned that the rosuvastatin/atorvastatin split is about 50/50 right now in BETonMACE. Good to know.

BearDownAZ