Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (April 2025)
29 slides from Gain Therapeutics's investor presentation, April 2025
Slide 1: Gain Therapeutics Corporate Presentation April 2025
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Presentation April 2025 NASDAQ: GANX
Slide 2: Forward-Looking Statements
Forward-Looking Statements Certain statements set forth in this presentation are forward-looking andreflect the Company’s plans, beliefs, expectations and current views with respect to, among other things, future events and financial performance (collectively referred to herein as “forward-looking s
Slide 3: GANX Corporate Highlights
GANX Corporate Highlights • Allosteric modulator of glucocerebrosidase enzyme (GCase) Lead Product GT-02287 • Disease modifying potential: slowing/stopping progression of motor and cognitive decline in GBA1 and Advancing into Parkinson’s idiopathic PD Disease Patients • Safe and well tolerated in Ph
Slide 4: Experienced Leadership: Extensive Biotech And Pharma Experience
Experienced Leadership: Extensive Biotech And Pharma Experience Gene Mack, MBA Jonas Hannestad, Gianluca Fuggetta MD, PhD Senior Vice President, Chief Executive Officer Chief Medical Officer Finance Terenzio Ignoni, Joanne Taylor, PharmD PhD SVP Technical SVP Research Operations © 2025 - GA I N T HE
Slide 5: Experienced Board of Directors: Extensive Biotech And Pharma Experience
Experienced Board of Directors: Extensive Biotech And Pharma Experience (>$30b Transactions) Khalid Islam, PhD Claude Nicaise, MD Eric I. Richman Gwen Melincoff Founder and Independent Member Independent Member Independent Member Chairman Hans Peter Dov Goldstein, Jeffrey Riley Hasler MD Independent
Slide 6: Gain Therapeutics Pipeline
Gain Therapeutics Pipeline ASSET INDICATION TARGET DISCOVERY RESEARCH PRECLINICAL PHASE 1 Parkinson’s Disease GCase Gaucher’s Disease GCase GT-02287 Dementia with Lewy Bodies GCase Alzheimer’s Disease GCase Multiple GALC Lysosomal Storage Disorders Undisclosed GLB1 Undisclosed Metabolic Diseases AAT
Slide 7: Lead Clinical Program GT-02287 GBA1 Parkinson's Disease
Lead Clinical Program GT-02287 GBA1 Parkinson’s Disease 7
Slide 8: Parkinson's Disease – Market Opportunity
Parkinson’s Disease – Market Opportunity Parkinson’s Disease US Market Potential: $4B Parkinson’s diseaseis the second most common neurodegenerative disease1 Current therapies only treat symptoms and do not prevent disease progression Parkinson’s Disease Up to15% US Market Potential: $3B (PD) 1M+ of
Slide 9: Dysfunctional GCase triggers disease cascade leading to neurodegeneration
Dysfunctional GCase triggers disease cascade affecting multiple organelle functions and leads to neurodegeneration Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron Mutated Nucleus GBA1 Gene GCase protein GBA1 Gene misfolding Defective GCase Normal GCase • Increased ER stress and ER -associat
Slide 10: GT-02287 restores GCase function, improves disease cascade and neuronal survival
Allosteric modulator GT-02287 restores GCase function, which improves disease cascade and neuronal survival Dopaminergic Neuron with Reduced ER Stress GCase Activity Toxic Substrate Restoration Depletion Restored GCase Function 1.0 8 (relative to untreated) 100 BiP protein level fibroblasts (ng/mg %
Slide 11: GT-02287 decreases aggregated alpha-synuclein, neuroinflammation and improves motor function
GT-02287 decreases aggregated ⍺-synuclein, neuroinflammation, and plasma NfL, and increases neuronal survival, striatal dopamine levels, and motor function GBA1-PD Model: CBE causes partial knockdown and Improves of GCase Motor Function activity in the GBA1-PD Mouse Model • CBE is an irreversible GC
Slide 12: GT-02287 Improves Motor and Cognitive Performance in Therapeutic Model of GBA1-PD
GT-02287 Improves Motor and Cognitive Performance in Therapeutic Model of GBA1-PD Improvements in Nest-building PerformanceNest-building behaviour test ✱✱✱ Nest Building Score Wire Hang Test wire day 28 Nest building is a non- ✱✱✱ ✱✱✱✱ invasive test to study A1. ✱✱ ✱✱✱✱ 400 activities of daily livin
Slide 13: GT-02287 displays a disease-modifying effect on motor function
GT-02287 displays a disease-modifying effect on motor function GBA1- PD Model (CBE+PFFs) Idiopathic PD Model (PFFs only) α-Syn PFFs α-Syn PFFs Mouse Wire Hang CBE No drug GT-02287 Washout No drug GT-02287 Washout © 2025 - GA I N T HE RAP EUT I C S 13
Slide 14: GT-02287 displays a disease modifying effect on cognitive function
GT-02287 displays a disease modifying effect on cognitive function GBA1-PD Model (CBE+PFFs) Idiopathic PD Model (PFFs only) Mouse Nest Building WASHOUT WASHOUT © 2025 - GA I N T HE RAP EUT I C S 14
Slide 15: Single- and Multiple-ascending Dose First-in-human Phase 1 Study
