Here are 1-3 brief analogies to describe Gain Therapeutics (GANX):

• Gain Therapeutics is like Vertex Pharmaceuticals, but focused on fixing *misfolded proteins* for a range of rare genetic and neurological disorders.
• Gain Therapeutics is like Sarepta Therapeutics, but instead of exon-skipping, they use small molecules to correct *misfolded proteins* for various rare genetic and neurological diseases.

• Morquio B Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat Morquio B disease.
• GM1 Gangliosidosis (GM1) Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat GM1 gangliosidosis.
• Neuronopathic Gaucher Disease Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat neuronopathic Gaucher disease.
• GBA1 Parkinson's Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat GBA1 Parkinson's disease.
• Krabbe Disease Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat Krabbe disease.
• Mucopolysaccharidosis Type 1 Therapy Candidate: A structurally targeted allosteric regulator candidate in development to treat Mucopolysaccharidosis type 1 disease.

Gain Therapeutics (GANX) is a biotechnology company focused on the early-stage development of therapies for rare genetic diseases and neurological disorders. As of its current stage, the company is primarily engaged in research and development and has not yet brought any products to market.

Therefore, Gain Therapeutics does not currently have major customers in the traditional sense of selling approved products or therapies to either other companies or individuals.

In the biotechnology industry, companies like Gain Therapeutics typically aim to develop drug candidates through various pre-clinical and clinical stages. Should these candidates prove successful, the company would then typically seek to partner with larger pharmaceutical companies through licensing agreements, collaborations, or co-development deals. These partners would eventually be responsible for the commercialization, manufacturing, marketing, and sale of any approved therapies to healthcare providers or patients.

Gene Mack President and Chief Executive Officer

Gene Mack was appointed President and Chief Executive Officer of Gain Therapeutics in January 2025, having previously served as interim CEO since June 2024 and Chief Financial Officer since April 2024. He brings over 25 years of experience in the life sciences sector, encompassing clinical research, financing and capital markets, investing, corporate strategy, and business development. Prior to joining Gain Therapeutics, Mr. Mack served as CFO at privately held Imcyse SA from 2021 to 2023. Before that, he was CFO at OncoC4, a privately held biotechnology company that spun out of Merck & Co.'s $475 million acquisition of OncoImmune in 2020, where he had also served as CFO. He has held CFO roles for several development- and commercial-stage biopharmaceutical companies, raising over $350 million through IPOs and other equity transactions. Earlier in his career, he spent nearly 15 years as a senior publishing analyst covering biotechnology and life sciences for investment banks, including Gruntal & Co, Lazard, Mizuho, and HSBC. Mr. Mack holds a B.S. in Biochemistry and an M.B.A. in Finance from Fordham University.

Gianluca Fuggetta Senior Vice President of Finance and Principal Financial Officer

Gianluca Fuggetta was appointed Senior Vice President of Finance and Principal Financial Officer of Gain Therapeutics in January 2025. He joined the company in July 2022 and has progressively taken on greater responsibilities, including Finance Director, Senior Finance Director, and Vice President of Finance, Principal Accounting Officer since January 2023.

Jonas Hannestad, M.D., Ph.D. Chief Medical Officer

Dr. Jonas Hannestad joined Gain Therapeutics as Chief Medical Officer in March 2024. He brings 25 years of translational and early clinical development experience in the Central Nervous System (CNS). Dr. Hannestad's academic work focused on immuno-neurology, particularly the effect of systemic inflammation on myeloid cells in the brain. His drug development expertise spans multiple indications, including Parkinson's, Alzheimer's, and ALS. Previously, he served as Chief Medical Officer at Tranquis Therapeutics and Capacity Bio, Senior Vice President of Clinical Development at Alkahest, Medical Director at Denali Therapeutics, and Director of Neuroscience Discovery Medicine at UCB Pharma. Prior to his industry career, Dr. Hannestad led the Clinical Neuroscience Research Unit at the Yale School of Medicine.

Terenzio Ignoni Chief Tech/Sci/R&D Officer

Terenzio Ignoni serves as the Chief Tech/Sci/R&D Officer at Gain Therapeutics.

Key Risks to Gain Therapeutics (GANX):

1. Clinical Trial Failure and Regulatory Risk: Gain Therapeutics' core business risk lies in the success or failure of its clinical programs, particularly its lead candidate, GT-02287, for Parkinson's disease. The company's valuation is heavily reliant on positive clinical trial outcomes. Any unexpected safety issues, negative trial results, or delays in advancing GT-02287 through its planned Phase 2 trial could be catastrophic and significantly erode investor confidence. Furthermore, the company faces regulatory hurdles, needing to obtain necessary approvals from bodies like the FDA for its therapies to reach the market.

2. Limited Financial Runway and Need for Future Capital: As a clinical-stage biotechnology company, Gain Therapeutics currently has no revenue-generating products and operates at a significant loss, consistently burning through cash to fund its research and development activities. As of Q3 2025, the company's cash and cash equivalents were insufficient to fund operations for a full year, raising substantial doubt about its ability to continue as a going concern without securing new capital. This necessitates frequent capital raises through public offerings, which can lead to the dilution of existing shareholders.

3. Intense Competition: The market for treatments targeting neurodegenerative diseases, particularly Parkinson's disease, is highly competitive. Gain Therapeutics' lead candidate, GT-02287, must compete with established therapies and numerous other novel candidates in development from other companies. To succeed, Gain Therapeutics will need to demonstrate clear and superior clinical benefits and safety profiles to differentiate its products in this crowded therapeutic landscape.

