Ann: Neuren to present at autism research meeting, page-2

Neuren (NEU) – ASX Announcement 6 February 2025
Neuren to present at autism research meeting
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced the acceptance of an
abstract for presentation at the 2025 International Society for Autism Research (INSAR) Annual Meeting,
being held 30 April - 3 May 2025, in Seattle Washington. Details of the presentation are as follows:
“Many Apparent False Negatives in Detection of Mutations in Gene Associated with Autism Spectrum
Disorders”
• Presentation Type: Poster
• Date: Thursday, 1 May
All INSAR Annual Meeting content to be presented or published is embargoed until the time of the
actual presentation. Posters will be embargoed until Thursday, 1 May.
Neuren Vice President of Clinical Development Nancy Jones PhD commented: “The importance of
accurate genetic testing for children with developmental delay cannot be overstated. We hope that in
bringing to light the shortcomings in current genetic testing, methodologies will improve and more
children and families can receive a diagnosis.”
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.