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IF we're successful with stage 3, what are the steps/actions that we need to undertake following that?

In the case of Acadia, if the Phase 3 trial of trofinetide in Rett syndrome is successful, Acadia will need to put together a New Drug Application (NDA) for US marketing approval. This step is likely to take a number of months. Prior to submitting the NDA, a review meeting has to be held with the FDA. Acadia would then need to file the NDA, along with a request for Priority Review and a Rare Paediatric Disease Priority Review Voucher. There is a 60 day wait before the FDA communicates whether it will accept the filed NDA and whether it will grant priority review. If priority review is granted, the NDA review process is shortened to 6 months. If the FDA’s decision is positive for trofinetide in Rett syndrome and marketing approval is received, it is then Acadia’s responsibility to organise manufacture, distribution and marketing in North America. Acadia is also responsible for post-market monitoring, which requires periodic safety updates to be submitted to the FDA.

In the case of Neuren, if the Phase 3 trial is successful, Neuren will need to negotiate a deal/s with interested parties in licensing trofinetide outside of North America, using the Phase 3 trial data. With the costly pivotal trial successfully completed and all data fully accessible, one would expect that Neuren would be positioned to negotiate an upfront/s well in excess of the UA$13.6 m paid by Acadia in 2018.

In addition, if the Phase 3 trial is successful and the FDA grants marketing approval, Neuren will also need to bank the development milestones that will be payable by Acadia. There is likely one payable on either completion or success of the trial and another on FDA approval and together, these payments are likely to exceed A$50 m.