GT-02287 / Gain Therap - LARVOL DELTA

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

Gain Therapeutics Announces Oral Presentation at AD/PD 2025 (GlobeNewswire) - "Gain Therapeutics, Inc...today announced that Joanne Taylor, Ph.D., Senior Vice President of Research at Gain, will give an oral presentation on the Company’s clinical stage lead drug candidate GT-02287 at the AD/PD 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders. AD/PD 2025 is being held April 1-5, 2025, in Vienna, Austria."

Preclinical • Parkinson's Disease

GANX-001-V102: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: Gain Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Parkinson's Disease

Gain Therapeutics Announces Formation of Clinical Advisory Board to Support Continued Advancement of Lead Drug Candidate GT-02287 (GlobeNewswire) - "Gain Therapeutics, In...announced the formation of its Clinical Advisory Board (CAB) to support the continued development of the Company’s lead drug candidate, GT-02287, in Parkinson’s disease. The CAB is composed of leading experts in Parkinson’s disease trial design, genetics, and biomarkers and will work closely with Gain leadership to progress GT-02287 through potential Phase 2 and Phase 3 clinical development."

Clinical • Parkinson's Disease

Gain Therapeutics Initiates Phase 1b Clinical Trial of Lead Candidate GT-02287 in People with GBA1 and Idiopathic Parkinson’s Disease (GlobeNewswire) - "Gain Therapeutics...announced it has received approval in Australia to initiate a Phase 1b trial. Gain will be working with local Parkinson’s disease (PD) advocacy groups to support enrollment and expects enrollment to complete in the spring of 2025 with data from the study expected mid-2025. GT-02287 is the Company’s lead allosteric small molecule in clinical development for the treatment of PD with or without a GBA1 mutation. The primary goal of the Phase 1b trial is to assess the safety and tolerability of GT-02287 in people with PD."

P1 data • Trial status • Parkinson's Disease

GANX-001-V102: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: Gain Therapeutics, Inc.

New P1 trial • CNS Disorders • Movement Disorders • Parkinson's Disease

Gain Therapeutics Reports Financial Results for Third Quarter 2024 and Provides Corporate Update (GlobeNewswire) - "Upcoming Milestones: Initiation of Phase 1b trial evaluating GT-02287 in Parkinson’s disease patients expected in Q4 2024; Pre-IND meeting with the U.S. Food and Drug Administration (FDA) anticipated by the end of 2024."

FDA event • New P1 trial • CNS Disorders • Parkinson's Disease

GT-02287, a clinical stage GCase regulator, demonstrates disease modifying capacity in both GBA1 and idiopathic Parkinson’s disease models (Neuroscience 2024) - "GT-02287 treatment was then withdrawn from half the treated animals, and we were able to demonstrate that a treatment effect in all three behavioural tests was maintained even in the absence of drug candidate, presumably due to a disease-modifying effect of GT-02287. This study supports the potential of GT-02287 as a disease modifying therapy for both idiopathic and GBA1 Parkinson’s disease."

Clinical • Late-breaking abstract • CNS Disorders • Cognitive Disorders • Parkinson's Disease • GBA

GT-02287, a GCase modulator and Gain Therapeutics’ PD drug candidate prevents Tau accumulation in a cellular model (Neuroscience 2024) - "Notably, the rescuing effect was also observed on the GCase wild-type background. Based on these findings, GT-02287 shows promise as a potential disease-modifying treatment option for AD and other tauopathies."

Late-breaking abstract • Alzheimer's Disease • CNS Disorders • Parkinson's Disease

GT-02287, a clinical stage GCase regulator, improves mitochondrial function and provides a neuroprotective effect in GBA1-Parkinson's disease models (Neuroscience 2024) - "Delayed administration of GT-02287 reduced the levels of Miro1 in the substantia nigra and plasma levels of emerging neurodegeneration biomarker neurofilament light chain (NfL) as well as completely restoring motor function to control levels. These data highlight the ability of GT-02287 not only to repair lysosomal dysfunction, but also to alleviate mitochondrial dysfunction which adds to the body of evidence supporting GT-02287 as a potential disease-modifying therapy for Parkinson’s disease."

