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]

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]

Financial Performance - Net loss for the three months ended June 30, 2025, was $5,809,087, compared to a net loss of $8,143,078 for the same period in 2024, indicating a reduction of approximately 28.8%[16] - The company reported a comprehensive loss of $5,105,139 for the three months ended June 30, 2025, compared to $8,152,479 for the same period in 2024, reflecting a decrease of approximately 37.5%[20] - For the six months ended June 30, 2024, the net loss was $12,157,272, compared to a net loss of $10,339,145 for the same period in 2025, indicating a decrease in losses year-over-year[31] - The company incurred net losses of $5.8 million and $10.3 million for the three and six months ended June 30, 2025, respectively[170] - Net loss for the three months ended June 30, 2025, was $5.8 million, a decrease of $2.3 million compared to a net loss of $8.1 million for the same period in 2024[187] Assets and Liabilities - Total assets decreased from $12,123,884 as of December 31, 2024, to $9,833,980 as of June 30, 2025, representing a decline of approximately 19.9%[13] - Current liabilities increased significantly from $3,911,321 as of December 31, 2024, to $5,197,044 as of June 30, 2025, an increase of about 32.9%[13] - Cash and cash equivalents decreased from $10,385,863 as of December 31, 2024, to $6,694,136 as of June 30, 2025, a decline of about 35.0%[13] - Total stockholders' equity decreased from $7,339,574 as of December 31, 2024, to $3,696,050 as of June 30, 2025, a decrease of approximately 49.7%[13] - As of June 30, 2025, accounts payable amounted to $2.7 million, an increase from $0.9 million as of December 31, 2024[116] Cash Flow and Financing - Cash used in operating activities for the six months ended June 30, 2024, was $9,342,986, while for the same period in 2025, it was $8,920,509, showing a slight improvement in cash flow management[31] - Cash provided by financing activities for the six months ended June 30, 2025, was $4.9 million, primarily from the issuance of shares under the 2024 ATM Program[203] - The company raised $4,910,240 from the issuance of shares in an ATM offering during the six months ended June 30, 2025[31] - The public offering completed in June 2024 resulted in gross proceeds of $11.5 million, including $1.2 million in underwriting commissions and expenses[131] - The company completed a public offering in July 2025, raising gross proceeds of $7.0 million from the sale of 4,501,640 shares and warrants[150] Research and Development - Research and development expenses for the six months ended June 30, 2025, were $5,015,983, down from $6,944,537 in the same period of 2024, a decrease of about 27.8%[16] - The company plans to continue advancing existing research programs and initiate additional programs targeting allosteric binding sites identified with its Magellan™ platform[154] - The clinical stage product candidate GT-02287 is being developed for the treatment of Parkinson's disease, with preclinical data showing restoration of glucocerebrosidase function and improvement in dopaminergic neuron survival[35] - GT-02287, a clinical stage product candidate for Parkinson's disease, demonstrated a 53% mean increase in GCase activity among healthy volunteers in a Phase 1 study[155] - The company anticipates significant research and development expenses to support ongoing clinical trials and product development[161] Capital Requirements and Going Concern - The company anticipates needing substantial additional capital to fund operations and develop its product candidates[46] - There is substantial doubt about the Company's ability to continue as a going concern, necessitating additional capital to sustain operations beyond the first quarter of 2026[49] - The company has substantial doubt about its ability to continue as a going concern without additional financing[195] - Future funding requirements will depend on various factors, including operational needs for preclinical studies and clinical trials, which are currently uncertain[207] - The company expects to finance operations through public and private equity offerings, debt financings, and collaborations until substantial product revenue is generated[208] Expenses and Cost Management - General and administrative expenses decreased by $1.4 million to $2.3 million for the three months ended June 30, 2025, compared to $3.7 million for the same period in 2024[189] - The total stock-based compensation expense for the six months ended June 30, 2025, was $941,523, a decrease from $1,673,325 in the same period of 2024[142] - Management plans to optimize expenditures and improve cash burn rate by reviewing the cost structure and seeking strategic collaborations and funding opportunities[50] - The primary use of cash is to fund operating expenses, including research and development and general administrative expenditures[206] Grants and Funding - The Company received a grant of $1.3 million from Eurostars and Innosuisse, with $0.45 million allocated to the Company, which was later terminated, resulting in a repayment of $0.21 million[98] - In May 2023, the Company was awarded a grant of $2.8 million under the Swiss Accelerator program, later amended to approximately $2.0 million due to project scope changes[99] - The Company recorded a reduction to research and development expenses of $0.3 million and $0.2 million for the three months ended June 30, 2025 and 2024, respectively, due to grants received[100] Risks and Uncertainties - The company faces risks common to early-stage biotechnology firms, including the need for significant additional research and development efforts prior to regulatory approval[38] - Increased costs may arise from global conditions such as high interest rates and supply chain disruptions, impacting overall funding requirements[210] - Management's estimates and judgments regarding financial conditions are based on historical experience and known trends, which may differ from actual results[209] Stock and Equity - The weighted average common stock for basic and diluted net loss per share increased from 19,215,582 in Q2 2024 to 30,341,523 in Q2 2025[16] - The company granted 1,204,325 stock options during the six months ended June 30, 2025, with a weighted average exercise price of $2.28[138] - The company had research commitments totaling $3.7 million as of June 30, 2025, for activities to be performed within one year[148] - As of June 30, 2025, the company had 4,903,778 outstanding warrants with a weighted average exercise price of $2.73[134] - The company’s stock options outstanding increased to 4,339,920 as of June 30, 2025, with a weighted average exercise price of $3.76[138]

