Exhibit 99.1
Alterity Therapeutics to Host Webcast Today on New Data from the bioMUSE Natural History Study
- New bioMUSE Data Informs ATH434-202 Study Endpoints –
- Webcast to Include Key Opinion Leader, Daniel Claassen, M.D., M.S., Vanderbilt University -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA – 30 May 2024: Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, will be hosting an investor webcast today on Thursday, 30 May 2024 in Australia / Wednesday, 29 May 2024 in the United States. Details are listed below and registration is required.
The call will feature Alterity’s CEO Dr. David Stamler who will discuss the bioMUSE Natural History Study results and their impact on the endpoints for the ATH434-202 Phase 2 clinical trial. Joining the call will be key opinion leader Daniel Claassen, M.D., M.S., Professor of Neurology, Vanderbilt University Medical Center. Dr. Claassen is the Principal Investigator of bioMUSE and the Coordinating Investigator of the ATH434-201 Phase 2 clinical trial.
Webcast details
AUSTRALIA PARTICIPANTS:
| Date: | Thursday, 30 May 2024 |
| Time: | 10:00 a.m. AEST (Sydney/Melbourne) |
UNITED STATES PARTICIPANTS:
| Date: | Wednesday, 29 May 2024 | |
| Time: | 5:00 p.m. Pacific Time | |
| 8:00 p.m. Eastern Time |
Register for the Zoom webcast: https://bit.ly/3yFXHMp
Registration is required and dial in details will be sent directly upon registration.
Investors will have the opportunity to ask questions during the call, at the conclusion of the presentation. Investors are also able to submit questions in advance to Hannah Howlett at we- aualteritytherapeutics@we-worldwide.com
About Alterity Therapeutics Limited
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company’s lead asset, ATH434, has the potential to treat various Parkinsonian disorders and is currently being evaluated in two Phase 2 clinical trials in Multiple System Atrophy. Alterity also has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company’s website at www.alteritytherapeutics.com.
Authorisation & Additional information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
Investor and Media Contacts:
Australia
Hannah Howlett
we-aualteritytherapeutics@we-worldwide.com
+61 450 648 064
U.S.
Remy Bernarda
remy.bernarda@iradvisory.com
+1 (415) 203-6386
David Stamler, MD CEO Webinar – bioMUSE Data Release May 29/30, 2024
Forward Looking Statements 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 2023 Form 20 - F, filed with US Securities and Exchange Commission, in particular Item 3 , Section D, titled “Risk Factors . ’’ 2
Objectives of Today’s Webinar • Review new data from bioMUSE natural history study • Discuss relevance of bioMUSE for ATH434 - 202 study • Expert perspective on Multiple System Atrophy (MSA) • Review timing of Phase 2 data 3
PARTNER Promising Portfolio in Neurodegenerative Diseases PARTNER PHASE ASSET PARTNER / COLLABORATOR PHASE 2 PHASE 1 NATURAL HISTORY PRE - CLINICAL DISCOVERY INDICATION PROGRAM mplete Enrollment Co Multiple System Atrophy Early Stage ATH434 - 201 mplete Enrollment Co Multiple System Atrophy Advanced ATH434 - 202 Parkinson’s Disease ATH434 Multiple System Atrophy Natural History Study bioMUSE Neurodegenerative Diseases Drug Discovery 4
Multiple System Atrophy (MSA): Rare, Highly Debilitating and Rapidly Progressive Neurodegenerative Disease • Parkinsonian disorder with no approved treatment • Orphan disease • Disease characteristics • Motor: Parkinsonism, uncoordinated movements, balance problems/falls • Autonomic dysfunction: blood pressure maintenance, bladder control, bowel function • Brain atrophy in multiple regions • Median survival 7.5 years after symptom onset 0 6 9 3 Years Premotor MSA Clinically Established MSA Death can occur 5 Sources: Fanciulli, Wenning. NEJM 2015 | Wenning, et al . Mov Disorders 2022 | Coon, et al. Brain 2015 | Claassen,et al. Mov Disorders 2021 Clinically Probable MSA
