rexaceract (GT-02287) / Gain Therap - LARVOL DELTA

Long-Term Open-Label Data on the GCase Modulator GT-02287 in People with Parkinson's Disease (MDS Congress 2026) - No abstract available

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease

Upcoming Anticipated Milestones (The Manila Times) - "Phase 2 clinical trial of rexaceract in people with Parkinson’s disease expected to begin in Q3 2026. Final results from Phase 1b clinical study of rexaceract expected in Q4 2026. GT-04686 initial target indication expected in Q4 2026."

New P2 trial • Pipeline update • Parkinson's Disease

Rexaceract accepted as the International Nonproprietary Name (INN) for GT-02287, establishing the compound's official generic name as it continues to advance through clinical development. (The Manila Times)

Commercial • Parkinson's Disease

Gain Therapeutics Receives FDA Clearance of IND to Advance GT-02287 into Phase 2 Clinical Development for Parkinson’s Disease (GlobeNewswire) - "The FDA’s decision will allow initiation of the Company’s Phase 2 clinical development of GT-02287 in Parkinson’s disease with or without a GBA1 mutation in the U.S....The planned Phase 2a study of oral GT-02287 in treated and untreated participants with early Parkinson’s disease is expected to enroll participants across sites in the United States, Australia, and Europe. The Company expects initiation of the Phase 2a in 3Q26."

IND • New P2a trial • Parkinson's Disease

Gain Therapeutics Presents Positive Interim Data from Phase 1b Clinical Study of GT-02287 at 3rd International GBA1 Meeting 2026 (GlobeNewswire) - "The oral presentation outlined new clinical data from the Phase 1b study of GT-02287 further supporting disease-modifying potential in both idiopathic and GBA1 Parkinson’s disease (PD)....To date, all 16 participants remain on study and have completed five months of dosing (Day 150). As previously reported, in all participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased by an average of 81% after 90 days of treatment with GT-02287. Elevated GluSph, a result of glucocerebrosidase (GCase) dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial and lysosomal function in neurons."

P1 data • Parkinson's Disease

Upcoming Anticipated Milestones (GlobeNewswire) - "FDA clearance of our IND submission, expected in Q2 2026, facilitating Phase 2 clinical development of GT-02287 to include clinical sites in the United States; Phase 2 clinical trial of GT-02287 in people with Parkinson’s disease expected to begin in Q3 2026; Results from Phase 1b clinical study of GT-02287 expected in Q4 2026."

IND • New P2 trial • P1 data • Parkinson's Disease

Gain Therapeutics Announces Oral Presentation at 3rd International GBA1 Meeting 2026 (Gain Therapeutics Press Release) - Presenter: Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain Session Name: In the pipeline: updates from pharma on ongoing preclinical studies and clinical trials Date: May 22, 2026 Time:4:15-4:30pm MST."

Preclinical • Trial status • Parkinson's Disease

Upcoming Anticipated Milestones (GlobeNewswire) - "Clearance of IND submission to FDA, expected in 2Q26, facilitating Phase 2 clinical development of GT-02287 to include clinical sites in the United States; Phase 2 clinical trial of GT-02287 in people with PD expected to begin in 3Q26; Results from Phase 1b clinical study of GT-02287 expected in 4Q26."

IND • New P2 trial • P1 data • Parkinson's Disease

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... (GlobeNewswire) - "As of March 2026, 14 of 16 participants enrolled in Phase 1b nine-month extension had completed 5 months of dosing (Day 150); MDS-UPDRS scores remained stable over 150 days of dosing; In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased by an average of 81% after 90 days of treatment with GT-02287; In participants with elevated baseline levels of GluSph in CSF, levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with GT-02287."

P1 data • Parkinson's Disease

NOVEL ALLOSTERIC GCASE MODULATORS, DIFFERENT FROM THE CLINICAL-STAGE GT-02287, FOR THE TREATMENT OF PARKINSON'S DISEASE. (ADPD 2026) - "We have developed a new chemical series of GCase modulators ready for IND-enabling studies for the treatment of Parkinson's disease, to serve as a back-up to clinical candidate GT-02287. The new series also has the potential to be developed for other indications involving GCase deficit. Gain Therapeutics' Magellan™ platform has demonstrated robust predictive capabilities by identifying multiple, structurally diverse chemical scaffolds that served as high-quality starting points for medicinal chemistry optimization."

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

AN OPEN-LABEL PHASE 1B STUDY OF GT-02287 IN PARKINSON'S DISEASE (ADPD 2026) - "GT-02287 has successfully completed a Phase 1 study in healthy volunteers and a 90-day Phase 1b study in people with PD. A 9-month extension is ongoing, and a randomized, placebo-controlled Phase 2 trial is being planned."

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

Gain Therapeutics Announces Presentations at AD/PDTM 2026… (GlobeNewswire) - "Interim analysis of Phase 1b clinical study of GT-02287, including biomarker and clinical endpoints, will be presented in oral symposium....Dr. Hannestad will present data on GT-02287 demonstrating CNS target engagement, beneficial effects on downstream pathway abnormalities, early signs of clinical improvement in patients, and additional data from the extension phase of the study. Dr. Garcia-Colazzo will present data on a novel glucocerebrosidase (GCase) modulator which is chemically distinct from GT-02287 with promising pre-clinical data in Parkinson’s disease models."

Biomarker • P1 data • Preclinical • Parkinson's Disease

Regulatory Update on GT-02287 (GlobeNewswire) - "The Company submitted an IND to conduct a placebo-controlled, dose-range-finding Phase 2 clinical trial of GT-02287 in the United States. In March 2026, the Company submitted a response to the FDA’s request for additional preclinical data. The Company has been working closely with the FDA and expects a response over the next few weeks. The Company expects to begin its Phase 2 clinical trial in 3Q 2026, as previously reported."

IND • New P2 trial • Parkinson's Disease

Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287 (Gain Therapeutics Press Release) - "In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF) at baseline, GluSph decreased by an average of 81% after 90 days of treatment with GT-02287. 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. 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)."

Biomarker • P1 data • Parkinson's Disease

Gain Therapeutics to Host Virtual KOL Event on GT-02287 for Parkinson’s Disease (Gain Therapeutics Press Release) - "Event to be held January 6th, 2026, at 10 a.m. EST....The event will also review biomarker results from the Phase 1b clinical study of GT-02287, demonstrating disease modifying potential in Parkinson’s disease patients with or without a GBA1 mutation."

Biomarker • P1 data • Parkinson's Disease

Gain Therapeutics Announces Positive Results in Key Exploratory Endpoint from its Phase 1b Clinical Study of GT-02287 in People with Parkinson’s Disease (Gain Therapeutics Press Release) - "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."

P1 data • Parkinson's Disease

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