A De-risked bio-tech with 4X upside - Karst Peak Capital, page-65

Hottod thanks for your excellent contributions both here and on the FX thread - much appreciated.

Re: the competitive landscape, I’ve been monitoring Anavex since their recent contentious P3 results with adult Retts patients. Despite controversy around the changed end points and reclassification from P2 to P3, their CEO claims it’s all legit and they're preparing an NDA for the FDA and other global agencies including our own TGA. They’re running a larger P3 trial with paediatric Retts sufferers with results supposedly due end of 2022. Their CEO has cited trofinetide against their drug blarcamesine's tolerability with minimal diarrhea and vomiting. They’re also into trials in Alzheimer’s Disease and have stated an agenda including Fragile X.

When I raised Anavex’s impact on trofinetide with Jon P in the recent Q&A, he took it in his stride and responded re: the question marks over the contentious issues above. Last week’s FDA sanctioned Patient Focused meeting included positive references for trofinetide but no mention of Anavex’s blarcamesine and given recent commentary from the Acadia team, first to market advantage seems to be clearly with trofinetide.

Hashan in his recent article also states, “we believe Anavex’s drug for Rett Syndrome is unlikely to receive FDA approval and thus not competitive given the controversy surrounding its ‘Phase 3’ trial.” He also notes Anavex’s forecast of a total addressable Retts market of US$2-5b, based on 10,000 patients with pricing at US$200,000-500,000 in the US alone. Mel from Trail to Texas, stated recently that she was going to comment on Anavex’s P3 “success”.

Hottod/others: do you have any insights/opinion into Anavex’s pathway and impact on trofinetide’s market (particularly after possible success with their paediatric trial) and the benefits for Acadia/Neuren in being 1^st to market?

OralAnavex 2-73 Safely Eases Rett Symptoms, Severity in Phase 3 Trial – RettSyndrome News