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]

Core Viewpoint - Gain Therapeutics has appointed Gene Mack as President and Chief Executive Officer, effective January 6, 2025, to ensure continuity in leadership as the company advances its lead drug candidate, GT-02287, through clinical development [1][2]. Leadership Changes - Gene Mack has been promoted from Chief Financial Officer and interim Chief Executive Officer to President and Chief Executive Officer, and he will also join the Board of Directors [1][2]. - Gianluca Fuggetta has been appointed as Senior Vice President, Finance and Principal Financial Officer [1]. Gene Mack's Background - Gene Mack brings over 25 years of experience in the life sciences sector, including roles in clinical research, financing, corporate strategy, and business development [3]. - Prior to joining Gain Therapeutics, he served as CFO at Imcyse SA and OncoC4, and has experience as a senior publishing analyst at various investment banks [3]. Drug Candidate Overview - GT-02287 is in clinical development for treating Parkinson's disease, targeting both GBA1 mutation and idiopathic forms [4][7]. - The drug is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [4]. Clinical Development and Support - GT-02287 has shown promising preclinical results, including restoration of GCase function and reduction of neuroinflammation and neuronal death [4][5]. - The program has received funding support from The Michael J. Fox Foundation and other organizations, indicating strong backing for its development [6]. Future Plans - The ongoing Phase 1b trial for GT-02287 in Parkinson's disease patients is expected to continue into the first half of 2025, with preparations for potential Phase 2 activities in the second half of 2025 [3].

The Company has received approval to begin enrollment of the Phase 1b clinical trial in Australia Phase 1b clinical trial will assess safety and tolerability along with biomarkers during three months of dosing with GT-02287 in people diagnosed with Parkinson's disease Phase 1b clinical trial follows successful Phase 1 study in which GT-02287 was safe and well tolerated while demonstrating GCase target engagement BETHESDA, Md., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", ...

Core Insights - Gain Therapeutics has received approval to initiate a Phase 1b clinical trial for GT-02287 in Australia, targeting individuals diagnosed with Parkinson's disease [1][9] - The trial aims to assess the safety and tolerability of GT-02287 over three months, with enrollment expected to complete by spring 2025 and data anticipated by mid-2025 [1][2][10] - GT-02287 is designed to treat Parkinson's disease with or without a GBA1 mutation and has shown promising preclinical data indicating a potential to slow or stop disease progression [4][11] Company Developments - The transition from preclinical to clinical stage development was marked by the successful completion of healthy volunteer studies in 2024 [2] - The Phase 1 study demonstrated a favorable safety profile and significant target engagement, with over 50% increase in glucocerebrosidase (GCase) activity [9][13] - Gain Therapeutics is preparing for a Phase 2 trial in 2025, contingent on the results of the Phase 1b trial [2][10] Clinical Trial Details - The Phase 1b trial will be conducted at seven sites across major metropolitan areas in Australia, focusing on patients with GBA1-PD or idiopathic Parkinson's disease [10] - The trial will administer 13.5 mg/kg/day of GT-02287 and will evaluate secondary endpoints including pharmacokinetics and biomarker levels in plasma and cerebrospinal fluid [10][11] - The study is supported by funding from The Michael J. Fox Foundation and other organizations [12]

BETHESDA, Md., Dec. 11, 2024 (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 announced that Gene Mack, Chief Financial Officer and Interim Chief Executive Officer of Gain, will give a corporate presentation at Biotech Showcase 2025, being held January 13-15, 2025, at the Hilton San Francisco Union Square in San Francisco, CA. ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 | --- | --- | --- | --- | |----------------------------------------------------------------|--------------------------------------------------------------------------- ...

Exhibit 99.1 Gain Therapeutics Reports Financial Results for Third Quarter 2024 and Provides Corporate Update Initiation of Phase 1b Study of GT-02287 in Parkinson's patients expected by end of 2024 BETHESDA, Md., November 14, 2024 -- 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 reports financial results for the quarter ended September 30, 20 ...