Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (2023)
22 slides from Gain Therapeutics's investor presentation, 2023
Slide 1: Leading Computational Drug Discovery For Allosteric Small Molecule Therapies
Leading Computational Drug Discovery For Allosteric Small Molecule Therapies Corporate Presentation Q3 2023 © 2023 - GAIN THERAPEUTICS Corporate Presentation
Slide 2: Forward-Looking Statement
Forward-Looking Statement Forward-Looking Statements Certain statements set forth in this presentation contain forward-looking statements that reflect the Company’s plans, beliefs, expectations and current views with respect to, among other things, future events and financial performance (collective
Slide 3: Gain Therapeutics – Value Drivers
Gain Therapeutics – Value Drivers SEE-Tx® Drug Allosteric Protein Product Pipeline Discovery Platform Modulation Current drugs bind to known active sites Gain’s drugs bind to novel allosteric sites ▪ Fast – accelerates drug discovery ▪ Small molecule drugs designed to ▪ Platform-generated pipeline f
Slide 4: SEE-Tx Platform Uncovers Novel Allosteric Small Molecule Therapies
SEE-Tx® Platform Uncovers Novel Allosteric Small Molecule Therapies SEE-Tx® integrates AI, structural biology, physics-based models and supercomputing power 3D Protein Structure Allosteric experimentally-derived or Modulator Gain of Stabilization AI-generated Function Hit compounds selected to Desta
Slide 5: SEE-Tx Platform Unlocks Allosteric Drug Discovery
SEE-Tx® Platform Unlocks Allosteric Drug Discovery Small molecule drugs designed to restore or disrupt protein function Active Binding Site Allosteric Binding Site • Site where natural ligand • Any site that is not the active binding site binds to the protein • Emerging targeting approach unlocked •
Slide 6: Diversified Pipeline of Allosteric Modulators Across Four Therapeutic Areas
Diversified Pipeline of Allosteric Modulators Across Four Therapeutic Areas THERAPEUTIC AREA INDICATION GENE DISCOVERY RESEARCH PRECLINICAL PHASE 1 Neurodegenerative Diseases Parkinson’s Disease GBA1 Lysosomal Storage Disorders Gaucher Disease GBA1 GM1 Gangliosidosis GLB1 Krabbe Disease GALC Metabol
Slide 7: GT-02287 Parkinson's Disease
Lead Program GT-02287 Parkinson’s Disease Beta-glucocerebrosidase (GCase) © 2023 - GAIN THERAPEUTICS Corporate Presentation
Slide 8: Lead Indication - GBA1-Parkinson's Disease
Lead Indication - GBA1-Parkinson’s Disease Parkinson’s Disease Background ▪Parkinson’s disease (PD) ▪Affects approx. 10 million people worldwide and 1 million people in the US ▪PD symptoms develop when dopamine levels in the brain drop below normal levels ▪GBA1-Parkinson’s Disease (GBA1-PD) GBA1-PD
Slide 9: GBA1 PD Program: Drug Candidate GT-02287
GBA1 PD Program: Drug Candidate GT-02287 GT-02287 is a GCase-targeting small molecule with robust efficacy and good drug-like properties GCase Target in vitro in vivo Drug Engagement Proof of Concept Proof of Concept Properties • Dose-dependent and • Enhancement of • Enhancement of GCase • Oral admi
Slide 10: Mechanism of Action of GT-02287
Mechanism of Action of GT-02287 Restoring GCase Enzyme Function to Stop Disease Progression Misfolded GCase enzyme leads GT-02287 restores enzyme function, to cell damage and disease which prevents cell damage and development Mutated disease progression GBA1 gene • Misfolded GCase is targeted for •
Slide 11: Key Findings for GT-02287 in Patient-Derived Neuronal Cells
Key Findings for GT-02287 in Patient-Derived Neuronal Cells “Disease in a Dish” NUCLEUS Mutated GBA1 Gene Increased GCase Levels Misfolded Protein Rescue of Folding Increased Lysosomal GCase Levels In vitro data confirms MoA of GT-02287 LYSOSOME Enhanced Depleted α-Synuclein Enzymatic Activity Impro
Slide 12: Key Findings for GT-02287 in Parkinson's Disease Animal Models
