Acadia pays $100m to double up in Rett

Having the only product on the market for Rett syndrome is not enough for Acadia, it seems. The company has expanded its agreement with Neuren Pharmaceuticals over Daybue, which was approved for Rett in April, gaining ex-North America rights to the drug. It has also bought global rights to Neuren’s candidate NNZ-2591 in Rett and Fragile X syndromes, paying $100m up front across both agreements. Neuren is eligible for milestone and royalty payments for both Daybue and NNZ-2591, with the group due to receive $35m and $15m respectively on first Europe and Japan sales for Rett syndrome. There is not a great deal of active drug development for Rett, a rare neurodevelopment disorder with which around 4,500 patients are currently diagnosed in the US. Anavex has the most advanced clinical project, though a technical hit in its Avatar trial was greeted with a share price fall owing to confusion over a changed endpoint. A couple of gene therapies are in mid-stage work, and one of these could yield data shortly. NNZ-2591 itself has completed phase 2 trials in Rett, but some years ago; now that Acadia is on board its development hiatus is presumably over.

Agents in active development for Rett syndrome
Project Company Mechanism Status
ANAVEX 2-73 Anavex Life Sciences Muscarinic acetylcholine receptor regulator; sigma-1 receptor agonist Ph3 Avatar trial in adult patients posted technical hit Feb 2022, no filing yet; ph2/3 Excellence trial in paediatric patients could report H2 2023
TSHA-102 Astellas/Taysha Gene Therapies/Vyant Bio MECP2 gene therapy Ph1/2 Reveal trial in adult patients could report early 2024
NGN-401 Neurogene MECP2 gene therapy Ph1/2 trial in paediatric patients could report 2029
NNZ-2591 Acadia/Neuren Cyclo-glycyl-proline analogue Development for Rett syndrome yet to begin
Source: Evaluate Pharma &

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