Leonabio, Inc. · NASDAQ:LONA
Leonabio — Athira Pharma — Investor Presentation (April 2022)
32 slides from Leonabio's investor presentation, Apr 11, 2022
Slide 1: Corporate Presentation April 2022
Corporate Presentation APRIL 2022 ADVANCING NEW THERAPIES FOR NEURONAL HEALTH
Slide 2: Disclaimer
Disclaimer This presentation and the accompanying oral commentary contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on our management’s beliefs and assumptions and on information currently availab
Slide 3: Our Mission
OUR MISSION To restore lives by advancing bold therapies for neuronal health, thoughtfully and urgently 3
Slide 4: Investment Highlights
Investment Highlights Late-stage program fosgonimeton (ATH-1017) designed to Our novel small enhance Hepatocyte Growth Factor (HGF) and its receptor, MET molecule compounds • Well established HGF/MET pathway is critical to normal brain function and is are designed to act on compromised in Alzheimer’
Slide 5: Therapeutic Potential Across a Broad Range of Clinical Applications
Therapeutic Potential Across a Broad Range of Clinical Applications PRECLINICAL CLINICAL Discovery and Status and Anticipated Program Indication Phase 1 Phase 2 Phase 3 Development Upcoming Milestones LIFT-AD enrollment Open-Label Phase 3 Clinical Trial complete 3Q22; Extension topline data 1H23 Alz
Slide 6: Lead Program: Fosgonimeton Alzheimer's Disease (AD)
Lead Program: FOSGONIMETON Alzheimer’s Disease (AD)
Slide 7: Alzheimer's Disease Pathology
Alzheimer’s Disease Pathology MULTIFACTORIAL AND COMPLEX PATHOLOGIES ULTIMATELY LEAD TO NEURODEGENERATION Protein misfolding Neurodegeneration Nervous System Oxidative stress Synaptic Loss Synaptic dysfunction Loss of network Reduced cerebral Vasculature connectivity blood flow Alzheimer’s disease S
Slide 8: Synapse Loss in Alzheimer's Disease
Synapse Loss in Alzheimer’s Disease 1 • In AD, 25-36% of synapses are lost • Synapse loss is an early event in disease progression that impacts several brain regions, 2 including the hippocampus and frontal cortex, important for learning and memory MILD-TO-MODERATE ALZHEIMER’S DISEASE Non-demented E
Slide 9: HGF/MET System is Critical to Normal Brain Function
HGF/MET System is Critical to Normal Brain Function MET is one of the most stably expressed NEURONAL MET EXPRESSION IN ALZHEIMER’S DISEASE genes in the adult human brain Stable MET expression is a signature 12 1 of the healthy adult brain 10 Non-AD 9 Suggests that dysregulation of HGF/MET *** 8 AD c
Slide 10: Fosgonimeton: A Positive Modulator of the HGF/MET Neurotrophic System
Fosgonimeton (ATH-1017): A Positive Modulator of the HGF/MET Neurotrophic System MULTIMODAL, PROTECTIVE AND REGENERATIVE Fosgonimeton: • Small molecule prodrug that is rapidly converted to an active metabolite (Fosgo-AM) in plasma • Crosses the blood-brain barrier • Positively modulates HGF/MET • Ad
Slide 11: Fosgo-AM Enhances Synaptogenesis and Neurite Outgrowth
Fosgo-AM Enhances Synaptogenesis and Neurite Outgrowth PRECLINICAL DATA IN PRIMARY RAT HIPPOCAMPAL NEURONS HGF + 1 nM Fosgo-AM Vehicle Synaptic count (number of synapses) and synaptic strength (relative abundance of presynaptic vesicles per synapse) were both significantly enhanced with fosgo-AM Neu
Slide 12: Clinical Development Strategy Includes Measures Strongly Correlated with Cognition
Clinical Development Strategy Includes Measures Strongly Correlated with Cognition QUANTITATIVE EEG (qEEG) EVENT RELATED POTENTIALS (ERP): P300 Latency Functional measurement for working memory Translational tool from access and executive function rodents to humans Strongly correlated with memory im
Slide 13: Changes in P300 Latency Correlate with Cognitive Outcomes
Changes in P300 Latency Correlate with Cognitive Outcomes with Treatment of Approved Therapies in AD Subjects PREVIOUSLY PUBLISHED RESULTS SUPPORT THE CORRELATION OF P300 LATENCY AND COGNITION Donepezil Rivastigmine Vitamin E 40 390 390 34 390 34 38 380 380 380 33 33 36 370 370 370 32 32 34 360 360
Slide 14: Fosgonimeton Treatment Improved P300 Latency in AD Subjects
Fosgonimeton Treatment Improved P300 Latency in AD Subjects – CTAD 2019 PHASE 1B – AD SUBJECTS N=11 Randomized, Placebo, Fosgonimeton (40 mg) Subcutaneous, Daily, 8 days ERP OBSERVATIONS ERP analysis to-date suggests treatment effects on P300 latency • Gradual decrease in latency over time in the tr
Slide 15: Fosgonimeton Treatment Improved P300 Latency in AD Subjects
Fosgonimeton Treatment Improved P300 Latency in AD Subjects – CTAD 2019 PHASE 1B – AD SUBJECTS P300 LATENCY: ACTIVE v PLACEBO TREATED AD SUBJECTS • Group averages of AD subjects receiving fosgonimeton (N=7) Fosgonimeton demonstrate decreased P300 0 latency over time -10 -20 -30 Significant change fr
Slide 16: Fosgonimeton Treatment Improved P300 Latency in AD Subjects
Fosgonimeton Treatment Improved P300 Latency in AD Subjects – CTAD 2019 PHASE 1B – AD SUBJECTS P300 LATENCY: ACTIVE v PLACEBO TREATED AD SUBJECTS Alzheimer’s Subjects Alzheimer’s Subjects • Every AD subject receiving (Fosgonimeton 40mg) (Placebo) fosgonimeton had a level of improvement in P300 laten
Slide 17: Why Mild-to-Moderate AD Instead of Pre-Dementia?
