Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [6] - The company's 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 [6] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [6] Upcoming Event Participation - Gain Therapeutics will participate in the 2025 Maxim Growth Summit on October 22nd to 23rd at The Hard Rock Hotel NYC [1] - Gene Mack, President and CEO of Gain, will be part of a live discussion panel focused on innovative approaches to neurodegenerative diseases [2] - The Maxim Growth Summit will feature industry leaders and discussions on advancements across various sectors, including biotechnology [3][4] Innovative Approach - Gain Therapeutics employs a unique approach to discover novel allosteric small molecule modulators that can restore or disrupt protein function [7] - The company utilizes its advanced Magellan™ platform to accelerate drug discovery and develop disease-modifying treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [7]

Summary of Gain Therapeutics Conference Call on October 14, 2025 Company Overview - **Company**: Gain Therapeutics (NasdaqGM: GANX) - **Focus**: Development of GT-022287 for Parkinson's disease Key Industry Insights - **Parkinson's Disease Prevalence**: Second most prevalent neurodegenerative disease in the U.S. with approximately 1 million patients [7] - **Genetic Mutation**: 10-15% of Parkinson's patients have a mutation in the GBA1 gene, which is the target for GT-022287 [7] - **Mechanism of Action**: GT-022287 stabilizes the enzyme glucocerebrosidase (GCase), improving its function in lysosomes and mitochondria, which is crucial for cellular health [9][10] Clinical Study Updates - **Phase 1b Study**: Ongoing in Australia, focusing on safety and tolerability of GT-022287 in Parkinson's patients [3][11] - **Dosing Information**: Patients treated for 90 days at a dose of 13.5 mg/kg, with an extension phase for an additional 9 months [11][12] - **Enrollment**: 21 participants enrolled, with 16 completing the initial 90-day study [12][45] - **Adverse Events**: 93 total adverse events reported, with 85% classified as mild [50] - **Biomarker Engagement**: Preliminary data shows a 53% increase in GCase activity [12] Biomarkers and Clinical Endpoints - **Biomarker Importance**: Biomarkers are critical for understanding disease progression and treatment efficacy [15][20] - **Alpha-Synuclein**: A key biomarker for Parkinson's disease, with ongoing efforts to quantify its presence [21][28] - **MDS-UPDRS**: The Unified Parkinson's Disease Rating Scale is used to assess clinical outcomes, with a focus on both self-reported and clinician-rated measures [33][36] Future Directions - **Data Availability**: Full data set from the 90-day study expected by the end of the year, including safety, tolerability, and biomarker data [58] - **Phase 2 Study**: Planned for Q2 of the following year, aiming to confirm the efficacy of GT-022287 with a placebo-controlled design [59][60] Additional Insights - **GBA1 Mutation Impact**: Patients with GBA1 mutations may experience a more aggressive form of Parkinson's disease [7][70] - **Regulatory Considerations**: There is a growing emphasis on the clinical meaningfulness of self-reported outcomes in regulatory assessments [38][39] - **Long-Term Efficacy**: The drug's potential to slow disease progression rather than provide immediate symptomatic relief is a key focus [75] Conclusion Gain Therapeutics is making significant strides in the development of GT-022287 for Parkinson's disease, with ongoing studies aimed at understanding its safety, tolerability, and biological impact on disease progression. The integration of biomarkers and clinical endpoints will be crucial in evaluating the drug's effectiveness in future trials.

Core Insights - Gain Therapeutics, Inc. will host a virtual key opinion leader event on October 14, 2025, focusing on biomarkers and clinical endpoints related to their drug candidate GT-02287 for Parkinson's disease [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company specializing in the discovery and development of allosteric small-molecule therapies [12] - The lead drug candidate, GT-02287, is in clinical development for treating Parkinson's disease with or without a GBA1 mutation [6][12] Event Details - The event will feature KOLs Karl Kieburtz and Kenneth Marek, discussing recent data from a Phase 1b clinical study of GT-02287 [1][2] - A live Q&A session will follow the formal presentations [3] Clinical Study Insights - Data from the Phase 1b study indicates that GT-02287 was generally well tolerated, with no serious adverse events reported and improvements in MDS-UPDRS scores [2][10] - The primary endpoint of the ongoing Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [10] Drug Mechanism and Preclinical Data - GT-02287 is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase), which is impaired due to GBA1 mutations [6][7] - Preclinical models show that GT-02287 can restore GCase function and has a disease-modifying effect, potentially slowing or stopping the progression of Parkinson's disease [8][9] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation and other organizations for the development of GT-02287 [11]

