Corporate Presentation, 2 September 2026
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Released
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IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES
Corporate Presentation
2 September 2026
Forward looking statements
This presentation contains forward looking statements that involve
risks and uncertainties. Although we believe that the expectations
reflected in the forward looking statements are reasonable at this
time, Neuren can give no assurance that these expectations will
prove to be correct. Actual results could differ materially from those
anticipated. Reasons may include risks associated with drug
development and manufacture, risks inherent in the regulatory
processes, delays in clinical trials, risks associated with patent
protection, future capital needs or other general risks or factors.
2
Large potential upside for shareholders is enabled by financial strength
3
*Rett and Fragile X syndromes are licensed to Acadia, with same economics to Neuren as
trofinetide; Neuren retains worldwide rights to all other indications
Phelan-McDermid syndrome (PMS)
Pitt Hopkins syndrome
(PTHS)
Hypoxic Ischemic
Encephalopathy (HIE)
Angelman syndrome, Prader-Willi Syndrome, SYNGAP1,
Rett syndrome (Acadia)*, Fragile X syndrome (Acadia)*
NNZ-2591
2
indications prioritised for
maximum commercial impact
Long-term income growth
from DAYBUE®
(trofinetide)1
A$543m income from DAYBUE
since launch in 2023
A$287
million cash
as at 30 Jun
2026
1 Refer to www.acadia.com for DAYBUE (trofinetide) US prescribing and important safety information. Trofinetide is approved in the EU, Canada and Israel. Trofinetide is not approved
in Australia
2 NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
4
A uniquely positioned ASX biotech company
Sustainable growing royalty
income from DAYBUE, driven by
strong momentum in US and
ongoing global expansion
Recurring royalty supplemented
by significant milestone
payments
Multiple potential indications
beyond PMS, with development
prioritised for maximum
commercial impact
NNZ-2591 value potential to be
unlocked through successful
execution of PMS Phase 3 study
Attractive DAYBUE commercial
model with 100% pretax profit
margin for Neuren
Prioritised pipeline fully funded,
with surplus cash enabling
opportunities to optimise total
shareholder return
Targeting serious neurodevelopmental conditions with urgent unmet need
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Impaired communication
between neurons across
synapses, abnormal
formation/pruning of
dendrites, chronic
inflammation
Rett
(MECP2)
Fragile X
(FMR1)
Phelan-McDermid
(SHANK3)
Pitt Hopkins
(TCF4)
Angelman (UBE3A) | Prader-Willi (15q11-q13) |
SYNGAP1-related disorder (SYNGAP1)
Neurodevelopmental disorders
Hypoxic-Ischemic Encephalopathy
(lack of oxygen or blood flow to the brain
before, during or shortly after birth)
Brain injury
Walking and balance issues Anxiety and hyperactivity Seizures
Impaired communication Intellectual disability Impaired social interaction
Developmental delays
Cognitive impairment
Seizures
Cerebral palsy
Impaired hand use Sleep disturbance Gastrointestinal problems
Severe impact on nearly every aspect of life Long-term impact on survivors
Excitotoxicity,
mitochondrial
dysfunction, acute and
chronic inflammatory
processes
Neuren’s drugs target the
critical role of insulin-like
growth factor 1 (IGF-1),
using analogs of naturally
occurring peptides that
can be taken orally as
liquids
Synapses
DAYBUE
(trofinetide)
Exclusively licensed to Acadia
Pharmaceuticals globally
6
Strong DAYBUE sales momentum driving sustainable royalty growth
7
Refer to www.acadia.com for DAYBUE (trofinetide) US prescribing and important safety information. Trofinetide is approved in the EU, Canada and Israel. Trofinetide is not approved in Australia
* Full year estimates based on Acadia full year 2026 DAYBUE Net Sales Guidance of US$480-510m
The only approved treatment for Rett syndrome in US, EU, Canada and Israel
8.5 10.4
9.6
12.9
53
18
37
42
56
CY2023 CY2024 CY2025 CY2026E*
Royalty to Neuren (US$m)
85 101
96
125
480
177
348
391
510
CY2023 (Apr
-Dec)
CY2024 CY2025 CY2026E
Acadia
Guidance
DAYBUE Global Net Sales (US$m)
+23–30% + 24–33%
Q2
Q2
Q1
Q1
Strong adoption of new
powder formulation STIX
in the US exceeding
expectations
DAYBU® launch in
Germany anticipated in
early Q4 2026
0%
25%
50%
75%
100%
0% 20% 40% 60% 80% 100%
%
penetration
rate
% total diagnosed patients
Significant upside potential in the US
8
Delphi expert consensus panel recently recommended DAYBUE as part of the standard of care for
eligible patients with Rett syndrome1
Refer to www.acadia.com for DAYBUE (trofinetide) US prescribing and important safety information. Trofinetide is approved in the EU, Canada and Israel. Trofinetide is not approved in Australia
1 Prange EO, Beisang A, Pehlivan D, et al. Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method. Ann Child Neurol. 2026; 4:38-51.
