Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (October 2024)
29 slides from Gain Therapeutics's investor presentation, October 2024
Slide 1: Corporate Presentation
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Presentation October 2024 NASDAQ: GANX
Slide 2: 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 herein as “forward-looking
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 Khalid Islam, PhD Gene Mack, MBA Jonas Hannestad, Founderand Interim CEO and CFO MD, PhD ExecutiveChairman Chief Medical Officer Joanne Taylor, Terenzio Ignoni, PhD PharmD SVP Research SVP Technical Operations © 2024 - GA IN T HE RAP EU
Slide 5: Experienced Leadership: Extensive Biotech And Pharma Experience (>$30b Transactions)
Experienced Leadership: Extensive Biotech And Pharma Experience (>$30b Transactions) Khalid Islam, PhD Claude Nicaise, MD Eric I. Richman Gwen Melincoff Founderand Independent Member Independent Member Independent Member ExecutiveChairman 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 disease is the second most common neurodegenerative disease1 Current therapies only treat symptomsand 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 Leading to Neurodegeneration Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron Mutated Nucleus GBA1 Gene GCase protein Loss of enzymatic GBA1 Gene misfolding activity Defective GCase Normal GCase • Activat
Slide 10: Allosteric Modulator GT-02287 Restores GCase Function
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 Improves Motor Effects of GCase Dysfunction in Animal Model
GT-02287 Improves Motor Effects of GCase Dysfunction in Animal Model of GBA1 Parkinson’s disease Two GBA1-PD Models: CBE and CBE+PFFs • CBE is an irreversible GCase inhibitor • Administrationof CBE models the effects of dysfunctional GCase seen in GBA1-Parkinson’s disease • Addition of PFFs causes a
Slide 12: GT-02287 Improves Cognitive Performance in Animal Model of GBA1-Parkinson's Disease
GT-02287 Improves Cognitive Performance in Animal Model of GBA1- Parkinson’s Disease plasma NFL Improvements in Nest-building Performance ✱✱ ✱ Nest-building behaviour test p=0.09 ✱✱✱ ns ✱✱ 1000 ✱✱ Nest building is a non- Vehicle Controls 800 6 invasive test to study A1. ✱✱✱ ns CBE + PFF CBE+PFF+GT-0
Slide 13: GT-02287 has a Long-Lasting Effect on Motor Function (Wire Hang)
GT-02287 has a Long-Lasting Effect on Motor Function (Wire Hang) GBA1-PD Model (CBE+PFFs) Idiopathic PD Model (PFFs only) α-Syn PFFs α-Syn PFFs CBE No drug GT-02287 Washout No drug GT-02287 Washout © 2024 - GA IN T HE RAP EUT IC S 13
Slide 14: GT-02287 has a Long-Lasting Effect on Cognitive Function
GT-02287 has a Long-Lasting Effect on Cognitive Function GBA1-PD Model (CBE+PFFs) Idiopathic PD Model (PFFs only) WASHOUT WASHOUT © 2024 - GA IN T HE RAP EUT IC S 14
Slide 15: 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 16: 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 17: GT-02287 was Generally Well-Tolerated
GT-00287 was Generally Well-Tolerated • No Serious Adverse Events • No severe (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 the MAD had 1 or
Slide 18: 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 © 2024 - GA IN T HE RAP EUT IC S 19
Slide 19: 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 20: 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 21: 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 22: Phase 1 SAD/MAD Trial Summary
Phase 1 SAD/MAD Trial Summary Safety ➢ GT-02287 is safe and generally well tolerated No SAEs, Grade 3 AEs, or clinically-significant changes in ECGs, BP/HR Plasma Exposure ➢ Plasma exposures in the projected therapeutic range Adequate safety margins based on toxicology studies CNS Exposure ➢ GT-0228
Slide 23: Planned Open-Label Phase 1b Trial in Parkinson's Disease Patients
Planned 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 Endp
Slide 24: Upcoming Anticipated Milestones and Potential Value Inflection Points
Upcoming Anticipated Milestones and Potential Value Inflection Points 2H 2024 1H 2025 2H 2024 Initiation of Phase 1b POC clinical trial in GBA1- Potential clinical POC based on biomarkers Parkinson’s disease of GBA1-Parkinson’s disease (interim data ▪ PD patients, 3-month treatment, open label from
Slide 25: GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease
GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease 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 Improves Lys
Slide 26: 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 27: 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 28: Company Background
Company Background C OR POR AT E BA C KGR OU ND FINANCIAL AND STOCK DATA Established in 2017 IPO (NASDAQ: GANX) − March 2021 26 employees in three locations: HQ in Bethesda, − Led by BTIG and Oppenheimer & Co. Maryland, Lugano, Switzerland, Barcelona, Spain CAPITAL STRUCTURE Founder and Executive Ch
Slide 29: Company Contacts
Company Contacts Gene Mack Interim-CEO, CFO gmack@gaintherapeutics.com Apaar Jammu Manager, Investor Relations and Public Relations ajammu@gaintherapeutics.com
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