Gain Therapeutics, Inc. · NASDAQ:GANX
Gain Therapeutics — Investor Presentation (October 2023)
19 slides from Gain Therapeutics's investor presentation, Oct 18, 2023
Slide 1: Developing Novel Transformative Medicines for Neurodegenerative and Other Diseases
DEVELOPING NOVEL TRANSFORMATIVE MEDICINES FOR PATIENTS WITH NEURODEGENERATIVE AND OTHER DISEASES Corporate Presentation Q4 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: Corporate Highlights
Corporate Highlights From Conception to Fruition: Clinical-Stage Lead Product (Phase 1) Shows Strong Potential to Slow or Stop Parkinson’s Disease Progression Lead candidate GT-02287 targets a genetic subpopulation of Parkinson’s disease with an opportunity to expand into other neurodegenerative dis
Slide 4: GT-02287 Genetically Defined Parkinson's Disease
Lead Clinical Program From Conception to Fruition GT-02287 Genetically Defined Parkinson’s Disease © 2023 - GAIN THERAPEUTICS Corporate Presentation
Slide 5: Parkinson's Disease
Parkinson’s Disease Parkinson’s disease is the second most 10 million people common neurodegenerative disease after globally Alzheimer’s disease and related dementias Symptoms develop when dopamine- 1 million producing neuronal cells die and dopamine people levels drop below normal levels in the U.S
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 Mutation in GBA1 gene, the biggest genetic risk factor for Affects up to 15% development of PD of all PD patients GT-02287 is targeting GCase, restores GCase function, GBA1 PD is more reduces neuroinfl
Slide 7: 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 GBA1 gene disease progression • Misfolded GCase causes ER stress a
Slide 8: GT-02287 Addresses All Pathophysiological Effects in Disease Cascade
GT-02287 Addresses All Pathophysiological Effects in Disease Cascade Resulting from GCase Dysfunction GT-02287 has best-in-class profile Neuro- • Restores GCase function ER Stress inflammation • Acts at the beginning of the disease cascade GBA1 Mitochondrial Toxic Lipid GCase Dysfunction Accumulatio
Slide 9: 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 10: Clinical Program Designed to Deliver Clinical Biomarker Proof of Concept Data
Clinical Program Designed to Deliver Clinical Biomarker Proof of Concept Data in First-in-Patient Study § Randomized, double-blind dose escalation study in healthy volunteers First-in-Human § Single Ascending Dose (SAD) in 40 subjects Data-driven value Phase 1 Clinical Trial § Multiple-Ascending Dos
Slide 11: Key Takeaways on Lead Program GT-02287
Key Takeaways on Lead Program GT-02287 v Mutation of GBA1 gene causes expression of misfolded and dysfunctional GCase enzyme, which results in cascade of altered pathological cellular events that cause neurodegeneration, neuroinflammation, and early onset and more severe form of Parkinson’s disease
Slide 12: Proprietary AI Platform Generates First-in-Class Small Molecule Therapies
Proprietary AI Platform Generates First-in-Class Small Molecule Therapies Combining Novel Biology with Novel Chemistry Gain’s platform specializes in identifying novel allosteric binding sites. These are unique sites that offer several therapeutic advantages and are highly differentiated from other
Slide 13: Company Background
Company Background FINANCIAL METRICS FUNDING AND SUPPORT IPO (Nasdaq: GANX) - March 2021 Corporate Background - Led by BTIG and Oppenheimer & Co. § Established in 2017 CASH POSITION § 35 employees in three locations: - $16.2 million at June 30, 2023 - HQ in Bethesda, Maryland - Lugano, Switzerland C
Slide 14: 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 Garcia CEO SVP, CFO CTO Chief Strategy Officer and SVP Research GM SVP Technical Operations
Slide 15: Strong Board of Directors
Strong Board of Directors (>$30B IN TRANSACTIONS) Khalid Islam 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 Independen
Slide 16: 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 GBA1-Parkinson’s Disease/ Neurodegenerative Diseases GBA1 Idiopathic Parkinson’s Disease Lysosomal Storage Disorders Gaucher Disease GBA1 GM1 Gangliosid
Slide 17: Gain Therapeutics - Key Takeaways
Gain Therapeutics - Key Takeaways v Gain’s clinical candidate GT-02287 has the potential to stop progression of Parkinson’s disease addressing a large market opportunity - GBA1 PD - $3B - idiopathic PD - $4B v GT-02287 will generate high value inflection points in 2024 - Safety and tolerability in h
Slide 18: Corporate Highlights
Corporate Highlights From Conception to Fruition: Clinical-Stage Lead Product (Phase 1) Shows Strong Potential to Slow or Stop Parkinson’s Disease Progression Lead candidate GT-02287 targets a genetic subpopulation of Parkinson’s disease with an opportunity to expand into other neurodegenerative dis
Slide 19: Company Contacts
Company Contacts Matthias Alder, CEO malder@gaintherapeutics.com Evan Ballantyne, CFO eballantyne@gaintherapeutics.com © 2023 - GAIN THERAPEUTICS Non-Confidential Presentation
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