Updated Investor Presentation and Webinar Reminder
Released
Released
Announcement PDF
Neurizon Therapeutics Limited
ABN 35 094 006 023
www.neurizon.com
Suite 2, Level 11, 385 Bourke Street, Melbourne, VIC 3000 Tel: +61 3 9692 7222
Neurizon Leadership Roundtable Webinar Reminder and Updated
Investor Presentation
Highlights:
Shareholders and interested investors are invited to join Neurizon’s Leadership Roundtable webinar
featuring Neurizon’s recently appointed Chief Executive Officer, Dr Chris Bremer and members of the
Company’s Board and Management team
The pre-recorded roundtable discussion will be followed by live Q&A, providing an opportunity to engage
directly with management
Neurizon releases an updated Investor Presentation providing background information on the
Company’s strategy, NUZ-001 and the foundations supporting its development
7 October 2026 – Melbourne Australia: Neurizon® Therapeutics Limited (ASX: NUZ; OTCQB: NUZTF) (“Neurizon” or
“the Company”), a late clinical-stage biotechnology company dedicated to advancing innovative treatments for
neurodegenerative diseases, is pleased to release its updated Investor Presentation and remind shareholders and
interested investors of its upcoming Leadership Roundtable webinar.
Updated Investor Presentation
The updated presentation provides an overview of Neurizon’s strategy as the Company advances its lead
investigational asset, NUZ-001, through late-stage clinical development. It highlights the 250-participant Phase 2/3
HEALEY ALS Platform Trial, with topline results expected in late Q2 CY2027, the clinical and scientific evidence
generated to date, the NIH-funded Expanded Access Program, and the Company’s manufacturing, regulatory and
commercial preparations ahead of the HEALEY readout.
The Investor Presentation has been released as background information for shareholders and investors ahead of the
roundtable. It is separate from the webinar and no slides will be presented during the event.
Neurizon Leadership Roundtable Webinar – The Road Ahead
The webinar will be held on Thursday, 8 October 2026 at 4:00pm AEDT and will feature a roundtable discussion with
Neurizon’s Chief Executive Officer (CEO) Dr Chris Bremer, Non-Executive Chairman Sergio Duchini and Chief Finance
Officer Dan O’Connell. The discussion will explore the Company’s recent progress, the advancement of NUZ-001 and
Neurizon’s priorities for its next phase.
The pre-recorded roundtable will be followed by a live Q&A, providing the opportunity to hear directly from the
leadership team. Participants are encouraged to submit questions in advance via registration form or by emailing
enquiries@neurizon.com.
Date: Thursday, 8 October 2026
Time: 4.00pm AEDT
Register: https://bit.ly/neurizonroundtable
A recording of the roundtable discussion will be made available following the event.
-ENDS-
This announcement has been authorised for release by Dr Chris Bremer, Chief Executive Officer of Neurizon
Therapeutics Limited.
For further information, please contact:
Marketing & Corporate Affairs
Lidija Damjanovic
Email: lidija@neurizon.com
Phone: +61 (0) 425 700 504
Investor Relations
Henry Jordan
Email: henry.jordan@sdir.com.au
Phone: +61 (0) 431 271 538
Neurizon Therapeutics Limited
ABN 35 094 006 023
www.neurizon.com
Suite 2, Level 11, 385 Bourke Street, Melbourne, VIC 3000 Tel: +61 3 9692 7222
About Neurizon Therapeutics Limited
Neurizon Therapeutics Limited (ASX: NUZ) is a late clinical-stage biotechnology company dedicated to advancing
treatments for neurodegenerative diseases. Neurizon is developing its lead drug candidate, NUZ-001, for the
treatment of ALS, which is the most common form of motor neurone disease. Neurizon’s strategy is to accelerate
access to effective ALS treatments for patients while exploring the potential of NUZ-001 for broader
neurodegenerative applications. Through international collaborations and rigorous clinical program, Neurizon is
dedicated to creating new horizons for patients and families impacted by complex neural disorders. NUZ-001 is an
investigational product and is not approved for commercial use in any jurisdiction.
Neurizon Investor Hub
We encourage you to utilise our Investor Hub for any enquiries regarding this
announcement or other aspects concerning Neurizon.
