Financial Performance - Gain Therapeutics reported a net loss of $0.15 per share for Q3 2025, an improvement from a loss of $0.17 per share in Q3 2024[11] - Net loss for Q3 2025 was $5,284,373, compared to a net loss of $4,485,692 in Q3 2024, representing a 17.8% increase in losses year-over-year[22] - Net loss per share attributable to common stockholders for Q3 2025 was $(0.15), compared to $(0.17) in Q3 2024[22] - The company reported a loss before income tax of $4,735,085 for Q3 2025, compared to $4,474,698 in Q3 2024[22] Expenses - Research and Development (R&D) expenses increased by $0.2 million to $2.8 million in Q3 2025, compared to $2.6 million in Q3 2024, primarily due to ongoing Phase 1b clinical trial costs[9] - General and Administrative (G&A) expenses rose by $0.1 million to $1.9 million for Q3 2025, attributed to higher stock-based compensation and personnel costs[10] - Total operating expenses for Q3 2025 were $4,785,777, an increase of 7.3% compared to $4,458,964 in Q3 2024[22] - Research and development expenses for the nine months ended September 30, 2025, were $7,865,493, down 17.8% from $9,566,259 in the same period of 2024[22] Clinical Trials - The Phase 1b study for GT-02287 in Parkinson's disease exceeded its enrollment target, with 21 participants enrolled, surpassing the original target of 15[8] - Approximately 80% of participants in the Phase 1b study expressed interest in continuing into the study extension phase[2] - Initial clinical data presented indicated a disease-slowing effect of GT-02287, with improvements in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores observed after 90 days of dosing[8] - The company received approval from Australian health authorities to extend the Phase 1b study duration to a total of 12 months[8] - The analysis of functional changes and biomarker activity from the Phase 1b study is expected to be available in Q4 2025[3] - An IND submission to the FDA is anticipated by the end of 2025 to facilitate expansion into Phase 2 clinical development of GT-02287[8] Financial Position - Cash and cash equivalents were reported at $8.8 million as of September 30, 2025, down from $10.4 million as of December 31, 2024[11] - Cash and cash equivalents as of September 30, 2025, were $8,807,353, a decrease from $10,385,863 as of December 31, 2024[24] - Total assets decreased to $10,971,680 as of September 30, 2025, from $12,123,884 as of December 31, 2024[24] - Total liabilities increased to $5,062,250 as of September 30, 2025, compared to $4,784,310 as of December 31, 2024[24] - The accumulated deficit as of September 30, 2025, was $81,194,908, up from $60,783,717 as of December 31, 2024[24] Stock Information - Weighted average common stock outstanding for Q3 2025 was 34,998,060, an increase from 26,531,747 in Q3 2024[22]

Core Insights - Gain Therapeutics, Inc. has presented initial data from its Phase 1b study indicating that GT-02287 shows a disease-slowing effect in Parkinson's disease, consistent with preclinical models and its proposed mechanism of action [1][4][15] - The company completed enrollment of 21 participants in the Phase 1b study, surpassing the initial target of 15 participants, and has received approval to extend the study duration to 12 months [1][3][12] - Financial results for Q3 2025 show an increase in R&D expenses to $2.8 million, primarily due to ongoing clinical trials and unfavorable currency translation [9][10] Clinical Program Highlights - The Phase 1b study of GT-02287 in Parkinson's disease has shown that 16 out of 21 participants completed 90 days of dosing, with the remaining participants expected to finish by December 2025 [3][12] - Initial clinical findings presented at the 2025 MDS conference suggest improvements in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores after approximately 30 days of administration [3][4] - The Phase 1b study extension commenced in September 2025, allowing participants to continue treatment for a total of 12 months, with a focus on long-term safety and tolerability [1][12][17] Financial Performance - R&D expenses increased by $0.2 million to $2.8 million for Q3 2025 compared to the same period in 2024, attributed to ongoing clinical trials and currency translation effects [9] - General and Administrative (G&A) expenses rose by $0.1 million to $1.9 million for Q3 2025, driven by higher stock-based compensation and personnel costs [10] - The net loss for Q3 2025 was reported at $5.3 million, or $0.15 per share, an improvement from a net loss of $4.5 million, or $0.17 per share, in Q3 2024 [11][13] Upcoming Milestones - Analysis of functional changes and biomarker activity from the Phase 1b study is expected to be available in Q4 2025 [2][4] - An IND submission to the FDA is anticipated by the end of 2025, facilitating the expansion into Phase 2 clinical development [12][18] - Results from the Phase 1b study extension are expected in the second half of 2026 [12]

Core Viewpoint - The focus is on long-term investment in growth markets, particularly in AI and biotech, while also exploring undervalued stocks with significant potential [1] Investment Focus - The investment strategy emphasizes a volatile portfolio with a concentration on semiconductor, mining, and biotech sectors [1] - The portfolio includes a diverse range of market capitalizations, from megacap to microcap [1] Investment Style - The investment approach is inspired by Warren Buffett but is applied to riskier investments [1] - The investor does not engage in cryptocurrency, banking, ETFs, retirement funds, or real estate [1] Market Coverage - The primary focus is on the U.S. market, with occasional coverage of European or Canadian stocks [1] Professional Background - The investor holds a Master's Degree in Law and serves as the deputy director general in a leading European sector organization [1]

Core Insights - Gain Therapeutics, Inc. is presenting its lead drug candidate GT-02287 at the Society for Neuroscience annual meeting, focusing on its potential in treating 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 [7] - The company is advancing GT-02287, which targets the lysosomal enzyme glucocerebrosidase (GCase) and is currently in a Phase 1b clinical trial for Parkinson's disease [7][5] Drug Candidate Details - GT-02287 is an orally administered small molecule that acts as an allosteric modulator, restoring GCase function impaired by GBA1 mutations or age-related stress [2] - Preclinical studies indicate that GT-02287 can reduce neuroinflammation, neuronal death, and restore motor function in models of both GBA1-PD and idiopathic PD [3][2] Clinical Trial Progress - A Phase 1 study showed favorable safety and tolerability, with increased GCase activity in participants receiving GT-02287 [4] - The ongoing Phase 1b trial aims to evaluate safety and tolerability over 3 months, with early observations suggesting disease-modifying effects [5] Funding and Support - Gain Therapeutics has received funding from The Michael J. Fox Foundation, The Silverstein Foundation, and the Eurostars-2 program, indicating strong support for its Parkinson's disease program [6] Research and Development Approach - The company utilizes its Magellan™ platform to discover novel allosteric small molecule modulators, targeting various disorders including neurodegenerative diseases and rare genetic disorders [8]

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]