Clinical • CNS Disorders • Parkinson's Disease • GBA • NEFL

Gain Therapeutics to Present at Michael J. Fox Foundation’s 16th Annual Parkinson’s Disease Therapeutics Conference (GlobeNewswire) - "Gain Therapeutics, Inc...announced that Jonas Hannestad...will give an oral presentation on the Company’s clinical stage lead drug candidate GT-02287 at the Michael J. Fox Foundation’s (MJFF) 16th Annual Parkinson’s Disease Therapeutics Conference on October 17, 2024, in New York City....'We look forward to presenting Phase 1 data for GT-02287, in development for the treatment of Parkinson’s disease, at the upcoming MJFF conference and appreciate the opportunity to contextualize these data with exciting preclinical data that have been generated recently for GT-02287'."

Clinical data • P1 data • Preclinical • Parkinson's Disease

Gain Therapeutics Presents Phase 1 GT-02287 Data at International Congress of Parkinson’s Disease and Movement Disorders Demonstrating Increase in GCase Activity in Healthy Volunteers (GlobeNewswire) - P1 | N=73 | ACTRN12623000860662 | Sponsor: Gain Therapeutics, Australia Pty Ltd | “The single and multiple dose levels tested were safe and generally well tolerated, with no serious adverse events or Grade 3 (severe) adverse events observed, and no other safety signals detected. The PK profile of GT-02287 was linear across the tested dose ranges, and plasma exposures at daily doses of 7.7 mg/kg and above were within the projected therapeutic range. GT-02287 was measurable in cerebrospinal fluid (CSF) at levels in line with rodent levels at effective doses, demonstrating CNS exposure. Notably, GCase activity in dried blood spots increased approximately 30% in subjects who received GT-02287 but not in those who received placebo, demonstrating target engagement and modulation of GCase enzyme."

P1 data • CNS Disorders • Parkinson's Disease

Gain Therapeutics To Host Virtual Webinar on Results From Phase 1 Study and Design of Upcoming Phase 1b Trial of GT-02287, a Novel GCase-Targeting Small Molecule Therapy for Parkinson’s Disease (GlobeNewswire) - "Gain Therapeutics, Inc...today announced it is holding a webinar to discuss data from the Phase 1 study of GT-02287, a novel GCase-targeting small molecule therapy for Parkinson’s disease, recently presented in a late-breaker at the International Congress of Parkinson’s Disease and Movement Disorders (MDS). The Company will also discuss the design of a planned Phase 1b trial of GT-02287 in Parkinson’s disease patients."

Clinical protocol • P1 data • CNS Disorders • Parkinson's Disease

The novel glucocerebrosidase chaperone GT-02287 in development for GBA-PD is safe and well tolerated in healthy volunteers at oral doses that produce plasma exposures in the projected therapeutic range (MDS Congress 2024) - "All single and multiple dose levels tested were well tolerated. AEs were mild (90%) or moderate (10%) in intensity and limited in duration. The most common AEs were headache and nausea."

Clinical • Late-breaking abstract • Alzheimer's Disease • CNS Disorders • Dementia • Pain • Parkinson's Disease

Gain Therapeutics to Present Preclinical GT-02287 Data in Parkinson’s Disease Models Including Two Late-Breakers at Neuroscience 2024 (GlobeNewswire) - "Gain Therapeutics, Inc...today announced presentations, including two late-breakers, at Neuroscience 2024 featuring preclinical data supporting development of GT-02287, the Company’s clinical stage lead drug candidate, in Parkinson’s disease with or without a GBA1 mutation. Neuroscience 2024, the Society for Neuroscience’s premier event, will be held at McCormick Place Convention Center in Chicago, IL from October 5-9, 2024."

Late-breaking abstract • Preclinical • CNS Disorders • Parkinson's Disease