This platform offers an opportunity to submit questions, share comments, and view
video summaries of key announcements.
To access Neurizon Investor Hub please scan the QR code or visit
https://investorhub.neurizon.com
Neurizon® is a registered trademark of Neurizon Therapeutics Limited
ABN 35 094 006 023
Advancing NUZ-001 in ALS
towards a key clinical
readout
Investor Presentation - October 2026
2
Disclaimer
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information provided in connection with it, as financial advice, financial product advice or advice
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This presentation is provided expressly on the basis that you will carry out your own independent
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The Company reserves the right to update, amend or supplement the information at any time in its
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Neither the Company, nor its related bodies corporate, officers, their advisers, agents and employees
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Future Matters
This presentation contains reference to certain intentions, expectations, future plans, strategy and
prospects of the Company. Those intentions, expectations, future plans, strategy and prospects may or
may not be achieved. They are based on certain assumptions, which may not be met or on which views
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Given the risks and uncertainties that may cause the Company’s actual future results, performance or
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Company does not warrant
or represent that the actual results, performance or achievements will be as expected, planned or
intended.
US Disclosure
This document does not constitute any part of any offer to sell, or the solicitation of an offer to buy, any
securities in the United States or to, or for the account or benefit of any “US person” as defined in
Regulation S under the US Securities Act of 1993 (“Securities Act”). The Company’s shares have not
been, and will not be, registered under the Securities Act or the securities laws of any state or other
jurisdiction of the United States, and may not be offered or sold in the United States or to any US
person without being so registered or pursuant to an exemption from registration including an
exemption for qualified institutional buyers.
Investor Presentation - October 2026
Our Vision
To lead the development of
treatments towards a promising
new horizon for people living with
neurodegenerative diseases.
Neurizon: A late clinical-stage biotech developing therapies for
neurodegenerative diseases
4
Lead ALS/MND program in Phase 2/3, with topline results expected in late Q2 CY2027
WHO WE ARE
Neurizon is developing
therapies for
neurodegenerative diseases,
with its lead investigational
therapy, NUZ-001, currently
being evaluated in ALS/MND.
NUZ-001 is an oral small molecule with
preclinical evidence demonstrating effects
on cellular protein clearance pathways
associated with neurodegeneration.
ASX: NUZ · OTCQB: NUZTF
LEAD ASSET: NUZ-001
Investigational oral small molecule being developed initially in ALS/MND and currently evaluated in
the Phase 2/3 HEALEY ALS Platform Trial.
NUZ-001 IN ALS/MND AT A GLANCE
ORAL
Once-daily
administration
PHASE 2/3
HEALEY ALS
Platform Trial
250
Regimen I participants,
randomised 3:1
LATE Q2 2027
Expected topline
BUILT AROUND THE CLINICAL OPPORTUNITY
CLINICAL
VALIDATION
250-participant
Phase 2/3 HEALEY
API SUPPLY
ESTABLISHED
Long-term supply agreement
with Elanco
EXPANDED
EVIDENCE
NIH-funded EAP
up to 200 participants
NEAR-TERM
CATALYST
Topline expected
late Q2 CY2027
BROADER POTENTIAL
Preclinical evidence in other
neurodegenerative diseases
Investor Presentation - October 2026
Why look now
5
A randomised Phase 2/3 readout in late Q2 2027 is the next value inflection
Late Q2 2027
Expected topline from the 250-
participant Phase 2/3 ALS trial.
Enrolled Under 5
Months
A record for the HEALEY ALS
Platform Trial.
Clinical advancement
A 250-participant randomised Phase 2/3
enrolled in less than 5 months – record
enrolment for the HEALEY ALS Platform Trial.
NIH-funded EAP for NUZ-001
Evidence-led development
Promising Phase 1 signals, with an exploratory
biomarker panel (including NfL and TDP-43) built
into ongoing Phase 2/3 trial.
HEALEY provides established clinical
infrastructure across 78 US sites led by Mass
General Brigham.
Supply chain readiness for scale
Long-term supply agreement with
Elanco for GMP API supply and
registration-scale manufacturing.
Strategic and commercial optionality
An upcoming, randomised Phase 2/3 dataset
that will inform Neurizon’s regulatory,
partnering and commercial strategy.
