This section highlights FDA-related milestones and regulatory updates for drugs developed by Gain Therapeutics (GANX).
Over the past two years, Gain Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
GT-02287 and GT-02287. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
GT-02287 - FDA Regulatory Timeline and Events
GT-02287 is a drug developed by Gain Therapeutics for the following indication: For Parkinson Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- GT-02287
- Announced Date:
- May 29, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced an oral presentation at the GBA1 Meeting 2025, being held June 5th-7th in Montreal, Canada.
AI Summary
Gain Therapeutics, Inc. announced an exciting update regarding its involvement in the upcoming GBA1 Meeting 2025, which will take place in Montreal, Canada from June 5th to 7th, 2025. The company’s Chief Medical Officer, Jonas Hannestad, M.D., Ph.D., is set to deliver an oral presentation during the event. The talk is scheduled for Thursday, June 5, 2025, at 11:20 p.m. EST, providing an opportunity for Gain Therapeutics to share insights about its latest developments and innovative approach to drug discovery.
As a clinical-stage biotechnology company, Gain Therapeutics is focused on developing next-generation allosteric therapies aimed at treating conditions such as Parkinson’s disease, among other neurological and genetic disorders. The presentation at GBA1 Meeting 2025 will highlight the potential and progress of these novel treatments, emphasizing the company’s commitment to unlocking advancements in disease-modifying therapies.
Read Announcement - Drug:
- GT-02287
- Announced Date:
- May 12, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc announced that a guided poster presentation was made at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, held May 7th-10th in New York City, NY.
AI Summary
Gain Therapeutics, Inc. presented a guided poster at the IAPRD 30th World Congress on Parkinson’s Disease and Related Disorders, which took place from May 7–10, 2025, in New York City, NY. The poster highlighted new preclinical evidence on GT-02287, the company’s lead drug candidate. Research showed that GT-02287 may provide a broad neuroprotective effect by improving both lysosomal and mitochondrial function while reducing key issues like α-synuclein aggregation and mitochondrial stress in dopaminergic neurons. These findings suggest that the drug candidate could serve as a disease-modifying therapy for Parkinson’s disease. The study underscored how GT-02287 stabilizes mitochondria through its allosteric modulation of glucocerebrosidase (GCase), an important enzyme involved in the disease pathology. This work adds to the growing scientific support for GT-02287’s potential role in slowing the progression of Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- April 30, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc 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.
AI Summary
Gain Therapeutics, Inc. announced it will deliver an oral poster presentation at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders. The event is scheduled for May 7th-10th in New York City. The presentation, titled “GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson's Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+,” will be led by Dr. Joanne Taylor, Senior Vice President of Research, on May 10, 2025, from 8:00-9:00 a.m. EST.
This session will focus on GT-02287, Gain Therapeutics’ promising lead drug candidate for Parkinson’s disease. The candidate, an orally administered small molecule, aims to restore the function of the glucocerebrosidase enzyme, potentially slowing or halting the progression of Parkinson’s disease, regardless of the presence of the GBA1 mutation. The company’s research continues to contribute valuable insights into innovative treatments for neurodegenerative disorders.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- April 10, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced that an oral presentation as well as a poster were presented at the AD/PD™ 2025 International Conference on Alzheimer's and Parkinson's Diseases and related neurological disorders held April 1-5, 2025, in Vienna, Austria.
AI Summary
Gain Therapeutics recently announced that an oral presentation and a poster were showcased at the AD/PD™ 2025 International Conference, held from April 1–5, 2025, in Vienna, Austria. During the conference, researchers presented new preclinical evidence highlighting the potential of their lead drug candidate, GT‑02287, to modify disease progression in both GBA1 and idiopathic Parkinson’s disease models. The oral presentation detailed how GT‑02287 helped improve motor functions and complex behaviors in animal studies, with lasting effects even after the treatment was stopped.
The poster presentation focused on the design of the ongoing Phase 1b clinical study in Parkinson’s patients. This study aims to assess the safety, tolerability, and pharmacokinetic profile of GT‑02287 while performing biomarker analyses to inform future Phase 2 trials planned for later in 2025. Overall, these presentations underscore the company’s commitment to advancing potential disease-modifying therapies for Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- March 27, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. provided a corporate update.
AI Summary
Gain Therapeutics, Inc. provided an update on its progress in developing GT-02287, a new treatment for Parkinson’s disease. The company recently began dosing patients in its Phase 1b trial, with the first analysis expected by the end of the second quarter of 2025. Gene Mack, the new President and CEO as of January 6, 2025, emphasized that results from this trial could mark a significant value inflection point for the program and will guide planning for a Phase 2 study later this year.
In addition, Gain Therapeutics is preparing to expand the clinical development of GT-02287 into the United States. The company is engaging in pre-Investigational New Drug discussions with the FDA to support this expansion, underlining its commitment to advancing its innovative therapy for the treatment of Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- March 14, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc 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.
