Do I only care about share price? We’re investors, so of course we care about whether or not the company we have invested in can develop drugs which go to market that are monetarily successful. That includes you. However we are also human. Meaning, most of us have sympathy for others, especially children, who suffer from devastating and life impairing diseases. Most, if not all of us know someone suffering from a horrific disease we wouldn’t wish on anyone. The result of having sympathy, is we hope and pray that drugs are developed as efficiently and quickly as possible. In this case, an investor here can both care about a return on a risky investment, AND, that suffering patients can as quickly as possible get some real relief from our drugs. I don’t think that is a strange or extreme concept. In fact, and you can call me crazy, but if Avatar and US show good dose response curve, I think the fda should approve immediately. That isn’t the investor in me talking, that’s the sympathetic nature in me.
Interim peeks happen all the time. Let’s recognize also that an interim peek in the AD ph2/3 is a whole different ball game then an interim peek for Rett Syndrome. Not all interim peeks are created equal. The reason being, and assuming Avatar data is good, we will have very positive stat sig results from 2 blinded placebo controlled Rett Syndrome trials, something we don’t have with AD. I will admit you are smarter then myself, however, knowing the results from 2 successful trials certainly lessons the negative impact of taking an interim look at Excellence, as we already know how terrifically the drug has previously performed in Rett. In AD, we are relying on a tiny open label trial, only. Furthermore, we all know the difficulty in enrolling trials for rare diseases. There are less patients and many trials of rare diseases never fully enroll. It simply isn’t possible; there aren’t enough patients. Taking an interim peek at a rare disease trial which technically may never reach full enrollment, is a reasonable action. And let’s not forget our trial n was expanded to account for properly powering the various age ranges in the study. As so, if the entire patient population shows statistical significance, the age cohort analysis won’t matter, and the expanded n won’t necessarily be beneficial, not necessary. Lastly, it’s extremely important to appreciate the simple fact that any interim peek won’t be independent of the fda. It will be agreed upon with the fda, and if the US low dose trial, and the Avatar high dose trial (assuming positive) are any indication, then it’s a safe bet that Excellence will be equally as good if not better. Even now on an interim basis. Great Avatar data should provide Missling the confidence to peek at this data. The same efficacy knowledge and confidence does not exist for AD. 2 different ballgames.
If Avatar data is great, it’s my opinion an interim peek at Excellence is warranted. Ethically and statistically (based on 2 other rett trials) seems like an appropriate gamble in order to get 2-73 more quickly accessible to all Rett patients suffering around the world. While I am invested here, my opinion is based on sympathy for suffering patients. Not share price or my own bank account. After all, I will hold whether or not there is an interim peek or not.
