Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (March 2026)
25 slides from Gain Therapeutics's investor presentation, March 2026
Slide 1: Corporate Deck March 2026
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Deck March 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
GANX Investment Summary • Completed Phase 1a in healthy volunteers and Phase1b open-label trial in PD (90-day) with Clinical PoC for GT-02287; 84% of patients electing to join 9-month extension study Disease-Modifying Treatment • Biomarker evidence from Phase 1b supports disease-modifying potential
Slide 5: Leadership: Extensive Biotech and Pharma Experience
Leadership: ExtensiveBiotech andPharma Experience Gene Mack, MBA Jonas Hannestad, Joanne Taylor, PhD MD, PhD SVP Research Chief Executive Officer Chief Medical Officer Terenzio Ignoni, Khalid Islam PharmD Gianluca Fuggetta Chairman, Co-Founder SVP Technical Senior Vice President, Operations Finance
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 © 2 0 2 6 - G A IN T H E R AP E U
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: GT-02287: A Disease-Modifying Therapy in Development for Parkinson's Disease
GT-02287: A Disease-Modifying Therapy in Development for Parkinson’s Disease Novel Mechanism GT-02287 binds at allosteric site, chaperones and modulates glucocerebrosidase enzyme (GCase) Disease Modification Restores GCase function and improves disease cascade and neuronal survival Efficacy Phase 1b
Slide 9: GT-02287: Clinical PoC as a Disease-Modifying Treatment for PD
GT-02287: Clinical PoC as a Disease-Modifying Treatment for PD Phase 1b 90-day data support biological rationale and validate disease-modifying MOA identified in pre-clinical studies GT-02287 was well- tolerated: 16/19 patients chose to remain on Patients with baseline elevation in CSF treatment for
Slide 10: GCase is Essential to Cell Pathways Involved in Maintenance and Energy Production
GCase is Essential to Cell Pathways Involved in Both Maintenance and Energy Production HealthyDopaminergicNeuron DiseasedDopaminergicNeuron 1 GBA1Gene Mutated GCaseprotein misfolding Nucleus GBA1Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endoplasmi
Slide 11: Allosteric Modulator GT-02287 Restores GCase Function
Allosteric Modulator GT-02287 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 Survival
Slide 12: GT-02287 Demonstrates a Disease-Modifying Effect in Animal Models of GBA1 and iPD
GT-02287 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 GT-02287 Washout No drug GT-02287 Washout 12 © 2 0 2 6 - G A IN T H E R AP E U T I CS 12
Slide 13: GT-02287 is Well-Tolerated in SAD/MAD and Bioavailability Studies
GT-02287 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 + 90 day 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: 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 16: GT-02287 Treatment Leads to a Decrease in CSF GluSph
GT-02287 Treatment Leads to a Decrease in CSF GluSph, a Prespecified Endpoint, Demonstrating CNS Target Engagement p<0.05* p<0.05* 400 300 GluSph (pg/ml) 200 100 * 0 t en 90 a tm ay D e tre Pr * Significant difference compared to pretreatment Two-way ANOVA P<0.05 Data are shown as mean + s.e.m. n=13
Slide 17: 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 (2) MDS-UPDRS not in ‘off’ state (1) CSF biomarkers and MDS- n=4 UPDRS n=13 High CSF GluS
Slide 18: PD Patients With High CSF GluSph at Baseline Show Greater Improvement in MDS-UPDRS
PD Patients With High Levels of CSF GluSph at Baseline Show a Markedly Greater Improvement in MDS-UPDRS Motor Scores Negative score indicates improvement of symptoms Part II Part III Part II+III All patients for which CSF GluSph and UPDRS scores available (n=13) -0.54 -2.00 -2.54 Part II Part III Pa
Slide 19: 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 GT-02287 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 to
Slide 20: Phase 2a Study
Phase 2a Study Dosing for 48 weeks 48-week, double blind, randomized, placebo- controlled Phase 2a study of 2 dose levels of High Dose oral GT-02287 in treated and untreated Study n=~37 participants with early PD Randomization stratified by Mid Dose Participants: Clinical diagnosis of PD according n
Slide 21: GT-02287: A Disease-Modifying Therapeutic in Development for Parkinson's Disease
GT-02287: A Disease-Modifying Therapeutic in Development for Parkinson’s Disease Mechanism Binds at allosteric site, chaperones and modulates glucocerebrosidase enzyme (GCase) Disease Modification Restores GCase function and improves disease cascade and neuronal survival Efficacy Pre-clinical: Demon
Slide 22: 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 23: Financials
Financials Established in 2017, IPO March 2021 Financials Headquartered in Bethesda, MD with offices • Nasdaq: GANX in Lugano, Switzerland, Barcelona, Spain • Cash: $20.8 million as of December 31, 2025 25 Employees • Cash Runway: through 1Q27 Founder and Executive Chairman: • No debt Dr. Khalid Isl
Slide 24: GANX Investment Summary
GANX Investment Summary Clinical PoC for GT- Multiple Near-Term Global Rights with Strong Financial and 02287, a Disease- Catalysts Strong IP Developmental Modifying Treatment Support from for Parkinson’s Scientific / Medical Disease Community © 2 0 2 6 - G A IN T H E R AP E U T I CS 24
Slide 25: Thank you
Corporate Presentation Thank you September 2024
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