Clinical Development - Full enrollment of the Phase 1b study for GT-02287 in Parkinson's disease was reached with 16 participants, with total enrollment not expected to exceed 20 participants [2] - Analysis of functional changes and biomarker activity from the Phase 1b study is expected to be available in Q4 2025 [14] - Approval from healthcare authorities in Australia to extend the dosing duration for participants in the Phase 1b study beyond the original 90 days [14] - The company plans to submit an Investigational New Drug (IND) application to the FDA by the end of 2025 to facilitate expansion of Phase 2 clinical development for GT-02287 in the U.S. [14] - Compelling preclinical data suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson's disease [10] Financial Performance - Net loss for Q2 2025 was $0.19 per share, compared to $0.42 per share for Q2 2024 [8] - The company reported a loss of $10,339,145 for the period, compared to a loss of $20,411,191 in the previous period, indicating an improvement of about 49.3% [22] Expenses - Research and Development (R&D) expenses decreased by $1.7 million to $2.8 million for Q2 2025, compared to $4.4 million for Q2 2024, primarily due to research grant income and optimization of pipeline costs [6] - General and Administrative (G&A) expenses decreased by $1.4 million to $2.3 million for Q2 2025, compared to $3.7 million for Q2 2024, mainly due to a reduction in stock-based compensation [7] Cash and Assets - Cash and cash equivalents were $6.7 million as of June 30, 2025, down from $10.4 million as of December 31, 2024 [8] - Cash and cash equivalents decreased from $10,385,863 to $6,694,136, a reduction of approximately 35.0% [22] - Total assets decreased from $12,123,884 to $9,833,980, a decline of approximately 19.0% [22] Liabilities and Equity - Current liabilities increased significantly from $3,911,321 to $5,197,044, representing a rise of about 32.9% [22] - Total stockholders' equity dropped from $7,339,574 to $3,696,050, a decrease of approximately 49.7% [22] - Additional paid-in capital increased from $88,779,318 to $94,642,942, reflecting a growth of about 6.5% [22] - The defined benefit pension plan liability rose from $443,623 to $522,676, an increase of approximately 17.8% [22] - Total noncurrent liabilities increased from $872,989 to $940,886, a rise of about 7.8% [22] - Accumulated deficit worsened from $(60,783,717) to $(81,194,908), indicating a decline of approximately 33.6% [22] Funding - Gain completed an underwritten public offering resulting in approximately $7.1 million of net proceeds, extending the cash runway beyond the completion of the Phase 1b study [14] Shareholder Information - The number of common shares issued and outstanding increased from 27,132,588 to 30,771,321, representing a growth of about 13.3% [22]