Neuren Media and Analyst Coverage, page-1677

Here is data from the study on clinical success rates that the Livewire authors cite -



They cite the overall P3 success rate of 59% but fail to mention the more relevant breakdown by therapeutic area which is 51.1% for CNS drugs. Although CNS is a bit of a catch all that includes neurodegenerative diseases (which have a notoriously high failure rate), it is generally accepted that neuro drugs have a below average success rate so the authors are justified using what they admit is a conservative figure of 50%.

Having said that, 2591 has some features which suggest it has an above average chance of success, although most conservative analysts won't include these. In particular:

1. 2591 has a similar mechanism of action (increasing availability of IGF-1) to trofinetide, which is approved for a related indication (Retts).
2. 2591 has encouraging data from 3 separate phase 2 trials in different indications, increasing confidence.

Basically, it is standard practice for analysts to use the industry averages. They note they are being conservative and it would be difficult to pin a number on the 2 points above in any case.