Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (October 2026)
27 slides from Gain Therapeutics's investor presentation, October 2026
Slide 1: Corporate Presentation October 2026
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Presentation October 2026 Nasdaq: GANX © 2026 - GA I N T HE RA P EUT 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
GANX Investment Summary • Completed Phase 1b open-label extension in PD; MDS-UPDRS scores in all 12 evaluable Clinical PoC for rexaceract; participants were stable over 12-month study period with rexaceract, demonstrating no Disease-Modifying Treatment clinically meaningful progression of motor symp
Slide 5: Leadership: Extensive Biotech and Pharma Experience
Leadership: ExtensiveBiotech andPharma Experience Terenzio Ignoni, PharmD Chief Operating Officer Gene Mack, MBA Joanne Taylor, PhD Chief Executive Officer Chief Scientific Officer Khalid Islam Chairman, Co-Founder © 2026 - GA I N T HE RA P EUT I CS 5
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’s Disease 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 Nucleus GCaseprotein misfolding GBA1 Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endoplasmic 3 Reticulum Lysosomal and mitochondr
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 Toxic Lipid Substrates ✓ ✓ ✓ Reduces Aggregated α-Synuclein ✓ ? ✓ GCase Mechanism of Improves Lysosomal Function ✓ ✓ ✓ Action Imp
Slide 12: Rexaceract Demonstrates 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: 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 15: Phase 1b Evaluable Population for CSF Biomarker and MDS-UPDRS Analysis
Phase 1b Evaluable Population for CSF Biomarker Analysis and MDS- UPDRS Scores at 12 Months *Last PD patient will complete dosing in October © 2026 - GA I N T HE RA P EUT I CS 15
Slide 16: No Clinically Meaningful Progression on MDS-UPDRS Motor Scores
12 Evaluable Patients Have Completed 12 Months of Dosing with Rexaceract and Show No Clinically Meaningful Progression of Parkinson's Disease on MDS-UPDRS Motor Scores • rexaceract was associated with no meaningful progression on either the MDS-UPDRS Part 2 or Part 3 scale • MDS-UPDRS Part II: Patie
Slide 17: UPDRS Part III Change from Baseline vs Placebo Arm from PASADENA
Patients Treated with Rexaceract for 12 Months Show No Meaningful Change in MDS-UPDRS Part III Compared to Placebo Arm from PASADENA Study Prasinezumab change in UPDRS III score rexaceract change in UPDRS III score (n=12) (n~105/group) 6 UPDRS Pt. III Change from Baseline 5 4 3 2 1 0 -1 -2 -3 Base-
Slide 18: Stabilization of MDS-UPDRS Motor Scores by Baseline CSF GluSph
By 12 Months of Rexaceract Treatment, Patients with Both High and Low Baseline CSF GluSph Display Stabilization of MDS-UPDRS Motor Scores High Baseline GluSph (n=4) Low Baseline GluSph (n=6) MDS-UPDRS 40 40 35 35 MDS-UPDRS Score MDS-UPDRS Score 30 30 25 25 20 20 15 15 10 10 5 5 0 0 -50 50 150 250 35
Slide 19: 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 accumulates due to Aggregation of alpha-synuclein than GluSph in i
Slide 20: Rexaceract Decreases CSF GluSph, Demonstrating CNS Target Engagement
Treatment with Rexaceract Leads to a Decrease in CSF GluSph, a Prespecified Endpoint, Demonstrating CNS Target Engagement p<0.05 400 300 GluSph (pg/ml) 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 21: 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 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 Summary of Rexaceract Evidence: and mitochondria • Patients with elevated baseline CSF GluSph had an average
Slide 23: Proposed Phase 2 Study Design
Proposed Phase 2 Study Design New Design: An adaptive dose-optimization and proof-of-concept study designed to identify the effective and safe dose for Phase 3 • Small sample size in Part 1 AND expansion to 111 patients sample size in Part 2 (90 evaluable at endpoint) • Primary Endpoint: Safety and
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 andChairman: 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 © 2026 - GA I N T HE RA P EUT I CS 26
Slide 27: Thank you
Corporate Presentation Thank you September 2024
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