Alterity Therapeutics Ltd. · ASX:ATH
Alterity Therapeutics — Conference Presentation (March 2021)
24 slides from Alterity Therapeutics's conference presentation, Mar 10, 2021
Slide 1: Alterity Therapeutics
Alterity Therapeutics (NASDAQ:ATHE, ASX:ATH) David Stamler, MD CEO March 2021
Slide 2: Forward Looking Statements
Forward Looking Statements fly. Alterity THERAPEUTICS 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
Slide 3: Our Purpose
Our Purpose o{lj- Alterity THERAPEUTICS 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.
Slide 4: H1 Progress and Financials
H1 Progress and Financials off. Alterity * 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
Slide 5: Review of Progress
Review of Progress 7th International Congress for MSA, coal THE MICHAEL ION :HAEL | FOX FOUNDA "FOR PARKINSON'S RESEARCH Cardiovascular data which strengthens the safety profile of ATH434 VANDERBILT WJ UNIVERSITY MEDICAL CENTER Michael J. Fox Foundation grant for ~US$500K for Parkinson’s disease MED
Slide 6: Parkinsonian Disorders — A Significant Unmet Need
Parkinsonian Disorders — A Significant Unmet Need fly. Alterity THERAPEUTICS » Parkinsonism is a syndrome of motor symptoms that includes slowness of movement, stiffness and tremor » Major source of disability » Parkinsonian disorders also include atypical variants such as Multiple system atrophy (I
Slide 7: Our Target is Alpha-Synuclein
Our Target is Alpha-Synuclein fly. Alterity A Major Focus of Treating Parkinsonian Disorders mnerapeencs N k le Pal # * Biogen AstraZeneca? = THE MICHAEL j. FOX FOUNDATION FOR PARKINSON’S RESEARCH Structure of a-synuclein * a-synuclein is an intracellular protein, abundantly expressed in nerve termi
Slide 8: Increased Brain Iron in Synuclein-related Diseases
Increased Brain Iron in Synuclein-related Diseases fly. Alterity Parkinson's disease Multiple System Atrophy Ol Patients {)) Healthy Substantia nigra (T) Cerebral cortex Substantia nigra (pc) Caudate nucleus 0 10000 20000 30000 Putamen (M nmol iron/g of human brain Deiior of a Bisin.taot —= (m) HE)
Slide 9: ATH434 Reduces Alpha-Synuclein-related Neuropathology
ATH434 Reduces Alpha-Synuclein-related Neuropathology Preserves Neurons and Improves Function in Parkinson’s and MSA Animal Models Parkinson’s disease model MSA model { a-Synuclein 3 a2 g € * 5 a * b1, * 0 UL VEH = ATH434 0.8. | £ Bos 3 0.4: 2 rs B02 & fd 0.0: Vehicle ATHA34 ATH434 Dose: 30 mg/kg Fi
Slide 10: 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 ; “14 Ny , ow 7 $ S. nigra AR2* MRI sequence (s*) * m DFP @ PBO Change in UPDRS motor score Motor Symptom Severity 3.0 4 2.0 4 -2.0 4
Slide 11: Excellent Progress with Lead Drug Candidate ATH434
Excellent Progress with Lead Drug Candidate ATH434 fly. Alterity THERAPEUTICS « 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 to
Slide 12: Clinical Development
fly. Alterity THERAPEUTICS Clinical Development 12
Slide 13: Phase 1 Completed: Clinical Pharmacokinetics of ATH434
Phase 1 Completed Clinical Pharmacokinetics of ATH434 ATH434 in plasma (ng/mL) Plasma Profile after Single Dose Administration 10000 1000 = ° So Hours post dose Rapid absorption after oral administration Dose dependent PK after single doses up to 600 mg and multiple doses up to 250 mg bid Mean elimi
Slide 14: Adverse Event Summary
Adverse Event Summary o{lj- Alterity THERAPEUTICS FEES 100 mg 300 mg 600 mg Soe (Nes) (N=6) (Ne6) Patients with = 1 AE 3 (38%) (e) 1 (17%) 1 (17%) Patients with AEs leading to Withdrawal ° i iy : 2 Patients with Serious AEs (e) 250 mg BID 2 PrwAaS ~ mg BID 200 mg BID 250 os 1=}10) Patients with = 1
Slide 15: Cardiovascular Data from 7th International MSA Conference
Cardiovascular Data from 7‘ International MSA Conference fly. Alterity ATH434 had no significant effect on Blood pressure or QT interval THERAPEUTICS Mean (SD) Change in Orthostatic SBP Mean (SD) Change in Orthostatic DBP. 2 20 2 20 S 3 Placebo 2 e = 100 mg bid 4 (10 5 10 ; 3 3 = 200 mg bid Steady s
Slide 16: Excellent Safety Profile
Excellent Safety Profile Bebe Alterity No clinically significant AEs Similar AE profile for adults and older adults (2 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 la
Slide 17: bioMUSE Natural History Study
bioMUSE Natural History Study oflj. Alterity THERAPEUTICS * 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 disea
Slide 18: Phase 2 Study Design
Phase 2 Study Design o{lj- Alterity THERAPEUTICS 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 s 3 years Sample size: 60 Tre
Slide 19: Clinical Development Timeline
Clinical Development Timeline tly. Alterity ATH434 in Multiple System Atrophy Natural History Study in early MSA Phase 2 Optimization 4 . 4 Phase 2 Study in early MSA i THERAPEUTICS 4 4 19
Slide 20: Commercial
fly. Alterity THERAPEUTICS Commercial 20
Slide 21: Commercial Opportunity — Multiple System Atrophy
Commercial Opportunity — Multiple System Atrophy Independent Analysis 'D. 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 oy underlying disease and not just the entrants targeti
Slide 22: Deal Landscape
Deal Landscape fly. Alterity THERAPEUTICS Biogen. Biogen )ALECTOR Sangame abbvie JENALI <p rothena Target / Therapeutic Neuro including F . AD &PD AD Neuro including PD atta AD, PD AD, PD Development phase Preclinical Phase 1 Phase 1 Preclinical Preclinical Royalties High-single to low Sub-teens to
Slide 23: Investment Summary
Investment Summary fly. Alterity THERAPEUTICS Y Targeting Orphan disease with no approved treatments * ATH434 has potential U.S. peak sales up to US$ 725 million Y Development team with proven track record at FDA v Lead drug candidate ATH434 * Completed Phase 1 with excellent safety profile ¢ Achiev
Slide 24: Contact
{fo Alterity THERAPEUTICS Contact: info@alteritytherapeutics.com www.alteritytherapeutics.com
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