Neuren Media and Analyst Coverage, page-490

When Sarepta’s Exondys 51 was launched in 2016 and market penetration and sales for the first full year were being estimated, a Leerink analyst expressed scepticism of other analysts’ modelling. Market consensus was modelling $215m sales in FY2017, which would have represented 19% market penetration.

The Leerink analyst argued that only a very small handful of rare disease drugs had ever breached the 20% penetration mark in their first full year of launch. He was tipping FY 2017 sales of $101m. Ultimately, the Leerink analyst fell well short in his first year sales estimate (it was actually $154m) but was right about Exondys 51 not reaching the lofty height of 19% market penetration in the first FY. Nevertheless, Exondys 51 was considered by the industry to be a highly successful orphan drug launch.

Which brings me to Daybue and analyst predictions.

In 2019, I highlighted an analyst’s predictions for trofinetide in Rett syndrome. Following an estimated December 2022 approval, the analyst was predicting just $57m for the second full year of sales  - that is, in 2024. “Peanuts”, according to the analyst.

Acadia’s only other asset is the insulin-like growth factor 1 regulator trofinetide, which the sellside sees making just $57m in 2024 in the neurological condition Rett syndrome – peanuts compared with a forecast total of $1.7bn for Nuplazid.

I’m not sure what analysts were predicting shortly after approval for Daybue’s first full year on market. Probably nothing as high as the 19% market penetration estimated for Exondys 51.

Well we’re not at the end of 2023 yet, yet alone 2024. The Evaluate analyst’s 2019 estimate of $57m for the second full year of sales has been blown out of the water by the ~$67m sales already reported in just the first full quarter. And 19% market penetration of US-registered Rett patients has already been achieved by the end of the first full quarter of sales.

I suspect that most analysts will have been caught off-guard by the success to date of Daybue’s launch. Who knows, some may even start recommending NEU to clients as their amazing new "undiscovered" stock tip!