Gain Therapeutics, Inc. Stock Price, News & Analysis

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.

Gain Therapeutics (NASDAQ: GANX) presented interim Phase 1b data for GT-02287 in Parkinson’s disease on October 6, 2025. Early dosing produced stability and trending improvements in MDS‑UPDRS Parts II and III by Day 90 in several participants while Part I remained unchanged.

GT-02287 was generally well tolerated with no treatment‑emergent serious adverse events, a consistent plasma PK profile within the projected therapeutic range, and DMC recommendations to continue the study. Australian authorities approved a study extension allowing treatment up to 12 months. A virtual KOL webinar is scheduled for October 14, 2025.

Gain Therapeutics (NASDAQ:GANX), a clinical-stage biotech company focused on developing next-generation allosteric small-molecule therapies, announced its participation in the Drug Discovery Innovation Programme 2025. Dr. Rinaldo Montalvão, the company's Director of Artificial Intelligence, will present a talk titled "Transforming Drug Discovery: Generative Models and Electrostatic Complementarity" during the AI and Automation in Drug Discovery track on September 26, 2025, at 9:20am CEST in Barcelona, Spain.

Gain Therapeutics (NASDAQ:GANX) has initiated a Phase 1b extension study for its Parkinson's disease treatment GT-02287, allowing participants to continue dosing for an additional nine months beyond the initial 90-day schedule. The original Phase 1b study, which enrolled 21 participants, is set to complete in December 2025.

The company will present early results from the Phase 1b study at the International Congress of Parkinson's Disease and Movement Disorders on October 7, 2025. Additional data, including MDS-UPDRS functional changes and biomarker data from cerebrospinal fluid and blood, will be available in Q4 2025. Management reports encouraging early observations regarding safety, tolerability, pharmacokinetics, and functional impact.