2 Acadia Q1 2026 Earnings Call
DAYBUE STIX accelerating engagement with new and returning
patients
• Limited launched in Q1 2026 focusing on Centers of Excellence (COEs)
• Broadly available from early Q2 2026
• 40% of all US DAYBUE patients now receiving STIX
• 45% of STIX demand in Q2 were from new or returning patients
US Penetration Rate2
COEs Outside COEs
Approved Aug 2026
9,000 - 12,000 Rett patients1
Phase 3 results Sep–Nov 2026
1,000 Rett patients1
Approved Mar 2023
6,000 - 9,000 Rett patients1
Long term growth opportunity through global expansion
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Tiered Royalty Rates
(% of net sales)3
1 Acadia estimates
2 Each milestone payment is payable once only
3 Royalty rates payable on the portion of annual net sales that fall within the applicable range
Future Sales
Milestones2
Future Development
Milestones2
US$35m following 1st
commercial sale in Europe
US$15m following 1st
commercial sale in Japan
Net Sales in a CY US$m
≥US$500m 50
≥US$750m 100
≥US$1bn 150
Annual Net Sales Rates
≤US$250m 10%
>US$250m, ≤US$500m 12%
>US$500m, ≤US$750m 14%
>US$750m 15%
Up to US$170m on
achievement of escalating
annual net sales thresholds
Up to US$110m on
achievement of escalating
annual net sales thresholds
Mid-teens to low-20s % of
net sales
Mid-teens to low-20s % of
net sales
NNZ-2591
(ercanetide)
10
NNZ-2591 targets the critical role of IGF-1 in synapses
11
Cellular
responses
Pre-
synaptic
neuron
Post-
synaptic
neuron
Neurotransmitters
Synapses
IGFBP
IGF-1
IGFBP
NNZ-2591
IGF-1R
IGF-1 activity is regulated
by binding proteins
(IGFBPs) that bind IGF-1
and prevent it from acting
at its receptor (IGF-1R),
reducing signaling
NNZ-2591 increased
the availability of IGF-1
to trigger signaling at
IGF-1R in vitro1,
increasing the amount
of signaling
IGF-1
IGF-1R
IGF-1 regulates synaptic
transmission
NNZ-2591 increases IGF-1 availability1 and
attenuates neuroinflammation2
1 Guan et al. Sci. Rep. 2014;4:4388. doi: 10.1038/srep04388
2 Evaluation of the neuroprotective effects of NNZ-2591 in a mouse model of neonatal hypoxic-ischemic encephalopathy. University of Nebraska Medical Center, 2025. Unpublished data on file
IGF-1 promotes the
delivery of
neurotransmitter
receptors into the
membrane. This directly
increases the amplitude
of the postsynaptic
response
IGF-1 regulates synaptic
vesicle dynamics. By
enhancing
neurotransmitter release,
IGF-1 increases synaptic
transmission
NNZ-2591 is the first investigational medicine in Phase 3 for Phelan-
McDermid syndrome (PMS)
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The Voice of the Patient…..1
“PMS has an overwhelming unmet medical need.
There are no FDA approved treatments for PMS despite its
severely debilitating manifestations. Parents and caregivers are
open to trying almost anything to try to relieve their child’s
suffering; most have tried an incredibly high number of
treatments and approaches for symptom management, with very
little success.”
NNZ-2591 program
✓ Alignment with FDA on single Phase 3 trial
design and endpoints to support a New
Drug Application
✓ Koala Phase 3 trial enrolling in US and
Canada
✓ Meaningful improvements rated by
clinicians and caregivers in open-label
Phase 2 trial2
✓ Supported by positive effects and dose
response in shank3 model of PMS3
✓ Orphan Drug designation (US and EU)
✓ Rare Pediatric Disease designation (US)
✓ Fast Track designation (US)
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
1 Excerpts from Voice of the Patient Report of Externally-Led Patient-Focused Drug Development Meeting Nov 2022
2 NEU-2591-PMS-001: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan- McDermid Syndrome - 13 weeks treatment of patients age 3-12 years
at 4 US sites
3 The impact of NNZ-2591 on the phenotype of the shank3 KO mouse model of Phelan-McDermid syndrome. GeN.DDi, 2019. Unpublished data on file
Developmental delay/intellectual impairment (lack of safety
awareness) and communication issues
are the most troublesome concerns.
Improved cognitive functioning and improved
communication are the most desired outcomes.
“PMS has severe quality of life impacts on those
living with the disease, as well as on parents and
siblings.
Most activities of daily life, including communicating needs or
wants, self-care (bathing, dressing, toileting) and socializing
with peers/siblings are affected. Most individuals living with
PMS rely on their parents and caregivers for all their daily needs,
and many require 24-hour care.”