Total addressable market (TAM) opportunity of
~US$5bn1
1
3
2
4
NIH-Funded EAP
for NUZ-001 for 200
participants up to 96 weeks.
1 Illustrative US TAM of approximately US$5 billion based on projected 2026 US ALS prevalence of approximately 34,720 adults (CDC National ALS Registry) and
assumed annual net pricing of US$150,000 per patient. Investor Presentation - October 2026
Leadership for the next stage
6
Dr Chris Bremer commencing as Chief Executive Officer on 1 October 2026
Investor Presentation - October 2026
CHIEF EXECUTIVE OFFICER
Dr Chris Bremer
Effective 1 October 2026. Leads Neurizon
through the HEALEY readout and the decisions
that follow.
CLINICAL DEVELOPMENT
John Clark · Amanda Seiz
· Dr Chris Freitag
SCIENCE AND REGULATORY
Dr Martin Engel · Linda McBride
FINANCE AND CORPORATE
Dan O’Connell · Sergio Duchini
EXPERIENCED BOARD
Dr Ross Murdoch, Dr Katie
MacFarlane, Marcus Hughes, Dr
Steve Nanchen (Elanco, Observer)
DRAWING ON DEEP SECTOR EXPERIENCE:
EXPERIENCE TO LEAD THE NEXT PHASE
20+ years of global biopharmaceutical leadership
SCIENCE STRATEGY TRANSACTIONS
Drug
development
Portfolio &
commercialisation
Licensing &
Partnerships
MD | PhD | MBA | INSEAD
7
NUZ-001: from scientific origin to clinical execution
From an established veterinary molecule to a randomised Phase 2/3 in ALS
NUZ-001 is a well-characterised molecule, with novel neurodegenerative biology, now advancing through a Phase 2/3 ALS clinical
programme
Investor Presentation - October 2026
2005 - 2023
1
Translation to human disease
and Neurizon formation
• Monepantel: proven veterinary
drug with deep safety data
• Moved from veterinary use into
neurodegeneration
• Neurizon formed as a focused
neurodegeneration company
2024 – 2025
2
Clinical validation and
HEALEY selection
• NUZ-001 Phase 1 showed encouraging
signals across exploratory clinical
measures in people with ALS
• Results drove inclusion in the
HEALEY ALS Platform Trial
• 78 US sites, led by Mass General
Brigham
NOW TO LATE Q2 2027
3
Clinical execution
• Phase 2/3 HEALEY trial in active
execution
• Topline results expected late Q2
2027
• Treatment appeared safe and
generally well tolerated.
About ALS
ALS contributes to progressive degeneration of motor neurons,
leading to increasing loss of voluntary muscle function
ALS AT A GLANCE
2–5 YEARS
Typical life expectancy following
diagnosis*
~90%
of ALS cases are without family history
NO CURE
Significant unmet
medical need remains
~35,000
people living with ALS in the US1
No approved therapy has substantially altered the course of ALS
progression for the broad patient population.
A therapy that slows ALS meaningfully, in a broad population, with oral
dosing, would address a substantial need in the ALS/MND market.
*Disease course varies considerably between individuals. Sources for final deck: ALS Association / peer-reviewed literature; verify wording and citations before publication.
.1. US prevalence estimate as used in company materials.
SPEECH
Loss of ability
to speak
SWALLOWING
Increasing
difficulty
swallowing
MOVEMENT
Progressive loss
of muscle
function
BREATHNG
Increasing
difficulty
breathing
ALS is a rapidly progressive neurodegenerative disease with
significant unmet need
9
Investor Presentation - October 2026
10
FUS = Fused in Sarcoma; SOD1 = Superoxide Dismustase 1; TDP-43 = Transactive response DNA-binding protein 43
1. Ling SC, Polymenidou M, Cleveland DW. Converging mechanisms in ALS and FTD: disrupted RNA and protein
homeostasis. Neuron. 2013 Aug 7;79(3):416-38. doi: 10.1016/j.neuron.2013.07.033.
