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]

Financial Performance - Net loss for the three months ended March 31, 2025, was $4.53 million, compared to a net loss of $4.01 million for the same period in 2024, reflecting an increase in loss of approximately 12.9%[16] - Comprehensive loss for Q1 2025 was $4.40 million, compared to $4.24 million in Q1 2024, indicating an increase of about 3.9%[20] - Cash used in operating activities for Q1 2025 was $3.82 million, compared to $3.59 million in Q1 2024, an increase of approximately 6.4%[28] - The company has incurred an accumulated deficit of $81.2 million as of March 31, 2025, with recurring losses and negative cash flows from operations[165] - Net loss for the three months ended March 31, 2025, was $4.5 million, an increase of $0.5 million compared to a net loss of $4.0 million for the same period in 2024[182] Assets and Liabilities - Total assets decreased to $11.62 million as of March 31, 2025, from $12.12 million as of December 31, 2024, representing a decline of approximately 4.1%[13] - Current liabilities increased significantly to $5.03 million as of March 31, 2025, compared to $3.91 million as of December 31, 2024, marking an increase of about 28.6%[13] - Total stockholders' equity decreased to $5.78 million as of March 31, 2025, from $7.34 million as of December 31, 2024, a decline of approximately 21.2%[13] - Total prepaid expenses and other current assets increased to $1,833,659 as of March 31, 2025, up from $945,536 on December 31, 2024, representing a growth of 93.9%[104] - Accounts payable increased to $2.2 million as of March 31, 2025, compared to $0.9 million on December 31, 2024, indicating a rise of 144.4%[112] Cash and Financing - Cash and cash equivalents at the end of Q1 2025 were $9.07 million, down from $10.39 million at the end of Q4 2024, a decrease of approximately 12.7%[13] - As of March 31, 2025, the Company has $9.1 million in cash and cash equivalents, which is insufficient to fund operations for the next 12 months[48] - The company raised gross proceeds of $2.49 million from the sale of 1,094,701 shares under the 2024 ATM Program at an average price of $2.28 per share[122] - The public offering completed in June 2024 resulted in gross proceeds of $11.5 million, including $1.2 million in underwriting commissions and expenses[126] - Cash provided by financing activities was $2.4 million for the three months ended March 31, 2025, due to net proceeds from the issuance of shares[197] Research and Development - The Company is developing GT-02287 for Parkinson's disease, showing significant preclinical efficacy in restoring GCase function and improving neuronal health[34] - In a Phase 1 study with 72 participants, GT-02287 was safe and well tolerated at the highest planned dose, supporting further development in GBA1 Parkinson's patients[34] - Research and development expenses decreased to $2.26 million in Q1 2025 from $2.51 million in Q1 2024, a reduction of about 9.9%[16] - The Company started a Phase 1 Clinical Trial for its lead program in Parkinson's disease in Australia, supported by the R&DTI program[98] - The company plans to continue advancing existing research programs and initiate additional programs targeting allosteric binding sites identified with the Magellan™ platform[150] Grants and Income - The Company received a grant of $1.3 million from Eurostars and Innosuisse, with $0.45 million allocated to the Company for its alpha-1 antitrypsin deficiency program[95] - 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[96] - The Company recorded deferred grant income of $0.2 million and $0.3 million as of March 31, 2025 and December 31, 2024, respectively[97] - The R&DTI program provided a cash refund based on a percentage of eligible research and development activities, resulting in a reduction to research and development expenses of $0.2 million during the three months ended March 31, 2025[98] Risks and Concerns - The Company anticipates incurring additional losses until it can generate significant sales from its product candidates currently in development[45] - If unable to secure additional funding, the Company may need to suspend or terminate ongoing development activities, impacting shareholder investments[51] - The company reported substantial doubt about its ability to continue as a going concern due to insufficient cash resources to fund operations for one year from the issuance of the financial statements[189] - The Company faces risks typical of early-stage biotechnology firms, including reliance on key personnel and the need for significant additional capital[37] Stock and Compensation - The weighted average common stock increased to 28,685,417 shares in Q1 2025 from 17,978,951 shares in Q1 2024, an increase of approximately 59.7%[16] - The total stock-based compensation expense for the three months ended March 31, 2025, was $419,504, significantly higher than $190,424 for the same period in 2024[137] - The company granted 1,099,325 stock options during the three months ended March 31, 2025, with a weighted average grant date fair value of $1.67[133] - As of March 31, 2025, the company had unrecognized compensation costs associated with stock options grants amounting to $4.3 million, expected to be recognized over 4.0 years[133] Management and Controls - Management plans to raise additional capital through private/public equity financings and is reviewing cost structures to optimize expenditures[49] - The company maintains effective disclosure controls and procedures as of March 31, 2025, ensuring timely reporting and management communication[208] - No changes in internal control over financial reporting were reported during the period that materially affected internal controls[210] - Management acknowledges that controls can only provide reasonable assurance and are subject to inherent limitations[211]

