Leonabio, Inc. · NASDAQ:LONA
Leonabio — Athira Pharma — Investor Presentation (January 2021)
31 slides from Leonabio's investor presentation, Jan 11, 2021
Slide 1: Restoring Lives by Advancing Bold Therapies
Restoring Lives by Advancing Bold Therapies Corporate Presentation, January 2021 © Athira Pharma, Inc. All Rights Reserved. Exhibit 99.1
Slide 2: Forward-looking statements
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 available to our m
Slide 3: Overview of Athira Pharma
Overview of Athira Pharma Pipeline focused on regeneration of neuronal damage in CNS and peripheral diseases to restore function ATH-1017 LEAD INDICATION: Alzheimer’s disease POTENTIAL FOLLOW-ON INDICATIONS: Parkinson’s dementia, ALS, MS, neuropathy, and neuropsychiatric (additional compounds in dev
Slide 4: ATH Compounds Have Therapeutic Potential in a Broad Range of Clinical Applications
Alzheimer’s disease (AD) Parkinson’s disease Dementia (PDD) Multiple Sclerosis (MS) Amyotrophic Lateral Sclerosis (ALS) ATH Compounds Have Therapeutic Potential in a Broad Range of Clinical Applications 4 Neuropathy CNS Degenerative Disorders Peripheral Disorders Neuropsychiatric Indications Depress
Slide 5: Current Development Stage of ATH Compounds and Discovery Research Programs
5 Current Development Stage of ATH Compounds and Discovery Research Programs to Improve Neuronal Health (1) RoA: route of administration; SC: subcutaneous; PO: oral. (2) ATH-1017 for AD is moving from Phase 1b to a Phase 2/3 clinical trial that may provide pivotal data in support of registration bas
Slide 6: Athira's Target, HGF/MET, is a Vital Neuronal Growth Factor
*Historically termed Vital neurotrophic factor system Critical to neuron function, learning, and memory Athira’s Target, HGF/MET, is a Vital Neuronal Growth Factor that Promotes Neuronal Health and Regeneration Alleviation of Aβ-induced cognitive impairment Prevention of onset of Parkinson’s disease
Slide 7: Promoting the HGF/MET Neurotrophic System Has Multiple Downstream Beneficial Effects
Promoting the HGF/MET Neurotrophic System has been Shown to have Multiple Downstream Beneficial Effects 7 NMDA Receptor Acute and Sustained Effects on Synaptic and Network Function Fast-acting positive modulator Protective and regenerative Procognitive (Symptomatic) EEG biomarker ATH Platfor
Slide 8: Lead Program: ATH-1017 – Dementia
Lead Program: ATH-1017 – Dementia 8
Slide 9: ATH-1017 Program Summary
ATH-1017 Program Summary 9 In Phase 1a/b trial ATH-1017 was generally well-tolerated with no serious adverse events (SAEs) Data showing ERP P300, a functional measure that is highly correlated with cognition, improved with treatment (p<0.05) Completed nonclinical GLP long term toxicology and safety
Slide 10: Clinical Development Plan Includes Predictive Measures of Cognitive Improvement
10 Clinical Development Plan Includes Predictive Measures of Cognitive Improvement and Translatable Tools to Guide Dose Selection Pathological changes in P300 latency correlate with cognitive impairment EEG measures electrical activity from firing neurons in the brain QUANTITATIVE EEG (qEEG) EVENT R
Slide 11: P300 Latency as a Functional Measure of Working Memory Processing Speed
P300 Latency as a Functional Measure of Working Memory Processing Speed that Highly Correlates with Cognition 11 Pathological changes in P300 latency correlate with cognitive impairment Recording brain activity while a subject is presented with a task reveals neural activity related to cognitive pro
Slide 12: Changes in P300 Latency Correlate with Cognitive Outcomes in Approved Therapies
12 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 in AD subjects Donepezil and Rivastigmine Improvement in cognition (ADAS-cog ) is correlated with reduc
Slide 13: ATH-1017 Treatment Improved P300 Latency in AD Subjects
ERP OBSERVATIONS ERP analysis to date suggests treatment effects on P300 latency 13 Decreased latency on pre-dose recordings (arrows) taken 24 hours after the last dose on the previous day, indicates sustained improvement Phase 1b – AD Subjects ATH-1017 – 40 mg (SC, OD, 8 days, n=7) Gradual decrease
Slide 14: P300 Latency: AD Subject ATH-1017 Treated and Placebo
P300 Latency: AD Subject ATH-1017 Treated and AD Subject Placebo ATH-1017 Treatment Improved P300 Latency in AD Subjects-CTAD 2019 14 Phase 1b – AD Subjects Group averages of AD subjects receiving ATH-1017 (n=7) demonstrate decreased P300 over time Significant change from baseline observed on Day 8
Slide 15: Every AD subject receiving ATH-1017 had improvement in P300 latency
15 Every AD subject receiving ATH-1017 had a level of improvement in P300 latency AD patients receiving placebo had no consistent response from baseline to end of study Phase 1b – AD Subjects P300 Latency: AD Subject ATH-1017 Treated and AD Subject Placebo Note: P300 data from FZ, CZ, and PZ electro
