Acadia Pharmaceuticals: I Remain Cautiously Bullish After Q3...

Acadia Pharmaceuticals: I Remain Cautiously Bullish After Q3 Updates

https://seekingalpha.com/article/4553380-acadia-pharmaceuticals-i-remain-cautiously-bullish-after-q3-updates

Excerpt below from the above Seeking Alpha article -

The Trofinetide Rett Syndrome Opportunity

^{As mentioned, Acadia's NDA for Trofinetide has been accepted by the FDA and the PDUFA date arrives next March.}

^{Results from Acadia's 187-patient LAVENDER study of the drug in girls and women between the ages of 5-20 - released in December last year - were positive, with statistically significant improvements observed vs. placebo in both endpoints of Rett Syndrome Behavior Questionnaire ("RSBQ") and Clinical Global Impression–Improvement ("CGI-I").}

^{It was an impressive readout given that there are no approved therapies for Rett Syndrome and that several biotechs had tried and failed to develop drugs in this indication before Acadia. The market opportunity is apparently rated as a potentially blockbuster (>$1bn sales per annum), although $500m is the figure suggested by Acadia management.}

^{The FDA has (so far) not requested that an advisory committee be convened to rule on whether to approve Tofinetide, which is potentially a good sign, and should the drug be approved, Acadia is in line to receive a rare pediatric disease review voucher, which it could use itself, or trade to another company, potentially for a triple-digit million sum.}