Alterity Therapeutics Ltd. · ASX:ATH
Alterity Therapeutics — HC Wainwright Global Life Science Conference 2021
26 slides from Alterity Therapeutics's conference presentation, Mar 10, 2021
Slide 1: Alterity CEO presents to US investors at the HC Wainwright Global Life Science Conference
Alterity CEO Dr David Stamler presents to US investors at the HC Wainwright Global Life Science Conference MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA – 10th March 2021: Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”) CEO Dr David Stamler is presenting to US investors th
Slide 2: Contact and About Alterity Therapeutics and ATH434
Contact: Investor Relations Greig King, WE Communications E: WE-AUAlterity@we-worldwide.com / info@alteritytherapeutics.com About Alterity Therapeutics Limited and ATH434 Alterity’s lead candidate, ATH434 (formerly PBT434), is the first of a new generation of small molecules designed to inhibit the
Slide 3: Alterity Therapeutics
Alterity Therapeutics (NASDAQ:ATHE, ASX:ATH) David Stamler, MD CEO March 2021 CONFIDENTIAL 1
Slide 4: Forward Looking Statements
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, objec
Slide 5: Our Purpose
Our Purpose We exist to create an alternate future for people living with neurodegenerative diseases. An alternate, healthier life. We’re here to disrupt the trajectory for people with these diseases. 3
Slide 6: H1 Progress and Financials
H1 Progress and Financials • Cash balance of $35M • Cash burn in line with expectations of $7.3M • Significant progress: • bioMUSE Natural History study for MSA patients progressing well • New patent to support new compound development across neurodegenerative diseases • Michael J Fox grant of US$50
Slide 7: Review of Progress
Review of Progress Cardiovascular data which strengthens Commence enrolling Multiple System 7th International the safety profile of ATH434 Congress for MSA Atrophy patients in bioMUSE Study Michael J. Fox Foundation grant for ATH434 reduces α-synuclein pathology, ~US$500K for Parkinson’s disease pre
Slide 8: Parkinsonian Disorders: A Significant Unmet Need
Parkinsonian Disorders A Significant Unmet Need Parkinsonism is a syndrome of motor symptoms that includes slowness of movement, stiffness and tremor Major source of disability Parkinsonian disorders also include MSA atypical variants such as Multiple system atrophy (MSA) and Parkinson’s Pro
Slide 9: Our Target is Alpha-Synuclein
Our Target is Alpha-Synuclein A Major Focus of Treating Parkinsonian Disorders Structure of α-synuclein • α-synuclein is an intracellular protein, abundantly “We conclude that alpha-synuclein remains one of the expressed in nerve terminals most compelling therapeutic targets for Parkinson's • Critic
Slide 10: Increased Brain Iron in Synuclein-related Diseases
Increased Brain Iron in Synuclein-related Diseases Parkinson’s disease Multiple System Atrophy n=7 * Patients Substantia nigra (T) n=9 Healthy n=8 n=8 * Cerebral cortex Substantia nigra (pc) n = 11 n=3 n = 13 n=8 * Cerebellum Caudate nucleus n = 24 n = 10 0 10000 20000 30000 n=8 * Putamen (M) n = 10
Slide 11: ATH434 Reduces Alpha-Synuclein-related Neuropathology
ATH434 Reduces Alpha-Synuclein-related Neuropathology Preserves Neurons and Improves Function in Parkinson’s and MSA Animal Models ↓ α-Synuclein Preserves nigral neurons Improves motor function 6000 *** *** Time (secs) Parkinson’s * ** 3000 disease model 0 0 1 3 10 30 80 C ATH434 (mg/kg) Total N SNp
Slide 12: Strategy Supported by Proof of Concept in Parkinson's
Strategy Supported by Proof of Concept in Parkinson’s 6 month placebo-controlled data from study of Deferiprone – a drug for treating iron overload Brain Iron by MRI Motor Symptom Severity Deferiprone • Binds iron with very high affinity • Boxed Warning for hematological toxicity Worsening Ligand Kd
Slide 13: Excellent Progress with Lead Drug Candidate ATH434
