Ann: NEU receives FDA minutes and re-confirms Phase 3 endpoints, page-2

Neuren (NEU) – ASX Announcement 13 May 2025
Neuren receives FDA minutes and re-confirms primary endpoints for Phase 3 trial
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today confirmed that it has received the
official minutes of its Type C Meeting with the US Food and Drug Administration (FDA), held on 8 April to
discuss efficacy endpoints for its upcoming Phase 3 clinical trial of NNZ-2591 to treat Phelan-McDermid
syndrome.
As announced last month by Neuren following the Meeting, the co-primary endpoints for the single
Phase 3 pivotal trial will be the change from baseline in the Receptive Communication sub-domain of the
Vineland Adaptive Behavior Scales, Third Edition (VABS-3 Receptive-Raw Score) and the overall score in
the Phelan-McDermid Syndrome Assessment of Change (PMSA-C, previously referred to as CGI-I in
Neuren’s Phase 2 trial). Both measures were robustly positive with clinically meaningful improvement in
Neuren’s Phase 2 open-label clinical trial. 16 out of 18 children showed improvement measured by the
VABS-3 Receptive-Raw Score, with mean improvement of 7.5 from a mean baseline of 29.0 (Wilcoxon
signed rank test p=0.0001) and 16 out of 18 children showed improvement from baseline measured by
the PMSA-C with a mean score of 2.4 (Wilcoxon signed rank test p<0.0001).
Neuren remains on-track to commence the Phase 3 trial mid-year 2025, subject to FDA review of the
final version of the trial protocol. Neuren’s financial strength means that no additional funding is
required to execute the program.

About Phelan-McDermid syndrome (PMS)
Phelan-McDermid syndrome is caused by a deletion or other change in the 22q13 region of chromosome
22, which includes the SHANK3 gene, or a mutation of the gene. PMS is also known as 22q13 deletion
syndrome. The SHANK3 gene codes for the shank3 protein, which supports the structure of synapses
between nerve cells in the brain. It is estimated that between 1 in 8,000 and 1 in 15,000 people have
PMS. There are no medications, drugs, or therapies specifically for PMS, which has an overwhelming
unmet medical need. PMS has severe quality of life impacts on those living with it, as well as on parents
and siblings. The most common characteristics are moderate to severe developmental and intellectual
impairment and developmental delay, delayed or absent speech, symptoms of autism, low muscle tone,
motor delays, mild to severe epilepsy, behavioural problems and difficulties with socialization, activities
of daily living and self-care. Further information about PMS is available at: www.pmsf.org and
www.cureshank.org



https://www.neurenpharma.com/pdf/4e...-minutes-and-reconfirms-Phase-3-endpoints.pdf