GANX

BETHESDA, Md., May 11, 2026 (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 quarter ended March 31, 2026, and provided a corporate update. “The first quarter of 2026 marked another exciting quarter for Gain, as we continued to build on the important advancements achieved in 2025 related to both the scientific understanding and clinical development of our lead candidate GT-02287, in development for the treatment of Parkinson’s disease with or without a GBA1 mutation. This is underscored by the promising biomarker and early clinical evidence from our ongoing Phase 1b study supporting the potential disease modifying properties of GT-02287 that we presented at AD/PD™ 2026 in Copenhagen this past March,” said Gene Mack, President and CEO of Gain Therapeutics. Mr. Mack added, “The analysis of biomarkers and clinical evidence of efficacy is ongoing throughout the nine-month extension of the Phase 1b study, which is expected to complete in October 2026. MDS-UPDRS scores have remained durable to date, and we look forward to sharing more details at the upcoming 3rd International GBA1 Meeting later this month in Phoenix, Arizona. We believe the totality of the data supports the potential of GT-02287 to treat both idiopathic and GBA1 Parkinson’s disease, and we remain focused on helping to shift the treatment paradigm from managing symptoms of Parkinson’s disease towards disease modification and addressing the underlying biology causing those symptoms. We expect to receive FDA clearance of our IND during Q2 2026 and remain on track to begin our Phase 2 study in Q3 2026.” First Quarter 2026 and Recent Corporate and Pipeline Highlights Pipeline Updates GT-02287 Presented additional data from the Phase 1b clinical study of GT-02287 demonstrating central target engagement, beneficial effects on downstream pathway abnormalities, and clinical improvement in participants, that supported the disease-modifying potential of GT-02287 at AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Disease and Related Neurological Disorders in March 2026 in Copenhagen, Denmark. As of March 2026, 14 of 16 participants enrolled in Phase 1b nine-month extension h...

BETHESDA, Md., March 26, 2026 (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, 2025, and provided a corporate update. “We are encouraged by the progress made in 2025, as we made important advancements related to both the scientific understanding and clinical development of our lead candidate GT-02287, in development for the treatment of Parkinson’s disease with or without a GBA1 mutation. The promising impact GT-02287 has on the causative biology of Parkinson’s disease has been further elucidated from the analysis of functional and biomarker changes from the Phase 1b study,” commented Gene Mack, President and CEO of Gain Therapeutics. Mr. Mack continued, “We will continue to follow patients in the Phase 1b nine-month extension study that is expected to complete in September 2026 and look forward to presenting longer-term follow up at additional scientific conferences throughout the balance of the year. To date, MDS-UPDRS scores remained stable and durable across the overall study population after 150 days of treatment with GT-02287 and are becoming more encouraging with the passage of time. We believe the totality of the data continues to support the potential of GT-02287 in both idiopathic and GBA1 Parkinson’s disease and we hope to one day shift the treatment paradigm away from symptomatic relief and to disease modification.” Fourth Quarter 2025 and Recent Corporate and Pipeline Highlights Pipeline Updates Presented initial data from the Phase 1b clinical study of GT-02287 demonstrating the disease-modifying potential of GT-02287 in people with Parkinson’s disease (PD) at the International Congress of Parkinson’s Disease and Movement Disorders® in October 2025 in Honolulu, HI. Presented preclinical data demonstrating evidence that GT-02287 beneficially impacts mitochondrial health in addition to lysosomal health and endoplasmic reticulum stress at Neuroscience 2025 in November 2025 in San Diego, CA. Presented additional data from the Phase 1b clinical study of GT-02287 demonstrating central target engagement, beneficial effects on downstream pathway abnormalities, and clinical improvement in partici...

