Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins

1 of 2 Neuren (NEU) – ASX Announcement 19 February 2025 Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins syndrome Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for NNZ-2591 for the treatment of Pitt Hopkins syndrome (PTHS). Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious conditions. Currently there are no treatments approved to treat PTHS. In Neuren’s Phase 2 clinical trial of NNZ-2591 in children with PTHS, 82% of participants showed improvement, including communication, social interaction, cognition and motor abilities (https://www.neurenpharma.com/pdf/045de136-df58-4616-8edf-10e713265b8a/Phase-2-trial-shows- significant-improvements-in-Pitt-Hopkins.pdf). About FDA Fast Track designation Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. A drug that receives Fast Track designation is eligible for some or all of the following: • More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval • More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers • Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met • Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA (https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority- review/fast-track) About Pitt Hopkins syndrome Pitt Hopkins syndrome (PTHS) is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18.  The incidence of PTHS has been estimated at between 1 in 34,000 and 1 in 41,000 people.  Characteristics of PTHS are a range of developmental delay with moderate-to-severe intellectual disability and behavioral differences, hyperventilation and/or breath- holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features.  Some individuals with PTHS are diagnosed with autism. Further information about PTHS is available at: www.pitthopkins.org 2 of 2 About Neuren Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug” designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases. DAYBUE™ (trofinetide) is approved by the US Food and Drug Administration (FDA) and Health Canada for the treatment of Rett syndrome. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide. Neuren’s second drug candidate, NNZ-2591, is in development for multiple neurodevelopmental disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome. Contact: [email protected] Jon Pilcher, CEO: +61 438 422 271 ASX Listing Rules information This announcement was authorized to be given to the ASX by the Board of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 Forward-looking Statements This announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.

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