In most ALS/MND, nuclear TDP-43 is lost and accumulates in the cytoplasm, disrupting RNA processing, axonal
maintenance and communication with muscles
TDP-43 dysfunction & aggregation links ALS pathology to
motor neuron failure
Investor Presentation - October 2026
1 2 3
<1%
FUS Mutation
~2%
SOD1 Mutation
97%
TDP-43 Aggregation
A SHARED PATHOLOGY
of ALS cases
show
TDP-43
pathology1
TDP-43 IS LOST FROM THE NUCLEUS
AND BUILDS UP IN THE CELL
Nuclear TDP-43 Loss
Cellular TDP-43 Aggregation
The neuron loses normal function
while harmful protein burden increases.
THE MOTOR NEURON–MUSCLE
CONNECTION BREAKS DOWN
A
B
C
Impaired axon maintenance and
synaptic vesicle release Æ unreliable
muscle control
Cytoplasmic toxic aggregates trap
essential proteins Æ impair axon
transport
Cytoplasmic aggregates stress
cellular machinery Æ starve
motor neurons
NUZ-001 shown in preclinical studies
to enhance cellular protein clearance
mechanisms to support motor neuron
proteostasis
A
B C
ALS: high unmet need with significant value potential
11
Three decades of approvals have delivered limited clinical benefit
Key ALS Value Reference Point:
Shionogi – RADICAVA (2026)
US$2.5B
Upfront for global rights paid by Shionogi to Tanabe
Pharma
~US$700M
Expected annual global sales
• Commercial, revenue-generating ALS franchise
• Oral product US patent until 2039
• Rare disease commercial platform foundation
Current US ALS treatment landscape
2017 IV
2022 ORAL
RILUZOLE
Broad ALS population | Oral
RADICAVA® / RADICAVA ORS®
Broad ALS population | IV & oral
QALSODY®
SOD1-ALS only | Intrathecal
1995 2023
~3 months
median survival extension
across randomized
controlled trials
~6 months
median survival extension
across randomized
controlled trials
~3 years
median survival extension
in pivotal VALOR trial
Protects neurons from
overactivation-induced
death
Reduces neuron
degeneration as
antioxidant in the nervous
system
Binds to mRNA of SOD1 to
prevent toxic protein
cluster formation in
neurons
1 2 3
4
1. Hinchcliffe et al. 2017, Degenerative Neurological and Neuromuscular Disease 7 (May): 61–70.
2. Takahashi et al. 2025, Muscle & Nerve 72 (4): 586–96.
3. Miller et al. 2026 JAMA Neurology 83 (2): 115–25.
4. Shionogi press release, 22 December 2025; transaction completion announced April 2026
Investor Presentation - October 2026
Science Behind NUZ-001
Therapeutic Approaches Addressing ALS Biology
13
Addressing cellular quality-control
systems involved in clearing toxic
protein aggregates.
Protein Homeostasis
Gene-Targeting & RNA
Modulation
Silencing or correcting disease-
causing gene transcripts at the RNA
level.
Dampening toxic glial activation
and immune-driven neuron injury.
Neuroinflammation &
Immune Regulation
Neutralise toxic free radicals and
protect vital cellular components.
Oxidative Stress
Gene translation
Investor Presentation – October 2026
Pre-clinical Evidence Supporting NUZ-001 Potential in ALS/MND
14
Prevents cellular
TDP-43 aggregation
Enhances protein clearance
mechanisms (autophagy &
proteosomal system)
Preserves STMN2
expression to support
neuronal health
Restores NfL axonal health
and synaptic signalling
+ NUZ-001
NfL = Neurofilament Light, STMN2 = Stathmin-2; TDP-43 = Transactive response DNA-binding protein 43
Consistent effects
observed across models:
MND-patient
motor neurons
Zebrafish
Human
neurospheres
NUZ-001 Enhances Protein Clearance Pathways to Remove Toxic Protein Aggregates
Investor Presentation – October 2026
NUZ-001 Expansion potential : Platform Optionality for
Proteinopathy-Driven Diseases
15
TDP-43
ALS/AD/PD/FTD/CTE
Amyloid beta
AD/PD
Tau
AD/PD/FTD/CTE/PSP
alpha Synuclein
AD/PD
NUZ-001 is a mechanism-driven, disease-agnostic approach to
treating neurodegeneration by rebalancing proteostasis and
addressing core cellular dysfunction.