Results showed CNS target engagement with reduction in GCase substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), a prespecified endpoint The reduction in GluSph in CSF, a first-ever observation following the administration of a GCase modulator to PD patients, suggests increased GCase activity in the brain, which is expected to impact the progression of Parkinson’s disease (PD) KOL event planned for early January to discuss the results; registration information herein BETHESDA, Md., Dec. 18, 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 provided evidence, for the first time in Parkinson’s Disease (PD), of a reduction in glucocerebrosidase (GCase) substrate in cerebrospinal fluid (CSF). Decreased GluSph in CSF is an indication of increased GCase activity in the brain All individuals with elevated levels of glucosylsphingosine (GluSph) in the CSF displayed large decreases back towards levels observed in healthy individuals after 90 days of treatment with GT-02287. Importantly, the change observed in GluSph in CSF was a prespecified exploratory endpoint of this Phase 1b study. Elevated GluSph, a hallmark of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial function and other intracellular processes in neurons. Gene Mack, president and CEO of Gain Therapeutics, commented on the results, stating, “We are excited by the unfolding biomarker evidence of GT-02287 activity and central nervous system target engagement. To our knowledge, GT-02287 is the first GCase modulator to demonstrate a reduction of GluSph in CSF in people with PD, providing downstream evidence of GCase enhancement in the brain. Further, we believe that reduction in brain GluSph levels will have a direct impact on neuronal health and translate to clinically observable improvements.” Mr. Mack continued, “With a capital position sufficient to fund operations through the end of the Phase 1b extension and year-end 2026, we look forward to presenting longer follow-up from the study at the AD/PD™ conference in March 2026, observing the effect of GT-02287 treatment on MDS-UPDRS scores in the participants who continued in the nine-month extensi...

Presented initial data from Phase 1b study suggesting GT-02287 has a disease-slowing effect consistent with the preclinical models in vivo and the proposed mechanism of action Completed enrollment of 21 participants in Phase 1b study evaluating GT-02287 in Parkinson’s Disease with or without GBA1 mutations during 3Q 2025 Received approval from the Australian health authorities to extend the duration of the Phase 1b study, allowing participants to continue treatment with GT-02287 for a total of 12 months; with a majority of subjects electing to participate in the study extension Analysis of functional changes and biomarker activity at 90 days expected to be available during 4Q 2025 BETHESDA, Md., Nov. 12, 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 quarter ended September 30, 2025, and provided a corporate update. “We are encouraged by the progress made during the third quarter of 2025 and remain on track to report the analysis of both functional changes and biomarker activity during the fourth quarter of 2025. We are excited to better understand the impact GT-02287 has on the biology of Parkinson’s disease from this analysis and look forward to presenting what we learn. We presented early clinical findings last month at the 2025 MDS conference in Hawaii suggesting GT-02287 has a disease-slowing effect as evidenced by stabilization and improvement in Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) scores that appears after approximately 30 days of administration according to our data,” said Gene Mack, President and CEO of Gain. “We are further encouraged by the strong engagement we have seen in the Phase 1b study, with approximately 80% of participants having joined or confirmed their interest in the study extension phase. We look forward to generating additional feedback from this study extension that we can incorporate into our Phase 2 planning, which is already underway.” Important highlights during the quarter included completion of enrollment for the company’s Phase 1b study evaluating lead candidate GT-02287 in Parkinson’s disease with or without a GBA1 mutation. Of the 21 participants enrolled, wh...

Reached full enrolment of Phase 1b study evaluating GT-02287 in Parkinson’s Disease (PD) with or without GBA1 mutations during 2Q 2025, earlier than anticipated Analysis of functional changes and biomarker activity at 90 days will be available during 4Q 2025 In July 2025 following the close of 2Q 2025, Gain completed an underwritten public offering that resulted in approximately $7.1 million of net proceeds BETHESDA, Md., Aug. 12, 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 quarter ended June 30, 2025, and provided a corporate update. Important highlights during the quarter included earlier-than-anticipated completion of target enrollment for the company’s Phase 1b study evaluating lead candidate GT-02287 in Parkinson’s disease with or without a GBA1 mutation. As a result, Gain expects the analysis of participants’ functional changes scored according to the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and biomarker activity from cerebrospinal fluid at 90 days to be available during 4Q 2025. As of June 30, 2025, a total of 16 patients were enrolled in the Phase 1b study. However, enrollment has been kept open to any participants screened prior to July 31 who meet the criteria for the study. Total enrollment of the Phase 1b is not expected to exceed 20 participants. Additionally, the company recently submitted a request to extend the dosing duration of the Phase 1b study beyond the current 90 days allowable in the original protocol; an update on the status of that request is expected in the coming weeks. “Reaching target enrollment in the Phase 1b study of GT-02287 during 2Q 2025 was an exciting achievement for Gain and we look forward to reporting the analysis of both functional changes and biomarker activity during 4Q 2025, which will go a long way in planning the design of our Phase 2 clinical trial for GT-02287, an effort that has already begun in earnest,” said Gene Mack, President and CEO of Gain. “We are further encouraged by clinicians’ request to extend the screening window for participants by another month and their support to extend the dosing duration for participants in the study who wish...