Sources: Kaindlstorfer, J. Alzheimers Dis. 2018; Bengoa - Vergniory N, Acta Neuropathol. 2017 6 Iron is Critical in Disease Pathogenesis Iron and α - Synuclein are important contributors to MSA pathology • Adverse impact of excess labile iron • Leads to α - synuclein aggregation ڮ neuronal dysfunction • Oxidative stress with intracellular damage • Cell death • Hallmark of MSA pathology • α - synuclein aggregates in neurons and glial (support) cells • Neuron and glial cells loss • Atrophy in multiple brain regions Oxidative Stress Neuroinflammation Iron Imbalance Stored iron Labile iron • Needed for cellular metabolism • Generates damaging free radicals
7 Clinical Observations Preserve Neurons/ Function α - Synuclein Midbrain* Iron Model Target Disease Improved motor performance á â â Mouse MPTP Parkinson’s disease Improved motor performance á â â Mouse A53T Parkinson’s disease Improved motor performance á â â Mouse tau knockout Parkinson’s disease Improved motor performance á â â PLP - α - syn MSA Improved motor performance á â â PLP - α - syn MSA Improved motor performance á n/a â Monkey MPTP Parkinson’s disease Accumulated Evidence of ATH434 Efficacy Source: Finkelstein, Acta Neuropath Comm 2017; Beauchamp et al, Neurotherapeutics 2022; Heras - Garvin et al, Mov Disord. 2021; Finkelstein et al, J Park Dis 2022; Bradbury et al, Am Acad. Neurol 2024 ATH434 consistently improved motor performance across diverse animal models of disease by redistributing iron and preserving neurons * includes s. nigra
Approach: Address Underlying Pathology of Disease Potential Disease Modifying Therapy for MSA Redistribute excess labile iron in the CNS Reduce α - synuclein aggregation and Oxidative stress Rescue brain cells/ neuronal function 8
Multiple System Atrophy Clinical Development Program 9
Clinical Studies in MSA ATH434 - 202 – Phase 2 ATH434 - 201 – Phase 2 bioMUSE – Natural History Study Single arm, open - label Randomized, double - blind Observational Design Efficacy and safety of ATH434 Efficacy and safety of ATH434 Characterize early - stage MSA Objectives Advanced MSA Early - stage MSA Early - stage MSA Population N = 10 N = 77 N = 21 Sample Size 12 months treatment 12 months treatment 12 months Observation/Treatment Same as bioMUSE Same as bioMUSE Iron, volume, glial pathology Brain MRI Biomarkers Same as bioMUSE Same as bioMUSE NFL, Aggregated α - synuclein Fluid Biomarkers ─ Same as bioMUSE Wearable movement sensors Other Biomarkers Same as bioMUSE Same as bioMUSE Motor exam, autonomic function, activities of daily living, global measures Clinical Measures 10
11 Sources: Presentations/Posters on file, including Claassen et al, MDS Conference 2021 and 2022. BioMUSE Natural History Study Learnings to Date Advanced MRI methods Optimize Patient Selection Precision Endpoint Assessment 100 1000 10000 100000 1000000 Fluorescence PD MSA Time → Differentiate MSA from PD α - synuclein in CSF New MRI Template Improve precision of structural MRI Novel strategies for measuring brain iron in individual regions Iron distribution in MSA 0 Iron content [ppb] 300 Identify “iron signature” of early MSA Revised selection criteria in ATH434 - 201 and ATH434 - 202 protocols to exclude PD patients State of the art methods enabled precise measurements of iron and brain volume with MRI
25 Adults 30 to 78 yo with MSA assessed for eligibility 4 Excluded • 2 Unable to complete procedures • 1 Exclusionary MRI findings • 1 Unreliable 21 Enrolled 17 Early Stage (Clinically Probable MSA) 4 Advanced (Clinically Established MSA) Followed for 12 months 10 MSA 5 Parkinson 2 Undefined Participant Flow in bioMUSE Single timepoint Ô Refine biomarker endpoints Source 13
Status How Assessed Biomarker 12 mo data reported today MRI/QSM Iron content 12 mo data reported today MRI/T1w Volumes Preliminary data reported in 2023 MR spectroscopy N - acetylaspartate/ myoinositol In process MRI Neuromelanin BioMUSE Brain Imaging Biomarkers Assessed in MSA Regions 14
