Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (September 2026)
27 slides from Gain Therapeutics's investor presentation, September 2026
Slide 1: Corporate Presentation
Corporate Presentation September 2024 GAIN THERAPEUTICS September 2026 Nasdaq: GANX © 2 0 2 6 - G A IN T H E R AP E U T I CS 1
Slide 2: Forward-Looking Statements
Forward-LookingStatements 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 s
Slide 3: Gain Therapeutics At-a-Glance
Gain Therapeutics At – a - Glance Established in 2017, IPO March 2021 Proprietary Platform enables novel therapeutic candidates Headquartered in Bethesda, MD with offices Our proprietary drug discovery platform, in Lugano, Switzerland, Barcelona, Spain Magellan , integrates AI-supported structural b
Slide 4: GANX Investment Summary for Parkinson's Disease
GANX Investment Summary • Completed Phase 1a in healthy volunteers and Phase1b open-label trial in PD (90-day) with Clinical PoC for rexaceract; 84% of patients electing to join 9-month extension study Disease-Modifying Treatment • Biomarker evidence from Phase 1b supports disease-modifying potentia
Slide 5: Leadership: Extensive Biotech and Pharma Experience
Leadership: ExtensiveBiotech andPharma Experience Terenzio Ignoni, Gene Mack, MBA PharmD Joanne Taylor, PhD Chief Executive Officer Chief OperatingOfficer Chief Scientific Officer Khalid Islam Gianluca Fuggetta Chairman, Co-Founder Senior Vice President, Finance © 2 0 2 6 - G A IN T H E R AP E U T I
Slide 6: Magellan: One Platform, a Broadening Pipeline
Magellan : One Platform, a Broadening Pipeline ASSET INDICATION TARGET DISCOVERY RESEARCH PRECLINICAL PHASE 1 Parkinson’s Disease GCase Gaucher’s Disease GCase rexaceract Dementia with Lewy Bodies GCase Alzheimer’s Disease GCase GT-04686 TBD GCase Multiple GALC Lysosomal Storage Disorders Undisclose
Slide 7: Parkinson's Disease: High Need for Disease-Modifying Therapies
Parkinson’s Disease: High Need for Disease-Modifying Therapies Existing therapies address symptoms without slowing progression Parkinson’sDisease GBA1-PD USMarket Potential: US Market Potential: $4B $3B Parkinson’s Disease Up to15% • Second most common 1M+US Patients of PD • Most common genetically-
Slide 8: Rexaceract: A Disease-Modifying Therapy in Development for Parkinson's Disease
Rexaceract: A Disease-Modifying Therapy in Development for Parkinson’s Disease Novel Mechanism rexaceract binds at allosteric site, and chaperones glucocerebrosidase (GCase) enzyme to lysosomes and mitochondria Disease Modification Restores GCase function and improves disease cascade and neuronal su
Slide 9: Dysfunctional GCase Affects Multiple Organelles
Dysfunctional GCase Affects Multiple Organelles HealthyDopaminergicNeuron DiseasedDopaminergicNeuron 1 GBA1Gene Mutated GCaseprotein misfolding Nucleus GBA1Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endoplasmic 3 Reticulum Lysosomal and mitochondri
Slide 10: Allosteric Modulator Rexaceract Restores GCase Function
Allosteric Modulator Rexaceract Restores GCase Function: Improves Disease Cascade to Promote Neuronal Survival (i.e., Disease-Modifying) Dopaminergic Neuron with GCase Activity Restored Lysosomal Toxic Substrate Restored GCase Function Restoration Function Depletion Mutated GBA1 Gene Neuronal Surviv
Slide 11: Rexaceract has Best-in-Class Profile for GBA1-Parkinson's Disease
Rexaceract has Best-in-Class Profile for GBA1-Parkinson's Disease Effect on Disease Cascade rexaceract BIA 28-6156 VQ-101 Increases Lysosomal GCase Activity ✓ ? ✓ Reduces ER Stress ✓ ? ? GCase Reduces Toxic Lipid Substrates ✓ ✓ ✓ Mechanism of Reduces Aggregated α-Synuclein ✓ ? ✓ Action Improves Lyso
Slide 12: Rexaceract Demonstrates a Disease-Modifying Effect in Animal Models of GBA1 and iPD
Rexaceract Demonstrates a 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 Mouse Wire Hang No drug rexaceract Washout No drug rexaceract Washout rexaceract 60mg/Kg rexaceract 90mg/Kg Control CBE/PF
Slide 13: Rexaceract is Well-Tolerated in SAD/MAD and Bioavailability Studies
Rexaceract is Well-Tolerated in SAD/MAD and Bioavailability Studies Demonstrated CNS Exposure and GCase Engagement 79 Healthy Volunteers • Single and multiple dose levels tested were generally well-tolerated, with no SAEs or Grade 3 (severe) adverse events observed; no other safety signals detected
Slide 14: Human AUC from MAD Study: Day 1 and Day 14
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 15: Phase 1b Study Overview and Open-Label Extension
