As I’ve said before, I think Neuren will be acquired by Acadia. I think that’s likely to happen before any development of NNZ-2591 in Rett. I’m in agreement with turbo that NNZ-2591 is likely to be used to evergreen trofinetide in Rett.
More broadly, I think Baker Brothers and Acadia are not planning to limit themselves to building a Rett franchise as Steve Davis has talked about, but rather a rare neurodevelopmental disease franchise that “owns” a number of diseases. They will license and acquire in order to get there.
Two examples of rare disease franchises are Vertex and Alexion.
Vertex (VRTX) is focused on rare diseases and has multiple assets in each indication. The lead indication is cystic fibrosis. Vertex now has eight different assets for cystic fibrosis, with four already approved. Viewed across a 15 year horizon, VRTX has grown from a market cap of US$3 bn to a current market cap of US$92 bn.
Alexion (ALXN) also had a market cap of US$3 bn in 2008. It was acquired by Astra Zeneca in 2021 for US$39 bn. Alexion’s particular focus is complement inhibition, which it applies to a broad range of immune-mediated rare diseases.
Perhaps not surprisingly, Baker Brothers was the largest single shareholder in Alexion at the time of acquisition and Felix Baker was a member of the Alexion Board. Baker had been a Board member since rare disease company Syngeva (in which Baker Brothers was again the largest shareholder) was acquired by Alexion for US$8.4 bn in 2015, netting Baker Brothers a US$1.4 bn profit. At the time, Syngeva’s lead product was still to be approved.
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