Welcome to our dedicated page for Gain Therapeutics news (Ticker: GANX), a resource for investors and traders seeking the latest updates and insights on Gain Therapeutics stock.
Gain Therapeutics, Inc. (Nasdaq: GANX) is a clinical-stage biotechnology company whose news flow is closely tied to the development of its lead drug candidate, GT-02287, and to progress in its broader allosteric small molecule pipeline. The company focuses on next generation allosteric therapies targeting neurodegenerative diseases, rare genetic disorders and oncology, with a primary emphasis on Parkinson’s disease.
News about Gain Therapeutics frequently covers clinical milestones for GT-02287, including updates from its Phase 1 and Phase 1b studies in people with Parkinson’s disease with or without a GBA1 mutation. Press releases have detailed safety and tolerability findings, exploratory biomarker data such as reductions in glucosylsphingosine (GluSph) in cerebrospinal fluid, and observations related to Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) scores. These items provide insight into how the company interprets GT-02287’s potential disease-modifying effects and mechanism of action.
Investors and observers can also expect regular announcements on preclinical research and scientific presentations. Gain Therapeutics reports data from preclinical models that explore GCase modulation, mitochondrial and lysosomal health, and neuroprotection in Parkinson’s disease. Poster presentations at scientific meetings, such as Neuroscience conferences and Parkinson’s-focused congresses, are common subjects of the company’s news releases.
Additional news items include corporate updates and financial disclosures, such as quarterly financial results, capital raises, changes in board composition, and shareholder meeting outcomes. The company also issues notices about participation in investor conferences, key opinion leader (KOL) events, and webinars that discuss biomarkers, clinical endpoints and emerging data from GT-02287 studies.
For readers following GANX, the news page provides a centralized view of clinical trial progress, scientific data, regulatory and corporate developments, and event participation related to Gain Therapeutics’ efforts in allosteric small molecule drug development.
Gain Therapeutics (Nasdaq: GANX) management will present at the Oppenheimer 36th Annual Healthcare Life Sciences Conference on Wednesday, February 25, 2026, including a fireside chat at 1:20 p.m. ET.
A replay will be made available via the company website and investors can request one-on-one meetings through Oppenheimer or by contacting ir@gaintherapeutics.com.
Gain Therapeutics (NASDAQ: GANX) announced its participation in events during the week of the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, Jan 11–14, 2026.
Planned events include: the 9th Annual Sachs Neuroscience Innovation Forum on Jan 11, 2026 (presentation and one-on-ones; presentation at 2:40 p.m., Track C — Rosenberg Room, Marine's Memorial Club), the Demy-Colton/Informa Biotech Showcase on Jan 12–14, 2026 (presentation and one-on-ones; Hilton San Francisco Union Square), and the LifeSci Advisors 15th Annual Corporate Access Event on Jan 14, 2026 (one-on-one meetings; The Beacon Grand).
Investors can request in-person meetings via the Biotech Showcase partnering system or by registering/contacting LifeSci Advisors representative Alex Grossman (alex@lifesciadvisors.com).
Gain Therapeutics (Nasdaq: GANX) reported Phase 1b biomarker and clinical data for GT-02287 on January 6, 2026, showing biochemical and clinical signals after 90 days of dosing.
Key findings: in participants with elevated baseline cerebrospinal fluid glucosylsphingosine (GluSph), GluSph fell 81% on average after 90 days; as of Nov 30, 2025, 19 patients completed Part 1 (90 days), and 15 evaluable patients showed an average -2.20 point change in the sum of MDS‑UPDRS Part II+III (Part II: -0.6; Part III: -1.6). Four patients were excluded from MDS‑UPDRS analysis (two alpha-synuclein assay negative; two missing ‘off’ state scores). The company describes the GluSph reduction as a first-ever CSF observation after a GCase modulator and says results support continued development and evaluation in the dosing extension.
Gain Therapeutics (Nasdaq: GANX) will host a virtual key opinion leader event on January 6, 2026 at 10:00 a.m. EST titled “Understanding GCase Substrates in Parkinson’s Disease: Perspectives on Biomarkers and Disease Modification”.