AI Summary
Gain Therapeutics, Inc. announced that it has begun dosing the first participant with Parkinson’s disease in its Phase 1b clinical trial of GT-02287, the company’s lead allosteric small molecule therapy for Parkinson’s disease, with or without a GBA1 mutation. This open-label, multi-center study will enroll up to 20 participants who will receive daily doses over a three‐month period to evaluate the drug’s safety, tolerability, and other key markers such as pharmacokinetics and relevant biomarkers in plasma and cerebrospinal fluid. Gene Mack, President and CEO, said this milestone is a significant step toward developing a potential disease-modifying therapy for Parkinson’s. The company anticipates an interim analysis toward the end of the second quarter of 2025 to further assess the therapy’s impact.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- December 23, 2024
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced it has received approval in Australia to initiate a Phase 1b trial.
AI Summary
Gain Therapeutics, Inc. has received approval in Australia to start a Phase 1b clinical trial for its lead candidate, GT‐02287, aimed at treating Parkinson’s disease. The trial will enroll patients with either idiopathic Parkinson’s or Parkinson’s associated with GBA1 mutations at seven sites across major Australian cities. Over three months, the study will focus on evaluating the safety, tolerability, and impact on key biomarkers, including glucocerebrosidase (GCase) activity levels. The trial builds on earlier positive results from a Phase 1 study in healthy volunteers, which showed that GT‐02287 was well tolerated and demonstrated significant target engagement. Enrollment is expected to be completed by spring 2025, with trial data anticipated to be available by mid-2025. This step marks a critical development for Gain Therapeutics as they move forward with clinical investigation into disease-modification options for Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- October 7, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced the presentation of new evidence supporting the disease-modifying activity of GT-02287 in preclinical animal models of both GBA-1 and idiopathic Parkinson's disease during a late-breaker poster session yesterday at the Society for Neuroscience (SfN) 2024 conference that is being held October 5-9 in Chicago, IL.
AI Summary
Gain Therapeutics, Inc. presented new preclinical evidence at the Society for Neuroscience (SfN) 2024 conference in Chicago, IL, showing that its drug candidate, GT‑02287, has potential to change the course of Parkinson’s disease. The company’s late-breaking poster highlighted that GT‑02287 improved motor and cognitive functions in animal models of both GBA1-associated and idiopathic Parkinson’s disease. Notably, even after the treatment was discontinued for more than a week, the improvements remained, suggesting a lasting disease-modifying effect.
Additional results revealed that GT‑02287 enhances mitochondrial function and offers neuroprotective benefits. These findings support the possibility that GT‑02287 could slow or halt disease progression by targeting underlying mechanisms of neurodegeneration, making it a promising candidate for further development in the treatment of Parkinson’s disease and potentially other neurodegenerative conditions.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- September 30, 2024
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced the presentation of a late-breaking abstract at the International Congress of Parkinson's Disease and Movement Disorders.
AI Summary
Gain Therapeutics recently presented a late-breaking abstract at the International Congress of Parkinson’s Disease and Movement Disorders. The presentation focused on data from a Phase 1 first‐in‐human study of their lead drug candidate, GT‑02287. Results from the trial in healthy volunteers showed that the drug was safe, well tolerated, and led to a roughly 30% increase in GCase enzyme activity compared to placebo. GT‑02287 also demonstrated exposure in the central nervous system, suggesting it can reach the brain effectively. The study reported a linear pharmacokinetic profile across tested dose levels, supporting the projected therapeutic range. Gain Therapeutics is encouraged by these findings and plans to initiate a trial in Parkinson’s patients later in 2024 to further assess safety, tolerability, and target engagement, with the goal of developing a treatment that could slow or halt the progression of Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- September 26, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. 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.
AI Summary
Gain Therapeutics, Inc. announced an upcoming webinar on September 30, 2024, at 8:30 AM ET to discuss key data from its Phase 1 study of GT-02287, a new therapy for Parkinson’s disease. GT-02287 targets the enzyme glucocerebrosidase (GCase), which can malfunction in Parkinson’s patients. The data, recently showcased as a late-breaking presentation at the International Congress of Parkinson’s Disease and Movement Disorders® (MDS), suggests the therapy may improve enzyme function and potentially slow disease progression. During the webinar, the company will also outline plans for a Phase 1b trial designed to further evaluate GT-02287’s safety and effectiveness in Parkinson’s disease patients. This discussion is expected to provide valuable insights into the new approach for treating Parkinson’s disease and set the stage for future research in this area.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- September 19, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. 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.
AI Summary
Gain Therapeutics, Inc. announced that it will present new research data at Neuroscience 2024, the premier event hosted by the Society for Neuroscience, held at McCormick Place Convention Center in Chicago from October 5-9, 2024. The company will showcase preclinical findings supporting the development of its clinical-stage lead drug candidate, GT-02287, for treating Parkinson’s disease, with or without a GBA1 mutation. Two late-breaking presentations will highlight the potential of GT-02287 as a GCase modulator. Early data indicate that the drug may help restore key enzymatic functions, prevent harmful protein accumulation, and reduce neurodegeneration, possibly slowing disease progression. This research involves collaborations with scientists from leading institutions, further supporting GT-02287’s promise as a novel treatment that could improve motor and cognitive functions in Parkinson’s disease patients.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- August 29, 2024
- Estimated Event Date Range:
- October 1, 2024 - December 31, 2024
- Target Action Date:
- Q4 - 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced that GT-02287, in Development for GBA1 Parkinson's Disease, on Track to Initiate Phase 1b Trial in Patients in Q4 2024
AI Summary
Gain Therapeutics, Inc. announced that its lead candidate GT-02287, developed for Parkinson’s disease associated with or without a GBA1 mutation, is progressing well. The company revealed positive Phase 1 results showing that the drug was safe and well tolerated in healthy volunteers. GT-02287 reached therapeutic plasma levels and effectively engaged its target in both the brain and peripheral tissues.
