Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (February 2024)
28 slides from Gain Therapeutics's investor presentation, February 2024
Slide 1: Unfolding the Next Generation of Allosteric Small Molecule Therapies
UNFOLDING THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES TO MEET PATIENT NEEDS Corporate Presentation February 2024 © 2024 - GAIN THERAPEUTICS Corporate Presentation
Slide 2: Forward-Looking Statements
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
Slide 3: Corporate Highlights
Corporate Highlights From Conception to Fruition: Clinical-Stage Lead Product GT-02287 (Phase 1) Shows Strong Potential to Slow or Stop Parkinson’s Disease Progression Lead indication for GT-02287 is targeting a genetic subpopulation of Parkinson’s disease with an opportunity to expand into other ne
Slide 4: Lead Clinical Program - From Conception to Fruition GT-02287
Lead Clinical Program - From Conception to Fruition GT-02287 Genetically Defined Parkinson’s Disease © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 5: Parkinson's Disease
Parkinson’s Disease Our goal is to develop a next-generation therapy to prevent disease progression § Parkinson’s disease is the second most 10 million common neurodegenerative disease after people globally Alzheimer’s disease and related dementias § Symptoms develop when dopamine- 1 million produci
Slide 6: Lead Indication for GT-02287 - GBA1 Parkinson's Disease
Lead Indication for GT-02287 - GBA1 Parkinson’s Disease GBA1 and GCase are fully validated targets GT-02287 is targeting GCase, restores GCase function, reduces neuroinflammation, demonstrates neuroprotection, and has potential to slow or halt disease progression § Mutations in GBA1 gene are the big
Slide 7: Clinical Candidate GT-02287 Corrects Dysfunctional GCase Enzyme
Clinical Candidate GT-02287 Corrects Dysfunctional GCase Enzyme GT-02287 restores GCase function – targeting root cause of GBA1-PD Normal GCase No Disease GBA1 Gene GCase Enzyme Elevated Levels Dysfunctional GCase • Toxic substrates PD • Alpha-synuclein Triggers disease cascade resulting • Neuroinfl
Slide 8: Dysfunctional GCase Triggers Disease Cascade Leading to Neurodegeneration
Dysfunctional GCase Triggers Disease Cascade Affecting Multiple Organelle Functions and Leading to Neurodegeneration Healthy Dopaminergic Neuron Diseased Dopaminergic Neuron GBA Gene Mutated GBA Gene Nucleus Nucleus Normal Defective GCase GCase Endoplasmic Endoplasmic Reticulum Reticulum Lysosome Ly
Slide 9: Treatment with GT-02287 restores GCase Function
Treatment with GT-02287 restores GCase Function, which improves Disease Cascade and Neuronal Survival Dopaminergic Neuron with Restored GCase Function Confirmatory Data Generated with GT-02287 ER Stress Marker GCase Activity Substrate Depletion Mutated 8 100 [Poster presentation 80 6 GBA Gene at upc
Slide 10: Preclinical Proof of Concept of GT-02287 in Parkinson's Disease
Preclinical Proof of Concept of GT-02287 in Parkinson’s Disease Best-in-class profile in industry-standard assays and models demonstrates disease-modifying potential GBA1 gene mutation expresses Effect of GT-02287 in Mechanism misfolded GCase Effect of GT-02287 in of Action Animal Models of Cell-bas
Slide 11: GT-02287 addresses the Primary Pathophysiological Effects in Disease Cascade
GT-02287 addresses the Primary Pathophysiological Effects in Disease Cascade Resulting from GCase Dysfunction GT-02287 has best-in-class profile of known GCase-targeting drug candidates Motor § Brain-penetrant small molecule that acts at the ER Stress Deficits beginning of the disease cascade § Only
Slide 12: GT-02287 Increases Neuronal Survival and Improves Motor Function in CBE Mouse Model
GT-02287 Increases Dopaminergic and Cortical Neuronal Survival, Decreases NfL in Plasma, and Improves Neuro-muscular and Motor Function in the CBE Mouse Model Design: 8-10 weeks of age male C57BL/6 mice received daily CBE i.p. injections and oral q.d. drug treatment for 14 days. Wire hang test and b
Slide 13: GT-02287 has the potential to slow or stop disease progression
In Vitro and In Vivo Results Support Disease Modifying Potential of GT-02287 in GBA1-Parkinson’s Disease ➤ Increased GCase enzyme levels ➤ ➤ Increased GCase transport to the lysosome Depletion of toxic substrate Restoring Enzyme Function ü ➤ Depletion of α-synuclein ➤ Reduced ER stress ➤ ➤ Improved
Slide 14: Upcoming Expected Milestones and Value Inflection Points