Gain Therapeutics (symbol: GANX) is developing therapies for several rare genetic diseases and neurological disorders. The addressable markets for their main products or services are as follows:

• Morquio B Disease: The global Morquio Syndrome (MPS IV, which includes Morquio A and B) treatment market was valued at USD 164.62 million in 2024 and is projected to grow to USD 273.44 million by 2033, exhibiting a compound annual growth rate (CAGR) of 5.8%. North America is expected to be the largest market for Morquio Syndrome (MPS-IV) drugs, accounting for over 43.7% of the market share in 2025.
• GM1 Gangliosidosis (GM1): The global GM1 gangliosidosis market size was valued at USD 1.11 billion in 2024 and is expected to reach USD 7.27 billion by 2032, with a CAGR of 26.50%. North America dominated the GM1 gangliosidosis market, holding the largest revenue share of 42.3% in 2024.
• Neuronopathic Gaucher Disease: The global Gaucher disease treatment market, which includes neuronopathic types (Type 2 and Type 3), was valued at USD 2.14 billion in 2024 and is expected to register a CAGR of 2.8%. The Type 3 segment, which encompasses neuronopathic Gaucher disease, is projected to grow substantially due to increasing awareness and diagnosis. North America accounted for 40.42% of the global revenue share in 2024.
• GBA1 Parkinson's Disease: While a specific market size for GBA1 Parkinson's Disease alone was not available, GBA1 mutations are identified as a significant genetic risk factor for Parkinson's disease, found in about 10.4% of Parkinson's patients globally. The global Parkinson's disease therapeutics market was valued at USD 6.2 billion in 2024 and is projected to reach USD 13.3 billion by 2034, growing at a CAGR of 8.1%. North America dominated this market with a 39.5% share in 2024.
• Krabbe Disease: The global Krabbe Disease Treatment Market size was valued at USD 3.04 billion in 2025 and is likely to reach USD 6.04 billion by 2035, demonstrating a CAGR of more than 7.1%. North America is anticipated to account for approximately 35.2% of the revenue share by 2035.
• Mucopolysaccharidosis Type 1 (MPS I) Disease: The global mucopolysaccharidosis type I market was valued at US$ 625 million in 2022 and is expected to register a revenue CAGR of 7.5% to reach US$ 1,288 million by 2031. North America held the largest revenue share of the global mucopolysaccharidosis type I market in 2022. Separately, the Mucopolysaccharidosis I Market Size in the 7MM (United States, EU4, UK, and Japan) was approximately USD 145 million in 2024 and is projected to grow to USD 297 million by 2034, with a CAGR of 8.0%. The United States accounted for 48% of the total 7MM market in 2024.

For Gain Therapeutics (GANX), a clinical-stage biotechnology company, the expected drivers of future revenue growth over the next 2-3 years are intrinsically tied to advancing its drug candidates through development and securing strategic partnerships, rather than immediate product sales, as the company is currently pre-revenue.

1. Successful Progression of GT-02287 in Clinical Trials for Parkinson's Disease: Gain Therapeutics' lead drug candidate, GT-02287, is being developed for Parkinson's disease (PD). The company anticipates initiating a placebo-controlled Phase 2 clinical trial for GT-02287 in the third quarter of 2026. Positive results from this and subsequent clinical trials are critical for demonstrating the drug's efficacy and safety, which are necessary steps toward regulatory approval and eventual commercialization or out-licensing. Successful advancement of GT-02287, which has shown potential as a disease-modifying therapy by targeting the underlying biology of PD, would be a significant driver of future value and potential revenue.
2. Expansion and Advancement of its Pipeline through the Magellan™ Platform: Gain Therapeutics leverages its proprietary Site-Directed Enzyme Enhancement Therapy (SEE-Tx) or Magellan™ computational platform to discover novel allosteric binding sites and identify small molecules for a range of diseases beyond Parkinson's, including other neurodegenerative disorders, lysosomal storage disorders (such as Morquio B, GM1 gangliosidosis, neuronopathic Gaucher disease, Krabbe, and Mucopolysaccharidosis type 1), metabolic diseases, and oncology. The ability to identify and advance additional promising drug candidates from its diverse preclinical pipeline into clinical development, or to form partnerships based on these assets, will broaden the company's future revenue potential.
3. Formation of Strategic Partnerships and Licensing Agreements: As a clinical-stage biotechnology company with no commercialized products, a significant driver of future revenue will come from strategic collaborations and licensing agreements with larger pharmaceutical companies. These partnerships can provide crucial non-dilutive funding through upfront payments, milestone payments upon achieving specific development or regulatory goals, and potential future royalties on product sales. Such collaborations validate the company's technology and pipeline, accelerating the development and commercialization of its drug candidates.

Share Issuance

• Gain Therapeutics raised approximately $46 million in gross proceeds from its Initial Public Offering (IPO) in March 2021, which included the full exercise of the underwriters' overallotment option.
• In June 2024, the company announced the pricing of a public offering of common stock and pre-funded warrants, expecting to receive approximately $11.0 million in gross proceeds.
• In July 2025, Gain Therapeutics announced the pricing of an underwritten public offering of common stock and warrants, anticipated to generate approximately $7.0 million in gross proceeds.

Inbound Investments

• Gain Therapeutics receives grant support from organizations such as the Michael J. Fox Foundation for Parkinson's research, The Silverstein Foundation for Parkinson's with GBA research, and Innosuisse (Swiss Innovation Agency).

Capital Expenditures

• In Q3 2024, Gain Therapeutics invested $945 in capital expenditures.
• For the nine months leading up to September 30, 2025, the company's operating cash burn was approximately $13.8 million, largely driven by Research and Development (R&D) expenses for its clinical pipeline.
• Research and Development expenses were $2.8 million in Q3 2025, reflecting the costs associated with the ongoing Phase 1b clinical trial for its lead product candidate, GT-02287.