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.
Select a button below to view the list of FDA events for that drug.
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:
- April 24, 2026
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced an oral presentation at the 3rd International GBA1 Meeting 2026, being held May 22-23, 2026, in Phoenix, AZ.
AI Summary
Gain Therapeutics announced it will deliver an oral presentation at the 3rd International GBA1 Meeting 2026, taking place May 22–23, 2026 in Phoenix, AZ. Company leaders will also attend the 7th World Parkinson’s Congress there May 24–27, 2026, to engage with the research and patient communities.
Gain’s lead candidate, GT-02287, showed favorable safety and tolerability in a Phase 1 study of healthy volunteers, with plasma and central nervous system exposures in the projected therapeutic range and evidence of target engagement via increased GCase activity at clinically relevant doses.
Early Phase 1b results in people with Parkinson’s disease showed central nervous system target engagement, reduction of glucosylsphingosine (GluSph) to baseline, and improvement or stabilization on MDS‑UPDRS scores. The program has received funding from The Michael J. Fox Foundation, The Silverstein Foundation, and co‑funding via Eurostars‑2/Horizon 2020 and Innosuisse. Gain uses its Magellan™ platform to discover allosteric small molecules aimed at disease‑modifying treatments.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- March 18, 2026
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced the presentation of an oral presentation and poster at AD/PD™ 2026 International Conference on Alzheimer's and Parkinson's Disease and Related Neurological Disorders , being held March 17-21, 2026, in Copenhagen, Denmark.
AI Summary
Gain Therapeutics, Inc. announced an oral presentation and a poster at the AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Disease, March 17–21, 2026 in Copenhagen. The company will present new clinical and preclinical data on its GCase allosteric modulators, including GT-02287 and a novel chemical series led by GT-04686.
New biomarker analyses showed higher cerebrospinal fluid (CSF) DOPA decarboxylase (DDC—an emerging Parkinson’s biomarker) at baseline, with DDC levels falling after 90 days of GT-02287 in people who had high baseline CSF glucosylsphingosine (GluSph). Those participants also showed substantial GluSph reductions after 90 days. Additional MDS‑UPDRS data showed durable effects at day 150. Phase 1 and Phase 1b results support safety, CNS exposure, GCase target engagement, reduction of GluSph to baseline, and stabilization or improvement in clinical scores.
Gain says its Magellan™ discovery platform produced the novel series and that lead GT‑04686 is ready for IND‑enabling studies. The company is following patients in a nine‑month Phase 1b extension expected to complete in September 2026 and plans further data presentations.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- March 12, 2026
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced presentations at the AD/PD ™ 2026 International Conference on Alzheimer's and Parkinson's Disease and Related Neurological Disorders, being held March 17-21, 2026 in Copenhagen, Denmark.
AI Summary
Gain Therapeutics announced it will present data at the AD/PD™ 2026 conference in Copenhagen, March 17–21, 2026. An oral presentation titled "An Open-Label Phase 1b Study of GT-02287 in Parkinson's Disease" will be given by Jonas Hannestad, M.D., Ph.D., Chief Medical Officer, on March 18, 2026, 3:15–3:30pm CET in Hall 180-181 during the session "Modulating Neuroinflammation, α-Synuclein, LRRK2, And Dopaminergic Repair: Early Human Data."
An accompanying poster, "Novel Allosteric GCase Modulators, Different From the Clinical Stage GT-02287, for the Treatment of Parkinson's Disease," will be presented by Ana Maria Garcia-Collazo, Ph.D. (Poster SHIFT 02-178). The oral presentation will report an interim analysis from the Phase 1b study, including biomarker and clinical endpoints showing CNS target engagement, reduction of disease-related biomarkers, early signs of clinical improvement, and additional extension-phase data.
Gain says it submitted additional preclinical data requested by the FDA in support of an IND and expects a response soon. The company remains on track to begin a placebo-controlled Phase 2 trial of GT-02287 in 3Q26.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- January 6, 2026
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. today provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287 in people with Parkinson's disease (PD) with or without a GBA1 mutation that further supports the disease-modifying potential of GT-02287.
AI Summary
Gain Therapeutics reported new biomarker and clinical data from a Phase 1b study of GT-02287 in people with Parkinson’s disease (with or without a GBA1 mutation). The company says the data further support the drug’s potential to modify disease biology.
