Many are getting really excited about this statement from the news release concerning the DSMB: "The DSMB reviewed available study data and noted that no safety or efficacy concerns were identified." This is of course good news from a safety standpoint, but not too surprising. The Phase 2 ASSURE and SUSTAIN trials treated patients for ~6 months. The first patients to start dosing in BETonMACE in November 2015 have now been treated for 9 months. But the trial is still actively enrolling and is still a ways off from being fully enrolled. Most patients in BETonMACE to date are likely treated less than 9 months.....probably an average of under 6 months. So from a DSMB standpoint, we don't really have too much new information yet from BETonMACE in terms of long-term safety since not too many patients have likely dosed beyond 6 months. The new information though is that in the diabetic, low-HDL, recent ACS (within 90 days) event population of BETonMACE, the drug seems safe. Patients in ASSURE and SUSTAIN were not required to have diabetes or have a recent ACS event, and only a small number of patients in ASSURE/SUSTAIN were diabetic, low-HDL, recent ACS.

Now as for the efficacy portion of the statement, I think many are reading way too much into this. There are not enough patients enrolled yet and dosing has not gone on long enough for a large enough number of events to have occured at this point to permit too much of a conclusion, IMO. Yes, the post-hoc analysis of MACE in the 6-month long ASSURE/SUSTAIN showed the impressive MACE relative risk reduction, but this was 5-point MACE. Recall, very few of these events were of the more strict 3-point MACE variety. Most likely, the DSMB is just making sure that RVX-208/apabetalone is not increasing the event rate compared to the placebo group. Based upon the anticipated 3-point MACE event rate in the patient population, and the anticipated rate of enrollment, and the projection of ~30% RRR in the RVX-208 group, it was estimated that the 125 event point would be reached mid-2017. If we call that June 2017, then we are still 9-10 months away from the projection of hitting 125 events.

So good news from a safety standpoint, but I wouldn't get too excited from an efficacy standpoint at this point in time.

BearDownAZ