First Biomarker Analysis from Phase 1b Study of GT-02287 in Parkinson’s Disease Expected Mid-2025 BETHESDA, Md., May 14, 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 quarter ended March 31, 2025, and provided a corporate update. “The first quarter of 2025 marked yet another exciting milestone for Gain with the first administration of GT-02287, our lead drug candidate in development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation, to participants with Parkinson’s disease in our ongoing Phase 1b study which began enrollment in early March,” said Gene Mack, President and CEO of Gain. “We are encouraged by enrollment of the study to date and expect to complete enrollment by the end of July 2025. In the meantime, we look forward to sharing results from a first biomarker analysis of the study in mid-2025. This readout is of significance as we believe that these observations will further elucidate the mechanism of GT-02287, which we believe goes beyond activation of lysosomal GCase to stabilize and chaperone the enzyme through various cellular compartments where it functions to promote neuroprotection. We look forward to incorporating what we learn from the Phase 1b into our Phase 2 planning for GT-02287 in 2H25. As we progress through the year, we will continue to engage with the FDA and anticipate IND submission by year end.” First Quarter 2025 and Recent Corporate and Clinical Program Highlights Corporate Updates Gene Mack was appointed President and Chief Executive Officer effective January 6, 2025. Prior to the appointment Gene served as Chief Financial Officer from April 2024. Since Mr. Mack’s appointment, Gianluca Fuggetta has assumed the role of Senior Vice President Finance and Principal Financial Officer. Announced formation of a Clinical Advisory Board composed of leading experts in Parkinson’s disease to support the advancement of GT-02287 through late-stage clinical development. including Parkinson’s Initiated enrollment of a Phase 1b study for GT-02287, which is now being administered for the first time to individuals with Parkinson’s disease (PD) regardless of GBA1 status. The open-label study wil...

First Analysis from Phase 1b Study of GT-02287 in Parkinson’s Disease Expected in Q2 2025 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 clinical development of our lead candidate GT-02287, in development for the treatment of Parkinson’s disease with or without GBA1 mutation,” said Gene Mack, President and CEO of Gain. “Building upon this momentum in 2025, we have recently initiated dosing in our Phase 1b trial of GT-02287 and look forward to sharing results from a first analysis of the study anticipated for the end of the second quarter of 2025. We believe the observations from this analysis will mark a significant value inflection point for the GT-02287 program and importantly, inform the planning of our Phase 2 study during the second half of 2025. At the same time, we are working towards expanding GT-02287 clinical development to the U.S. and continue to have productive dialogue with the FDA. We look forward to providing continued updates on our progress as we aim to achieve these milestones throughout the year.” Fourth Quarter 2024 and Recent Corporate Highlights Pipeline Updates First participant dosed in the Phase 1b trial of GT-02287 in Parkinson’s disease. First analysis is anticipated at the end of the second quarter 2025. Presented new clinical data from a Phase 1 study of GT-02287 in healthy volunteers that demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement with a statistically significant >50% increase in glucocerebrosidase (GCase) activity among those receiving GT-02287 at clinically relevant doses during the Michael J. Fox Foundation’s 16th Annual Parkinson’s Disease Therapeutics Conference in October 2024 in New York, NY. Presented preclinical data elucidating a broader impact on the biology of Parkinson’s disease than was previously understood to be associated with GT-02287 stabilization and modulati...