Phase 1b Study Overview + 90 day and Open-Label Extension Phase 1b Open-Label Extension Study Completed Nov 2025 Ongoing until Q3 2026 90 days 9 months Enrolled: n=21 Enrolled: n=16 Completed: n=19 84% of eligible patients elected to join the 9- month extension study Overview: • 3-month open label m
Slide 16: Decreased GCase Leads to Excess GluCer and Increased GluSph in Parkinson's
Decreased GCase Leads to Excess GluCer and Consequently Increased GluSph in Parkinson’s Excess GluCer is Mitochondrial dysfunction converted to GluSph Acid ceramidase GluSph Lysosomal dysfunction GluCer is >1000x GluCer more abundant Aggregation of alpha-synuclein accumulates due to than GluSph in i
Slide 17: Treatment with Rexaceract Leads to a Decrease in CSF GluSph
Treatment with Rexaceract Leads to a Decrease in CSF GluSph, a Prespecified Endpoint, Demonstrating CNS Target Engagement p<0.05 400 GluSph (pg/ml) 300 200 100 0 t en 90 tm ay a D re et Pr Data are shown as CSF GluSph levels for individual patients before and after treatment. Gray lines represent pa
Slide 18: Higher CSF DDC Levels in Participants with High CSF GluSph at Baseline
Higher CSF DDC Levels in Participants Who Had High CSF GluSph at Baseline DDC Decreases Following Rexaceract Treatment in High GluSph Group Baseline DDC Levels Change in DDC Levels at D90 p<0.05 p=0.06 DDC is elevated 150 Change in DDC levels (pg/ml) 20 in CSF in people with Parkinson’s, likely due
Slide 19: Phase 1b Evaluable Population for CSF Biomarker Analysis and MDS-UPDRS Scores
Phase 1b Evaluable Population for CSF Biomarker Analysis and MDS- UPDRS Scores Completed 90 days dosing n=19 CSF alpha synuclein SAA negative n=2 Confirmed PD patients No CSF sample (1) n=17 Blood in CSF sample (1) MDS-UPDRS not in ‘off’ state (1) CSF biomarkers and MDS- n=3 UPDRS n=14 High CSF GluS
Slide 20: High CSF GluSph Patients Show Greater MDS-UPDRS Improvement After 90 Days
PD Patients With High Levels of CSF GluSph at Baseline Show a Markedly Greater Improvement in MDS-UPDRS Motor Scores After 90 Days of Dosing with Rexaceract Change in MDS-UPDRS from Baseline at Day 90 Part II Part III Part II+III All patients for which CSF GluSph and UPDRS scores available (n=14) -0
Slide 21: High CSF GluSph Patients Show Greater MDS-UPDRS Improvement After 150 Days
PD Patients With High Levels of CSF GluSph at Baseline Show a Markedly Greater Improvement in MDS-UPDRS Motor Scores After 150 Days of Dosing with Rexaceract Change in MDS-UPDRS from Baseline at Day 150 Part II Part III Part II+III All patients for which CSF GluSph and UPDRS scores available (n=10)
Slide 22: Rexaceract: A Disease-Modifying Therapeutic in Development for Parkinson's Disease
Rexaceract: A Disease-Modifying Therapeutic in Development for Parkinson’s Disease Mechanism Rexaceract binds at allosteric site, and chaperones glucocerebrosidase (GCase) enzyme to lysosomes and mitochondria Rexaceract Evidence: • Patients with elevated baseline CSF Disease Modification Restores GC
Slide 23: Phase 2a Study
Phase 2a Study Dosing for 48 weeks 48-week, double blind, randomized, placebo- High Dose controlled Phase 2a study of 2 dose levels of Study n=~37 oral rexaceract in participants with early PD Randomization stratified by Mid Dose Participants: Clinical diagnosis of PD according n=~111 n=~37 treatmen
Slide 24: KOL Events and Industry Support
KOL Events and Industry Support JANUARY 2026 OCTOBER 2025 UNDERSTANDING GCASE SUBSTRATES IN BIOMARKERS, CLINICAL ENDPOINTS, AND PARKINSON’S DISEASE – PERSPECTIVES THE PATH TO DISEASE MODIFICATION: ON BIOMARKERS AND DISEASE CONTEXUALIZING THE EMERGING DATE MODIFICATION FROM GT-02287 Featured Key Opin
Slide 25: Financials
Financials Established in 2017, IPO March 2021 Financials Headquartered in Bethesda, MD with offices • Nasdaq: GANX in Lugano, Switzerland, Barcelona, Spain • Cash: $13.1 million as of June 30, 2026 23 Employees • Cash Runway: through 1Q27 • No debt Scientific Founder and Chairman: Dr. Khalid Islam
Slide 26: GANX Investment Summary
GANX Investment Summary Clinical PoC for Multiple Near-Term Global Rights with Strong Financial and rexaceract, a Catalysts Strong IP Developmental Disease-Modifying Support from Treatment for Scientific / Medical Parkinson’s Disease Community © 2 0 2 6 - G A IN T H E R AP E U T I CS 26
Slide 27: Thank you
Corporate Presentation Thank you September 2024
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