Based on these encouraging findings, Gain Therapeutics is on track to start a Phase 1b trial in patients with Parkinson’s disease in the fourth quarter of 2024. This upcoming study aims to evaluate the safety, tolerability, and key mechanisms of GT-02287 in a patient population, with initial results expected by mid-2025. The positive early data and advancing clinical milestones underscore the potential of GT-02287 as a promising treatment for Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- August 29, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announces positive results from the Phase 1 study conducted to evaluate the safety, tolerability and pharmacokinetics of GT-02287.
AI Summary
Gain Therapeutics, Inc. announced positive results from a Phase 1 study of GT-02287, their lead drug candidate for treating Parkinson’s disease. The study, which involved 72 healthy volunteers aged up to 64 years, showed that both single and multiple doses of GT-02287 were safe and generally well tolerated. No serious adverse events or discontinuations were reported. The study also revealed that GT-02287 successfully reached the cerebrospinal fluid, demonstrating its ability to engage the target in the body. These findings support that the oral doses provided therapeutic plasma levels, central nervous system exposure, and effective target engagement, which are key for further development. Based on these encouraging results, Gain Therapeutics plans to start a Phase 1b trial in Parkinson’s patients by the fourth quarter of 2024, aiming to confirm the drug’s safety, tolerability, and proof-of-mechanism through relevant biomarkers.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- June 27, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announces the presentation of a poster at the Federation of European Neuroscience Societies (FENS) Forum 2024.
AI Summary
Gain Therapeutics, Inc. recently presented an important poster at the FENS Forum 2024 in Vienna, Austria. The poster highlighted promising preclinical results using their lead drug candidate, GT-02287, in a mouse model of GBA1 Parkinson’s disease. Researchers showed that daily oral administration of GT-02287 improved rodent nest-building behavior and motor performance, which are seen as signals for enhanced cognitive abilities and better daily functioning.
Dr. Joanne Taylor, Senior Vice President of Research at Gain Therapeutics, emphasized that these findings support the company’s belief in GT-02287’s potential to slow or stop Parkinson’s disease progression. The presentation at FENS Forum 2024 provided a significant platform for sharing these insights with the neuroscience community, fostering further discussions and collaborations in the field.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- May 30, 2024
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc announced that a late-breaking abstract with new data on GT-02287 has been accepted for presentation at FENS Forum 2024 being held June 25-29, 2024 in Vienna, Austria.
AI Summary
Gain Therapeutics, Inc. announced that a late-breaking abstract featuring new data on its drug candidate, GT-02287, has been accepted for presentation at the FENS Forum 2024 in Vienna, Austria. Scheduled to take place from June 25-29, 2024, this prominent neuroscience congress will host the presentation on June 27, 2024, at 11:30 am. The abstract, titled “GT-02287, a clinical-stage GCase Enhancer, improves activities of daily living and cognitive performance in a preclinical model of GBA1 Parkinson’s disease,” details how GT-02287 helped improve cognitive function and everyday activities in a preclinical model of GBA1 Parkinson’s disease. The data highlights promise in addressing cognitive aspects of the disease by restoring lysosomal enzyme activity impaired due to a GBA1 mutation, underscoring the potential impact of GT-02287 on managing symptoms in Parkinson’s disease patients.
Read Announcement
GT-02287 + GT-02329 - FDA Regulatory Timeline and Events
GT-02287 + GT-02329 is a drug developed by Gain Therapeutics for the following indication: Gaucher and GBA1 Parkinson's Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- GT-02287 + GT-02329
- Announced Date:
- March 6, 2025
- Indication:
- Gaucher and GBA1 Parkinson's Disease
Announcement
Gain Therapeutics, Inc 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.
AI Summary
Gain Therapeutics, Inc. announced that Joanne Taylor, Ph.D., Senior Vice President of Research, will deliver an oral presentation at the AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases. The event will take place from April 1-5, 2025, in Vienna, Austria. Dr. Taylor’s presentation, scheduled for April 5 at 6:10 pm CET in Hall B, will focus on the company’s clinical stage lead drug candidate, GT-02287. This allosteric small molecule therapy is designed to restore the function of glucocerebrosidase, a key enzyme impaired by mutations associated with Parkinson’s disease. The presentation will highlight preclinical evidence that GT-02287 has the potential to modify disease progression by improving enzyme activity, reducing neuroinflammation, lowering aggregated proteins, and enhancing motor and cognitive performance. This work underscores the promise of GT-02287 in addressing both GBA1-related and idiopathic Parkinson’s disease.
Read Announcement
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