Gain Therapeutics, Inc. · NASDAQ:GANX

Gain Therapeutics — Investor Presentation (February 2026)

19 slides from Gain Therapeutics's investor presentation, Feb 3, 2026

  1. Slide 1: Gain Therapeutics Corporate Deck February 2026

    Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Deck February 2026 NASDAQ: GANX

  2. Slide 2: Forward-looking statements

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 22 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 per

  3. Slide 3: GANX Corporate Highlights

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 33 GANXCorporateHighlights Lead Program GT-02287 Moving to Phase 2 Clinical Evaluation in Parkinson’s Disease Multiple Assets in Discovery andPreclinical Development StrongIntellectual Property Estate Upcoming Mile

  4. Slide 4: Leadership: Extensive Biotech and Pharma Experience

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 44 Leadership: ExtensiveBiotechandPharma Experience Gene Mack, MBA Chief Executive Officer Jonas Hannestad, MD, PhD Chief Medical Officer Gianluca Fuggetta Senior Vice President, Finance Joanne Taylor, PhD SVPResea

  5. Slide 5: Gain Therapeutics Pipeline

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 55 GainTherapeuticsPipeline ASSET INDICATION TARGET DISCOVERY RESEARCH PRECLINICAL PHASE 1 GT-02287 Parkinson’s Disease GCase Gaucher’s Disease GCase Dementia with Lewy Bodies GCase Alzheimer’s Disease GCase Multip

  6. Slide 6: GT-02287 – Parkinson's Disease

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 66 Well-tolerated in both healthy volunteers and PD patients with therapeutic CNS exposures achieved Mechanism Binds at allosteric site, chaperones and modulates glucocerebrosidase enzyme (GCase) Disease Modificati

  7. Slide 7: Parkinson's Disease – Unmet Medical Need for Disease Modifying Therapy

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 77 Parkinson’s Disease –Unmet Medical Need for Disease Modifying Therapy 7 Parkinson’sDisease GBA1-PD • Most common genetically-defined subpopulation of PD • GBA1variantscause misfolding of glucocerebrosidase (GCas

  8. Slide 8: GCase is Essential to Cell Pathways Involved in Maintenance and Energy Production

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 88 GCase is Essential to Cell Pathways Involved in Both Maintenance and Energy Production Nucleus Endoplasmic Reticulum Lysosomeand Mitochondria GBA1Gene Normal GCase GCaseMaintainsCellHealth • Prevents buildup oft

  9. Slide 9: Allosteric Modulator GT-02287 Restores GCase Function

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 99 AllostericModulatorGT-02287RestoresGCaseFunction: ImprovesDiseaseCascadeto PromoteNeuronalSurvival (i.e., Disease Modifying) DopaminergicNeuron with RestoredGCaseFunction Mutated GBA1 Gene Defective GCase GT-022

  10. Slide 10: GT-02287 Displays a Disease Modifying Effect in Animal Models of GBA1 and iPD

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1010 GT-02287Displaysa Disease ModifyingEffectin Animal Models of GBA1 and iPD α-SynPFFs α-SynPFFs No drug GT-02287 Washout Low Level CBE No drug GT-02287 Washout GBA1-PDModel (CBE+PFFs) Idiopathic PD Model (PFFs o

  11. Slide 11: GT-02287 is Well-Tolerated in SAD/MAD and Shows GCase Engagement

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1111 GT-02287 is Well-Tolerated in SAD/MAD and Bioavailability Study and Shows GCaseEngagement GCase Activity in Dried Blood Spots (DBS) • Single and multiple dose levels tested were generally well tolerated, with

  12. Slide 12: Phase 1b Study Overview

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1212 Phase 1b Study Overview: 90 day (Part 1) and Open-Label Extension (Part 2) • GANX-001-V102 is a two-part, open-label, Phase 1b study in people with Parkinson’s disease • Objectives: Safety and tolerability, pl

  13. Slide 13: Decreased GCase Leads to Excess GluCer and Increased GluSph in Parkinson's

    © 2 0 2 6 - G A I N T H E R A P E U T I C S 13 Decreased GCaseLeads to Excess GluCerand Consequently Increased GluSphin Parkinson’s Sphingosine GCase GluSph Ceramide GCase is very inefficient in cleaving GluSph into sphingosine and glucose Excess GluCer is converted to GluSph Acid ceramidase GluCer

  14. Slide 14: CSF biomarkers and MDS-UPDRS

    © 2 0 2 6 - G A I N T H E R A P E U T I C S 14 CSF biomarkers and MDS-UPDRS n=13 High CSF GluSph MDS-UPDRS correlation n=6 Low CSF GluSph MDS-UPDRS correlation n=7 CSF alpha synuclein SAA negative n=2 Confirmed PD patients n=17 No CSF sample (1) Blood in CSF sample (2) MDS-UPDRS not in ‘off’ state (

  15. Slide 15: GT-02287 Treatment Leads to a Decrease in CSF GluSph

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1515 GT-02287 Treatment Leads to a Decrease in CSF GluSph, a Prespecified Endpoint, Demonstrating CNS Target Engagement Pretreatment Day 90 0 100 200 300 400 GluSph (pg/ml) CNS GluSph N=13 * * Significant differenc

  16. Slide 16: PD Patients With High CSF GluSph Show Greater MDS-UPDRS Improvement

    2/3/2026 © 2 0 2 6 - G A I N T H E R A P E U T I C S 16 16 PD Patients With High Levels of CSF GluSph at Baseline Show a Markedly Greater Improvement in MDS-UPDRS Motor Scores Part II Part III Part II+III -0.54 -2.00 -2.54 All patients for which CSF GluSph and UPDRS scores available (n=13) Part II P

  17. Slide 17: GT-02287 – Parkinson's Disease

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1717 GT-02287 – Parkinson’s Disease Well-tolerated in both healthy volunteers and PD patients with therapeutic CNS exposures achieved Mechanism Binds at allosteric site, chaperones and modulates glucocerebrosidase

  18. Slide 18: Phase 1b conclusions

    © 2 0 2 6 - G A I N T H E R A P E U T I C S 18 • 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 disea

  19. Slide 19: Company Background, Analyst Coverage, Financial and Stock Data

    © © 2 0 2 6 2 0 2 5 - -G AG A I N T H E R A P E U T I C S I N T H E R A P E U T I C S 1919 Company Background Analyst Coverage BTIG Tom Shrader, Ph.D., CFA Oppenheimer & Co Jay Olson, CFA H.C. Wainwright Ram Selvaraju, Ph.D. Maxim Jason McCarthy, Ph.D. ROTH Boobalan Pachaiyappan, Ph.D. Financial and

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