Clinical Program Designed to Deliver Clinical Biomarker Proof of Concept Data in Phase 1 Study § Randomized, double-blind dose escalation study in healthy volunteers § Single Ascending Dose (SAD) in up to 40 subjects Data-driven First-in-Human § Multiple-Ascending Dose (MAD) in up to 32 subjects (14
Slide 15: Key Takeaways on Lead Program GT-02287
Key Takeaways on Lead Program GT-02287 Mutation of GBA1 gene leads to misfolded and dysfunctional GCase enzyme, which results in cellular events that cause neurodegeneration, neuroinflammation, and early onset and more severe form of Parkinson’s disease Clinical lead candidate GT-02287 restores phys
Slide 16: Magellan Platform
Magellan Platform TM Exploring uncharted Biology and Vast Chemical Space to Discover Allosteric Small Molecule Therapies © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 17: Proprietary Magellan Platform Identifies Potential First-in-Class Therapies
Proprietary Magellan Platform Identifies Potential First-in-Class Small Molecule Therapies MagellanTM is the next generation of Gain’s original SEE-Tx® (Site-Directed Enzyme Enhancement Therapy) platform Combines physics-based methods and AI/ML technology Enhanced with virtual screening capabilities
Slide 18: Combines AI with Physics-Based Methods
Proprietary Magellan Platform Identifies Potential First-in-Class Small Molecule Therapies Combines AI with Physics-Based Methods Gain’s platform specializes in identifying novel allosteric binding sites. These are unique sites that offer several therapeutic advantages and are highly differentiated
Slide 19: Magellan Platform Built to Screen Trillions of Compounds
Gain’s Magellan Platform Built to Screen Trillions of Compounds Magellan uses AI to screen virtual libraries with 50 billion TM compounds, resulting in the following benefits: § Initial hits are novel and patentable and can be chemically synthesized quickly § Larger selection of compounds results in
Slide 20: Corporate Background
Corporate Background © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 21: Company Background
Company Background F I N A N C I A L A N D S T O C K D ATA A N A LY S T C O V E R A G E IPO (Nasdaq: GANX) BTIG: - March 2021 Tom Shrader: TP $10.00 - Buy - Led by BTIG and Oppenheimer & Co. Oppenheimer & Co. C O R P O R AT E B A C K G R O U N D Hartaj Singh: TP $9.00 - Buy § Established in 2017 CAS
Slide 22: Supported by Industry Leading Foundations and KOLs
Supported by Industry Leading Foundations and KOLs FUNDING AND SUPPORT InnoSuisse grant in Q2 2023 $2.8 EuroStars grant in Q2 2023 $0.5 million million to fund GT-02287 clinical program © 2024 - GAIN THERAPEUTICS Corporate Presentation Page 22
Slide 23: Novel Pipeline Generated from Platform
Novel Pipeline Generated from Platform THERAPEUTIC AREA INDICATION TARGET DISCOVERY RESEARCH PRECLINICAL PHASE 1 Parkinson’s Disease GCase Neurodegenerative Diseases Dementia with Lewy Bodies GCase Alzheimer’s Disease Lysosomal Storage Disorders Gaucher Disease GCase GM1 Gangliosidosis GLB1 Krabbe D
Slide 24: Experienced Leadership
Experienced Leadership EXTENSIVE BIOTECH AND PHARMA EXPERIENCE (>$15B TRANSACTIONS) C. Evan Ballantyne Manolo Bellotto, PhD Terenzio Ignoni, AnaTaylor, Joanne MariaPhD Matthias Alder PharmD CEO SVP, CFO Chief Strategy Officer and SVPGarcia Research GM SVP Technical Operations Collazo, PhD VP of Rese
Slide 25: Strong Board of Directors
Strong Board of Directors (>$30B IN TRANSACTIONS) Khalid Islam, PhD Matthias Alder Eric I. Richman Gwen Melincoff Hans Peter Hasler Dov Goldstein, MD Jeffrey Riley Claude Nicaise, MD Founder and Chairman CEO and Member Independent Member Independent Member Independent Member Independent Member Indep
Slide 26: Gain Therapeutics – Key Takeaways
Gain Therapeutics – Key Takeaways © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
Slide 27: Gain Therapeutics - Key Takeaways
Gain Therapeutics - Key Takeaways Gain’s clinical candidate GT-02287 has the potential to slow or stop progression of Parkinson’s disease addressing a large market opportunity • GBA1 PD - $3B • Idiopathic PD - $4B GT-02287 is expected to generate significant clinical data-based value inflection poin
Slide 28: Company Contacts
Company Contacts: Matthias Alder, CEO malder@gaintherapeutics.com Evan Ballantyne, CFO eballantyne@gaintherapeutics.com © 2024 - GAIN THERAPEUTICS Non-Confidential Presentation
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