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].

Exhibit 99.1 Gain Therapeutics Reports Financial Results for the Fourth Quarter and Year End 2024 and Provides Corporate Update First Analysis from Phase 1b Study of GT-02287 in Parkinson's Disease Expected in Q2 2025 BETHESDA, Md., March 27, 2025 -- Gain Therapeutics, Inc. (Nasdaq: GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today reported financial results for the fourth quarte ...

BETHESDA, Md., March 27, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today reported financial results for the fourth quarter and year ended December 31, 2024, and provided a corporate update. "2024 was a year of significant progress for Gain, as we made important advancements related to both the scientific understanding and ...

Core Viewpoint - Gain Therapeutics, Inc. has initiated dosing in a Phase 1b clinical trial for its lead drug candidate GT-02287, aimed at treating Parkinson's disease, marking a significant step in the development of a disease-modifying therapy for this condition [1][2]. Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [8]. - The company’s 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 [5][8]. Clinical Trial Details - The Phase 1b trial is an open-label, multi-center study evaluating the safety and tolerability of GT-02287 in participants with GBA1-PD and idiopathic PD, with secondary endpoints including pharmacokinetics and biomarker analysis [3]. - The trial will enroll up to 20 participants who will receive GT-02287 daily for three months, with interim data expected by the end of Q2 2025 [3][4]. Previous Study Results - A prior Phase 1 study in healthy volunteers demonstrated a favorable safety and tolerability profile for GT-02287, with significant target engagement indicated by over 50% increase in glucocerebrosidase (GCase) activity [4][8]. - Preclinical data showed that GT-02287 restored GCase function, reduced neuroinflammation, and improved motor and cognitive functions in models of Parkinson's disease [5][6]. 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, co-funded by the European Union Horizon 2020 research and Innosuisse [7].

Core Viewpoint - Gain Therapeutics, Inc. has initiated dosing in its Phase 1b clinical trial for GT-02287, aimed at treating Parkinson's disease, marking a significant step in the development of a disease-modifying therapy [1][2]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric therapies [8]. - The lead drug candidate, GT-02287, is being evaluated for Parkinson's disease treatment, specifically targeting both GBA1 mutation carriers and idiopathic cases [5][8]. Clinical Trial Details - The Phase 1b trial is an open-label, multi-center study designed to assess the safety and tolerability of GT-02287 in participants with GBA1-PD and idiopathic PD, with secondary endpoints including pharmacokinetics and biomarker analysis [3]. - Up to 20 participants will receive GT-02287 daily for three months, with interim data expected by the end of Q2 2025 [3][4]. Previous Study Results - A prior Phase 1 study in healthy volunteers demonstrated a favorable safety profile and significant target engagement, with over 50% increase in glucocerebrosidase (GCase) activity [4][8]. - Preclinical data indicated that GT-02287 restored GCase function, reduced neuroinflammation, and improved motor and cognitive functions in models of Parkinson's disease [5][6]. Funding and Support - Gain's Parkinson's disease program has received funding from The Michael J. Fox Foundation, The Silverstein Foundation, and the Eurostars-2 program, co-funded by the European Union Horizon 2020 research and Innosuisse [7].

Core Viewpoint - Gain Therapeutics, Inc. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, with promising preclinical data suggesting disease-modifying potential [1][5]. Group 1: Presentation Details - Joanne Taylor, Ph.D., will present on GT-02287 at the AD/PD™ 2025 International Conference, scheduled for April 1-5, 2025, in Vienna, Austria [1][2]. - The presentation will focus on GT-02287's potential in preclinical models of both GBA1 and idiopathic Parkinson's disease [2]. Group 2: Drug Candidate Overview - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric modulator of the glucocerebrosidase enzyme, which is often impaired due to GBA1 mutations [4][7]. - Preclinical studies have shown that GT-02287 restores GCase function, reduces neuroinflammation, and improves motor and cognitive functions [4][5]. Group 3: 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 research initiatives [6]. Group 4: Company Background - Gain Therapeutics is a clinical-stage biotechnology company focused on developing next-generation allosteric therapies for neurodegenerative diseases and other challenging disorders [8]. - The company utilizes its Magellan™ platform to discover novel small molecule modulators aimed at restoring protein function [8].

