Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (August 2024)
23 slides from Gain Therapeutics's investor presentation, August 2024
Slide 1: Transforming the Treatment of Parkinson's Disease
NASDAQ: GANX TRANSFORMING THE TREATMENT OF PARKINSON’S DISEASE Corporate Presentation August 2024 © 2024 - GAIN THERAPEUTICS Corporate Presentation
Slide 2: Forward-Looking Statements
Forward-Looking Statements Forward-Looking Statements Certain statements set forth in this presentation are forward-looking and reflect the Company’s plans, beliefs, expectations and current views with respect to, among other things, future events and financial performance (collectively referred to
Slide 3: GANX Corporate Highlights
GANX Corporate Highlights Lead Product GT-02287 Advancing into GBA1 Parkinson’s Disease Patients • Restoring motor function and improving cognition – potential to slow or stop PD progression • Safety and tolerability has been established in healthy volunteers with further PK analysis forthcoming • T
Slide 4: Experienced Leadership
Experienced Leadership EXTENSIVE BIOTECH AND PHARMA EXPERIENCE (>$15B TRANSACTIONS) Khalid Islam, PhD Gene Mack, MBA Jonas Hannestad, MD, PhD Joanne Taylor, PhD Terenzio Ignoni, PharmD Founder and Executive Interim – CEO Chief Medical Officer SVP Research SVP Technical Operations Chairman Chief Fina
Slide 5: Strong Board of Directors
Strong Board of Directors (>$30B IN TRANSACTIONS) Khalid Islam, PhD Claude Nicaise, MD Eric I. Richman Gwen Melincoff Hans PeterHasler Dov Goldstein, MD JeffreyRiley Founder and IndependentMember IndependentMember IndependentMember IndependentMember IndependentMember Independent Member Executive Cha
Slide 6: Novel Pipeline Generated from Magellan Platform
Novel Pipeline Generated from Magellan Platform THERAPEUTIC AREA INDICATION TARGET DISCOVERY RESEARCH PRECLINICAL PHASE 1 Parkinson’s Disease GCase Neurodegenerative Diseases Dementia with Lewy Bodies GCase Alzheimer’s Disease Lysosomal Storage Disorders Gaucher Disease GCase GM1 Gangliosidosis GLB1
Slide 7: Lead Clinical Program GT-02287 GBA1 Parkinson's Disease
Lead Clinical Program GT-02287 GBA1 Parkinson’s Disease © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 8: GBA-Parkinson's – A Genetically Defined Subpopulation of Parkinson's Disease
GBA-Parkinson’s – A Genetically Defined Subpopulation of Parkinson’s Disease Parkinson’s Disease • Parkinson’s disease is the second most common neurodegenerative disease1 Up to • Current therapies only treat symptoms and do not prevent neuronal cell death GBA-Parkinson’s Disease • Largest genetic r
Slide 9: Dysfunctional GCase Triggers Disease Cascade
Dysfunctional GCase Triggers Disease Cascade Affecting Multiple Organelle Functions and Leading to Neurodegeneration Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron GBA Gene Mutated GBA Gene Nucleus Nucleus Normal Defective GCase GCase Endoplasmic Endoplasmic Reticulum Reticulum Lysosome Mi
Slide 10: Treatment with GT-02287 restores GCase Function and Neuronal Survival
Treatment with GT-02287 restores GCase Function, which improves Disease Cascade and Neuronal Survival Dopaminergic Neuron with Restored GCase Function Confirmatory Data Generated with GT-02287 ER StressMarker GCase Activity Substrate Depletion Mutated 1 00 8 GBA Gene 80 6 60 40 4 Nucleus 20 0 2 0 De
Slide 11: Animal PoC Studies – GT-02287 Improves Effects of GCase Dysfunction
Animal PoC Studies – GT-02287 Improves Effects of GCase Dysfunction CBE PD Model Aggregated α-syn (SN) Iba-1 (SN) NfL ✱✱✱✱ ✱✱✱✱ ✱ ✱ ✱✱ ✱✱✱✱ ✱✱✱ ✱ ✱ ✱✱ ✱✱✱✱ ✱✱✱✱ ✱ ✱ ✱✱ ✱✱✱✱ 0.025 ✱✱✱✱ ✱ ✱ ✱✱ 200000 (normalised IBA-1/GAPDH) 6 00 0 ✱✱✱✱ ✱✱✱✱ ✱ ✱✱ ✱ Fluorescence intensity Fluorescence Intensity 0.020 1
Slide 12: GT-02287 improves activities of daily living and cognitive performance in animal model