Slide 16: Changes in P300 Latency Have Been Predictive of Changes in Cognition
16 Previously published results support the correlation of P300 latency and cognition in AD subjects Note: Results from donepezil and rivastigmine adapted from Thomas et al., 2001; results from memantine adapted from Sallach et al., 2011; and results from scopolamine adapted from Potter et al., 2000
Slide 17: ATH-1017 Phase 2/3 Trial for AD Initiated in Sep-2020
17 ATH-1017 Phase 2/3 Trial for AD Initiated in Sep-2020 Trial may provide pivotal evidence to support product registration LIFT-AD: N=240-300 mild-to-moderate AD dementia subjects (55-85 years; CDR 1 and 2; MMSE 14-24 incl.) Potential pivotal study design If both key secondaries are positive If key
Slide 18: ATH-1017 Phase 2 Trial for AD Initiated in Nov-2020
18 ATH-1017 Phase 2 Trial for AD Initiated in Nov-2020 Trial can help strategic decisions around additional clinical trials
Slide 19: ATH-1019/20 – Neuropsychiatric Indications
19 ATH-1019/20 – Neuropsychiatric Indications
Slide 20: ATH Small Molecules Enhance HGF/MET Activity
ATH small molecules promote HGF/MET, which has the potential to promote multiple beneficial effects and improve neuronal function Activity can apply to promote nerve cell health in several indications Improvement of neuronal health and function may be relevant to several neuropsychiatric indications
Slide 21: Rationale for Targeting HGF/MET for Neuropsychiatric Indications
Rationale for Targeting HGF/MET for Neuropsychiatric Indications 21 Preclinical studies demonstrate enhancing HGF/MET activity has anti-depressant and anxiolytic effects in rodents Isogawa et al., 2005; Wakatsuki et al., 2007 Clinical trials show an association between reduced HGF/MET expression lev
Slide 22: Novel ATH Potential Candidates ATH-1019 and ATH-1020
Novel ATH Potential Candidates 22 ATH-1019 and ATH-1020 are in development as potential candidates for neuropsychiatric indications, including depression, anxiety, and potentially schizophrenia Activate HGF/MET Orally bioavailable Distribute to the brain Neuroactive in animal models Procognitive eff
Slide 23: ATH-1019 and ATH-1020 Demonstrated Anti-Depressant Effects in Animal Models
Treatment with ATH-1019 and ATH-1020 led to significant improvement from depressive-like behaviors in the rat forced swim model of depression ATH-1019 and ATH-1020 were delivered orally ATH-1019 and ATH-1020 Demonstrated Anti-Depressant Effects in Animal Models *p<0.05 compared to Vehicle * * * *
Slide 24: ATH-1019/20 Potential Product Profile
ATH-1019/20 Potential Product Profile 24 INDICATIONS Neuropsychiatric disorders MOA Small molecule agonist of HGF/MET DELIVERY MODE Oral REGIMEN Targeting once per day
Slide 25: Current Development Stage of ATH Compounds and Discovery Research Programs
25 Current Development Stage of ATH Compounds and Discovery Research Programs to Improve Neuronal Health (1) RoA: route of administration; SC: subcutaneous; PO: oral. (2) ATH-1017 for AD is moving from Phase 1b to a Phase 2/3 clinical trial that may provide pivotal data in support of registration ba
Slide 26: Thank you!
© Athira Pharma, Inc. All Rights Reserved. Thank you!
Slide 27: Appendix
Appendix >athiga
Slide 28: ATH-1017 Phase 1a/b Trial Overview
Randomized, placebo-controlled, double-blinded, single-ascending dose (Part A) and multiple-ascending dose (Part B) (SC, OD) Safety, pharmacokinetics, and pharmacodynamics measures, i.e., qEEG/ERP 28 Phase 1 – Active IND SAD MAD ATH-1017 Phase 1a/b Trial Overview
Slide 29: EEG as a Translatable Measure of Functional Recovery in the Brain
29 EEG as a Translatable Measure of Functional Recovery in the Brain 29 Noninvasive EEG recordings reflect brain activity and function EEG captures electrical activity in the brain and displays these electrical impulses as waves Gamma waves are the faster, higher frequency waves associated with lear
Slide 30: ATH-1017 Increased High Frequency Gamma Power Across All Treated Cohorts
ATH-1017: single dose EEG, 1-hour post-dose Placebo, n=12 Low doses pooled (2 & 6 mg), n=12 Mid doses pooled (20 & 40 mg), n=12 High doses pooled (60 & 90 mg), n=11 Gamma at 20, 40, 60, 90 mg (>50%) Dose-dependent increase Statistically significant at 90 mg (n=6) Indicate CNS penetration and targe
Slide 31: ATH-1019 Demonstrated Procognitive Activity in Animal Models
ATH-1019 Demonstrated Procognitive Activity in Animal Models 31 Oral delivery of ATH-1019 significantly improved performance of rats in the Morris water maze test of spatial memory Scopolamine (SCO) treatment blocks spatial memory formation ATH-1019 treatment reversed the effects of scopolamine, ind