Investor presentation, 26 August 2026
Released
Released
Announcement PDF
IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES
H1 2026 Results Webinar
26 August 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$287
million cash
as at 30 Jun
2026
1 Refer to www.acadia.com for DAYBUE (trofinetide) US prescribing and important safety information. Trofinetide is approved in the EU, Canada and Israel. Trofinetide is not approved
in Australia
2 NNZ-2591 is an investigational medicine and is currently not approved for sale in any country
Optimising total shareholder return with first-ever dividend program
4
Dividend program anchored to growing royalty
income:
• Semi-annual payment
• Targeting payout ratio of 70 –
100% of the after-tax amount of
royalty income less corporate and
administrative costs (“Available Pool”)
• Franked to max extent possible
Declared interim dividend of 15c per share:
• Fully franked
• Representing 90% of H1 26 Available
Pool
• To be paid on 7 Oct 2026
A$m unless otherwise stated H1 2026 actual Full year 2026
estimates
Royalty income US$m 23.3 52.6-56.2
AUD/USD exchange rate 0.702 0.70
Royalty income 33.2 75.1-80.3
Corporate & admin (3.0) (6.6)
Net total before tax 30.2 68.5-73.7
Applicable tax @30% (9.0) (20.6-22.1)
Available Pool 21.1 48.0-51.6
Payout ratio 90%
Payout Amount 19.0
Dividend per share A$ 0.15
H1 2026 highlights
5
29
2.3
15.4 16.0
20.8
18.1
24.2
23.3
33
1H23 2H23 1H24 2H24 1H25 2H25 1H26 2H26E*
Royalty income to Neuren
US$ million
* Range estimates based on full year 2026 royalty income estimates of US$53 – 56 million assuming Acadia meets its full year 2026 DAYBUE net sales guidance of US$480 – 510 million, less H1 26 actual royalty
income of US$23.3 million
CY26E US$53-56m
CY25 US$42m
CY24 US$37m
CY23 US$18m
On track for record FY 26 royalty income
US$53-56m, with US$23.3m
already earned in H1 26
H1 26 R&D investment of A$27.4mdriven
by the expected progression of the Koala Phase
3 study in PMS
Modest corporate & admin cost of A$3.0m
for H1 26, fully offset by interest income of
A$5.6m
Strong balance sheet with A$287m cash &
short-term investments at 30 Jun 2026
All NNZ-2591 programs are fully funded
6
Milestones achieved since Jan 2026
Q1 2026 Q2 2026
FDA feedback on PTHS and HIE
Q3 2026
PMS Phase 2 published in
Neurology Genetics
Acadia request CHMP re-
examination following
negative opinion
1st patient dosed in 13-week placebo-
controlled Koala Phase 3 study
STIX limited launch in the US
STIX broadly available in the US CHMP recommended
DAYBU’s approval in EU
after re-examination
PMSF family
conference
presented by Neuren
Published study supported
by Neuren estimating the
prevalence of PMS to be 1
in 7,300 people
DAYBUE
approved in Israel
Growing sales in the US and through global named patient programs
Koala active trial sites expanded from 2 to 15; 8 sites now active for open label extension (OLE)
PRV program reauthorized by US Congress
Acadia upgraded CY26
DAYBUE guidance
1st patient enrolled in Koala OLE after
completing placebo-controlled study
Delphi expert consensus
recommended DAYBUE as standard
of care for Rett in the US
EC approval
of DAYBU
DAYBUE
(trofinetide)
1
NNZ-2591
(ercanetide)
2
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
DAYBUE
(trofinetide)
Exclusively licensed to Acadia
Pharmaceuticals globally
7
Strong DAYBUE sales momentum driving sustainable royalty growth
8
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$480-510m
1 Prange EO, Beisang A, Pehlivan D, et al. Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method. Ann Child Neurol. 2026; 4:38-51
Record quarterly sales in Q2 and upgraded 2026 guidance by Acadia
Delphi expert consensus panel
recommended DAYBUE as part
of the standard of care for
eligible patients with Rett
syndrome1
DAYBUE STIX accelerating
engagement with new and
returning patients
8.5 10.4
9.6
12.9
53
18
37
42
56
CY2023 CY2024 CY2025 CY2026E*
Royalty to Neuren (US$m)
85 101
96
125
480
177
348
391
510
CY2023 (Apr
-Dec)
CY2024 CY2025 CY2026E
Acadia
Guidance
DAYBUE Net Sales (US$m)
+23–30% + 24–33%
Q2
Q2
Q1
Q1
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
9
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
Neuren is leading the development of a first treatment for Phelan-
McDermid syndrome (PMS)
11
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 (ercanetide) program
✓ Orphan Drug designation (US and EU)
✓ Rare Pediatric Disease designation (US)
✓ Meaningful improvements rated by
clinicians and caregivers in open-label
Phase 2 trial2
✓ Alignment with FDA on single Phase 3 trial
design and endpoints to support a New
Drug Application
✓ Fast Track designation (US)
✓ Koala Phase 3 trial enrolling in US and
Canada
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
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.”
12
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
13
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 America1)
Honoured to support and participate in the
biannual PMSF Family Conference as the
Presenting Sponsor
Koala Phase 3 study2 continues to gain
momentum
15 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 Estimates based on United Nations population data 2025, derived by applying the estimated prevalence rate to the populations under 60 years
2 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
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A milestone-rich roadmap
Q3 2026 Q4 2026 2027
JP 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
JP NDA submission
Acadia Q4 26 results
Commencement of
juvenile animal toxicity
study to support HIE IND
FDA meeting 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
15
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