Core Viewpoint - Gain Therapeutics, Inc. is actively participating in key healthcare conferences to showcase its advancements in allosteric small molecule therapies, particularly focusing on its lead drug candidate GT-02287 for Parkinson's disease [1][2]. Conference Participation - Gain Therapeutics will attend the 44th Annual J.P. Morgan Healthcare Conference and other related events, including the Sachs Neuroscience Innovation Forum on January 11, 2026, and the Demy-Colton/Informa Biotech Showcase from January 12-14, 2026 [1][3]. - The company will conduct presentations and one-on-one meetings with management during these events, providing opportunities for investors and stakeholders to engage directly [3][4]. Drug Development - GT-02287 is in clinical development for treating Parkinson's disease, targeting both patients with and without a GBA1 mutation [5][8]. - The drug functions as an allosteric enzyme modulator, aiming to restore the function of the glucocerebrosidase enzyme, which is often impaired due to genetic mutations or age-related stress [5]. - In preclinical models, GT-02287 has shown promising results, including restoration of enzymatic function and reduction of neurodegeneration biomarkers [5][6]. Clinical Trials - GT-02287 is currently undergoing a Phase 1b clinical trial across seven sites in Australia, focusing on safety and tolerability over a three-month period [6]. - An extension of the Phase 1b study allows participants to continue treatment for up to 12 months, indicating a commitment to long-term evaluation of the drug's effects [6]. Funding and Support - The development of GT-02287 has received funding from notable organizations, including The Michael J. Fox Foundation and the Eurostars-2 program, highlighting the drug's potential and the support it has garnered [7]. Company Overview - Gain Therapeutics is a clinical-stage biotechnology company specializing in the discovery and development of next-generation allosteric therapies, with a focus on neurodegenerative diseases and other challenging disorders [8][10]. - The company employs its proprietary Magellan™ platform to accelerate drug discovery and develop novel treatments for various conditions, including lysosomal storage disorders and solid tumors [10].

Core Insights - Gain Therapeutics, Inc. has provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287, indicating its potential as a disease-modifying therapy for Parkinson's disease [1] Group 1: Clinical Data and Results - In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased by an average of 81% after 90 days of treatment with GT-02287, suggesting increased GCase activity in the brain [2] - Out of 19 patients who completed Part 1 of the study, 15 patients showed an average improvement of 2.20 points in the sum of MDS-UPDRS Part II and Part III scores after treatment [3] - The study observed stabilization in MDS-UPDRS scores and anecdotal improvements in specific functional areas such as balance, gait, and sense of smell after 90 days of dosing with GT-02287 [4] Group 2: Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [6] - Preclinical models have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease [6][7] - The drug is currently being evaluated in a Phase 1b clinical trial, with the primary endpoint focused on safety and tolerability after three months of dosing [9] Group 3: Future Developments and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research and other organizations to advance the development of GT-02287 [10] - The company is hosting a virtual event to discuss the results of the Phase 1b clinical study and engage with key opinion leaders in the field [5]

Core Viewpoint - Gain Therapeutics, Inc. is advancing its lead drug candidate GT-02287 for the treatment of Parkinson's disease, with a focus on its disease-modifying potential and biomarker data from a Phase 1b clinical study [1][2]. Company Overview - Gain Therapeutics, Inc. is a clinical-stage biotechnology company specializing in the discovery and development of next-generation allosteric therapies [10]. - The company is currently evaluating GT-02287 in a Phase 1b clinical trial for Parkinson's disease, targeting patients with or without a GBA1 mutation [10]. Drug Candidate Details - GT-02287 is an orally administered, brain-penetrant 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 enzymatic function and reduce various pathologies associated with Parkinson's disease, including neuroinflammation and neuronal death [5][6]. - The drug has demonstrated a disease-modifying effect in preclinical models, suggesting potential to slow or stop the progression of Parkinson's disease [6]. Clinical Study Insights - The Phase 1 study of GT-02287 in healthy volunteers indicated favorable safety and tolerability, with target engagement reflected in increased GCase activity [7]. - The ongoing Phase 1b clinical trial has enrolled participants across seven sites in Australia, focusing on the safety and tolerability of GT-02287 after three months of dosing [8]. - An extension of the Phase 1b study allows participants to continue treatment for up to 12 months [8]. Funding and Support - Gain Therapeutics has received funding support for its lead program from The Michael J. Fox Foundation for Parkinson's Research and The Silverstein Foundation for Parkinson's with GBA, among others [9].

