Replies to post #331433 on Anavex Life Sciences Corp (AVXL)

[tredenwater2]

From an investors standpoint an ultra rare disease limits the number of candidates

Perhaps with our safety proven this allows them to use historical controls for placebo and with a limited number of patients available, perhaps his thought process is that the trial process be accelerated thus if efficacy is shown early it may allow us get to market faster…maybe even simeoultaneously with pediatric Rett Syndrome.

All patients, rare or ultra rare are important in the eyes of God. Kudos to Dr Missling and his team for his ability to humble himself and work with each of these Rare disease groups designing the trials around meeting their needs. Im not sure how big pharma has approached this in the past but it is in the best interest of all to do so.

[Steady_T]

I can think of several reasons for choosing an ultra rare disease.

No SOC to compete with.

Lots of pressure from advocacy groups to get it approved if it works.

Good will in general for Anavex.

Small trial numbers so it should be a cheap trial to run.

If Rett trial is as good as we think, could be a one trial to an NDA.

Broadens the MOA to a general CNS drug by showing the disparate indications that it has efficacy upon.

Might obtain a high price for the drug to offset the low numbers of people being treated.