Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (October 2025)
24 slides from Gain Therapeutics's investor presentation, October 2025
Slide 1: Corporate Presentation
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Presentation October 2025 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: altering progression of motor/cognitive decline in GBA1 and iPD Being Evaluated in • Safe and well tolerated in Phase 1 SAD/MAD study and demonstrated target eng
Slide 4: Leadership: Extensive Biotech And Pharma Experience
Leadership: Extensive Biotech And Pharma Experience Gene Mack, MBA Jonas Hannestad, Gianluca Fuggetta MD, PhD Chief Executive Officer Senior Vice President, Chief Medical Officer Finance Terenzio Ignoni, Joanne Taylor, PhD PharmD SVP Research SVP Technical Operations © 2025 - GA I N T HE RAP EUT I C
Slide 5: 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 6: Lead Clinical Program
Lead Clinical Program GT-02287 GBA1 Parkinson’sDisease
Slide 7: Parkinson's Disease – Market Opportunity
Parkinson’s Disease – Market Opportunity Parkinson’sDisease GBA1-Parkinson’s Disease USMarket Potential: US Market Potential: $4B $3B Up to15% Genetically defined subpopulation of Parkinson’s disease is the second most common of PD Parkinson's disease neurodegenerative disease1 Parkinson’s GBA1mutat
Slide 8: GCase Plays Integral Role in Organelle and Cellular Health
GCase Plays Integral Role in Organelle and Cellular Health Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron 1 GBA1 Gene Mutated Nucleus GCase protein misfolding GBA1 Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endoplasmic 3 Reticulum Lysosom
Slide 9: Allosteric modulator GT-02287 restores GCase function
Allosteric modulator GT-02287 restores GCase function, which improves disease cascade and neuronal survival DopaminergicNeuron with GCase Activity Restored Lysosomal Toxic Substrate RestoredGCase Function Restoration Function Depletion Mutated GBA1 Gene Neuronal Survival GT- 02287 Defective GCase No
Slide 10: GT-02287 displays a rescue and disease-modifying effect in animal models
GT-02287 displays a rescue and disease-modifying effect in animal models of GBA1 and iPD GBA1- PD Model (CBE+PFFs) Idiopathic PD Model (PFFs only) α-Syn PFFs α-Syn PFFs Low Level CBE No drug GT-02287 Washout No drug GT-02287 Washout Mouse Wire Hang Rescue & Washout Day Day Data is shown as Mean ± S.
Slide 11: Nesting Score Unaffected by Drug Washout
Nesting Score Unaffected by Drug Washout Evidence of disease modifying effect on complex behaviors CBE+PFFs (GBA1-PD) PFFs only (iPD) WASHOUT WASHOUT Mouse Nesting Building Washout $ $$ $$$ $$$ $$$ $$$$ $$$$ $$$$ $$$$ Data is shown as Mean ± S.E.M.(n= 7). $Significant difference as compared to sham
Slide 12: Brain biomarker changes maintained following GT-02287 washout
Brain biomarker changes maintained following GT-02287 washout Data from CBE+PFF Model support GT-02287’s disease modifying effect No Washout Washout Day 20 – Day 29 IRE-1 LAMP1 MIRO-1 Aggregated GCase Activity GCase Activity ER Stress Lysosomal Integrity Mitochondrial Health a-Syn $$$$ $$$$ **** ***
Slide 13: Translational plasma-based biomarkers of GT-02287 rescue effect
Translational plasma-based biomarkers of GT-02287 rescue effect Plasma GlcCer Plasma GlcSph Plasma NfL Control CBE + PFFs +GT-02287 (30mg/kg) +GT-02287 (60mg/kg) +GT-02287 (90mg/kg) Data shown as Mean ± S.E.M. (n=10). Significant difference as compared to PFFs or PFFs + CBE. © 2025 - GA I N T HE RAP
Slide 14: 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 blood spots
Slide 15: GT-02287 was Generally Well-Tolerated, No Serious Adverse Events Observed
GT-02287 was Generally Well-Tolerated, No Serious Adverse Events Observed SAD GT-02287 SAD Placebo MAD GT-02287 MAD Placebo All GT-02287 All Placebo TEAE N=30 N=10 N=25 N=8 N=55 N=18 Any 17 (56.7%) 4 (40.0%) 16 (64.0%) 4 (50.0%) 33 (60%) 8 (44%) Related to Study Drug 10 (33.3%) 1 (10%) 11 (44.0%) 0
Slide 16: Adverse Event Profile
Adverse Event Profile Most common TEAEsin 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 16
Slide 17: GT-02287 Demonstrates GCase Target Engagement In Healthy Volunteers
GT-02287 Demonstrates GCase Target Engagement In Healthy Volunteers Healthy Volunteer Results GCase Activity in Dried Blood Spots (DBS) • GCase activity in dry blood spots was measured in MAD Cohort 4 • In GT-02287 subjects, 5 out of 6 had increased GCase activity • In placebo subjects, no increase
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 Mean CSF level Total brain level Mean plasma Cmax Species Timepoint Dose (mg/kg) (ng/mL) (ng/mL) (ng/mL) mean Day 14 Human (MAD4) 3.1 (1.7-4.9) Not sampled 850 Day 13 13.5 PO Mouse 4 6592 2414 15 min 10 IV Rat 3 2441 680 1 hou
Slide 20: Upcoming Milestones
Upcoming Milestones Phase 1b analysis of functional changes and full biomarker Q4 2025 analysis in CSF and blood 2H 2025 Start of Phase 2 planning and regulatory filings in US/EU © 2025 - GA I N T HE RAP EUT I C S 20
Slide 21: Design of Phase 1b Trial in Parkinson's Disease Patients
Design of Phase 1b Trial in Parkinson’s Disease Patients An Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Participants with Parkinson’s Disease With or Without a Pathogenic GBA1 Mutation Study Extension Treatment Period Follow-up Period 13
Slide 22: Phase 1b Study Objectives
Phase 1b Study Objectives Study Objectives Endpoints Primary To evaluate the safety and tolerability of GT-02287 To characterize the single-dose and steady state Secondary plasma PK profile of GT-02287 To assess levels of GT-02287 in CSF after at least 12 Concentration of GT-02287 in CSF at 4 hours
Slide 23: 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 DiseaseCascade GT-02287 BIA 28-6156 VQ-101 Increases LysosomalGCase Activity ? Reduces ERStress ? ? Reduces ToxicLipid Substrates GCase Mechanism Reduces Aggregated α-Synuclein ? of Action Improves LysosomalFunction Improves M
Slide 24: Company Background
Company Background Corporate Background Financial and Stock Data • Established in 2017 IPO (NASDAQ: GANX) − March 2021 • 27 employees in three locations: HQ in Bethesda, − Led by BTIG and Oppenheimer & Co. Maryland, Lugano, Switzerland, Barcelona, Spain • Founder and Executive Chairman: Dr. Khalid I
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