AD = Alzheimer’s Disease; ALS = Amyotrophic Lateral Sclerosis; CTE = Chronic Traumatic Encephalopathy; FTD = Frontotemporal Dementia; HD = Huntington Disease; PD = Parkinson’s Disease; PSP =
Progressive Supranuclear Palsy
Strong mechanistic rationale for potential efficacy across a wide array of neurodegenerative diseases. Impaired proteostasis
contributes to the formation of intracellular inclusion bodies of misfolded proteins, a hallmark of neurodegenerative diseases.
mHTT
HD
New preclinical data show NUZ-001 increases activity of multiple protein
clearance pathways in neuronal models: autophagy and proteasomal systems.
Investor Presentation – October 2026
Clinical Development
Phase 1b and Open Label Extension Study
Early Clinical Development of NUZ-001 in MND/ALS started in Australia
17
PRIMARY OUTCOME
• Long-term treatment with NUZ-001 at the
recommended Phase 2/3 dose appeared
safe and generally well-tolerated (n=12)
SLOWED FUNCTIONAL
DECLINE
RESPIRATORY
STABILISATION
SURVIVAL*
• Sustained slowing in disease progression
by 39% (P1b) and 31% (P1b & OLE)
compared to PRO-ACT matched controls
• No study discontinuations
• Several participants still accessing NUZ-
001 under the TGA’s Special Access
Scheme
Highlights (Based on a study population of n=12 patients)
• Reduction in the risk of death by 71%
compared to PRO-ACT matched
controls
• Estimated life extension of ~16 months
• 49% reduction (P1b) and 43% reduction
(P1b & OLE) in VC PP compared to PRO-
ACT matched controls
* Neurizon Phase 1 and Open Label Extension Study data from September 2025
BIOMARKER TRENDS
• Stable plasma NfL and urinary p75ECD
PATIENT SATISFACTION
Investor Presentation – October 2026
-2.93 -1.65
-5
-4.5
-4
-3.5
-3
-2.5
-2
-1.5
-1
-0.5
0
PRO-ACT Matched Controls
(n=420) NUZ-001 (n=12)
-1.21 -0.83
-2
-1.8
-1.6
-1.4
-1.2
-1
-0.8
-0.6
-0.4
-0.2
0
PRO-ACT Matched Controls
(n=360) NUZ-001 (n=12)
ALSFRS-R
Slope
(points/month)
Phase 1b and Open Label Extension Results
Preliminary Signals of Efficacy in ALSFRS-R and SVC
18
Treatment with NUZ-001 across combined Phase 1 and OLE studies slowed the progression of ALS in all patients by 31% for ALSFRS-R and 43%
for SVC percent predicted (PP) when compared to matched controls from the PRO-ACT historical database1
1. Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, Walker J, Katsovskiy I, Schoenfeld D, Cudkowicz M, Leitner M. The PRO-ACT database: design, initial analyses, and predictive features.
Neurology. 2014 Nov 4;83(19):1719-25. doi: 10.1212/WNL.0000000000000951.Epub 2014 Oct 8. PMID: 25298304; PMCID: PMC4239834
2. Forced vital capacity (FVC) was used when SVC was not collected
3. Matched on time since onset, baseline ALSFRS-R, pre-baseline slope, and disease onset location
Motor Function – ALSFRS-R
Difference
0.37 points/month (p=0.145)
Difference
1.27 points/month (p=0.078)
31% 43%
Respiration – SVC PP2
3 3
VC
PP
Slope
(points/month)
Investor Presentation – October 2026
Phase 1b & Open Label Extension
NUZ-001 shows improved survival vs matched controls
19
NUZ-001 treatment resulted in statistically
significantly longer survival of participants with
ALS, (X2=11.35, p=0.00075), compared to matched
controls1 from the PRO-ACT database2
1. Matched on time since onset, baseline ALSFRS-R, pre-baseline ALSFRS-R slope, and disease onset location
2. Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, Walker J, Katsovskiy I, Schoenfeld D, Cudkowicz M, Leitner M. The PRO-ACT database: design, initial
analyses, and predictive features. Neurology. 2014 Nov 4;83(19):1719-25. doi: 10.1212/WNL.0000000000000951.Epub 2014 Oct 8. PMID: 25298304; PMCID:
PMC4239834
Analysis* suggests that treatment with NUZ-001
reduces the risk of death by 71%:
Hazard ratio of 0.29 (95% CI: (0.131, 0.644), p = 0.0024)
Investor Presentation – October 2026
* Neurizon Phase 1 and Open Label Extension Study data from September 2025
Clinical development progress from Phase 1b to Phase 2/3
20
Phase 1b established preliminary safety profile & informed the hypothesis; HEALEY is designed to
provide the controlled evidence
Investor Presentation – October 2026
NUZ-001 was selected for inclusion in HEALEY following independent ALS expert review of Neurizon’s preclinical and clinical evidence package.
