Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [6] - The company'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 [6] - Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors [6] Upcoming Event Participation - Gain Therapeutics will participate in the 2025 Maxim Growth Summit on October 22nd to 23rd at The Hard Rock Hotel NYC [1] - Gene Mack, President and CEO of Gain, will be part of a live discussion panel focused on innovative approaches to neurodegenerative diseases [2] - The Maxim Growth Summit will feature industry leaders and discussions on advancements across various sectors, including biotechnology [3][4] Innovative Approach - Gain Therapeutics employs a unique approach to discover novel allosteric small molecule modulators that can restore or disrupt protein function [7] - The company utilizes its advanced Magellan™ platform to accelerate drug discovery and develop disease-modifying treatments for challenging disorders, including neurodegenerative diseases and rare genetic disorders [7]

Core Insights - Gain Therapeutics, Inc. presented early safety and tolerability findings from the Phase 1b clinical study of GT-02287 for Parkinson's disease at the International Congress of Parkinson's Disease and Movement Disorders [2][3] - The study showed improvements in MDS-UPDRS scores among participants, indicating a potential disease-slowing effect of GT-02287 [3][5] - GT-02287 was well-tolerated with no serious adverse events reported, and the study has been recommended for continuation by independent data monitoring committees [3][11] Study Findings - The Phase 1b study enrolled 21 participants, with varying treatment backgrounds, including treatment-naïve individuals and those on existing Parkinson's medications [4] - Mean MDS-UPDRS scores at baseline were recorded as 5.8, 7.4, and 24.7 for Parts I, II, and III respectively, with notable improvements in Parts II and III after 90 days of dosing [5] - The pharmacokinetics of GT-02287 were consistent across participants and within the projected therapeutic range, comparable to previous Phase 1 studies in healthy volunteers [6][10] Future Developments - Gain Therapeutics plans to host a webinar on October 14 to discuss the results from the study in more detail [7] - The Phase 1b study has received approval for an extension, allowing treatment for up to 12 months [3][11] - GT-02287 is positioned as a potential treatment for Parkinson's disease with or without a GBA1 mutation, and has shown promise in preclinical models for various neurodegenerative conditions [8][9][13]

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]

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 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]

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]