Excellent Progress with Lead Drug Candidate ATH434 Robust efficacy in animal Completed Phase 1 Qualitative research with models of disease Orally bioavailable, brain penetrant U.S. neurologists completed Evidence of neuroprotection with ATH434: safe and well tolerated 15 movement disor
Slide 14: Clinical Development
off. Alterity Clinical Development 12
Slide 15: Clinical Pharmacokinetics of ATH434
Phase 1 Completed Clinical Pharmacokinetics of ATH434 Plasma Profile after Single Dose Administration CSF and Plasma Concentrations 1-2 hrs after dosing Plasma (unbound, ng/mL) ATH434 in plasma (ng/mL) • CSF concentrations at well-tolerated doses • Rapid absorption after oral administration exceeded
Slide 16: Adverse Event Summary
Adverse Event Summary Placebo 50 mg 100 mg 300 mg 600 mg Single Ascending Doses (N=8) (N=6) (N=6) (N=6) (N=6) Patients with ≥ 1 AE 3 (38%) 0 0 1 (17%) 1 (17%) Patients with AEs leading to 0 0 0 0 0 Withdrawal Patients with Serious AEs 0 0 0 0 0 250 mg BID Placebo 100 mg BID 200 mg BID 250 mg BID Mul
Slide 17: Cardiovascular Data from 7th International MSA Conference
Cardiovascular Data from 7th International MSA Conference ATH434 had no significant effect on Blood pressure or QT interval Baseline-adjusted, placebo-corrected change in QTcF ΔΔQTcF (msec) ΔΔQTcF vs. ATH434 plasma (ΔΔQTcF) = 1.68 ms (90% CI: 0.386 to 3.75 ms), concentration well below the threshold
Slide 18: Excellent Safety Profile
Excellent Safety Profile • No clinically significant AEs • Similar AE profile for adults and older adults (≥ 65 years) • All AEs with ATH434 were mild to moderate in severity • Most common AEs reported in ATH434 subjects was headache • No significant findings observed in vital signs, clinical labora
Slide 19: bioMUSE Natural History Study
bioMUSE Natural History Study • Design: Observational (no treatment) • Objective: De-risk Phase 2 study • Identify biomarker(s) suitable for endpoint in treatment study • Evaluate the change in biomarkers and clinical manifestations in patients with early MSA to track disease progression • Populatio
Slide 20: Phase 2 Study Design
Phase 2 Study Design • Design: Randomized, double-blind, placebo controlled • Objectives • Assess target engagement and preliminary efficacy of ATH434 • Evaluate safety and tolerability of ATH434 • Population: Early MSA patients with motor symptoms ≤ 3 years • Sample size: 60 • Treatment: 6 months d
Slide 21: Clinical Development Timeline
Clinical Development Timeline ATH434 in Multiple System Atrophy 2021 2022 Natural History Study in early MSA Phase 2 Optimization E.g., Data to inform P2 Phase 2 Study in early MSA 2H ’20 1H ’21 2H ’21 1H ’22 2H ’22 European Conduct First patient Last patient Top line National Feasibility enrolled i
Slide 22: Commercial
ofiys Alterity THERAPEUTICS Commercial 20
Slide 23: Commercial Opportunity – Multiple System Atrophy
Commercial Opportunity – Multiple System Atrophy Independent Analysis STRONG INTENT TO SUBSTANTIAL UNMET NEED PRESCRIBE Severely debilitating, fatal illnesses with Motivated by efficacy in treating the no current treatments are ripe for new underlying disease and not just the entrants targeting what
Slide 24: Deal Landscape
Deal Landscape Target / Therapeutic Neuro including AD & PD AD Neuro including PD area AD, PD AD, PD Development phase Preclinical Phase 1 Phase 1 Preclinical Preclinical Royalties High-single to low Sub-teens to Undisclosed Undisclosed Double-digit (% on net sales) double-digit High single-digit $1
Slide 25: Investment Summary
Investment Summary Targeting Orphan disease with no approved treatments • ATH434 has potential U.S. peak sales up to US$ 725 million Development team with proven track record at FDA Lead drug candidate ATH434 • Completed Phase 1 with excellent safety profile • Achieved CSF concentrations assoc
Slide 26: Contact
Contact: info@alteritytherapeutics.com www.alteritytherapeutics.com
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