15 • Based on routine (e.g., structure/volume) and specialized MRI techniques • Bioengineer evaluated MRI processing algorithms to optimize measurement 1,2 • Method selected which is reproducible and minimizes artifacts between MRIs 1 Marques. QSM reconstruction challenge 2.0. Magn Reson Med . 2021 2 QSM consensus Committee. Recommended implementation of QSM. Magn Reson Med . 2024 MRI Methods to Measure Brain Iron We have established standardized methods to analyze brain iron in bioMUSE and will apply same methods in the Phase 2 studies
6M 12M - 0.02 0.00 0.02 0.04 0.06 Substantia Nigra Change from BL ** 6M 12M - 0.02 - 0.01 0.00 0.01 0.02 Putamen Change from BL 6M 12M - 0.02 - 0.01 0.00 0.01 0.02 Pallidum Change from BL 6M 12M - 0.02 - 0.01 0.00 0.01 0.02 Cerebellar Dentate Nucleus Change from BL Statistically significant increase in iron over 12 months in the substantia nigra ** p < 0.01 BioMUSE: Change in Iron Content by MRI Brain Regions affected in MSA 16
• Multiple System Atrophy = atrophy (shrinking) of brain structures • Routine MRI are analyzed in 2 dimensions (2D) • We are interested in measuring brain volume (in 3D) • Needed a new method to measure volumes in MSA affected areas Method Development • Neuroradiologist manually traced 2D images for each bioMUSE subject • 2D MRI ڮ 3D rendering ڮ measure volume • Human time: 10 hours per MRI • Bioengineer employed Machine Learning/AI to teach computer to do the same • Computer time: 5 minutes per MRI Allows measurement of brain volumes with precision in all Phase 2 participants 3D rendering 2D image Optimizing MRI Methods to Measure Brain Volumes 17
18 Statistically significant decreases in brain volume observed in all regions at 12 months and in 3 of 4 regions at 6 months 6M 12M - 6 - 3 0 Cerebellum % Change from BL ** ** 6M 12M - 8 - 6 - 4 - 2 0 Brainstem % Change from BL ** ** 6M 12M - 12 - 9 - 6 - 3 0 Putamen % Change from BL ** 6M 12M - 12 - 9 - 6 - 3 0 Pallidum % Change from BL * ** * p < 0.05 ** p < 0.01 BioMUSE: Change in Brain Volume by MRI Brain Regions affected in MSA
6M 12M - 6 - 4 - 2 0 MSA % Change from BL ** ** New Imaging Biomarker MSA Volume Index 6M 12M - 6 - 4 - 2 0 PD % Change from BL Combine volume of these 4 regions affected in MSA BioMUSE: Change in Brain Volume by MRI Brain Atrophy in MSA 19
• Observational Study in MSA • Goals: improve patient selection and choose endpoints for Phase 2 • Iron content: Significant increase in iron observed at 12 months in one brain region (s. nigra) • Brain volume: Significant decrease in volume observed over 12 months in all four MSA regions ❖ Novel Imaging Biomarker: MSA Volume Index BioMUSE Summary 20
Implications of bioMUSE Data on ATH434 - 202 Study 21
We target endpoints with the largest change over the planned study period to more readily demonstrate a drug effect Endpoint Selection in Treatment Trials Biomarker Endpoint Selection Relevant to Tracks with disease Preclinical indication underlying disease progression of drug effect 22
ATH434 - 202 Key Study Endpoints Change in MSA Volume Index by MRI at 12 months • Based on the significant decrease in volume in relevant brain regions in MSA Primary Endpoint Change in Iron content in the substantia nigra by MRI at 12 months • Based on the significant increase in iron in relevant brain region in MSA Secondary Endpoint 23
ATH434 - 201 Phase 2 Trial Study Complete Feb 25 Jan 24 Feb 24 Mar 24 Apr 24 May 24 Jun 24 Jul 24 Aug 24 Sep 24 Oct 24 Nov 24 Dec 24 Jan 25 Key Milestones ATH434 MoA data BioMUSE Data ATH434 - 202 Preliminary 6 mo Data Scientific Meeting Presentations ATH434 - 201 Phase 2 Trial Topline Data 24
Professor of Neurology Vanderbilt University Medical Center Research focus: Neuroimaging/Therapeutics Principal Investigator of bioMUSE Coordinating Investigator of the ATH434 - 201 Clinical Trial Daniel Claassen, M.D., M.S. 25
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