Why Mild-to-Moderate AD Instead of Pre-Dementia? Medical need: Reduced development risk: Mild-to-moderate AD The point of most accelerated Clinical, syndromal 1,2 5 disease progression diagnosis is possible Currently marketed drugs Increased likelihood of in mild-to-moderate space tangible placebo d
Slide 18: Fosgonimeton Phase 2 Trial (ACT-AD)
Fosgonimeton Phase 2 Trial (ACT-AD) PROOF-OF-CONCEPT TRIAL TO HELP BETTER UNDERSTAND NATURE OF NOVEL INTERVENTION POPULATION TREATMENT DURATION ENDPOINTS AND TIMELINE PRIMARY ENDPOINT 26-week randomized, ACT-AD: N=77 (recruitment complete) double-blind treatment, mild-to-moderate AD dementia subject
Slide 19: Fosgonimeton Phase 3 Trial (LIFT-AD)
Fosgonimeton Phase 3 Trial (LIFT-AD) TRIAL MAY PROVIDE PIVOTAL EVIDENCE TO SUPPORT PRODUCT REGISTRATION POPULATION TREATMENT DURATION ENDPOINTS AND TIMELINE PRIMARY ENDPOINT 26-week randomized, LIFT-AD: Target N=~420 double-blind treatment, • Global Statistical Test (GST) – unbiased mild-to-moderate
Slide 20: Fosgonimeton – Potential First-Line Therapy to Improve Cognition
Fosgonimeton – Potential First-Line Therapy to Improve Cognition Over 100 million 6.2 million treatment eligible 35 million globally by 2050 patients in the US in 2021 based Estimated Alzheimer’s cases 1 worldwide on prevalence data ~900,000 new patients diagnosed annually in 2,3 Growing at 3% per y
Slide 21: Fosgonimeton: PDD and DLB
FOSGONIMETON Parkinson’s Disease Dementia (PDD) and Dementia with Lewy Bodies (DLB)
Slide 22: Phase 2 Trial in PDD and DLB
Phase 2 Trial in PDD and DLB PROOF-OF-CONCEPT TRIAL TO UNDERSTAND THE POTENTIAL OF FOSGONIMETON BEYOND ALZHEIMER’S DISEASE POPULATION TREATMENT DURATION ENDPOINTS AND TIMELINE PRIMARY ENDPOINT N=~75 mild-to-moderate • Global Statistical Test (combining P300 and 26-week randomized, PDD or DLB subject
Slide 23: PDD and DLB – Critical Unmet Need and Significant Opportunity
PDD and DLB – Critical Unmet Need and Significant Opportunity DLB is the third most Nearly 1 million $51.9B common cause of dementia people in the US and more Economic burden of PD than 10 million people accounting for 5-15% of all 4 globally are living with 2,4 overall in the US, as of 2017 dementi
Slide 24: Fosgonimeton Program Summary
Fosgonimeton Program Summary CHANGING THE TREATMENT PARADIGM TO RESTORE NEURONAL HEALTH Significant market potential Based on strong science using novel approach to leverage naturally occurring repair mechanism • Over 2.5 million mild-to-moderate AD patients in the US being treated with therapies wi
Slide 25: ATH-1020 Neuropsychiatric Indications
ATH-1020 Neuropsychiatric Indications
Slide 26: ATH-1020 – Addressing Neuronal Connectivity
ATH-1020 – Addressing Neuronal Connectivity PHYSIOLOGICAL CHANGES IN THE BRAIN AFFECT BEHAVIOR AND EMOTION • Preclinical data demonstrate enhancing HGF/MET activity has 1,2 anti-depressant and anxiolytic effects Our novel approach is • Human clinical trials also show an association between reduced f
Slide 27: Preclinical Studies in Rodent Models of Depression and Schizophrenia
Preclinical Studies Demonstrate Improvement in MMN Response is Rodent Models of Depression and Schizophrenia Translatable from Rodent Models to Human Clinical Schizophrenia Reduction of Depression-related Behaviors in ATH-1020 Rescues the MMN Deficit the Rodent Forced-Swim Test Model Seen in the Rat
Slide 28: Market Opportunity: Neuropsychiatric Indications
Market Opportunity: Neuropsychiatric Indications Global Market Size Depression Schizophrenia 3 for Depression Worldwide population 1 3.8% affected by depression 2021: 2028 Projected: ~20 million people across the globe are affected $11.9B $15.4B 1 with schizophrenia ~280 million people of all ages s
Slide 29: Corporate
Corporate > Athira PHARMA
Slide 30: Athira Management Team with Significant CNS Product Development Experience
Athira Management Team with Significant CNS Product Development and Approval Experience EXECUTIVE LEADERSHIP Mark Litton, PhD Rachel Lenington Hans Moebius, MD, PhD Kevin Church, PhD Glenna Mileson Mark Worthington, JD President and CEO COO CMO Executive Vice President, CFO General Counsel Research
Slide 31: Achievements and Upcoming Milestones
Achievements and Upcoming Milestones RECENT ACHIEVEMENTS ✓ Enrollment completed for Phase 2 ACT-AD trial in Oct LOOKING AHEAD 2021 → Phase 2 ACT-AD Trial: Data readout expected ✓ Strong enrollment to-date in the Phase 3 LIFT-AD trial by end of 2Q22 ✓ Open label extension trial underway for ACT-AD an
Slide 32: Thank You
Thank You > Athira PHARMA