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]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3][4] - The company will present at The Citizens Life Sciences Conference on May 8, 2025, with CEO Gene Mack participating in one-on-one meetings [1] - Gain'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 [3][4] Company Overview - Gain Therapeutics specializes in discovering and developing allosteric therapies, with a unique approach to creating small molecule modulators that can alter protein function [3][4] - The company utilizes its advanced Magellan™ platform to accelerate drug discovery for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4] Clinical Development - GT-02287 is being evaluated for Parkinson's disease treatment, with further potential in conditions like Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease [3] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [3]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3][4] - The company will present at The Citizens Life Sciences Conference on May 8, 2025, with one-on-one meetings available for interested parties [1][2] Company Overview - Gain'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 [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. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, showcasing promising preclinical and early clinical data that suggest potential disease-modifying effects [1][3][4]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric therapies [7]. - The company is leveraging its Magellan™ platform to accelerate drug discovery for various difficult-to-treat disorders, including neurodegenerative diseases and rare genetic disorders [8][9]. Drug Candidate Details - GT-02287 is an allosteric enzyme modulator aimed at restoring the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired in Parkinson's disease due to GBA1 mutations [2][7]. - The drug is currently in a Phase 1b clinical trial, evaluating its safety and tolerability in patients with Parkinson's disease [5]. Clinical Data - Preclinical studies have shown that GT-02287 can restore GCase activity, reduce neuroinflammation, and protect dopaminergic neurons from mitochondrial toxins [2][3]. - A Phase 1 study in healthy volunteers indicated favorable safety and tolerability, with a greater than 50% increase in GCase activity observed at clinically relevant doses [4]. Funding and Support - Gain Therapeutics has received funding support from notable organizations, including The Michael J. Fox Foundation for Parkinson's Research and the Eurostars-2 joint program, which is co-funded by the European Union [6].

Core Viewpoint - Gain Therapeutics, Inc. announced new evidence supporting the disease-modifying potential of its lead drug candidate GT-02287 in preclinical models of GBA1 and idiopathic Parkinson's disease, presented at the AD/PD 2025 conference [2][6][12]. Group 1: Clinical Study Updates - The ongoing Phase 1b study is assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of GT-02287 in individuals with Parkinson's disease, with biomarker analysis expected in Q2 2025 [2][3]. - Enrollment for the Phase 1b study is robust, with completion anticipated by the end of Q2 2025 [5][14]. - The primary endpoint of the Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [14]. Group 2: Preclinical Findings - In animal models, GT-02287 demonstrated the ability to rescue motor deficits and prevent the development of complex behavioral deficits, indicating its disease-modifying potential [6][12]. - Treatment with GT-02287 resulted in statistically significant reductions in several biomarkers of disease progression, including aggregated α-synuclein and markers of ER stress and neuroinflammation [6][11]. Group 3: Future Planning - An early biomarker analysis from the Phase 1b study participants will inform Phase 2 planning, expected to commence in the second half of 2025 [3][8]. - The results from the Phase 1b study will guide the design of a subsequent randomized, placebo-controlled, double-blind Phase 2 study to evaluate the efficacy of GT-02287 [8]. Group 4: Company Background - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric therapies, with GT-02287 being evaluated for Parkinson's disease and other related conditions [16][17]. - The company has received funding support from various organizations, including The Michael J. Fox Foundation and the Eurostars-2 joint program [15].

Core Insights - Gain Therapeutics, Inc. is presenting a poster on the Phase 1b clinical trial design for its drug candidate GT-02287 aimed at treating Parkinson's disease at the AD/PD™ 2025 conference [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [5] - The lead drug candidate, GT-02287, is designed to treat Parkinson's disease with or without a GBA1 mutation and is an orally administered, brain-penetrant small molecule [2][5] Drug Candidate Details - GT-02287 functions as an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase), which is often impaired due to GBA1 gene mutations [2] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce aggregated α-synuclein, decrease neuroinflammation and neuronal death, and improve both motor and cognitive functions [2][3] - The drug has also been noted to significantly lower plasma neurofilament light chain (NfL) levels, a biomarker for neurodegeneration [2] Clinical Development - The Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety, tolerability, and target engagement with the GCase enzyme [5] - Compelling preclinical data suggest that GT-02287 may have a disease-modifying effect, potentially slowing or stopping the progression of Parkinson's disease [3] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation for Parkinson's with GBA, and the Eurostars-2 program co-funded by the European Union Horizon 2020 research and Innosuisse [4]