In participants who had high baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid, GluSph fell by an average of 81% after 90 days of treatment. Elevated GluSph is linked to impaired GCase enzyme activity, greater alpha‑synuclein aggregation, and mitochondrial problems. The observed GluSph reduction—reported as a first following a GCase modulator—suggests increased GCase activity in the brain and a possible effect on disease progression.
As of Nov. 30, 2025, 19 patients completed 90 days of dosing. After excluding four patients for assay or assessment issues, 15 patients showed an average 2.20‑point improvement on the combined MDS‑UPDRS Parts II and III (0.6 and 1.6 points, respectively). The trial was not designed to prove efficacy, but investigators noted stabilization and anecdotal gains in balance, gait, and smell.
Gain says these findings support continued clinical development and a longer dosing extension of up to 12 months.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- December 18, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. today provided evidence, for the first time in Parkinson's Disease (PD), of a reduction in glucocerebrosidase (GCase) substrate in cerebrospinal fluid (CSF).
AI Summary
Gain Therapeutics reported the first evidence in Parkinson’s disease of a reduction in the glucocerebrosidase (GCase) substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF) after treatment with GT-02287. Lower CSF GluSph is taken as a sign of increased GCase activity in the brain, which could reduce alpha‑synuclein aggregation and other processes that harm neurons in PD.
In the Phase 1b study, people with elevated CSF GluSph showed large decreases toward levels seen in healthy individuals after 90 days of dosing with GT-02287; this change was a prespecified exploratory endpoint. The trial enrolled 21 participants, 19 completed the 90‑day period, and 15 entered a nine‑month extension. GT‑02287 was generally well tolerated at projected therapeutic exposures, and the data monitoring committee recommended continuing the study unchanged.
Gain plans to present longer follow‑up and functional outcome data at upcoming conferences and will host a January KOL webinar to discuss these biomarker findings and their implications for disease modification in PD.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- December 18, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced that it will host a virtual key opinion leader (KOL) event, "Understanding GCase Substrates in Parkinson's Disease: Perspectives on Biomarkers and Disease Modification, Contextualizing emerging biomarker data from the Phase 1b clinical study of GT-02287".
AI Summary
Gain Therapeutics announced a virtual key opinion leader (KOL) event titled "Understanding GCase Substrates in Parkinson's Disease: Perspectives on Biomarkers and Disease Modification, Contextualizing emerging biomarker data from the Phase 1b clinical study of GT-02287." The session will be held Tuesday, January 6, 2026 at 10:00 a.m. EST and features Roy Alcalay, M.D., M.Sc., and Peter Lansbury, Ph.D. Registration is available through the company.
The event will review emerging biomarker results from the Phase 1b study of GT-02287 and discuss their implications for disease modification in Parkinson's disease patients with or without a GBA1 mutation. Presentations will focus on GCase substrates, biomarker interpretation, and how those data may reflect target engagement and potential clinical benefit. A live question-and-answer session will follow the formal talks, giving clinicians and researchers a chance to probe the findings and implications for future research and therapy development.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- November 20, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced a poster presentation was made at the Society for Neuroscience annual meeting, Neuroscience 2025, held November 15th-19th in San Diego, CA.
AI Summary
Gain Therapeutics announced a poster presentation at the Society for Neuroscience annual meeting, Neuroscience 2025, held November 15–19 in San Diego. The poster presented new preclinical data supporting the company’s lead candidate, GT-02287, and its proposed mechanism of helping the enzyme glucocerebrosidase (GCase) reach mitochondria and lysosomes.
In three models, GT-02287 reduced mitochondrial stress and boosted neuron survival in rat dopaminergic neurons treated with the toxin MPP+ by lowering reactive oxygen species and preventing cytochrome C release. In a mouse Parkinson’s model with injected α‑synuclein fibrils, the drug decreased MIRO1 staining, a marker of damaged mitochondria. In patient-derived fibroblasts with the L444P GBA1 mutation, GT-02287 raised mitochondrial GCase levels, improved complex I activity, and restored membrane potential.
The poster, presented on-site by Joanne Taylor, Ph.D., is available as a PDF on Gain’s website and supports GT-02287’s potential as a disease‑modifying therapy for Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- October 30, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc announced a poster presentation at the Society for Neuroscience annual meeting, Neuroscience 2025, being held November 15th–19th in San Diego, CA.
AI Summary
Gain Therapeutics, Inc., a clinical-stage biotech company, announced a poster presentation at Neuroscience 2025 in San Diego, CA, on November 19, 2025. The poster, PSTR438, titled “GT-02287, a small molecule allosteric modulator of lysosomal enzyme glucocerebrosidase (GCase), acts at both lysosomes and mitochondria,” will be shown from 1:00 PM to 5:00 PM in SDCC Halls B-H.
