Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (February 2026)
19 slides from Gain Therapeutics's investor presentation, February 2026
Slide 1: Gain Therapeutics Corporate Deck
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Deck February 2026 NASDAQ: GANX
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: GANX Corporate Highlights
GANX Corporate Highlights Lead Program GT-02287 Moving • Studies completed in both Phase 1a healthy volunteers and Phase1b open-label trial to Phase 2 Clinical Evaluation in in PD (90-day) with 9-month open-label extension ongoing Parkinson’s Disease • Biomarker evidence from Phase 1b supports disea
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 SVPResearch SVPTechnical Operations © ©2 022052 6- -G A G IANI NT H T
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 GLB1 Undisclosed Undisclosed Metabolic Diseases AAT
Slide 6: GT-02287 – Parkinson's Disease
GT-02287 – Parkinson’s Disease Mechanism Binds at allosteric site, chaperones and modulates glucocerebrosidase enzyme (GCase) Disease Restores GCase function and improves disease cascade and neuronal survival Modification Pre-clinical: Demonstrates a disease modifying effect in models of GBA-1 and i
Slide 7: Parkinson's Disease – Unmet Medical Need for Disease Modifying Therapy
Parkinson’s Disease – Unmet Medical Need for Disease Modifying Therapy Parkinson’sDisease GBA1-PD U.S. Market Potential: U.S. Market Potential: $4B $3B • Second most common Parkinson’s Disease • Most common genetically-defined neurodegenerative disease 1M+ people in U.S. Up to15% subpopulation of PD
Slide 8: 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 Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron 1 GBA1 Gene Mutated Nucleus GCaseprotein misfolding GBA1 Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endo
Slide 9: 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 RestoredGCase Function Restoration Function Depletion Mutated GBA1 Gene Neuronal Survival
Slide 10: GT-02287 Displays a Disease Modifying Effect in Animal Models of GBA1 and iPD
GT-02287 Displays 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 No drug GT-02287 Washout Mouse Wire Hang No drug GT-02287 Washout Washout 10 © ©2 022052 6- -G A G IANI NT H T EH R E AR PA EP U
Slide 11: GT-02287 is Well-Tolerated in SAD/MAD and Shows GCase Engagement
GT-02287 is Well-Tolerated in SAD/MAD and Bioavailability Study and Shows GCase Engagement 96 Healthy Volunteers in SAD/MAD and Bioavailability Studies GCase Activity in Dried Blood Spots (DBS) • Single and multiple dose levels tested were • GCase activity in dry blood spots was generally well toler
Slide 12: Phase 1b Study Overview: 90 day (Part 1) and Open-Label Extension (Part 2)
Phase 1b Study Overview: 90 day (Part 1) and Open-Label Extension (Part 2) Part 1 Part 2 Completed Nov 2025 Ongoing until Q3 2026 Screened: 9 months 90 days N=27 Enrolled: N=16 Enrolled: N=21 Completed: N=19 N=3 did not want to continue in Part 2 N=1 wanted to continue but had travel abroad planned
Slide 13: 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 converted to GluSph dysfunction Acid ceramidase Lysosomal GluSph dysfunction GluCer is >1000x GluCer Aggregation of more abundant than accumulates due to insufficient alpha-synuclei
Slide 14: 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-UPDRS n=4 n=13 High CSF GluSph
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 CNS GluSph N=13 400 300 GluSph (pg/ml) 200 * 100 * 0 t en 90 tm ay a D re et Pr Significant difference compared to pretreatment Two-way ANOVA P<0.05 Data are shown as mean + s.e.m. n=13
Slide 16: PD Patients With High CSF GluSph Show Greater Improvement in MDS-UPDRS Motor Scores
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 17: GT-02287 – Parkinson's Disease
GT-02287 – 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 Pre-clinical: Demonstrates a disease modifying effect in models of GBA-1 and i
Slide 18: GT-02287 – Translation From Animal Models to Clinic
GT-02287 – Translation From Animal Models to Clinic • Disease modifying properties GT-02287 demonstrated in earlier studies and preclinical animal models are now reflected in our Phase 1b clinical biomarker and functional readouts, supporting successful translation into individuals with Parkinson’s
Slide 19: Company Background
Company Background Corporate Background Financial and Stock Data • Established in 2017 IPO (NASDAQ: GANX) − March 2021 • 25 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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