Core Insights - Gain Therapeutics, Inc. presented early safety and tolerability findings from the Phase 1b clinical study of GT-02287 for Parkinson's disease at the International Congress of Parkinson's Disease and Movement Disorders [2][3] - The study showed improvements in MDS-UPDRS scores among participants, indicating a potential disease-slowing effect of GT-02287 [3][5] - GT-02287 was well-tolerated with no serious adverse events reported, and the study has been recommended for continuation by independent data monitoring committees [3][11] Study Findings - The Phase 1b study enrolled 21 participants, with varying treatment backgrounds, including treatment-naïve individuals and those on existing Parkinson's medications [4] - Mean MDS-UPDRS scores at baseline were recorded as 5.8, 7.4, and 24.7 for Parts I, II, and III respectively, with notable improvements in Parts II and III after 90 days of dosing [5] - The pharmacokinetics of GT-02287 were consistent across participants and within the projected therapeutic range, comparable to previous Phase 1 studies in healthy volunteers [6][10] Future Developments - Gain Therapeutics plans to host a webinar on October 14 to discuss the results from the study in more detail [7] - The Phase 1b study has received approval for an extension, allowing treatment for up to 12 months [3][11] - GT-02287 is positioned as a potential treatment for Parkinson's disease with or without a GBA1 mutation, and has shown promise in preclinical models for various neurodegenerative conditions [8][9][13]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric therapies, with its lead drug candidate GT-02287 currently in a Phase 1b clinical trial for Parkinson's disease [4][5] Company Overview - Gain Therapeutics is leading the discovery and development of allosteric small-molecule therapies, utilizing its advanced Magellan™ platform to accelerate drug discovery [4][5] - The company has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [4] Upcoming Event - Dr. Rinaldo Montalvão, Director of Artificial Intelligence at Gain Therapeutics, will present at the WORLD BI sponsored Drug Discovery Innovation Programme (DDIP) 2025 on September 26, focusing on "Transforming Drug Discovery: Generative Models and Electrostatic Complementarity" [1][2] - The event aims to connect global leaders in the pharma, biotech, and life sciences industries, facilitating networking and knowledge sharing [2][3]

Core Insights - Gain Therapeutics, Inc. has announced the extension of its Phase 1b study for GT-02287, allowing participants to continue treatment for an additional nine months after the initial 90-day dosing schedule [1][4] - The Phase 1b study has enrolled 21 participants, with the last participant expected to complete dosing in December 2025 [2][8] - Early data from the Phase 1b study will be presented at the International Congress of Parkinson's Disease and Movement Disorders in October 2025 [2][4] Study Details - The Phase 1b extension study will enable participants to continue dosing with GT-02287, with more than half of the participants agreeing to continue treatment [1][4] - The primary endpoint of the Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [8] - Additional analysis from participants enrolled as of June 30, 2025, will include functional changes and biomarker data, expected in Q4 2025 [3] Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule designed to treat Parkinson's disease, targeting the lysosomal enzyme glucocerebrosidase (GCase) [5][10] - Preclinical models have shown that GT-02287 can restore GCase function and reduce various pathologies associated with Parkinson's disease [5][6] - Results from a Phase 1 study in healthy volunteers indicated favorable safety, tolerability, and significant increases in GCase activity [7] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation, and the Eurostars-2 joint program [9]

Core Viewpoint - Gain Therapeutics has received approval to extend the dosing period of its Phase 1b clinical trial for GT-02287, allowing participants to continue treatment for a total of 12 months, which reflects positive early feedback from patients and clinicians [1][4][2] Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing allosteric small molecule therapies, with its lead candidate GT-02287 targeting Parkinson's disease [10][11] - The company has received funding support from organizations such as The Michael J. Fox Foundation and the Eurostars-2 joint program [9] Clinical Trial Details - The ongoing Phase 1b trial of GT-02287 is designed to evaluate safety and tolerability in participants with Parkinson's disease, with the primary endpoint being assessed after 3 months of dosing [8] - The independent data monitoring committee (DMC) has reviewed interim safety data and found no safety concerns, recommending the continuation of the study without changes [3][4] Drug Mechanism and Preclinical Data - GT-02287 is an orally administered small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [5] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce neuroinflammation, and improve motor function in models of both GBA1-PD and idiopathic PD [6][5] Future Development Plans - The additional data from the extended dosing period is intended to support planning for Phase 2 trials, focusing on long-term safety, tolerability, and functional changes in patients [4][1]

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 June 30, 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 (Stat ...

Exhibit 99.1 Gain Therapeutics Reports Financial Results for Second Quarter 2025 and Provides Corporate Update Reached full enrolment of Phase 1b study evaluating GT-02287 in Parkinson's Disease (PD) with or without GBA1 mutations during 2Q 2025, earlier than anticipated Analysis of functional changes and biomarker activity at 90 days will be available during 4Q 2025 In July 2025 following the close of 2Q 2025, Gain completed an underwritten public of ering that resulted in approximately $7.1 million of net ...

Core Insights - Gain Therapeutics has completed full enrollment of its Phase 1b study for GT-02287 in Parkinson's Disease earlier than expected, with results anticipated in Q4 2025 [1][12] - The company raised approximately $7.1 million in net proceeds from a public offering, extending its cash runway [1][12] - The Phase 1b study aims to evaluate the safety and tolerability of GT-02287, with a total enrollment capped at 20 participants [2][13] Clinical Program Highlights - The Phase 1b study has enrolled 16 patients as of June 30, 2025, with the possibility of including additional participants screened before July 31 [2][12] - The company has submitted a request to extend the dosing duration of the Phase 1b study beyond the original 90 days, with an update expected soon [2][3] - Preclinical data presented at various conferences indicate GT-02287's potential to modify disease progression in Parkinson's models [4][10] Financial Results - Research and Development (R&D) expenses decreased by $1.7 million to $2.8 million for Q2 2025 compared to $4.4 million in Q2 2024, attributed to grant income and optimized costs [6] - General and Administrative (G&A) expenses also decreased by $1.4 million to $2.3 million for Q2 2025 compared to $3.7 million in Q2 2024, mainly due to reduced stock-based compensation [7] - The net loss for Q2 2025 was $0.19 per share, down from $0.42 per share in Q2 2024, with cash and cash equivalents at $6.7 million as of June 30, 2025 [8][19] Upcoming Milestones - Analysis of functional changes and biomarker activity from the Phase 1b study is expected in Q4 2025 [1][12] - The company anticipates submitting an Investigational New Drug (IND) application to the FDA by the end of 2025 to facilitate Phase 2 clinical development [12]