GT-02287 / Gain Therap - LARVOL DELTA

Efficacy, Safety, Tolerability, and Biomarker Effects of GT-02287 in Early Parkinson's Disease (clinicaltrials.gov) - P2 | N=111 | Not yet recruiting | Sponsor: Gain Therapeutics, Inc.

Biomarker • New P2 trial • CNS Disorders • Movement Disorders • Parkinson's Disease

Gain Therapeutics Presents Preclinical GT-02287 Data at Neuroscience 2025 (GlobeNewswire) - "GT-02287 reduces mitochondrial stress and enhances neuronal survival in MPP+ treated rat dopaminergic neurons. GT-02287 decreases staining for MIRO1, suggesting improved mitochondrial health in α-synuclein-induced mouse PD model."

Preclinical • Parkinson's Disease

Upcoming Anticipated Milestones (The Manila Times) - "Analysis of functional changes scored according to MDS-UPDRS and biomarker levels in cerebrospinal fluid and blood samples from participants who completed 90 days of the Phase 1b study evaluating GT-02287 in people with Parkinson’s disease expected in the fourth quarter of 2025; IND submission to FDA, expected by year end 2025, facilitating expansion into Phase 2 clinical development of GT-02287 to include clinical sites in the United States; Results from Phase 1b study extension expected the second half of 2026."

IND • P1 data • Parkinson's Disease

Completed enrollment in the Phase 1b study for GT-02287 in people with Parkinson’s disease regardless of GBA1 status. 21 participants have been enrolled as of September 30, 2025, surpassing original target enrollment of 15 participants. (The Manila Times)

Enrollment closed • Parkinson's Disease

GT-02287, a small molecule allosteric modulator of the lysosomal enzyme glucocerebrosidase (GCase), also affects GCase at the level of the mitochondria (Neuroscience 2025) - "2023, 14: 1930 . https://doi.org/10.1038/s41467-023-37454-4"

CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Glucocerebrosidase Target Engagement and Therapeutic Plasma and Cerebrospinal Fluid Levels After GT-02287 Administration in Healthy Volunteers. (PubMed, Mov Disord) - "GT-02287 was safe and well-tolerated in healthy volunteers. Plasma and CSF levels were consistent with levels in rodents that modulate PD biology."

Journal • CNS Disorders • Movement Disorders • Pain • Parkinson's Disease • GBA1

Gain Therapeutics to Present at Neuroscience 2025 (The Manila Times)

Clinical data • Parkinson's Disease

GT-02287 in Parkinson's Disease: Interim Data from a Phase 1b Study (MDS Congress 2025) - "GT-02287 is a GCase-targeting small molecule that successfully completed Phase 1 in healthy volunteers and that is currently being evaluated in people with PD. Based on the genetic association of GBA1 variants with PD and extensive preclinical data demonstrating that GT-02287 modulates molecular pathways implicated in PD, this novel compound has the potential to slow disease progression in GBA1-PD and possibly in idiopathic PD."

P1 data • CNS Disorders • Movement Disorders • Parkinson's Disease • NEFL

Gain Therapeutics Presents Initial Data from Phase 1b Clinical Study of GT-02287 in Parkinson’s Patients at International Congress of Parkinson’s Disease and Movement Disorders (GlobeNewswire) - "Mean MDS-UPDRS scores at baseline were 5.8, 7.4, and 24.7 for Parts I, II, and III, respectively. Importantly, several participants experienced an improvement in their UPDRS Part II and III scores after 90 days of dosing with GT-02287 while mean Part I scores remained unchanged. The mean improvement in Parts II and III by Day 90, which was not observed by Day 30, suggests that GT-02287 has a disease-slowing effect..."

P1 data • Movement Disorders • Parkinson's Disease

Gain Therapeutics, Inc…announced the start of the Phase 1b extension study allowing participants to continue treatment for an additional nine months. (Gain Therapeutics Press Release) - "More than half of the participants have agreed to continue treatment. The Phase 1b study enrolled 21 participants and is expected to complete in December 2025. Early data from the Phase 1b study will be presented on October 7, 2025, at the International Congress of Parkinson’s Disease and Movement Disorders® in Honolulu, HI."

P1 data • Trial status • Parkinson's Disease

Upcoming Anticipated Milestones (GlobeNewswire) - "Approval from healthcare authorities in Australia to extend the dosing duration for participants in the Phase 1b beyond the 90 days specified under the current protocol; IND submission to FDA expected by year end 2025 facilitating expansion of Phase 2 clinical development for GT-02287 to include clinical sites in the United States."

