Corporate Presentation May 2026
Released
Released
Announcement PDF
May 2026
Corporate
Presentation
Forward looking statements
2
This presentation may contain some statements that may be considered “Forward-Looking
Statements”, within the meaning of the US Securities Laws. Thus, any forward-looking
statement relating to financial projections or other statements relating to the Company’s
plans, objectives, expectations or intentions involve risks and uncertainties that may cause
actual results to differ materially. For a discussion of such risks and uncertainties as they relate
to us, please refer to our 2025 Form 20-F, filed with US Securities and Exchange Commission,
in particular Item 3, Section D, titled “Risk Factors.’’
Alterity is a late clinical stage biopharmaceutical
company dedicated to developing treatments
for neurodegenerative diseases
Our goal is to slow the course
of disease progression
We strive to create an
alternate future and improve
patient quality of life
Alterity means the
state of being different
3
Redefining Neurodegenerative Disease Therapy
A Potential First-in-Class, Disease-Modifying Therapy for Multiple System Atrophy (MSA)
4
MSA is a rare, rapidly progressive disease (up to 50,000 U.S. patients)
Independent assessment supports ~$US2.4B global peak sales opportunity in MSA
End-of-Phase 2 FDA meeting planned for mid-2026 to align on Phase 3 design
Veteran development team with 3 FDA approvals in Neurology area
Up to 48% slowing of disease progression v. placebo (p<0.05) on FDA-endorsed endpoint*
Favorable safety profile with no drug-related serious adverse events
*modified Unified MSA Rating Scale Part 1 (mUMSARS)
Compelling Phase 2 Efficacy
on FDA-Endorsed Endpoint
Unmet Need with Significant
Commercial Potential
Pivotal Advancement in 2026
with Clear Regulatory Path
Differentiated, Disease-
Modifying Approach for MSA
Oral iron chaperone ATH434 targets excess reactive iron and α-synucleinopathies
Blood brain barrier penetrant small molecule
Program:
Discovery /
Pre-clinical
Phase 1
Natural
History
Phase 2 Phase 3 Collaboration
Multiple system atrophy
Double blind study
Multiple system atrophy
Open label study in
advanced MSA
Multiple system atrophy
Natural history study
Parkinson’s disease
Friedreich’s ataxia
Drug discovery
Broad opportunity for ATH434 in neurodegenerative disease
5
Planned
Multiple System Atrophy (MSA):
Parkinsonian disorder with no approved treatment
0
Years: 3 6 9
7.5 years
median survival
after symptom onset
Over 50%
require wheelchair
in 5 years
Rapidly progressive
Highly debilitating
Up to 50,000
patients in U.S.
Disease characteristics:
• Motor: Parkinsonism, uncoordinated
movements, balance problems, falls
• Autonomic dysfunction: blood pressure
maintenance, bladder control, bowel function
• Atrophy and α-synuclein accumulation in
multiple brain regions
Premotor MSA Clinically probable MSA Death can occur
Clinically established MSA
Urinary dysfunction
REM sleep behavior disorder
Orthostatic hypotension
Multidomain
autonomic failure
Pyramidal signs
Parkinsonism
Cerebellar features
Recurrent falls
Indwelling catheter
Frontal executive dysfunction
Tracheostomy
Bronchopneumonia
Urosepsis
Unintelligible speech
Gastrostomy
6
Sources: Fanciulli, Wenning. NEJM 2015 | Wenning, et al. Mov Disorders 2022 | Coon, et al. Brain 2015 |
Multiple System Atrophy: National Institute of Neurological Disorders and Stroke (nih.gov)
Sudden death
Targeting the pathology in
Parkinsonian disorders
8
Targeting key players in MSA pathology
Alpha-synuclein and iron balance in health and disease
Two forms of iron required for cellular function
• Neurotransmitter synthesis (e.g., dopamine)
• Myelin synthesis (allows fast signal transmission)
α-Synuclein protein
• Present in all neurons
• Enables neuronal communication
Fe2+
Fe3+
Fe2+
Reactive
Fe3+
Stable
In disease: α-Synuclein
aggregates in neurons in MSA
impairing communication and
leading to dysfunction
In disease: Excess reactive iron
drives α-synuclein aggregation
and oxidative injury
Pathology of Parkinsonian disorders
9
Dexter et al. Brain.1991
Parkinson’s disease
n = 24
n = 13
n = 9
n = 7 *
n = 3
n = 8 *
0 10000 20000 30000
S. nigra (T)
S. nigra (pc)
Cerebellum
nmol iron/g of human brain
n = 8
n = 8
n = 9
n = 8
n = 11
n = 8 *
n = 9
n = 6 *
n = 10
n = 8 *
0 10000 20000 30000
nmol iron/g of human brain
Putamen (M)
Putamen (L)
Globus pallidus (M)
Globus pallidus (L)
S. nigra (T)
Increased Brain Iron in Areas of Pathology
Patients Healthy controls
Multiple System Atrophy
Parkinson’s disease
10
Excess labile iron is a key driver of pathology causing
α-synuclein aggregation and oxidative injury
Iron
imbalance
Excess
labile iron
Fe2+ Fe2+
Fe2+
Fe2+
Fe2+
Fe3+
PCBP
Fe2+
Neuron death
and impaired
oligodendrocyte
function
monomer
or normal
oligomers fibrils
α-syn fibrils
damage multiple
organelles
α-syn fibrils
impair myelin
production in MSA
α-Synuclein aggregation
Mitochondrial dysfunction
ROS damage to cellular components.
