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ATH434

Phase 2

Multiple System Atrophy | Small molecule | Neurology |Alterity Therapeutics Limited|Last Updated: Jul 28, 2026

Target and mechanism

ModalitySmall molecule

Also known as ATH434 dose level 1

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials3
Total Enrollment92

FDA Designations

ORPHAN_DRUGFAST_TRACK

Clinical trial landscape

ATH434 · 3 trials · 1 indication

Phase 2 3
NCT07729852Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in FranceMultiple System Atrophy
NOT YET_RECRUITING5 Analytics
NCT05864365A Biomarker Study of ATH434 in Participants With MSAMultiple System Atrophy
COMPLETED10 Analytics
NCT05109091Study of ATH434 in Participants with Multiple System AtrophyMultiple System Atrophy
COMPLETED77 Analytics
PHASE2NOT YET_RECRUITING
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
Multiple System AtrophyUnlock trial analytics
PHASE2COMPLETED
A Biomarker Study of ATH434 in Participants With MSA
Multiple System AtrophyUnlock trial analytics
PHASE2COMPLETED
Study of ATH434 in Participants with Multiple System Atrophy
Multiple System AtrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence, Severity, and Relationship of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline to completion of study treatment, an average of 1 year.

All AEs will be reviewed at every participant contact and documented in the CRF. All SAEs will be reported within 24 hours of awareness in accordance with local regulatory requirements.

Change From Baseline in Clinical Laboratory Parameters
Baseline to completion of study treatment, an average of 1 year.

Clinical laboratory parameters include hematology and blood chemistry panels.

Change From Baseline in Vital Signs
Baseline to completion of study treatment, an average of 1 year.

Vital signs include blood pressure, pulse, temperature, and respiration rate.

Duration of Exposure to ATH434
Baseline to completion of study treatment, an average of 1 year.

Total duration of treatment with investigational product per participant.

Cumulative Dose of ATH434
Baseline to completion of study treatment, an average of 1 year.

Total cumulative dose of investigational product administered per participant.

Change in brain volume as measured by MRI
Change from Baseline to Week 52
Change in iron content as measured by brain MRI
Change from Baseline to Week 52

Secondary Endpoints

Change in iron content in substantia nigra as measured by MRI
Change from Baseline to Week 52
Change in Unified MSA Rating Scale (UMSARS) Score
Change from Baseline to Week 52
Change in Patient Global Impression of Change (PGI-C)
Change from Baseline to Week 52
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ATH434 75mg BIDEXPERIMENTALParticipants who completed the prior ATH434-201 Phase 2 trial will receive ATH434 75 mg orally twice daily (BID). Treatment consists of an initial 12-month treatment phase, followed by an ongoing extension phase based on investigator assessment of continued clinical benefit, safety, and tolerability
ATH434EXPERIMENTAL -
ATH434 Arm 1EXPERIMENTAL -
ATH434 Arm 2EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
ATH434DRUGATH434 75 mg tablets administered orally twice daily (BID).
ATH434 dose level 1DRUGATH434 taken BID
ATH434 dose level 2DRUGATH434 taken BID
PlaceboDRUGPlacebo taken BID
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Eligibility Criteria

Age Range30 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Completed Alterity protocol ATH434-201. * Expected to benefit from treatment from treatment with ATH434, in the opinion of the Investigator. Exclusion Criteria: * Discontinued prior ATH434 treatment (ATH434-201) for any reason. * Significant medical or psychiatric condition ...

Countries:FranceUnited StatesAustraliaItalyNew ZealandUnited Kingdom
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Recent Changes (Last 90 Days)

LOWJul 28, 2026NCT07729852NEW_TRIAL: changed
LOWJul 28, 2026NCT07729852NEW_TRIAL: changed

Frequently asked questions about ATH434

What is ATH434 used for in Multiple System Atrophy?

ATH434 is an investigational small molecule being developed for the treatment of Multiple System Atrophy (MSA), a rare neurodegenerative disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug has received Orphan Drug and Fast Track designations from the FDA.

What does ATH434 target?

ATH434 is a small molecule designed to target pathological processes in Multiple System Atrophy (MSA). While the specific molecular target is not disclosed in available information, it is being studied for its potential to modify the disease course. The drug is in Phase 2 clinical trials for MSA.

Who makes ATH434?

ATH434 is being developed by Alterity Therapeutics Limited, a biopharmaceutical company listed on the NASDAQ under the ticker symbol ATHE. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Multiple System Atrophy (MSA).

What phase is ATH434 in?

ATH434 is currently in Phase 2 clinical development for Multiple System Atrophy (MSA). It is an investigational drug that has not been approved by regulatory authorities. The FDA has granted it Orphan Drug and Fast Track designations, reflecting the serious nature of the condition it targets.

What clinical trials is ATH434 in?

ATH434 has been studied in two clinical trials. The first, NCT05864365, was a completed Phase 2 biomarker study in 10 participants with Multiple System Atrophy (MSA) in the United States. The second, NCT07729852, is a Phase 2 open-label access study for patients who completed the first trial, enrolling 5 participants in France.

Is ATH434 the same as any other drug?

ATH434 is the primary name for this investigational drug. No alternative names have been reported in the available clinical trial information. It is being developed by Alterity Therapeutics Limited for the treatment of Multiple System Atrophy (MSA) and is currently in Phase 2 trials.