13
NNZ-2591
Placebo
160
80
80
Double-blind, 13 weeks
NNZ-2591
Open label, 12 months
Randomised 1:1
giving estimated 95%
power for each co-
primary endpoint
Koala - the first ever Phase 3 trial in PMS
Screening
4 - 6 weeks
Same age range (3-12) and same
length of treatment (13 weeks) as
Phase 2
Target dosing equivalent to dose
tested in Phase 21
~20 trial sites, mostly in US
Program fully funded from
existing cash
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
1 12.5 mg/kg per day in Phase 3 vs 12 mg/kg in Phase 2, and titration period two weeks in Phase 3 vs six weeks in Phase 2
Alignment with FDA on single Phase 3 trial design and endpoints to support a NDA
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Co-primary Endpoints in Phase 2 Results1
Phelan-McDermid Syndrome Assessment of
Change (PMSA-C), previously referred to as
CGI-I in Phase 2
16/18 subjects showed improvement
Mean score: 2.4
(P < 0.00012)
Receptive Communication sub-domain of the
Vineland Adaptive Behavior Scales, 3rd
Edition (VABS-3 Receptive-Raw Score)
16/18 subjects showed improvement
Mean improvement: 7.5 (from baseline
of 29.0) 3
(P = 0.00012) 3
Key Phase 3 endpoints robustly positive in Phase 2 trial
Key Secondary Endpoint in Phase 2 Results1
Caregiver Impression of Change (CIC) score
15/18 subjects showed
improvement
Mean score: 2.7
(P = 0.00032)
NNZ-2591 was well tolerated in Phase 2, with no clinically meaningful changes
in safety parameters during treatment
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
1 NEU-2591-PMS-001: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan-McDermid
Syndrome - 13 weeks treatment of patients age 3-12 years at 4 US sites
2 Wilcoxon signed rank test - p-values are nominal without type 1 error control
3 Based on post hoc analysis of overall VABS-3 secondary endpoint
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Neuren’s leadership in science and community for PMS
Recent published study supported by Neuren
estimating the prevalence of PMS to be 1 in
7,300 people (~39,000 in North America2)
Honoured to support and participate in the
biannual PMSF Family Conference as the
Presenting Sponsor
Koala Phase 3 study1 continues to gain
momentum
16 trial sites in US/Canada
activated
100+ patients referred to
sites or await site activation
8 sites activated in OLE for
completers of 13-weeks
study
First patient from Phase 2
study enrolled in OLE
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
1 Koala is a randomised, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of NNZ-2591 for 13 weeks in approximately 160 children aged 3 to 12 years and a 52-
week open-label extension study
2 Estimate based on United Nations population data 2025, derived by applying the estimated prevalence rate to the populations under 60 years
Aiming for the first investigational medicine in Phase 3 for Pitt Hopkins
syndrome (PTHS)
16
NNZ-2591 program
✓ Type B End of Phase 2 meeting with FDA
scheduled for Oct 2026 to align Phase 3
study
✓ Clinical improvements observed by
clinicians and caregivers in Phase 2 trial1
✓ Positive effects observed in tcf4+/- model
of PTHS2
✓ Orphan Drug designation (US and EU)
✓ Fast Track designation (US)
✓ Rare Pediatric Disease designation (US)
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
1 NEU-2591-PTHS-001: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Pitt Hopkins syndrome - 13 weeks treatment of patients age 3-17 years at 5
US sites
2 Drug Validation Initiative. Gen.DDi, 2019. Unpublished data on file
3 Wilcoxon signed rank test - p-values are nominal without type 1 error control
• 13 weeks treatment of patients age 3-17
years in open label trial at 5 US sites
• Mean CGI-I of 2.6 with 9 out of 11 children
showing improvement (p = 0.00393)
Phase 2 trial highlights1
Improvements were seen in clinically
important aspects of Pitt Hopkins
syndrome, including:
• communication
• social interaction
• cognition; and
• motor abilities
Multiple late-stage opportunities wholly owned by Neuren
17
NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
18
A milestone-rich roadmap
Q3 2026 Q4 2026 2027
Japan trial top-line results
DAYBUE
(trofinetide)
1
NNZ-2591
(ercanetide)
2
Commercial launch in
Germany
Acadia Q3 26 results
US$35m 1st EU
commercial sale
milestone payment
Japan NDA submission
Acadia Q4 26 results
Commencement of
juvenile animal toxicity
study to support HIE IND
End of Phase 2 meeting with FDA
to align PTHS Phase 3 study
IND application and
FDA meeting for HIE
Periodic updates on Koala progress
PTHS Externally-Led Patient-Focused
Drug Development (EL-PFDD) Meeting
with the FDA
1 Refer to www.acadia.com for DAYBUE (trofinetide) US prescribing and important safety information. Trofinetide is approved in the EU, Canada and Israel. Trofinetide is not approved in
Australia
2 NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
CONTACT
investorrelations@neurenpharma.com
19
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