Lipid peroxidation
Increased
ROS
ROS
Fe2+
PCBP
PCBP: Poly(rC)-binding proteins
Fe3+ Stable
Fe3+
Fe2+ Reactive
Fe2+
ATH434 mechanism of action: Iron chaperone
ATH434 chaperones excess labile (reactive) iron
to reduce neuronal injury
Sources: Adapted from Yanatori et al., BBA 2020 and Philpott et al., Frontiers Mol Bio. 2023 |
Pall, et al. Metallomics, 2024.
Efflux iron from
cell (ferroportin)
Increase iron
storage (ferritin)
Buffering Fe2+
in labile iron pool
PCBP: Poly(rC)-binding proteins
1
2
Fe3+
Fe2+
ZIP8
ZIP14
Transferrin
receptor
Labile iron
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
Fe2+
ATH434
ATH434
PCBP
PCBP
1
Fe2+
PCBP
2
Fe
3+
Tf
Fe
3+
PCBP
Enzyme
PCBP
ATH434
ROS
Fe2+
Fe2+
Fe2+
3
11
Chaperone
mechanisms:
3
Treatment approach: Address underlying pathology
12
Based on
mechanism of
action, ATH434 is a
potential disease
modifying therapy
Preserve neurons
and oligodendrocyte
cell function
Reduce α-synuclein
aggregation and
oxidative injury
Chaperone excess
labile iron in the
CNS
Stabilize function or
slow decline
ATH434: Small molecule drug candidate
13
Broad treatment
potential
Potential to treat many neurodegenerative
diseases (e.g., Parkinson’s, Friedreich Ataxia)
Orphan & Fast Track
designations
US FDA Fast Track Designation and
Orphan drug designation in U.S. and EU
Blood-brain barrier
penetrant
Acts intracellularly to address underlying
pathology
Iron chaperone Moderate binding affinity, redistributes
excess labile iron in CNS
Oral administration Preferred by patients and doctors vs
infusions (IV, intrathecal) or injections
ATH434 binding
to labile iron
14
ATH434 consistently
improved motor
performance by
reducing α-synuclein
aggregation and
preserving neurons
Target
Disease
Model
Midbrain iron
(incl. s. nigra)
α-Synuclein
Preserve
neurons/
function
Clinical
observations
MSA1 PLP-α-syn
Improved motor
performance
MSA2 PLP-α-syn to
Improved motor
performance
Parkinson’s Monkey MPTP to n/a
Improved motor
performance
Parkinson’s Mouse MPTP
Improved motor
performance
Parkinson’s Mouse A53T
Improved motor
performance
Parkinson’s
Mouse tau
knockout
Improved motor
performance
Multiple models of neurodegenerative disease demonstrate
ATH434 efficacy
Stable
Sources: Bradbury et al, Am Acad. Neurol 2024; Finkelstein, Acta Neuropath Comm 2017; Beauchamp et
al, Neurotherapeutics 2022; 1 Heras-Garvin et al, Mov Disord. 2021; 2 Finkelstein et al, J Park Dis 2022
ATH434 clinical development
program in MSA
Diligent approach to de-risk development program
16
• Observational study in 21
participants with clinically
probable MSA
• Designed to de-risk clinical
development program
• Identify biomarkers to
improve accuracy of
patient selection
Phase 2
ATH434-201
ATH434-202
Randomized double-blind
placebo-controlled trial
Results: clinically meaningful efficacy on
MSA rating scale, measures of orthostatic
hypotension, disease severity
Natural History Study
Open label trial in advanced
MSA patients
Results: showed improved neurological
symptoms in more advanced patients and
favorable safety
bioMUSE
*Biomarkers of progression in Multiple System Atrophy (bioMUSE).; Biomarkers: Biomarkers:; MRI, Fluid, Digital
BioMUSE natural history study results
17
bioMUSE
Advanced MRI methods
Structural mapping
Sources: Presentations/Posters on file, including Claassen et al, MDS Conference 2021 and 2022.
