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]

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]

Core Insights - Gain Therapeutics, Inc. is participating in BTIG's Annual Virtual Biotechnology Conference on July 29-30, 2025, with a fireside chat scheduled for July 30 at 3:20 PM ET [1][2] Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric therapies [3] - The 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. has announced a public offering of 4,501,640 shares of common stock and warrants, aiming to raise approximately $7.0 million for the development of its lead product candidate GT-02287 and general corporate purposes [1][3][4]. Group 1: Offering Details - The public offering price is set at $3.11 for two shares of common stock and one warrant, translating to an effective price of $1.55 per share and $0.01 per warrant [1]. - The warrants will have an exercise price of $1.65 per share, are immediately exercisable, and will expire five years after issuance [1]. - The underwriter has a 30-day option to purchase up to an additional 15% of shares and/or warrants at the public offering price [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized for the clinical and nonclinical development of GT-02287, targeting neurodegenerative diseases such as GBA1 Parkinson's disease, as well as for general corporate purposes [4]. Group 3: Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric therapies, with GT-02287 currently in a Phase 1b clinical trial for Parkinson's disease [7]. - The company employs its Magellan™ platform to discover novel allosteric small molecule modulators aimed at treating various difficult-to-treat disorders, including neurodegenerative diseases and rare genetic disorders [8].

Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] 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 [4][9] - The drug is designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which is impaired due to mutations in the GBA1 gene, a common genetic abnormality associated with Parkinson's disease [4][5] Clinical Trial Details - The Phase 1b trial aims to evaluate the safety and tolerability of GT-02287 over a 90-day dosing period in participants with Parkinson's disease, with enrollment ongoing across seven sites in Australia [7][9] - The company plans to extend the screening window for participants until July 31, 2025, and is seeking to extend the dosing period beyond the current 90-day limit [2][3] Preclinical and Clinical Evidence - Preclinical studies have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease, suggesting a potential disease-modifying effect [5][6] - Results from a Phase 1 study in healthy volunteers indicated favorable safety and tolerability, with a greater than 50% increase in GCase activity observed [6][9] Funding and Support - Gain Therapeutics has received funding support from organizations such as The Michael J. Fox Foundation for Parkinson's Research and the Eurostars-2 joint program, which is co-funded by the European Union [8][9]

Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] Company Progress - The company plans to extend the screening window for participants until July 31, 2025, in response to clinician and patient interest [2] - Gain Therapeutics is also seeking to extend the dosing period of the Phase 1b trial beyond the current 90-day limit, with long-term toxicology studies nearing completion [2] - The company anticipates that the extension will provide valuable insights for the Phase 2 protocol design planned for early 2026 [2] Drug Candidate Overview - GT-02287 is an allosteric enzyme modulator aimed at treating Parkinson's disease, particularly in patients with or without a GBA1 mutation [4][9] - The drug has shown promise in preclinical models by restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) and addressing various pathological features associated with Parkinson's disease [4][5] - Previous Phase 1 studies indicated favorable safety and tolerability, with a significant increase in GCase activity observed [6][7] Market Context - There are over 1 million Americans diagnosed with Parkinson's disease, highlighting a significant unmet medical need for effective treatments that can modify disease progression rather than just alleviate symptoms [3][5] - Gain Therapeutics has received funding support from notable organizations, including The Michael J. Fox Foundation and the European Union, to advance its research and development efforts [8][9]

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [2][3] - The company’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 [2][3] Company Overview - Gain Therapeutics is pioneering the discovery of allosteric therapies, utilizing its advanced Magellan™ platform to accelerate drug discovery [3] - The company is also developing multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [2] Upcoming Events - Gene Mack, President and CEO, will present at the BIO 2025 conference on June 18, 2025, at 1:45 p.m. EST [1] - Additionally, Gene Mack will participate in a virtual fireside chat at the H.C. Wainwright 6th Annual Neuro Perspectives Hybrid Conference on June 17, 2025, at 7:00 a.m. EST [5]

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]