Neuren highlights participation in PMSF Family Conference
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Neuren (NEU) – ASX Announcement 24 July 2026
Neuren highlights participation in the PMSF Family Conference
Melbourne, Australia: Neuren Pharmaceuticals Limited (Neuren; ASX: NEU) was honoured to support
and participate in the biannual Phelan-McDermid Syndrome Foundation Family Conference as the
PresentingSponsor. The conference titled “The climb we make together” was held in Colorado from 15
July to 19 July, with more than 800 community members attending.
NeurenCEOJonPilcher addressedthe conference inboth the opening
and closing sessions and Chief Medical Officer Liza Squires presented
Neuren’s development program for its investigative drug NNZ-2591,
culminating in the ongoing Koala study - the first ever Phase 3 clinical
trial for Phelan-McDermid syndrome (PMS). The Neuren team of 12
people hosted both a Neuren corporate booth as well as a booth
dedicated to the Koala study, which were visited by many families.
Neuren CEO Jon Pilcher commented: “The PMSF Family Conference
was inspiring and energising for the Neuren team, with so many
conversations with parents and siblings caring for their loved ones with PMS. The conference
demonstrated how far the PMS community has come in the four years since Neuren started the first
ever multi-site clinical trial. As we strive to achieve a potential first approved treatment for PMS, the
courage, determination and support of PMS families and continuing the productive collaboration with
clinicians, researchers, PMSF and CureSHANK will be critical.”
Robbie Baker, CEO of the Phelan-McDermid Syndrome Foundation (PMSF), commented: “We are
gratefulto Neurenforservingas the PresentingSponsorof the 2026 PMSFFamily Conference. Neuren's
generous support has helped bring together families, researchers, and clinicians to learn from one
another and strengthen our shared commitment to advancing research and care for individuals with
PMS. We are in an unprecedented era of research and therapeutic development in PMS, including
Neuren’sPhase 3Koala study evaluating NNZ-2591. This landmark study is the first-ever Phase 3 clinical
trial conducted specifically for PMS. For a community navigating a rare disorder with no currently
approved treatments, this milestone represents both meaningful scientific progress and hope for the
future.”
The conference alsofeatured an overview by Tess Levy (Icahn School of Medicine, Mt Sinai, New York),
the lead author of the paperrecentlypublishedin the AutismResearch journal of a study estimating the
prevalence of PMS to be 1 in 7,300 people (https://onlinelibrary.wiley.com/doi/full/10.1002/aur.70297)
This is significantly higherthan previousestimates,reinforcingthe magnitude of the unmetneedandthe
importance of ongoing initiatives to accelerate diagnosis and improve genetic testing. The study was
supported by Neuren.
Neuren’s Koala Phase 3 clinical study in children aged 3 to 12 years with PMS now has 13 trial sites
activated to enrol participants, including the first site in Canada. Four more sites are scheduled to
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activate in August. More than 100 potential participants have beenreferredbyNeurento active sites or
await activation of closer sites. The first participants have completed 13-weeks of treatment in the
randomisedplacebo-controlledstudyandare continuing in the 52-week open-label extension study. In
addition, the first patient from Neuren’s previously completed Phase 2 open-label clinical study in
paediatric patients with PMS has now been able to re-commence treatment by enrolling in the open-
label extension study.
NeurenChief MedicalOfficerLiza Squires commented: “Neuren’s Koala Phase 3 study continues to gain
momentum. 13 sites across the US and Canada are now actively enrolling, and four additional sites are
scheduled to open next month. We are excited and proud to partner with the PMS community to
advance this important trial.”
Click here to view this announcement on our Investor Hub.
About Phelan-McDermid syndrome
Phelan-McDermid syndrome (PMS) 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 neurons in the brain. It is estimated that 1 in 7,300 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
impairmentand developmentaldelay,delayedorabsentspeech,symptomsof 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
About Neuren
Neuren Pharmaceuticals is developing new drug therapies to treat multiple serious neurological
disorders caused by genetic abnormalities or brain injury, that have no or limited approved treatment
options. Neuren’s therapies target the critical role of Insulin-like growth factor 1 (IGF-1) in the brain,
using orally administered analogs of naturally occurring peptides.
DAYBUE® (trofinetide) oral solution is approved by the US Food and Drug Administration (FDA), Health
Canada and the Ministry of Health in Israel and DAYBUE STIX (trofinetide) powder is approved by the
FDA forthe treatment of Rett syndrome. Neuren has granted an exclusive worldwide license to Acadia
Pharmaceuticals Inc. for the development and commercialisation of trofinetide.
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Neuren’s investigative drug candidate, NNZ-2591 (ercanetide), is in clinical development as an oral
solution treatmentfor multiple neurodevelopmental disorders, including Phelan-McDermid syndrome,
Pitt Hopkins syndrome and Angelman syndrome. Each of these programs has been granted “orphan
drug” designation in the United States and the European Union as well as Fast Track and Rare Pediatric
Disease designations from the FDA. Neuren is also developing NNZ-2591 for the treatment of hypoxic
ischemic encephalopathy (HIE), a serious condition caused by brain injury before or shortly after birth.
Currently, Neuren is conducting a Phase 3, randomized, double-blind, placebo-controlled clinical trial
(“Koala”) evaluating the safety and efficacy of NNZ-2591 in children aged 3 to 12 years with Phelan-
McDermid syndrome and a 52-week open-label extension study.
Investor contact:
Jon Pilcher, CEO & Managing Director
+61 438 422 271
investorrelations@neurenpharma.com
Media Contact:
Monsoon Communications
Rudi Michelson
rudim@monsoon.com.au
ASX Listing Rules information
This announcement was authorized to be given to the ASX by the CEO of Neuren Pharmaceuticals
Limited, Suite 1.01, 117 Camberwell Road, Hawthorn East, VIC 3123
Forward-looking Statements
This announcementcontainsforward-lookingstatementsthataresubjectto risksand 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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