Core Insights - Gain Therapeutics, Inc. is participating in BTIG's Annual Virtual Biotechnology Conference on July 29-30, 2025, with a fireside chat scheduled for July 30 at 3:20 PM ET [1][2] Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric therapies [3] - The lead drug candidate, GT-02287, is currently in a Phase 1b clinical trial for treating Parkinson's disease, with potential applications in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [3] - The company has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [3] Technology and Approach - Gain employs a unique approach to discover novel allosteric small molecule modulators that can restore or disrupt protein function [4] - The Magellan™ platform is utilized to accelerate drug discovery and develop disease-modifying treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4]

Core Viewpoint - Gain Therapeutics, Inc. has announced a public offering of 4,501,640 shares of common stock and warrants, aiming to raise approximately $7.0 million for the development of its lead product candidate GT-02287 and general corporate purposes [1][3][4]. Group 1: Offering Details - The public offering price is set at $3.11 for two shares of common stock and one warrant, translating to an effective price of $1.55 per share and $0.01 per warrant [1]. - The warrants will have an exercise price of $1.65 per share, are immediately exercisable, and will expire five years after issuance [1]. - The underwriter has a 30-day option to purchase up to an additional 15% of shares and/or warrants at the public offering price [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized for the clinical and nonclinical development of GT-02287, targeting neurodegenerative diseases such as GBA1 Parkinson's disease, as well as for general corporate purposes [4]. Group 3: Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric therapies, with GT-02287 currently in a Phase 1b clinical trial for Parkinson's disease [7]. - The company employs its Magellan™ platform to discover novel allosteric small molecule modulators aimed at treating various difficult-to-treat disorders, including neurodegenerative diseases and rare genetic disorders [8].

Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric small molecule therapies, with GT-02287 as its lead candidate for treating Parkinson's disease [4][9] - The drug is designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which is impaired due to mutations in the GBA1 gene, a common genetic abnormality associated with Parkinson's disease [4][5] Clinical Trial Details - The Phase 1b trial aims to evaluate the safety and tolerability of GT-02287 over a 90-day dosing period in participants with Parkinson's disease, with enrollment ongoing across seven sites in Australia [7][9] - The company plans to extend the screening window for participants until July 31, 2025, and is seeking to extend the dosing period beyond the current 90-day limit [2][3] Preclinical and Clinical Evidence - Preclinical studies have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease, suggesting a potential disease-modifying effect [5][6] - Results from a Phase 1 study in healthy volunteers indicated favorable safety and tolerability, with a greater than 50% increase in GCase activity observed [6][9] Funding and Support - Gain Therapeutics has received funding support from organizations such as The Michael J. Fox Foundation for Parkinson's Research and the Eurostars-2 joint program, which is co-funded by the European Union [8][9]

Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] Company Progress - The company plans to extend the screening window for participants until July 31, 2025, in response to clinician and patient interest [2] - Gain Therapeutics is also seeking to extend the dosing period of the Phase 1b trial beyond the current 90-day limit, with long-term toxicology studies nearing completion [2] - The company anticipates that the extension will provide valuable insights for the Phase 2 protocol design planned for early 2026 [2] Drug Candidate Overview - GT-02287 is an allosteric enzyme modulator aimed at treating Parkinson's disease, particularly in patients with or without a GBA1 mutation [4][9] - The drug has shown promise in preclinical models by restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) and addressing various pathological features associated with Parkinson's disease [4][5] - Previous Phase 1 studies indicated favorable safety and tolerability, with a significant increase in GCase activity observed [6][7] Market Context - There are over 1 million Americans diagnosed with Parkinson's disease, highlighting a significant unmet medical need for effective treatments that can modify disease progression rather than just alleviate symptoms [3][5] - Gain Therapeutics has received funding support from notable organizations, including The Michael J. Fox Foundation and the European Union, to advance its research and development efforts [8][9]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [2][3] - The company’s lead drug candidate, GT-02287, is currently in a Phase 1b clinical trial for treating Parkinson's disease and has potential applications in other neurodegenerative diseases [2][3] Company Overview - Gain Therapeutics is pioneering the discovery of allosteric therapies, utilizing its advanced Magellan™ platform to accelerate drug discovery [3] - The company is also developing multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [2] Upcoming Events - Gene Mack, President and CEO, will present at the BIO 2025 conference on June 18, 2025, at 1:45 p.m. EST [1] - Additionally, Gene Mack will participate in a virtual fireside chat at the H.C. Wainwright 6th Annual Neuro Perspectives Hybrid Conference on June 17, 2025, at 7:00 a.m. EST [5]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [1][2] - The company will present at the GBA1 Meeting 2025 on June 5, 2025, with a focus on its lead drug candidate, GT-02287, for treating Parkinson's disease [1][2] - Gain's innovative Magellan™ platform is designed to accelerate drug discovery for various difficult-to-treat disorders, including neurodegenerative diseases and rare genetic disorders [3] Company Overview - Gain Therapeutics is engaged in the discovery and development of allosteric therapies, with GT-02287 currently in a Phase 1b clinical trial for Parkinson's disease [2] - The company has additional potential applications for GT-02287 in treating Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [2] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [2] Presentation Details - The oral presentation is scheduled for June 5, 2025, at 11:20 p.m. EST, presented by Dr. Jonas Hannestad, Chief Medical Officer of Gain Therapeutics [2]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-40237 GAIN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 85-1726310 (Sta ...