Single- and Multiple-ascending Dose First-in-human Phase 1 Study Participants: SAD/MAD endpoints: MAD Cohort 4: • Healthy men and women ages 18-65 • Treatment-emergent adverse events • CSF drug levels • 8 subjects per cohort • Clinical labs, vital signs, ECGs, C-SSRS • GCase activity in dry • 2 plac
Slide 16: GT-02287 was Generally Well-Tolerated
GT-02287 was Generally Well-Tolerated • No Serious Adverse Events • No Severe Adverse Events (CTCAE Grade 3) • No discontinuations due to AE • 56.7% (n=17) of active and 40% (n=4) of placebo participants in the SAD had 1 or more TEAE • 64% (n=16) of active and 50% (n=4) of placebo participants in th
Slide 17: Adverse Event Profile
Adverse Event Profile Most common TEAEs in MAD: • Nausea 32% • Abdominal pain 8% • Diarrhea 8% • Headache 8% Nausea: • >90% of events were mild • >90% of events were <3h in duration • Incidence increased with dose level • Incidence decreased with continued dosing © 2025 - GA I N T HE RAP EUT I C S 1
Slide 18: Therapeutic Range: Phase 1 PK Data in Healthy Volunteers
Therapeutic Range: Phase 1 PK Data in Healthy Volunteers Human AUC from MAD Study : Day 1 and Day 14 12000 10000 R² = 0.9663 8000 R² = 0.9975 AUC: ng.h/mL 6000 4000 Onset of Therapeutic Effect 2000 (Animal Models) Linear Regression Day 14 0 Linear Regression Day 1 4 5.5 7 8.5 10 11.5 13 14.5 16 Dose
Slide 19: GT-02287 Demonstrates CNS Exposure Comparable to that Observed in Rodents
GT-02287 Demonstrates CNS Exposure Comparable to that Observed in Rodents • CSF levels in Humans comparable to those observed at efficacious dose levels in rodents • CSF levels are low in all species due to low aqueous solubility and high protein binding • Observed total brain levels in rodents are
Slide 20: GT-02287 Demonstrates GCase Target Engagement In Healthy Volunteers
GT-02287 Demonstrates Gcase Target Engagement In Healthy Volunteers • GCase activity in dry blood spots was GCase Activity (DBS) measured in MAD Cohort 4 ✱✱✱ ✱✱ • In GT-02287 subjects, 5 out of 6 had ✱ increased GCase activity 12 GCase activity (μmol/l/h) ns • In placebo subjects, no increase was 10
Slide 21: Phase 1 Blinded, Placebo Controlled SAD/MAD Trial
Phase 1 Blinded, Placebo Controlled SAD/MAD Trial Safety GT-02287 is safe and generally well tolerated in SAD (40 HV) and MAD (32 HV, daily dosing 14 days) No SAEs, Grade 3 AEs, or clinically-significant changes in ECGs, BP/HR Plasma Exposure Plasma exposures in the projected therapeutic range Adequ
Slide 22: Open-Label Phase 1b Trial in Parkinson's Disease Patients
Open-Label Phase 1b Trial in Parkinson’s Disease Patients Design 3 month dosing • Open-label • Idiopathic PD and GBA1 genetic mutation • Australia with potential to expand to other geographies Objectives Primary Endpoint: Safety and tolerability in Parkinson’s disease patients Secondary Endpoints: P
Slide 23: Upcoming Anticipated Milestones and Potential Value Inflection Points
Upcoming Anticipated Milestones and Potential Value Inflection Points 1H 2025 2H 2025 Potential clinical POC based on Start of Phase 2 planning biomarkers of GBA1-Parkinson’s disease (first analysis from Phase 1b clinical trial) © 2025 - GA I N T HE RAP EUT I C S 23
Slide 24: GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease
GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease Effect on Disease Cascade GT-02287 BIA 28-6156 VQ-101 Increases Lysosomal GCase Activity ? Reduces ERStress ? ? Reduces Toxic Lipid Substrates GCase Mechanism Reduces Aggregated α-Synuclein ? of Action Improves Lysosomal Function Improv
Slide 25: 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 Gain Therapeutics, Inc. GT-02287 allosteric GCase chaperone Vanqua Bio (Private) Allosteric GCase activator Bial (formerly LTI - acquired for $130M) Allosteric GCase
Slide 26: Large Market Potential for GT-02287 for Various Neurodegenerative Diseases
Large Market Potential for GT-02287 for Various Neurodegenerative Diseases Number of Indication Rationale Patients (US) Patients have dysfunctional GCase due to GBA1-Parkinson’s Disease 150,000 heterozygous GBA1 mutation Idiopathic Parkinson’s Disease Increase in GCase activity and target activation
Slide 27: Company Background
Company Background COR POR ATE BA CKGR OU ND FINANCIAL AND STOCK DATA Established in 2017 IPO (NASDAQ: GANX) 27 employees in three locations: HQ in Bethesda, − March 2021 − Led by BTIG and Oppenheimer & Co. Maryland, Lugano, Switzerland, Barcelona, Spain CAPITAL STRUCTURE Founder and Executive Chair
Slide 28: Company Contacts
Company Contacts Gene Mack President & CEO gmack@gaintherapeutics.com Apaar Jammu Manager, Investor Relations and Public Relations ajammu@gaintherapeutics.com
Slide 29: Appendix
Appendix 7
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