Phase 1b Foundation in 12
People Living with ALS
• Established 10 mg/kg daily dose
• Preliminary safety profile
observed over 3.5 years
• Early signals of efficacy; slowing
function decline and extended life
expectancy
• Mechanistic insights for
hypothesis-formation & trial
design
HEALEY PROVIDES THE EVIDENCE
Tailored Study Design
Randomised • double-blind • concurrent placebo • 36 weeks
SCALE + EXECUTION
250
Participants
78
US sites under a
single master
protocol
<5 months
to complete
enrolment
Filing-relevant evidence package
Function • survival • extended safety • CSF exposure • biomarkers
Clinical Questions to be
Determined
• Clinical efficacy against
matched placebo
• Reproducibility across
MND/ALS diversity
• Biological measures of disease
progression
Regimen I is designed to investigate clinical benefit
21
REGIMEN I AT A GLANCE
Design Double-blind, placebo-controlled adaptive Phase 2/3 under the HEALEY master protocol
Participants 250 enrolled at close of recruitment (protocol expanded from 160 to 240 in May 2026)
Randomisation 3:1 NUZ-001 to placebo — a fully concurrent, internally consistent placebo arm
Dose and route
10 mg/kg once daily, oral — the dose carried forward from the Phase 1b open-label
extension
Treatment period 36-week double-blind period, followed by a 36-week active treatment extension
36-week blinded period
Earlier HEALEY regimens read out at 24 weeks. A
longer blinded window gives more time for functional
curves to separate in a disease where treatment
effects accumulate slowly and variably, and optimises
the chance of showing a clinically meaningful
treatment effect.
Fully concurrent placebo arm
Regimen I carries its own placebo group, removing the
temporal-drift and comparability associated with
pooled placebos.
Primary and Secondary Endpoints
WHY THESE DESIGN CHOICES MATTER
Recruitment complete
July CY2026
All participants enrolled
and dosing commenced
RCT Completion
Early Q2 CY2027
36-week randomised
period, then active
extension
Database lock
Q2 CY2027
Data cleaning and
validation
Topline result
Late Q2 CY2027
Primary efficacy and safety
readout
P: Change in ALSFRS-R and survival from baseline to Week 36
S: Slow vital capacity from baseline to Week 36
S: Freedom from death or permanent assisted ventilation from
baseline to Week 36
Investor Presentation – October 2026
Exploratory Endpoints
Biological validation of disease progression and mechanism
of action through extensive exploratory biomarker panel
(NfL, mtDNA, protein panel, speech)
Regimen I Biomarker Portfolio
22
Connecting clinical response profile with biological measures provides development optionality
Investor Presentation – October 2026
Blood / CSF
response
to disease and treatment
Molecular
analysis -
disease activity
and
treatment
mechanism
Digital speech -
objective
signal of
disease
progression
Biomarker Insight
Identify subgroups
Clarify who may benefit
most in a
heterogeneous disease
Support regulatory
dialogue
Add biological context
to
the totality of evidence
De-risk future
development
Inform population
selection and next-
study design
Inform future payer
discussions
Build evidence for
differentiated
value and access
HEALEY
Regimen I
Large, diverse, well-
characterised ALS
population
Expanded access protocol extends NUZ-001 to patients outside
Regimen I
23
What the program does Why it matters
Access outside the trial
Provides NUZ-001 to people with ALS
who cannot access Regimen I.
Funded externally
NIH funded, highlighting further program
validation. Access is extended without
drawing on capital allocated to HEALEY.
