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]

Product Development and Clinical Trials - GT-02287, the lead product candidate for Parkinson's disease, is currently in a Phase 1b study, with enrollment expected to complete by summer 2025[18]. - In a Phase 1 clinical trial, GT-02287 demonstrated a 53% increase in GCase activity in subjects receiving the highest dose of 13.5 mg/kg, indicating target engagement[17]. - The Phase 1b trial will assess the safety and tolerability of 13.5 mg/kg/day of GT-02287 for three months in patients, with interim data expected by mid-2025[18]. - The Phase 1 clinical trial included 72 healthy volunteers, confirming the safety and tolerability of GT-02287 across all age groups[16]. - The Phase 1 clinical trial for GT-02287 was initiated in September 2023, assessing safety, tolerability, and pharmacokinetics in healthy participants[57]. - In preclinical models, GT-02287 demonstrated statistically significant improvements in GCase activity and reduced neuroinflammation, leading to increased survival of dopaminergic neurons[55]. - The company has initiated a chronic toxicity study lasting six months in rodents and nine months in non-rodents to support clinical studies exceeding twelve months[56]. - The company has commenced its first Phase 1 clinical trial but has not yet successfully completed any clinical trials or conducted sales and marketing activities necessary for commercialization[190]. - The company’s product candidates are still in development, requiring extensive testing and regulatory approvals before commercialization can occur[214]. Drug Discovery and Research Platforms - The Magellan™ platform has identified over 50 billion compounds for potential binding to allosteric sites, significantly enhancing drug discovery efficiency[27]. - The average success rate for experimentally validated compounds from the Magellan™ platform is 14%, over 100 times higher than traditional high-throughput screening methods[28]. - The company plans to advance existing research programs and initiate new ones targeting allosteric binding sites identified through the Magellan™ platform[21]. - The computational drug discovery platform Magellan™ has in-licensed a European patent expected to expire in 2032, related to binding site and binding energy determination[70]. - The GLB program has in-licensed a patent family in the U.S., Europe, and Japan, with expected expiration in 2037, covering composition of matter[71]. - The GBA program also has in-licensed a patent family in the U.S., Europe, and Japan, with similar expiration and coverage as the GLB program[72]. Regulatory Environment and Compliance - The FDA regulates drug products under the FD&C Act, requiring substantial time and financial resources for compliance with various regulations[76]. - The process for FDA approval involves extensive preclinical studies, submission of an IND application, and successful completion of clinical trials[79]. - Clinical trials are conducted in three phases, with Phase 3 trials providing statistically significant evidence of clinical efficacy[87]. - Post-approval trials, or Phase 4 trials, may be mandated by the FDA to gather additional safety data after initial marketing approval[88]. - The company must submit progress reports and safety reports to the FDA, including any serious adverse events[89]. - An NDA must be submitted to the FDA for marketing approval, containing proof of the drug's safety and efficacy[94]. - The FDA targets ten months for the initial review of a new molecular entity NDA and six months for priority review[106]. - Each NDA must be accompanied by a user fee, which is adjusted annually by the FDA[96]. - Orphan Drug Designation allows for a seven-year marketing exclusivity if the product receives the first FDA approval for the designated condition[103]. - The FDA may require post-market studies or clinical trials to assess new safety risks after approval[115]. - The FDA may impose restrictions on marketing or manufacturing based on post-approval findings, which can affect market potential[117]. - Fast Track designation provides increased opportunities for sponsor interactions with the FDA during drug development[107]. - Breakthrough Therapy designation offers intensive guidance on efficient drug development for serious conditions[108]. - The FDA may withdraw approval of a drug if confirmatory trials fail to verify its clinical benefit under Accelerated Approval[111]. - The company is subject to extensive and costly government regulations which are subject to change, impacting its operations and financial condition[181]. - The company is subject to extensive and costly government regulation, including oversight by the FDA and other regulatory authorities, which significantly increases the cost and risk of product development and commercialization[193]. - Noncompliance with regulatory requirements can lead to delays, refusals of applications, fines, product recalls, and other penalties[196]. Financial Considerations and Market Dynamics - The company has incurred operating losses since inception and expects to continue incurring losses for the foreseeable future, raising substantial doubt about its ability to continue as a going concern[182]. - The company plans to raise additional capital primarily through public and/or private equity financings and/or convertible debt financings, but financing may not be available on acceptable terms[183]. - The company expects substantial additional operating expenses over the next several years as research, development, and clinical trial activities increase[185]. - The company has not generated any significant revenues to date and does not expect to generate revenues from the commercial sale of products in the foreseeable future[188]. - Market acceptance of products is heavily influenced by third-party payor reimbursement decisions, which are increasingly focused on cost containment[141]. - The company may face civil, criminal, and administrative penalties if found in violation of healthcare laws, which could adversely affect its operations and financial results[134]. - The company faces risks from global macroeconomic conditions, including heightened inflation and high interest rates, which could impact funding and operational capabilities[197]. Challenges in Clinical Development - Delays in clinical trials can significantly increase development costs and hinder commercialization efforts, potentially leading to abandonment of trials[202]. - Enrollment challenges in clinical trials may arise due to the low prevalence of targeted disorders, affecting the ability to initiate or complete trials[209]. - The development of product candidates may be delayed by external factors, including budget cuts at federal agencies affecting the FDA's operations[199].