IND • Trial status • Parkinson's Disease

A clinical study to compare the safety and tolerability of two formulations of a new drug in Healthy Participants for Parkinson's disease. (ANZCTR) - P1 | N=24 | Completed | Sponsor: Gain Therapeutics, Australia Pty Ltd (local Sponsor) | Not yet recruiting ➔ Completed

Trial completion • CNS Disorders • Movement Disorders • Parkinson's Disease

Gain Therapeutics Announces Pricing of $7.0 Million Public Offering (GlobeNewswire) - "Gain Therapeutics...announced the pricing of an underwritten public offering of 4,501,640 shares of its common stock and warrants to purchase up to an aggregate of 2,250,820 shares of its common stock. The public offering price for each set of two shares of common stock and accompanying warrant to purchase one share of common stock is $3.11 per set of securities, yielding an effective price of $1.55 per share and $0.01 per warrant....Gain intends to use the net proceeds from the offering to continue clinical and nonclinical development of its lead product candidate GT-02287 for the treatment of neurodegenerative diseases including GBA1 Parkinson’s disease and for general corporate purposes."

Financing • Parkinson's Disease

Gain Therapeutics Announces Completion of Target Enrollment in Phase 1b Clinical Study Evaluating GT-02287 in People With Parkinson’s Disease With or Without a GBA1 Mutation (The Manila Times) - "Gain Therapeutics, Inc...provided an update on the progress of its ongoing Phase 1b clinical study evaluating the safety and tolerability of GT-02287 in people with Parkinson’s Disease with or without a GBA1 mutation. The study, which aimed to enroll 15-20 participants, reached 16 participants on June 30th, 2025....Analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) in all participants currently enrolled is now expected to also track earlier; available in 4Q 2025 instead of 1Q 2026; Gain will continue screening patients for enrollment through July 31st, 2025, at the request of clinical investigators and additional participants who wish to gain access to the study....Long-term chronic toxicology studies required to support this extension of dosing are near completion, and Gain is expected to provide an update on the progress of the extension before the end of 3Q25."

P1 data • Trial status • Parkinson's Disease

Gain Therapeutics Reports Financial Results for First Quarter 2025 and Provides Corporate Update (GlobeNewswire) - "Upcoming Anticipated Milestones: First biomarker analysis from the Phase 1b study evaluating GT-02287 in people with Parkinson’s disease expected mid-2025; Completion of Phase 1b study expected 4Q25; 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."

Biomarker • IND • New P2 trial • P1 data • Trial status • Parkinson's Disease

Gain Therapeutics Presents Preclinical Data at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders (GlobeNewswire) - "In cultured rat mesencephalic dopaminergic neurons treated with MPP+, a mitochondrial toxin that induces mitochondrial impairment by inhibiting mitochondrial complex 1, GT-02287 improved the function of both the lysosomes and mitochondria and exerted a broader neuroprotective effect. GT-02287 lowered α-synuclein aggregation, lysosomal dysfunction, and mitochondrial stress while also preventing the release of mitochondrial cytochrome C, a programmed cell death (apoptosis) signal, thereby promoting neuronal survival."

Preclinical • Parkinson's Disease

Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders. (PubMed, Int J Mol Sci) - "Using Site-directed Enzyme Enhancement Therapy (SEE-Tx®), two structurally targeted allosteric regulators (STARs), GT-02287 and GT-02329, were developed and tested in GD patient-derived fibroblasts with relevant GCase variants. This enhanced lysosomal function and reduced cellular stress. These findings validate the STARs' mechanism of action and highlight their therapeutic potential for GCase-related disorders, including GD, PD, and Dementia with Lewy Bodies."

Journal • Alzheimer's Disease • CNS Disorders • Dementia • Gaucher Disease • Genetic Disorders • Lewy Body Disease • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Rare Diseases • GBA • GBA1

Gain Therapeutics Announces Oral Poster Presentation at IAPRD 2025 30th World Congress on Parkinson's Disease and Related Disorders (GlobeNewswire) - "Gain Therapeutics, Inc...today announced an oral poster presentation at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, being held May 7th-10th in New York City, NY."