Exhibit 99.1 Gain Therapeutics Reports Financial Results for First Quarter 2025 and Provides Corporate Update Upcoming Anticipated Milestones Q1 2025 Financial Results Research and Development (R&D) expenses decreased by $0.2 million to $2.3 million for the three months ended March 31, 2025, as compared to $2.5 million for the three months ended March 31, 2024. The decrease in research and development expenses was primarily related to recognition of research grant income, a tax rebate in Australia, and opti ...

Core Insights - Gain Therapeutics, Inc. reported significant progress in its clinical development of GT-02287 for Parkinson's disease, with the first administration of the drug in a Phase 1b study and expectations for a biomarker analysis by mid-2025 [2][4][7] Corporate Updates - The Phase 1b study for GT-02287 began enrollment in early March 2025, with completion expected by the end of July 2025 [2][7] - Gene Mack was appointed as President and CEO effective January 6, 2025, succeeding the previous CFO [7] - A Clinical Advisory Board has been formed to support the advancement of GT-02287 through late-stage clinical development [7] Financial Results - Research and Development (R&D) expenses decreased by $0.2 million to $2.3 million for Q1 2025 compared to $2.5 million in Q1 2024, attributed to research grant income and cost optimization [4] - General and Administrative (G&A) expenses increased by $0.2 million to $2.1 million for Q1 2025 compared to $1.9 million in Q1 2024, primarily due to higher legal and professional fees [5] - The net loss for Q1 2025 was $0.16 per share, an improvement from a loss of $0.22 per share in Q1 2024 [6] Clinical Development Highlights - GT-02287 is being evaluated for the treatment of Parkinson's disease with or without a GBA1 mutation, with a focus on safety and tolerability after three months of dosing [11] - The drug has shown promising preclinical data indicating a potential disease-modifying effect in both GBA1-PD and idiopathic PD models [9] - The company anticipates submitting an Investigational New Drug (IND) application to the FDA by the end of 2025 [2][7] Asset and Liability Overview - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $9.1 million, down from $10.4 million at the end of 2024 [6][19] - Total liabilities increased to $5.84 million as of March 31, 2025, compared to $4.78 million at the end of 2024 [19]

Core Insights - Gain Therapeutics, Inc. announced new evidence supporting GT-02287's neuroprotective effects and potential as a disease-modifying therapy for Parkinson's disease at the IAPRD 30th World Congress [1][3] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric small molecule therapies, with GT-02287 as its lead candidate for treating Parkinson's disease [11][12] - GT-02287 is designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired due to mutations in the GBA1 gene, a common genetic abnormality associated with Parkinson's disease [5][11] Research and Development - Recent preclinical data indicate that GT-02287 improves lysosomal and mitochondrial function, reduces α-synuclein aggregation, and promotes neuronal survival by preventing apoptosis signals [3][5] - The drug has shown a disease-modifying effect in preclinical models of both GBA1-PD and idiopathic PD, suggesting it may slow or stop disease progression [6][11] - A Phase 1 study demonstrated favorable safety and tolerability, with over a 50% increase in GCase activity in participants receiving GT-02287 [7][8] Clinical Trials - GT-02287 is currently being evaluated in a Phase 1b clinical trial for Parkinson's disease, focusing on safety and tolerability after three months of dosing [8] - The trial is enrolling participants across seven sites in Australia [8] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research and other organizations to advance its lead program [9][10]