Core Insights - Gain Therapeutics, Inc. has reported a significant reduction in glucosylsphingosine (GluSph) levels in cerebrospinal fluid (CSF) of Parkinson's disease (PD) patients after treatment with their drug candidate GT-02287, indicating increased GCase activity in the brain, which may influence PD progression [1][2][3] Group 1: Study Results - The Phase 1b study of GT-02287 showed that all participants with elevated GluSph levels experienced substantial decreases towards levels seen in healthy individuals after 90 days of treatment [2] - The study enrolled 21 participants, with 19 completing the 90-day dosing period, and 15 (79%) opting to continue into a nine-month extension [4] - The data monitoring committee has recommended the continuation of the Phase 1b study with no changes, confirming the treatment's tolerability [5] Group 2: Future Plans and Events - A KOL event is scheduled for January 6, 2026, to discuss the results of the study and emerging biomarker data [6] - The company plans to present longer follow-up data at the AD/PD™ conference in March 2026, focusing on the effects of GT-02287 on MDS-UPDRS scores [3] Group 3: Drug Candidate Overview - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, aimed at restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [7][8] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce ER stress, and improve motor function in models of both GBA1-PD and idiopathic PD [9][10] Group 4: 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 [12]

Core Insights - Gain Therapeutics, Inc. announced promising preclinical data for its lead drug candidate GT-02287, which shows potential as a disease-modifying therapy for Parkinson's disease (PD) by enhancing mitochondrial and lysosomal health [1][2][7] Preclinical Data - GT-02287 demonstrated a reduction in mitochondrial stress and improved neuronal survival in rat dopaminergic neurons treated with MPP+, a mitochondrial toxin [3] - In a mouse PD model, GT-02287 decreased staining for MIRO1, indicating improved mitochondrial health [4] - The drug facilitated the trafficking of glucocerebrosidase (GCase) to mitochondria, enhancing mitochondrial function in patient-derived fibroblasts with GBA1 mutations [5][6] Mechanism of Action - The drug acts throughout the disease cascade resulting from dysfunctional GCase, contributing to neuroprotection by restoring GCase function and reducing neuroinflammation and neuronal death [2][6][7] - GT-02287's effects on mitochondrial health were highlighted as a significant aspect of its mechanism, addressing previously open questions in PD research [2][4] Clinical Development - GT-02287 is currently in a Phase 1b clinical trial for PD, with early observations suggesting disease-modifying effects consistent with preclinical findings [9] - The trial aims to evaluate safety and tolerability after 3 months of dosing, with an extension allowing treatment for up to 12 months [9] Funding and Support - The development of GT-02287 has received funding from notable organizations, including The Michael J. Fox Foundation and the Eurostars-2 joint program [10]

Core Insights - Gain Therapeutics, Inc. has presented initial data from its Phase 1b study indicating that GT-02287 shows a disease-slowing effect in Parkinson's disease, consistent with preclinical models and its proposed mechanism of action [1][4][15] - The company completed enrollment of 21 participants in the Phase 1b study, surpassing the initial target of 15 participants, and has received approval to extend the study duration to 12 months [1][3][12] - Financial results for Q3 2025 show an increase in R&D expenses to $2.8 million, primarily due to ongoing clinical trials and unfavorable currency translation [9][10] Clinical Program Highlights - The Phase 1b study of GT-02287 in Parkinson's disease has shown that 16 out of 21 participants completed 90 days of dosing, with the remaining participants expected to finish by December 2025 [3][12] - Initial clinical findings presented at the 2025 MDS conference suggest improvements in Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores after approximately 30 days of administration [3][4] - The Phase 1b study extension commenced in September 2025, allowing participants to continue treatment for a total of 12 months, with a focus on long-term safety and tolerability [1][12][17] Financial Performance - R&D expenses increased by $0.2 million to $2.8 million for Q3 2025 compared to the same period in 2024, attributed to ongoing clinical trials and currency translation effects [9] - General and Administrative (G&A) expenses rose by $0.1 million to $1.9 million for Q3 2025, driven by higher stock-based compensation and personnel costs [10] - The net loss for Q3 2025 was reported at $5.3 million, or $0.15 per share, an improvement from a net loss of $4.5 million, or $0.17 per share, in Q3 2024 [11][13] Upcoming Milestones - Analysis of functional changes and biomarker activity from the Phase 1b study is expected to be available in Q4 2025 [2][4] - An IND submission to the FDA is anticipated by the end of 2025, facilitating the expansion into Phase 2 clinical development [12][18] - Results from the Phase 1b study extension are expected in the second half of 2026 [12]

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]