Optimized patient selection in Phase 2 trials
Precision biomarker assessment
Differentiated MSA from
Parkinson’s disease (PD)
Identified ”iron signature”
of early MSA
Revised selection criteria in
ATH434‐201 and ATH434-202
protocols to exclude PD patients
Novel strategies for measuring
brain iron in individual regions
Improved precision of
volume measurements
State of the art methods enabled
precise measurements of brain
iron and volume with MRI
α-synuclein SAA in CSF
Iron content in pallidum
ATH434-201: Randomized, double-blind, placebo-controlled study
18
N = 77
Study design: Endpoints:
Patient criteria:
• Clinical diagnosis of MSA
• Motor symptoms ≤4 years
• No severe impairment
• Elevated brain iron on MRI
• Elevated plasma NfL
Placebo twice daily
50 mg twice daily
Key clinical endpoint:
MSA Rating Scale
75 mg twice daily
ATH434
ATH434-201
MRI - Magnetic Resonance Imaging; UMSARS - Unified MSA Rating Scale (UMSARS) Scale– measures activities of daily living
CGI-S - Clinical Global Impression-Severity scale; OHSA - Orthostatic Hypotension Symptom Assessment
Additional secondary endpoints:
CGI-S, OHSA, Wearable Sensors,
Safety
ATH434
12 months treatment
Key biomarker endpoint:
brain iron content by MRI
1 : 1 : 1
Importance of the Unified MSA Rating Scale Part I (UMSARS I)
19
ATH434-201
Speech Walking
Swallowing Falling
Handwriting Orthostatic symptoms
Cutting food Urinary function
Dressing Bowel function
Hygiene Sexual function^
Validated rating scale to assess MSA disease severity
Rates functional impairment in domains affected in MSA
Rated from 0 to 48
higher scores worse
^ Modified version of UMSARS I excludes sexual function item
UMSARS Part I Items:
UMSARS is the FDA
endorsed endpoint
to support approval
for the treatment of
MSA
Proposed primary
endpoint in Phase 3
Placebo ATH434-201
50 mg twice daily
ATH434-201
75 mg twice daily
N=22 N=25 N=24
Age (y) 61.3 (6.6) 63.1 (6.1) 63.9 (6.7)
Gender (% male) 63.6% 52.0% 62.5%
Duration of motor
symptoms (y)
2.5 (0.8) 2.6 (0.8) 2.3 (0.9)
Modified UMSARS I 16.9 (3.9) 15.2 (5.4) 14.4 (4.4)
Motor score
of Parkinson plus scale1
57.6 (14.2) 47.8 (18.4) 48.9 (16.8)
Plasma NfL (pg/mL) 34.9 (12.5) 31.1 (9.1) 32.3 (9.0)
CSF aggregating α-syn SAA (+) 91% 92% 96%
OH symptom assessment 13.5 (9.8) 13.8 (13.2) 15.0 (12.2)
Clinical phenotype: MSA-P (%) 59.1% 60.0% 70.8%
Severe orthostatic hypotension 4.5% 4.0% 29.2%
20
Baseline characteristics ATH434-201
1 Payan et al. PlosOne 2011
Groups balanced at
baseline except for
severe orthostatic
hypotension − a
predictor of rapid
disease progression
Clinical Analysis Population
!"
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%"
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!
