When looking at the Feb 12-13 presentation, two new pieces of information were made available. The first was the baseline characteristics of the the first 2000 or so BETonMACE enrolled patients. The second was the projected MACE rate of 8.0 pr. 100 patient years. The projected MACE rate was unchanged from previous guidance.

As we wait for the sample size reestimation analysis - due by mid 2018, according to guidance from RVX - I have made some calculations on what will happen if the actual event rate turns out to be 8.0 pr. 100 patient years and the background (i.e placebo) event rate is 9.5 pr. 100 patient years. The 9.5 pr. 100 patient years is taken from the 9.5% calculation on slide 16 (still talking about the Feb 12-13 presentation).

I used a chisquare-test, as the log-rank test that is normal for a case like this requires more information that I have available, and using this type of statistical test, I got a p-value of 0.007 and a relative risk reduction of 31.6%. This is highly statistically significant, but not high enough to warrant stopping the trial early for efficacy - such a threshold is typically p<0.001. However, doing the same calculation as above, I found that p<0.001 was achieved if the placebo MACE rate was 9.9% (i.e. 9.9 pr 100 patient years). The 9.9% target is really close to the event rate that RVX calculated for the placebo group, and given the fact that the HDL in the BETonMACE population is as low as 33 mg/dL (median), the initial estimate of 9.5% for the placebo group may be an underestimation.

Thus, while you should always do you own due diligence and not rely on any of my analyses, the possibility of the trial being stopped early for efficacy is definitely a possibility that should not be overlooked.

BR

B.