Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (June 2026)
25 slides from Gain Therapeutics's investor presentation, June 2026
Slide 1: Gain Therapeutics Corporate Deck
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Deck June 2026 NASDAQ: GANX © 2026 - GA I N T HE RA P EUT I CS 1
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: 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: Extensive Biotech and Pharma Experience Gene Mack, MBA Jonas Hannestad, Joanne Taylor, PhD MD, PhD Chief Executive Officer SVP Research Chief Medical Officer Terenzio Ignoni, Khalid Islam PharmD Gianluca Fuggetta Chairman, Co-Founder SVP Technical Senior Vice President, Operations Financ
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 © 2026 - GA I N T HE RA P EUT I CS
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 US Market Potential: US Market Potential: $4B $3B Parkinson’s Disease Up to15% • Second most common 1M+US Patients of PD • Most common geneticall
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, and chaperones glucocerebrosidase (GCase) enzyme to lysosomes and mitochondria Disease Modification Restores GCase function and improves disease cascade and neuronal surviv
Slide 9: Dysfunctional GCase Affects Multiple Organelles
Dysfunctional GCase Affects Multiple Organelles Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron 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 mitoch
Slide 10: 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 11: 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 11 © 2026 - GA I N T HE RA P EUT I CS 11
Slide 12: 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 13: 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 14: 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 15: 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<0.05 400 300 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 patients
Slide 16: 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 17: 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 GT-02287 Negative score indicates improvement of symptoms Part II Part III Part II+III All patients for which CSF GluSph and UPDRS scores available (n=14)
Slide 18: High CSF GluSph Patients Continue to Show Differential Response After 150 Days
PD Patients With High Levels of CSF GluSph at Baseline Continue to Show Differential Response After 150 Days of Dosing with GT-02287 Negative score indicates improvement of symptoms Part II Part III Part II+III All patients for which CSF GluSph and UPDRS scores available (n=10) 1.3 -1.0 0.3 Part II
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: 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 GT-02287 binds at allosteric site, and chaperones glucocerebrosidase (GCase) enzyme to lysosomes and mitochondria GT-02287 Evidence: Restores GCase function and improves disease • Patients with elevated baseli
Slide 21: 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 GT-02287 in treated and untreated Randomization participants with early PD stratified by Mid Dose n=~111 n=~37 treatment status and Participants: Cl
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: $16.5 million as of March 31, 2026 25 Employees • Cash Runway: through 1Q27 Founder and Executive Chairman: • No debt Dr. Khalid Islam
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 © 2026 - GA I N T HE RA P EUT I CS 24
Slide 25: Thank you
Corporate Presentation Thank you September 2024
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