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& !' %# '( )%
*++,
ATH434-201
worsening
Modified
UMSARS
I
Change ATH434 − Change Placebo
Change Placebo
Relative Treatment Effect:
Minimal clinically important
difference (MCID) on UMSARS I = − 1.5 points
21
Clinically Significant Efficacy on Modified UMSARS Part I
Change from Baseline to Week 52
ATH434
50 mg
N=25
Placebo
N=22
ATH434
75 mg
N=24
Difference v.
placebo
LS mean (SE)
Relative
treatment
effect
-3.7 (1.7) 46%
-2.7 (1.8) 34%
Clinical Analysis Population, incl CSF NfL as covariate
Efficacy on Clinical Global Impression of Severity (CGI-S) scale
Change from baseline to week 52
22
• CGI-S
• 7-point scale
• higher score indicates a worse outcome
• Assesses total picture over prior 28 days
• illness severity, impact of illness on
function, level of distress and any other
aspects of impairment
ATH434-201
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Clinical Analysis Population, incl CSF NfL as covariate
ATH434
50 mg
ATH434
75 mg
Placebo
LS mean (SE)
Orthostatic Hypotension Symptom Assessment (OHSA)
Change from baseline to week 52
10
5
-5
0
Change
In
OHSA
(week
S2‒
BL)
• Assesses symptoms of low blood
pressure when going from sitting to
standing (e.g., dizziness / feeling faint /
lightheadedness)
• Patient reported outcome
p=0.08
worsening
LS Mean (SE)
p=0.14
ATH434
50 mg
ATH434
75 mg
Placebo
23
Kaufmann, et al. Clin Auton Res 2012..
ATH434-201
Clinical Analysis Population
24
ATH434-201
Step count Total time walking
Sit to stand transitions
Pendant
Wearable movement sensor
ATH434-201
50 mg
ATH434-201
75 mg
Placebo
Bouts of walking
ATH434 preserved walking in outpatient setting
Change from baseline to week 52
Clinical Analysis Population, incl CSF NfL as covariate
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25
1 - Reporting one or more event
2 - None related to Study Drug
Placebo ATH434-201 ATH434-201
twice daily 50 mg 75 mg
Any Adverse Event (AE) 24 (92.3%) 21 (84.0%) 25 (96.2%)
N=25 N=26
N=26
ATH434-201
N (%) of subjects 1
• Similar rates of
AEs in ATH434
and placebo
participants
• No severe or
serious AEs
related to study
drug
• No hematologic
side effects
Severe AEs 2
8 (30.8%) 3 (12.0%) 6 (23.1%)
Serious AEs 2
10 (38.5%) 5 (20.0%) 7 (26.9%)
1 (3.8%) 3 (12.0%) 2 (7.7%)
2 (7.7%) 1 (4.0%) 5 (19.2%)
1 (3.8%) 6 (24.0%) 4 (15.4%)
8 (30.8%) 7 (28.0%) 8 (30.8%)
14 (53.8%) 10 (40.0%) 7 (26.9%)
Back pain
Fatigue
Covid-19
Fall
UTI
Adverse Events
Neuroimaging
Substantia Nigra
Midbrain axial view
Putamen / Globus Pallidus
Basal ganglia axial view
QSM: Quantitative Susceptibility Mapping І QSM overlay: warm colors = higher magnetic susceptibility (iron content)
Measuring Iron Content with MRI (QSM)
Regional increases in iron in MSA Patient
28
Iron Deposition on MRI in Placebo: Change Baseline → Week 52
ATH434-201
Mean ± 95% CI
Iron Deposition on MRI: ATH434 vs. Placebo
ATH434-201
MMRM with sex, age, baseline CSF NfL, and baseline iron as covariates
30
ATH434 decouples iron accumulation from clinical worsening
Correlation Between Change in Iron and Change in UMSARS I Score at Week 52
Controlling for Sex, Age, Baseline CSF NfL, Baseline QSM
Summary: ATH434 Interrupts the Pathologic Process in MSA
31
• Iron accumulates in the basal ganglia of MSA patients, most prominently in the
globus pallidus and putamen
• QSM allows quantitation of total brain iron but does not distinguish between
reactive (toxic in excess) and stable forms of iron
• In placebo-treated patients, change in iron strongly correlates with change in
disease severity
• In ATH434-treated patients, change in iron weakly correlates with change in
disease severity
- ATH434 leads to a decoupling of iron accumulation and clinical worsening as
assessed by UMSARS I
Measuring Brain Volume in MSA Regions of Interest
Subject 1
Change in Brain Volume*
0.0000
-0.1000
-0.2000
-0.3000
-0.4000
-0.5000
-0.6000
worsening
Z-score
ATH434-201
50 mg BID
ATH434-201
75 mg BID
Placebo
26 weeks 52 weeks
ATH434 showed trends in
preserving brain volume
* MSA Atrophy Index: composite z-score of putamen, GP, cerebellum
and brainstem vs. healthy age-matched population
Subject 2
ATH434-202: Open label study in advanced MSA
33
1 MSA affected areas by MSA-atrophy index. Trujillo, P. et al Annals of Clin and Trans Neuro, 2025