✓ Operates alongside the RCT
Separate from Regimen I; no effect on its
conduct, randomisation or blinding.
Observations are uncontrolled and are
not evidence of efficacy.
Investor Presentation – October 2026
Why it matters
HEALEY REGIMEN I (RCT)
Randomised,
blinded, 3:1
36-week blinded period,
placebo controlled
Topline
late Q2 CY2027
EXPANDED ACCESS PROTOCOL (EAP)
NIH-funded,
non-dilutive
Broader patient
population, all on active
Observations without
placebo control
WHAT THE EAP ADDS TO THE PROGRAM
Longer-term safety in a
broader population
Grant-funded drug supply
and safety systems
Wider clinician experience
with NUZ-001
EAP is complementary to HEALEY RCT and fully funded by US National Institute of Health (NIH)
✓
✓
The Road Ahead
Comparison of Key Outcomes Across Approved ALS Treatments & NUZ-001
Riluzole1
(Rilutek®)
Edaravone2
(Radicava®)
Amx00353,4
(Relyvrio®)
Tofersen6
(Qalsody®)
01
02
03
04
ALSFRS-R
(Improvement
Compared to
Placebo)
Not evaluated Not evaluated
FVC/SVC
(Improvement
Compared to
Placebo)
Survival
(Improvement
Compared to
Placebo)
NUZ-0017
~3 months
33.20% (p=0.0013) 23.43% (FVC, p=0.0942) ~6 months
23.27% (p=0.030) 22.37% (SVC, p=0.080) ~6.5 months5
14.74% (p=0.970) 35.59% (SVC, p>0.05) Not evaluated
39% (p=0.080)10 48% (SVC, p=0.058)10
31% (p=0.145)10 41% (SVC, p=0.078)10
~16 months10
24 weeks
24 weeks
28 weeks
OLE: 116 weeks9
MEND: 40 weeks8
1. N Engl J Med. 1994 Mar 3;330(9):585–9;2. Lancet Neurol 201716: 505-12; 3. N Engl J Med 2020; 383:919-30; 4. Voluntary removal from the market; 5. Muscle Nerve. 2021
Apr;63(4):31–39.6. Engl J Med 2022:387:1099-110; 7. Neurizon data on file; 8. Median treatment period in Phase 1 MEND Study; 9. Median treatment period for combined
Phase 1 MEND Study and OLE Study; 10. Comparison to matched untreated controls in PRO-ACT Database. 11. Research Reports World 2024; 12. BioPharma Dive,
ALS Drug Development, September 30, 2025; 13. Biogen 2025 Annual Report; Verified Market Report 2025;
(2017)
(1995)
(2022)
(2023)
Cross-trial comparisons are descriptive only; differences in study design, patient populations, and endpoints limit
direct comparability.
52 weeks n=77
n=69
n=89
n=72
n=12
Investigational
25
Investor Presentation - October 2026
Annual Sales
Generic, ~US$40m
in sales11
~US$1 bn in
peak sales11
>US$400m
sales12
~US$86.9m
sales13
NA
A Trial Pathway Built To Carry Advanced Regulatory Discussion
26
Potential pathway options only. Final strategy, approval pathway and label depend on the Regimen I trial data and regulator interaction.
Investor Presentation - October 2026
UNDER WAY
Blinded treatment
36-week randomised period, then an
active treatment extension
LATE Q2 CY2027
Phase 2/3 readout
Function, survival, respiratory, safety and
biomarker data across all participants and
pre-specified subgroups
FOLLOWING READOUT
Agency engagement
Discussion with the FDA, EMA, TGA
and PMDA on the data package
SUBJECT TO DATA
Registration path
Scope, route and timing are set in
the regulatory engagement.
WHAT REGIMEN I BRINGS INTO THAT DISCUSSION
A randomised, internally
controlled dataset
Regimen I carries its own concurrent placebo arm
rather than pooled platform controls, over a longer
duration vs other regimens.
Supporting evidence built
alongside the trial
An exploratory biomarker panel and real-world data
from the Expanded Access Program to support data
from the primary and secondary endpoints.
Regulatory and manufacturing
readiness
Orphan drug desi
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