GT-02287 improves activities of daily living and cognitive performance in animal model of GBA1 Parkinson’s disease • Nest building is a non- Nest-building behaviour test Wire Hang test invasive test to study ✱✱✱ wire day 28 activities of daily living ✱✱ ✱✱✱✱ and cognitive 6 ✱✱✱ ns ✱✱✱✱ performance i
Slide 13: GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease
GT-02287 has Best-in-Class Profile for GBA1-Parkinson’s Disease of known GCase- targeting candidates Effect on Disease Cascade GT-02287 Increases Lysosomal GCase Activity Reduces ER Stress GT-02287 restores GCase function and demonstrates Reduces Toxic Lipid Substrates effect on entire disease GCase
Slide 14: Phase 1 Clinical Trial
Phase 1 Clinical Trial Single Ascending Dose • Completed in March - 40 subjects treated • Generally well tolerated, no SAEs • Exposures in humans reached levels observed in preclinical efficacy studies Multiple Ascending Dose • Completed in July - 32 subjects • Generally well tolerated, no SAEs • Ac
Slide 15: Upcoming Anticipated Milestones and Potential Value Inflection Points
Upcoming Anticipated Milestones and Potential Value Inflection Points ▪ Start of Phase 1b POC clinical trial in GBA1-Parkinson’s disease - 3-month treatment, open label - Safety and tolerability in patients - Biomarkers of GCase disease cascade and PD disease ▪ Type B FDA Meeting 2H 2024 ▪ Potential
Slide 16: GT-02287 Differentiated Profile within Competitive Landscape
GT-02287 Differentiated Profile within Competitive Landscape © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 17: Clinical Stage Programs targeting GBA1 and GCase Disease Cascade
Clinical Stage Programs targeting GBA1 and GCase Disease Cascade Competitive landscape is uncluttered GCase-Targeting Small Molecules PHASE 1 Gain Therapeutics, Inc. GT-02287 allosteric GCase chaperone PHASE 2 Vanqua Bio (Private) Allosteric GCase activator Bial (formerly LTI - acquired for $130M) P
Slide 18: GT-02287 has Favorable Profile over other known MOAs and Modalities
GT-02287 has Favorable Profile over other known MOAs and Modalities GCase-Targeting Small Molecules α-Syn-Targeting α-Syn-Targeting Gene Therapy Modality Small Molecules Antibodies Upstream Effect Brain Penetration Convenience of Administration Safety Commercial (COGS/Pricing) Favorable Product Attr
Slide 19: GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease
GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease of known GCase- targeting candidates Effect on Disease Cascade GT-02287 BIA 28-6156 VQ-101 Increases Lysosomal GCase Activity Reduces ER Stress Reduces Toxic Lipid Substrates GCase Mechanism Reduces Aggregated α-Synuclein of Action Impr
Slide 20: Large Pharma is interested in GBA1-related Programs
Large Pharma is interested in GBA1-related Programs Recent acquisitions at >10x of Gain’s market cap Effect on Disease Cascade GT-02287 Increases Lysosomal GCase Activity $1.2B+ in big pharma acquisitions of Reduces ER Stress preclinical GBA programs in the last year Reduces Toxic Lipid Substrates G
Slide 21: Large Market Potential for GT-02287 Across Several Neurodegenerative Diseases
Large Market Potential for GT-02287 Across Several Neurodegenerative Diseases Number of Patients Total Addressable Indication Rationale (US) Market (US) • Patients have dysfunctional GCase due to GBA1-Parkinson’s Disease 150,000 $1.5B heterozygous GBA mutation • GT-02287 has shown effects in preclin
Slide 22: Company Background
Company Background F IN A N C IA L A N D STOC K DATA A N A LYST C OVE R A GE IPO (Nasdaq: GANX) BTIG: − March 2021 Tom Shrader, PhD, CFA: Buy − Led by BTIG and Oppenheimer & Co. C OR P OR AT E BA C KGR OU N D Oppenheimer & Co. Jay Olson, CFA: Outperform ▪ Established in 2017 CASH POSITION − $16.9 mi
Slide 23: Company Contacts
Company Contacts Gene Mack: interim-CEO, CFO gmack@gaintherapeutics.com Apaar Jammu: Manager, Investor Relations and Public Relations ajammu@gaintherapeutics.com © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
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