*ATH434-201 listed for comparative purposes, data are not from a head-to-head study
Design Single arm, open-label
Objective Assess safety and efficacy in advanced MSA
Population Advanced MSA (n=10)
Treatment ATH434 75 mg BID x 12 months
Brain MRI Biomarkers Volume, Iron
Key Clinical Measure UMSARS I
ATH434-202
The study indicates
the potential of
ATH434 to slow
disease progression
in advanced MSA
No serious Adverse Events (AEs) related to study drug
AEs consistent with underlying disease
Comparable efficacy observed at same dose in double blind study
Outcomes:
Carefully designed Phase 2 program demonstrates potential for
ATH434 in MSA
34
ATH434 demonstrated clinically significant efficacy in slowing disease progression in MSA
Both dose levels efficacious on UMSARS I
and important secondary endpoints
Demonstrated target engagement with
reduced iron accumulation in MSA affected
brain regions
Stabilized orthostatic hypotension, one of the
most challenging MSA symptoms to manage
Preserved walking in outpatient setting as
measured with objective digital biomarker
Open-label trial showed comparable
safety and efficacy in advanced MSA
No safety signals and well-tolerated
No serious AEs related to study drug
Proposed Phase 3 trial Design
35
N = ~200
Study design: Efficacy Endpoints:
Patient criteria:
• Clinical diagnosis of MSA
• Ambulatory without
assistance
• No severe impairment
• Brain atrophy in MSA
affected regions on MRI
• Elevated plasma NfL
1 : 1 *
Primary endpoint
Modified UMSARS I
FDA endorsed clinical endpoint
50 mg
twice daily
ATH434
MRI - Magnetic Resonance Imaging; UMSARS - Unified MSA Rating Scale (UMSARS) Scale– measures activities of daily living
CGI-S - Clinical Global Impression-Severity scale; OHSA - Orthostatic Hypotension Symptom Assessment; orthostatic SBP – orthostatic systolic blood pressure
Secondary endpoints
OHSA, Wearable Sensors,
Brain Volume by MRI
Placebo
12 months treatment
Finalize Phase 3 program after end-of-Phase 2 FDA feedback and then initiate trial activities by YE 2026
twice daily
* Randomization stratified by screening plasma NfL
Efficacy Analysis
Includes baseline CSF NfL as
covariate
Commercial assessment &
corporate overview
Independent commercial assessment in MSA
37
USD $2.4 Billion
Potential worldwide annual peak sales for ATH434 in MSA
Severely debilitating illness
with no approved treatment
ripe for new entrants
Critical need for a tolerable,
disease modifying therapy
Importance of inhibiting
α‐synuclein aggregation to
address the underlying
pathology of disease
Slowing disease progression is
key driver of physician interest
Stabilizing orthostatic
hypotension^, one of the most
challenging symptoms in MSA,
strongly positions ATH434
^Orthostatic hypotension is the drop in blood pressure on standing; Global peak sales number in U.S. Dollars;
Independent market research survey conducted in 2025. Peak sales estimate based on U.S. prevalence of ~19,000
Over 70% of neurologists
were “extremely likely” or
“very likely’ to prescribe
ATH434 based on its profile
Target product profile based on positive Phase 2 data
ü Reached alignment in Type C meeting related to clinical pharmacology and non-clinical elements
ü Reached alignment in Type C meeting related to Chemistry Manufacturing & Controls (CMC)
• On track for Type B End-of-Phase 2 meeting mid-year
38
• Initiate start-up activities for the trial by year end
• Clinical site identification and qualification
• Manufacture and package of clinical drug supply
• Expand intellectual property protection
• Evaluate additional high-value indications to grow the pipeline
• Strengthen the team to enhance organizational capabilities
Finalize Regulatory Strategy - Align with U.S. FDA on Phase 3 clinical trial
Phase 3 Readiness activities
Build for Scalable Growth
Well Positioned for 2026 Catalysts
ASX: ATH
NASDAQ: ATHE
APPENDIX
40
41
Inadequately chaperoned cellular iron drives MSA pathology
Promotes α-synuclein cross
linking1
Directly increases
α‐synuclein translation2
ODG toxicity due to limited
endogenous glutathione3
Free radicals promote
α‐synuclein aggregation4
Impaired lysosomal
autophagy5
1Duce JA, et al. Mol Neurodegener. 2017;12(1):45. 2Febbraro F, et al. NeuroReport. 2012;23(9):576-580. 3Thorburne SK, Juurlink BH. Neurochem.
