Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (December 2025)
21 slides from Gain Therapeutics's investor presentation, December 2025
Slide 1: Gain Therapeutics Corporate Presentation December 2025
Corporate Presentation September 2024 GAIN THERAPEUTICS Corporate Presentation December 2025 NASDAQ: GANX
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: GANX Corporate Highlights
GANX Corporate Highlights • Allosteric modulator of glucocerebrosidase enzyme (GCase) Lead Product GT-02287 • Disease modifying potential: altering progression of motor/cognitive decline in GBA1 and iPD Being Evaluated in • Phase 1b trial in GBA1and idiopathic PD patients ongoing; study extension ha
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 SVP Research SVP Technical Operations © 2025 - GA I N T HE RAP EUT I C
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 Undisclosed GLB1 Undisclosed Metabolic Diseases AAT
Slide 6: Lead Clinical Program
Lead Clinical Program GT-02287 GBA1 Parkinson’sDisease
Slide 7: Parkinson's Disease – Market Opportunity
Parkinson’s Disease – Market Opportunity Parkinson’sDisease GBA1-Parkinson’s Disease USMarket Potential: US Market Potential: $4B $3B Up to15% Genetically defined subpopulation of Parkinson’s disease is the second most common of PD Parkinson's disease neurodegenerative disease1 Parkinson’s GBA1mutat
Slide 8: GCase Plays Integral Role in Organelle and Cellular Health
GCase Plays Integral Role in Organelle and Cellular Health Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron 1 GBA1 Gene Mutated Nucleus GCase protein misfolding GBA1 Gene 2 Increased ER stress and ER- Normal GCase Defective associated protein degradation GCase Endoplasmic 3 Reticulum Lysosom
Slide 9: Allosteric modulator GT-02287 restores GCase function, which improves disease cascade and neuronal survival
Allosteric modulator GT-02287 restores GCase function, which improves disease cascade and neuronal survival DopaminergicNeuron with GCase Activity Restored Lysosomal Toxic Substrate RestoredGCase Function Restoration Function Depletion Mutated GBA1 Gene Neuronal Survival GT- 02287 Defective GCase No
Slide 10: GT-02287 displays a rescue and disease-modifying effect in animal models of GBA1 and iPD
GT-02287 displays a rescue and 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 No drug GT-02287 Washout Mouse Wire Hang Rescue & Washout Day Day Data is shown as Mean ± S.
Slide 11: Single- and Multiple-ascending Dose First-in-human Phase 1 Study
Single- and Multiple-ascending Dose First-in-human Phase 1 Study Participants SAD/MAD Endpoints MAD Cohort 4 • Healthy men and women ages 18-65 • Treatment-emergent adverse events • CSF drug levels • 8 subjects per cohort • Clinical labs, vital signs, ECGs, C-SSRS • GCase activity in dry blood spots
Slide 12: GT-02287 Demonstrates GCase Target Engagement In Healthy Volunteers
GT-02287 Demonstrates GCase Target Engagement In Healthy Volunteers Healthy Volunteer Results GCase Activity in Dried Blood Spots (DBS) • GCase activity in dry blood spots was measured in MAD Cohort 4 • In GT-02287 subjects, 5 out of 6 had increased GCase activity • In placebo subjects, no increase
Slide 13: GT-02287 Demonstrates CNS Exposure Comparable to that Observed in Rodents
GT-02287 Demonstrates CNS Exposure Comparable to that Observed in Rodents Mean CSF level Total brain level Mean plasma Cmax Species Timepoint Dose (mg/kg) (ng/mL) (ng/mL) (ng/mL) mean Day 14 Human (MAD4) 3.1 (1.7-4.9) Not sampled 850 Day 13 13.5 PO Mouse 4 6592 2414 15 min 10 IV Rat 3 2441 680 1 hou
Slide 14: Design of Phase 1b Trial in Parkinson's Disease Patients
Design of Phase 1b Trial in Parkinson’s Disease Patients An Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Participants with Parkinson’s Disease With or Without a Pathogenic GBA1 Mutation Study Extension Treatment Period Follow-up Period 13
Slide 15: Phase 1b Study Objectives
Phase 1b Study Objectives Study Objectives Endpoints Primary To evaluate the safety and tolerability of GT-02287 To characterize the single-dose and steady state Secondary plasma PK profile of GT-02287 To assess levels of GT-02287 in CSF after at least 12 Concentration of GT-02287 in CSF at 4 hours
Slide 16: Phase 1b Initial Data: Demographics and Baseline Characteristics
Phase 1b Initial Data: Demographics and Baseline Characteristics 27 individuals were screened and 21 enrolled from March through September 2025; enrollment in Part 1 is now complete The 21 participants include 3 women and 18 men, 2 treatment-naïve, 2 on DBS, and 18 on levodopa +/- dopamine agonists
Slide 17: Phase 1b Initial Data: Safety and Tolerability
Phase 1b Initial Data: Safety and Tolerability Adverse Events 18 participants have experienced 93 treatment emergent adverse events (TEAEs) as of 03 Sep 2025 The most common TEAEs were headache (n=6 participants), lab abnormalities (n=6), diarrhea (n=6), fatigue (n=4), and nausea (n=3) 85% of TEAEs
Slide 18: Phase 1b Initial Data: Plasma PK
Phase 1b Initial Data: Plasma PK Plasma concentration versus time on Day 1, n=14 Plasma exposures were within the projected therapeutic range and comparable to exposures observed in healthy volunteers in Phase 1 © 2025 - GA I N T HE RAP EUT I C S 18
Slide 19: Phase 1b Initial Data: MDS-UPDRS Changes
Phase 1b Initial Data: MDS-UPDRS Changes • No mean improvement by Day 30, but mean improvement by Day 90 • This indicates that there is no acute, dopaminergic, symptomatic effect, but that a slower effect may occur • Reduction of 1-2 points on UPDRS Part II and 4-8 points on Part III is clinically m
Slide 20: GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease
GT-02287 has Best-in-Class Profile for GBA1-Parkinson's Disease Effect on DiseaseCascade GT-02287 BIA 28-6156 VQ-101 Increases LysosomalGCase Activity ? Reduces ERStress ? ? Reduces ToxicLipid Substrates GCase Mechanism Reduces Aggregated α-Synuclein ? of Action Improves LysosomalFunction Improves M
Slide 21: 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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