Core Viewpoint - Gain Therapeutics is advancing the clinical development of GT-02287, a potential disease-modifying therapy for Parkinson's disease, with significant milestones expected in 2025 [1][9]. Clinical Development - The company received approval to initiate a Phase 1b clinical trial of GT-02287 in December 2024, following a successful study in healthy volunteers that showed a 53% increase in glucocerebrosidase (GCase) activity [2][3]. - The Phase 1b trial will enroll 15-20 individuals with GBA1 or idiopathic Parkinson's disease, who will receive daily oral doses of GT-02287 for three months [3]. - Key biomarkers to be evaluated include GCase enzyme activity and inflammatory markers, with results expected to provide insights into the drug's neuroprotective effects [3][4]. Preclinical and Mechanism Insights - The company is expanding its preclinical data on GT-02287, focusing on its mechanism of action and neuroprotective roles beyond the lysosome [4]. - Observations include mitochondrial stabilization associated with GT-02287 administration, which may enhance its therapeutic profile [4]. Regulatory Engagement - Gain Therapeutics is strengthening its collaboration with the FDA, having received positive feedback during a pre-IND meeting in December 2024, indicating no significant regulatory hurdles for Phase 2 preparations [5]. Financial Position - As of September 30, 2024, the company had approximately $12 million in cash, expected to finance operations through the interim analysis of the Phase 1b trial in 2Q 2025 [6]. - The company is exploring licensing partnerships and other financing sources to maintain control over GT-02287's development [6]. Market Context - Parkinson's disease is the second most common neurodegenerative disease, with current treatments only addressing symptoms, highlighting the potential impact of GT-02287 as a disease-modifying therapy [9][10]. - The company aims to progress GT-02287 through clinical trials and expand its preclinical data package, contributing to the broader field of Parkinson's disease research [9].

Core Insights - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing next-generation allosteric small molecule therapies [3][4] - The company's lead drug candidate, GT-02287, is being evaluated for the treatment of Parkinson's disease, showing favorable safety and tolerability in a Phase 1 study [3][4] - CEO Gene Mack will present at two upcoming investor conferences, providing opportunities for investors to engage with management [1][6] Company Overview - Gain Therapeutics specializes in discovering and developing allosteric therapies, utilizing its advanced Magellan™ platform to accelerate drug discovery [3][4] - The company aims to unlock novel treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [4] Upcoming Events - Gene Mack will present at the BIO CEO & Investor Conference on February 11, 2025, at 1:15 PM ET [1][6] - A virtual corporate presentation will take place at the Oppenheimer 35th Annual Healthcare Life Sciences Conference on February 12, 2025, at 10:40 AM ET [6]

Core Viewpoint - Gain Therapeutics, Inc. (GANX) has been upgraded to a Zacks Rank 1 (Strong Buy), indicating a positive outlook on its earnings estimates, which significantly influence stock prices [1][4]. Earnings Estimates and Revisions - The Zacks Consensus Estimate for Gain Therapeutics indicates an expected loss of -$1 per share for the fiscal year ending December 2024, reflecting a year-over-year change of 41.5% [9]. - Over the past three months, the Zacks Consensus Estimate for Gain Therapeutics has increased by 12.1%, suggesting a positive trend in earnings estimates [9]. Zacks Rating System - The Zacks rating system is based solely on a company's changing earnings picture, which is a critical factor in determining stock price movements [2][3]. - The Zacks Rank system classifies stocks into five groups, with Zacks Rank 1 stocks historically generating an average annual return of +25% since 1988 [8]. - Only the top 5% of Zacks-covered stocks receive a 'Strong Buy' rating, indicating superior earnings estimate revisions [10][11]. Market Implications - The upgrade to Zacks Rank 1 positions Gain Therapeutics among the top 5% of stocks in terms of estimate revisions, suggesting potential for near-term price increases [11]. - Rising earnings estimates and the corresponding rating upgrade imply an improvement in Gain Therapeutics' underlying business, which could lead to higher stock prices as investors respond positively [6].

Core Insights - Gain Therapeutics, Inc. has formed a Clinical Advisory Board (CAB) to support the development of its lead drug candidate, GT-02287, for Parkinson's disease [1][2] - The CAB consists of experts in trial design, genetics, and biomarkers related to Parkinson's disease, aiming to advance GT-02287 through Phase 2 and Phase 3 clinical development [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [9][10] - The lead drug candidate, GT-02287, is being evaluated for treating Parkinson's disease with or without a GBA1 mutation [9][10] Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [6][9] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce aggregated α-synuclein, and improve motor and cognitive functions [6][7] Clinical Development and Support - The drug candidate has received funding support from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA, among others [8] - Results from a Phase 1 study indicated favorable safety and tolerability, with plasma exposure in the projected therapeutic range [9]