Key Findings for GT-02287 in Parkinson’s Disease Animal Models NUCLEUS Mutated GBA1 Gene Increased GCase Levels Misfolded Protein Rescue of Folding Increased Lysosomal GCase Levels In vivo data confirms MoA Depleted α-Synuclein and Sphingolipids and LYSOSOME Improved Lysosomal Function demonstrates
Slide 13: GT-02287 decreases α-synuclein and microgliosis in a Rotenone Rat Model
GT-02287 decreases α-synuclein and microgliosis in the Substantia Nigra in a Rotenone Rat Model Aggregated Total α-synuclein Phosphorylated α-synuclein Iba-1 α-synuclein in SN, Vehicle vehicle Controls controls Rotenone + GT-02287 Rotenone rotenone rotenone(30mg/kg) + GT-02287 Rotenone (60 mg/kg) ro
Slide 14: GT-02287 increases TH staining and dopamine levels in striatum
GT-02287 increases TH staining and dopamine levels in striatum and improves rearing behavior in the Rotenone Rat Model Total Rearings Tyrosine Hydroxylase Staining Dopamine Tyroxine hydroxylase in Striatum Supported + Unsupported ✱ 5 ✱✱✱✱ ✱ IHC staining scoring 4 3 (0-4) 2 1 0 Vehicle vehicle Contro
Slide 15: GT-02287 decreases aggregated α-synuclein and improves motor strength in mouse model
GT-02287 decreases aggregated α-synuclein and microgliosis, increases TH staining and improves motor strength on the CBE + α-syn PFFs Mouse Model Aggregated Iba-1 Tyrosine Hydroxylase Wire Hang Test α-synuclein Wire hang test ✱✱✱✱ ✱✱✱✱ ✱✱ 300 Vehicle Latency to fall (sc) CBE (1 200 α-syn +GT-02 +GT-
Slide 16: GT-02287 Improves Locomotion in a Rotenone-induced Rat PD Model
GT-02287 Improves Locomotion in a Rotenone-induced Rat PD Model Differences in locomotion (open field test) obtained by video-tracking software system, of a representative rotenone-treated rat versus a rotenone-treated rat with oral admin of GT-02287 (60 mg/kg) for 7 days A. Representative videos of
Slide 17: Upcoming Program Milestones
Upcoming Program Milestones ▪ Completion of GLP-Toxicity Studies ✓ ✓ 1H 2023 ▪ Completion of CMC activities in preparation of Phase 1 Clinical Trial ▪ Commence Phase 1 Clinical Trial 2H 2023 ▪ Healthy volunteer study ▪ SAD/MAD design ▪ Conclusion of Phase 1 Clinical Trial 1H 2024 ▪ Pre-IND Meeting w
Slide 18: GT-02287 Therapeutic Potential in Multiple Neurodegenerative Diseases
GT-02287 has Therapeutic Potential in Multiple Neurodegenerative Diseases each with $1B+ Peak Sales Potential US Market Opportunity 3M+ NEURONOPATHIC GAUCHER DISEASE • Monogenic rare childhood disease caused by GBA1 mutations • No therapies available for neuronopathic GD 2M ✓ GT-02287 has shown effe
Slide 19: Key Takeaways and Upcoming Milestones
Key Takeaways and Upcoming Milestones Major Inflection Point – Transitioning to Clinical Stage Biotech Company SEE-Tx® Drug Allosteric Protein Product Pipeline Discovery Platform Modulation ▪ Fast – accelerates drug discovery ▪ Small molecule drugs designed to ▪ Platform-generated pipeline offers fr
Slide 20: Experienced Leadership
Experienced Leadership EXTENSIVE BIOTECH AND PHARMA EXPERIENCE (>$15B TRANSACTIONS) C. Evan Ballantyne Xavier Barril, PhD Manolo Bellotto, PhD Terenzio Ignoni, AnaTaylor, Joanne MariaPhD Matthias Alder PharmD SVP, CFO Chief Strategy Officer and Garcia SVP Research CEO CTO Collazo, PhD GM SVP Technic
Slide 21: Strong Board of Directors
Strong Board of Directors (>$30B IN TRANSACTIONS) Khalid Islam Matthias Alder Eric I. Richman Gwen Melincoff Hans Peter Hassler Dov Goldstein, MD Jeffrey Riley Claude Nicaise, MD Founder and Chairman CEO and Member Independent Member Independent Member Independent Member Independent Member Independe
Slide 22: Company Contacts
Company Contacts Matthias Alder, CEO malder@gaintherapeutics.com Evan Ballantyne, CFO eballantyne@gaintherapeutics.com © 2023 - GAIN THERAPEUTICS Non-Confidential Presentation