Clinical data • Parkinson's Disease

Gain Therapeutics Presents...Design of Phase 1b Clinical Study of GT-02287 at AD/PD 2025 and Provides Enrollment Update (GlobeNewswire) - "An early biomarker analysis from the currently enrolled participants in the Phase 1b study will be conducted mid-year and will include samples from participants both with and without the GBA1 mutations for purposes of informing Phase 2 planning during the second half of 2025. Complete analysis of the Phase 1b endpoints, available after all patients have completed 90 days of treatment, is anticipated sometime during the fourth quarter of 2025....The study will assess safety and tolerability of GT-02287 during three months of dosing in people with PD, using adverse events, laboratory tests, electrocardiograms, and vital signs as the key safety endpoints....Results from this Phase 1b study will guide the design of a subsequent randomized, placebo-controlled, double-blind dose-finding Phase 2 study to evaluate the efficacy of GT-02287 as measured by a clinically meaningful endpoint in PD over a longer treatment period.​ Phase 2 planning is expected to commence in the second..."

Clinical protocol • New P2 trial • Trial status • Parkinson's Disease

Gain Therapeutics Presents Additional Preclinical Data...of GT-02287 at AD/PD 2025 (GlobeNewswire) - "The new preclinical data that were presented additionally support a disease-modifying potential of GT-02287. Several biomarkers of disease progression, including aggregated α-synuclein, IRE-1 (a marker of ER stress), LAMP-1 (a marker of lysosomal integrity), Miro1 (a marker of damaged mitochondrial destined for mitophagy), phospho-Tau (a marker of neurodegeneration), and Iba-1 (a marker of neuroinflammation) were statistically reduced upon treatment with GT-02287 and remained reduced for several days following drug washout demonstrating a long-lasting effect."

Preclinical • Parkinson's Disease

DESIGN OF A PHASE 1B STUDY TO EVALUATE THE GCASE -TARGETING MOLECULE GT -02287 IN GBA1 -PD AND SPORADIC PD. (ADPD 2025) - "GT-02287 is a novel brain -penetrant, GCase -targeting small molecule that has successfully completed Phase 1 in healthy volunteers and that is now being evaluated in the first study in people with PD. Based on extensive preclinical data, GT -02287 has the po tential to slow disease progression in both GBA1 -PD and sporadic PD."

P1 data • CNS Disorders • Movement Disorders • Parkinson's Disease • NEFL

CLINICAL STAGE ALLOSTERIC GLUCOCEREBROSIDASE MODULATOR, GT-02287, SHOWS DISEASE MODIFYING POTENTIAL IN PRECLINICAL MODELS OF BOTH GBA1 AND IDIOPATHIC PARKINSON'S DISEASE (ADPD 2025) - "Conclusions This study further supports GT-02287's potential as a disease modifying therapy for both idiopathic and GBA1 Parkinson's disease. A clinical study including both types of patients is expected to conclude in H2 2025."

Preclinical • CNS Disorders • Cognitive Disorders • Developmental Disorders • Movement Disorders • Parkinson's Disease • GBA1

Gain Therapeutics Announces Poster Presentation at AD/PD 2025 (GlobeNewswire) - "Gain Therapeutics, Inc...announced that a poster detailing the design of the Phase 1b clinical trial of GT-02287 in Parkinson’s disease will be presented at the AD/PD 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders."

Clinical protocol • Parkinson's Disease

Gain Therapeutics Reports Financial Results for the Fourth Quarter and Year End 2024 and Provides Corporate Update (GlobeNewswire) - "Commenced pre-Investigational New Drug (IND) engagement with U.S. Food and Drug Administration (FDA) in preparation for expansion of GT-02287 Phase 2 clinical development to include the United States....IND submission to FDA expected by year end 2025....Research and development (R&D) expenses decreased by $0.7 million to $10.8 million for year ended December 31, 2024, as compared to $11.5 million for the year ended December 31, 2023....These increases were partially offset by higher costs during 2024 associated with the Phase 1 clinical trial of the Company’s lead program compound GT-02287 for the treatment of Parkinson’s disease."

Commercial • IND • Parkinson's Disease

Gain Therapeutics Doses First Participant in Phase 1b Clinical Trial of GT-02287 in Parkinson’s Disease (GlobeNewswire) - "Gain Therapeutics, Inc...today announced the dosing of the first participant with Parkinson’s disease (PD) in its Phase 1b clinical trial of GT-02287, the Company’s lead allosteric small molecule in development for the treatment of PD with or without a GBA1 mutation...Interim data from the Phase 1b trial are anticipated at the end of 2Q 2025."

P1 data • Trial status • Parkinson's Disease