1996;67(3):1014-1022. 4Souza JM, J Biol Chem. 2000;275(24):18344-18349. 5Tanji, K, et al. Neurobiol Dis. 2013;49:190-198.
doi 10.3233/JAD-170601 (2018); doi.org/10.3390/ijms25084269 (2024)
Christine Kaindlstorfer, Kurt A Jellinger, Sabine Eschlbök, Nadia Stefanova, Günter Weiss, Gregor K Wenning
Seojin Lee, Ivan Martinez-Valbuena , Anthony E. Lang, Gabor G. Kovacs
Iron converts native α-SYN into a β-sheet conformation and promotes
its aggregation either directly or via increasing levels of oxidative stress. The
disturbance of iron homeostasis leads to abnormal iron deposition in the brain
and causes neurotoxicity via generation of free radicals and oxidative stress.
Importantly, extensive evidence suggests a molecular relationship
between iron accumulation and α-syn pathology.
Cellular iron deposition patterns predict clinical subtypes of
multiple system atrophy
Neurobiology of Disease. 2024. https://doi.org/10.1016/j.nbd.2024.106535
Journal of Alzheimer’s Disease (2018) DOI 10.3233/JAD-170601
42
Reactive iron
Generates free radicals
Promotes α-synuclein aggregation
α-synuclein aggregation
Neuronal toxicity
Impaired myelin production
Oxidative stress
Disrupts multiple cellular functions
Promotes α-synuclein aggregation
Iron is a key driver of the MSA Pathology
ATH434 chaperones excess iron to reduce neuronal injury
Overwhelms
natural iron
buffering systems,
leading to iron
accumulation in
MSA brain regions
MSA Pathology Cycle
Disrupted
Control of
CNS Iron
4
Key disease
drivers
43
Correlation Between Change in Iron and Change in UMSARS I Score at Week 52
ATH434 decouples iron accumulation from clinical worsening
Controlling for Sex, Age, Baseline CSF NfL, Baseline QSM
ATH434-201: Populations and key endpoints
44
All patients in Imaging and Clinical analysis populations were randomized and treated
Imaging analysis
population
Clinical analysis
population
71
61
6 no post baseline UMSARS I
5 neg. CSF α-syn SAA
10 d/c before week 26
1 completed week 26 w/o MRI
Endpoint Change from BL to week 52 Population
Biomarker (Primary) Imaging
Clinical (Key secondary) Clinical
Iron content in s. nigra by MRI
Change in Modified UMSARS Part I
Randomized
77
ATH434-201
ATH434-202: Open label study in advanced MSA
45
Key objective was
to assess efficacy
and safety of
ATH434 75 mg dose
for comparison to
75 mg dose in 201
double-blind study
Parameter
ATH434-202
75 mg BID
N=10
ATH434-201
75mg BID
N=24
Age (yr) 64.5 (7.5) 63.9 (6.7)
Duration of motor symptoms (yr) 3.9 (1.8) 2.3 (0.9)
Modified UMSARS I1 19.2 (5.3) 14.4 (4.4)
Motor score of Parkinson Plus Scale2 57.5 (20.4) 48.9 (16.8)
Plasma NfL (pg/mL) 42.1 (14.1) 32.3 (9.0)
OH Symptom Assessment 16.7 (14.8) 15.0 (12.2)
Severe Orthostatic Hypotension 40.0% 29.2%
Mean (SD)
1 MSA affected areas by MSA-atrophy index. Trujillo, P. et al Annals of Clin and Trans Neuro, 2025
ATH434-202
Change over 12 Months
ATH434-202
75 mg BID
N=10
ATH434-201
75mg BID
N=24
Modified UMSARS I 3.5 (4.7) 5.6 (5.6)
Clinical global impression of change (% stable) 30% 21%
Patient global impression of change (% stable) 30% 26.4%
Brain volume1 -0.44 (0.14) -0.42 (0.29)
Mean (SD)
ATH434-202: Key data at 75 mg dose
Comparison to double blind study at 12 mo
46
• No serious AEs related to study drug
• AEs consistent with underlying disease
1 In MSA affected areas by MSA-atrophy index. Trujillo, P. et al. Annals of Clin and Trans Neuro, 2025
ATH434-202
The 75 mg dose
demonstrated
comparable efficacy
to that observed in
the double-blind
study
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