NEU Presentations at ACMG Annual Clinical Genetics Meeting
Released
Released
Announcement PDF
Neuren (NEU) – ASX Announcement
22 January 2025
Neuren Presentations at the ACMG Annual Clinical Genetics Meeting 2025
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced the acceptance of three
abstracts for presentation at the 2025 American College of Medical Genetics and Genomics (ACMG)
Annual Clinical Genetics Meeting, being held on 18–22 March 2025, in Los Angeles.
Details of the presentations are as follows:
“NNZ-2591, a Synthetic IGF-1 Metabolite Analog: Phase 2 Clinical Trial Results for Children and
Adolescents with Pitt Hopkins Syndrome”
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Presentation Type: Platform (O38)
Date/Time: Thursday, 20 March – 1:30 PM (Platform Session 2)
“The Evolving Genetic Landscape of Phelan-McDermid Syndrome and Implications for Diagnostics”
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Presentation Type: Poster (P672)
Date/Time: Friday, 21 March– 10:30-11:30 AM – Exhibit Hall A
“Safety, Efficacy, and Exposure-Response of NNZ-2591, a Synthetic Analog of an IGF-1 Metabolite, for
Phelan-McDermid Syndrome in Children and Adolescents”
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Presentation Type: Poster (P166)
Date/Time: Friday, 21 March – 10:30-11:30 AM – Exhibit Hall A
All ACMG Annual Clinical Genetics Meeting content to be presented or published is embargoed until the
time of the actual conference presentation. Posters will be embargoed until Wednesday, 19 March at
5:00 pm PT.
Neuren Chief Medical Officer Liza Squires M.D. commented: “We are very pleased to have three
presentations at ACMG this year which highlight our programs to develop NNZ-2591 as a much-needed
treatment for Phelan-McDermid syndrome and Pitt Hopkins syndrome. The presentations address the
evolving diagnostic landscape in Phelan-McDermid syndrome, and the encouraging results achieved in
our Phase 2 trials.”
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.
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Neuren’s second drug candidate, NNZ-2591, is in Phase 2 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
Investor Relations
investorrelations@neurenpharma.com
Jon Pilcher, CEO: +61 438 422 271
Scientific and Medical Enquiries
medicalinformation@neurenpharma.com
ASX Listing Rules information
This announcement was authorized to be given to the ASX by the CEO & Managing Director 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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