Neuren Pharmaceuticals (NEU) Investor update summary

Share buyback program and financial position

• Approved a new on-market buyback of up to 5% of shares, reflecting confidence in future prospects and strong cash position supported by DAYBUE franchise cash flows.

• Buyback will be conducted at management's discretion over up to 12 months, with flexibility to vary, suspend, or terminate based on market and operational factors.

• Shares bought back will be cancelled, reducing total shares on issue; daily notifications to ASX will be provided.

• Board views current share price as materially undervaluing assets, with analyst consensus at $24.40 per share and all analysts rating as buy.

• Buyback will not impact funding or progress of key development programs, and is seen as the right move given recent share price volatility.

Regulatory and clinical program updates

• Trofinetide received a negative trend vote from the CHMP in Europe; Acadia will request reexamination, with a new review team to be appointed.

• U.S. and international named patient programs remain the main revenue drivers, with 2023 royalty guidance of AUD 63–66 million based on Acadia’s net sales.

• DAYBUE STIX powder formulation launching in U.S. Centers of Excellence, expected to broaden patient uptake and improve persistence rates.

• DAYBUE and DAYBUE STIX are FDA-approved for Rett syndrome, with global licensing to Acadia Pharmaceuticals.

NNZ-2591 development priorities and trial progress

• Phelan-McDermid syndrome (PMS), Pitt Hopkins syndrome, and HIE are prioritized for maximum commercial and patient impact, with leadership positions targeted.

• PMS phase III (Koala trial) has dosed its first patient, with 160-patient enrollment target and multiple U.S. sites opening throughout H1 2024.

• FDA feedback for HIE and Pitt Hopkins provides clear paths, though additional juvenile animal data is required for HIE and further endpoint discussions for Pitt Hopkins.

• Targeting initiation of HIE and Pitt Hopkins phase II/III studies by year-end.

• NNZ-2591 is in Phase 3 for Phelan-McDermid syndrome and showed positive Phase 2 results in Pitt Hopkins and Angelman syndromes.