Featured speakers include Roy Alcalay, M.D., M.Sc. and Peter Lansbury, Ph.D.. The program will review biomarker results from the Phase 1b clinical study of GT-02287, described as demonstrating disease‑modifying potential in Parkinson’s disease patients with or without a GBA1 mutation. A live question-and-answer session will follow the presentations and registration is required.
Gain Therapeutics (Nasdaq: GANX) announced Phase 1b data showing first-ever reduction of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF) after 90 days of dosing with GT-02287, a GCase modulator for Parkinson’s disease. The prespecified exploratory endpoint suggests CNS target engagement and increased GCase activity. The 21-participant study had 19 completers; 15 (79%) entered a nine-month extension expected to finish in September 2026. GT-02287 was generally well tolerated and the DMC recommended continuation. A virtual KOL webinar is scheduled for Jan 6, 2026.
Gain Therapeutics (Nasdaq: GANX) presented preclinical data for lead candidate GT-02287 at Neuroscience 2025 (Nov 15–19, 2025) showing mitochondrial and neuronal benefits across three models.
Key findings: reduced mitochondrial ROS and prevented cytochrome C release in MPP+-treated rat dopaminergic neurons; reduced MIRO1 staining in an α-synuclein mouse PD model; and increased mitochondrial GCase, boosted complex I activity, and improved membrane potential in patient fibroblasts with the L444P GBA1 mutation. The company said these results support a mechanism of GCase trafficking to mitochondria and lysosomes and reinforce planned 90-day human biomarker analyses.
Gain Therapeutics (Nasdaq: GANX) reported Q3 2025 results and clinical updates for lead candidate GT-02287.
Highlights: completed enrollment of 21 participants in the Phase 1b Parkinson’s study (target 15), presented initial data showing MDS‑UPDRS stabilization/improvement after ~30–90 days, no treatment‑emergent serious adverse events, plasma PK within projected therapeutic range, and Australian approval to extend dosing to a total of 12 months. Analysis of functional changes and biomarkers at Day 90 is expected in Q4 2025. Company reported $8.8M cash at Sept 30, 2025, and completed a public offering netting approximately $7.1M.
Gain Therapeutics (Nasdaq: GANX) will present a poster at Neuroscience 2025 in San Diego, taking place November 15–19, 2025.
The poster (Session: Poster PSTR438) is titled “GT-02287, a small molecule allosteric modulator of lysosomal enzyme glucocerebrosidase (GCase), acts at both lysosomes and mitochondria”. Presentation is scheduled for Wednesday, November 19, 2025, 1:00 PM–5:00 PM in SDCC Halls B-H. The topic focuses on developing Parkinson’s disease therapeutic strategies using cellular models.
Gain Therapeutics (Nasdaq: GANX) will participate in the 2025 Maxim Growth Summit on October 22–23, 2025 at The Hard Rock Hotel NYC.
Gene Mack, President and CEO, will join a live discussion on October 22 titled “Neurodegenerative Diseases Panel – Under the Radar Innovators in One of the Most Challenging Therapeutics Categories.” The panel will cover innovative approaches for neurodegenerative diseases that are currently in active clinical trials. Investors or analysts interested in a meeting with Gain management are instructed to contact their Maxim sales representative to schedule.
Gain Therapeutics (NASDAQ: GANX) will host a virtual Key Opinion Leader event titled “Biomarkers, Clinical Endpoints, and the Path to Disease Modification: Contextualizing the emerging data from GT-02287” on October 14, 2025 at 4:00 p.m. EST.
Speakers include Karl Kieburtz, M.D., M.P.H., and Kenneth Marek, M.D. Presentations will review biomarkers, clinical outcome scales, and endpoints to contextualize Phase 1b data for GT-02287 in Parkinson’s disease. The Phase 1b data presented show GT-02287 was generally well tolerated with no treatment-emergent serious adverse events and trending improvements in MDS-UPDRS scores. A live Q&A will follow the presentations.