Anavex Life Sciences Corp (AVXL)

[frrol]

The company is not giving up on Rett. It is a difficult indication to credibly show efficacy, although Acadia did so with Trofinetide. Anavex is gonna do some post-hoc analysis of the Excellence data, and no doubt get Avatar OLE analyzed (like they did for the U.S. trial last June). But approval without a larger and successful phase 3 is a long shot. Regulators will balk at the lack of stat-significance (admittedly 'close' for RSBQ, but apparently not close for CGI and ADAMS), which Acadia was able to show. And insurers would be even less convinced, especially at orphan drug pricing.
If the company can show stat-sig reduction in seizure frequency (an element of the CGI Scale), that would be quite impressive. Seizures are distinctive and episodic, and alleviating them is huge for QOL. But they did not do so, though there is little doubt they looked for that as a glimpse of hope for the TLR. They only mentioned it in the anecdotal "RWE". Unfortunately that's not clinically credible. Seizures are distinctive, reasonably loggable, and fairly immune to patient placebo effect. A credible reduction would be in the data.
Maybe in the coming weeks the company will provide a call with Q&A on the Rett program.