Shareholding Meeting - Part 1
Overall great meeting. Missling graciously answered any and all questions that I had. And I had many. There were about 15 people there in total. I met Missling, Stephan Toutain, and Daniel Klamer. They were all very nice and seemed excited about the company and where it is heading. There were several other Anavex related people there including Clint from IR and a lawyer from K & L among others.
I arrived early and was able to get into a 20 minute discussion with Missling and 2 other investors from the Facebook group. Missling did a presentation with the usual slide deck and included a few new ones. After the presentation we were able to ask questions. I asked every question I could think of and they were all answered the best he could without giving away material information. After the meeting, I was able to meet with Christopher and a few other investors. We spoke about a variety of topics for about 20 minutes.
I’m not a science guy, so I will refer to others to clarify science related topics.
Rett
-Safety not an issue. In order to run the trial with these children, they had to do a Juvenile Toxicity Study required by the FDA. So NO issues there.
-Toutain said the trial would consist of 75 patients. During Missling’s presentation, he said it would be less than 100. So it sounds right.
-The specific patients in the trial are not selected yet. However, they have identified the patient pool from which the participants would be selected. Once the FDA gives the go ahead, screening will be done using the data collected.
-Delay is due to back and forth negotiations with the FDA. The 30 day clock seems more like a rolling clock, not set in stone. Because this is a new drug, using a unique trial design (especially for this indication), they want to do the most to get it done right. And also to get it done so it can be approved out of phase 2.
**All endpoints for all trials will be endpoints that were used in prior trials**. Meaning they are not creating new and improved endpoints.
-RWE will be used to determine effectiveness but will not be a primary endpoint. More likely a secondary peripheral measure and evidence of efficacy.
-There is a very low bar for Rett approval. Minor improvements will result in approval.
-Age range of children not determined yet.
Part 2 and 3 coming