GT-02287 is Gain’s lead candidate for Parkinson’s disease, with or without a GBA1 mutation. This oral, brain-penetrant small molecule restores misfolded GCase enzyme function, reduces cellular stress, and boosts mitochondrial health. In preclinical models, it lowered α-synuclein, reduced inflammation, and improved motor function in rodents.
After positive Phase 1 results showing safety and target engagement, GT-02287 is now in a Phase 1b trial in Parkinson’s patients. Early findings suggest disease-modifying effects. An extension allows participants to continue treatment for up to 12 months.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- October 9, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced that it will host a virtual key opinion leader (KOL) event, "Biomarkers, Clinical Endpoints, and the Path to Disease Modification: Contextualizing the emerging data from GT-02287" featuring Key Opinion Leaders (KOLs), Karl Kieburtz, M.D., M.P.H., Professor of Neurology, University of Rochester and Kenneth Marek, M.D., President and Senior Scientist, Institute for Neurodegenerative Disorders.
AI Summary
Gain Therapeutics, Inc. will host a virtual key opinion leader (KOL) event titled “Biomarkers, Clinical Endpoints, and the Path to Disease Modification: Contextualizing the emerging data from GT-02287” on Tuesday, October 14, 2025, at 4:00 p.m. EST. The live online meeting will explore how biomarkers and clinical outcome scales can guide the development of disease-modifying therapies.
The event will highlight data from Gain’s Phase 1b study of GT-02287 in people with Parkinson’s disease, with or without a GBA1 mutation. Early results show the drug was generally well tolerated with no serious adverse events and trends toward improved scores on the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS).
Key Opinion Leaders Karl Kieburtz, M.D., M.P.H., Professor of Neurology at the University of Rochester, and Kenneth Marek, M.D., President and Senior Scientist at the Institute for Neurodegenerative Disorders, will discuss these findings and their implications.
A live question-and-answer session will follow the presentations. Registration is available online.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- October 6, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. today presented a poster at the International Congress of Parkinson's Disease and Movement Disorders® summarizing early safety and tolerability findings, the primary endpoint, and observations from key exploratory endpoints from the Phase 1b clinical study of GT-02287 in people with Parkinson's disease (PD) with or without a GBA1 mutation.
AI Summary
Gain Therapeutics, Inc. presented a poster at the International Congress of Parkinson’s Disease and Movement Disorders summarizing early safety and tolerability findings, the primary endpoint, and observations from key exploratory endpoints in its Phase 1b study of GT-02287 in people with Parkinson’s disease, with or without a GBA1 mutation. Over 90 days of dosing, no treatment-emergent serious adverse events were observed, and two independent data monitoring committees recommended that the study continue without changes.
In the first nine participants assessed at Day 90, mean Movement Disorder Society–Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III scores improved compared to baseline, while Part I remained stable. These improvements, not seen at Day 30, suggest a potential disease-slowing effect of GT-02287. The remaining participants are now entering their 90-day follow-up to confirm these trends.
Plasma pharmacokinetic profiles were consistent across patients, stayed within the projected therapeutic range, and matched exposures seen in a prior Phase 1 healthy volunteer study. Overall, GT-02287 was generally well tolerated, supporting its continued development as a potential Parkinson’s therapy.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- September 18, 2025
- Indication:
- For Parkinson Disease
Announcement
Gain Therapeutics, Inc. announced the start of the Phase 1b extension study allowing participants to continue treatment for an additional nine months. More than half of the participants have agreed to continue treatment.
AI Summary
Gain Therapeutics has launched a Phase 1b extension study that lets participants continue taking GT-02287 for nine more months after the initial 90-day dosing period.
The Phase 1b study involves 21 subjects at seven sites in Australia, and all will complete dosing by December 2025. Early data on safety, tolerability, and brain exposure will be shared at the International Congress of Parkinson’s Disease and Movement Disorders in October 2025.
More than half of the study participants have agreed to continue treatment with GT-02287 in the extension phase. This strong support lets researchers gather extra data on the drug’s long-term safety and impact on Parkinson’s symptoms.
Gain plans to report additional results, including movement scores and biomarker findings, in the fourth quarter of 2025. These insights will guide the next steps in developing GT-02287 for Parkinson’s disease.
Read Announcement- Drug:
- GT-02287
- Announced Date:
- September 4, 2025
- Indication:
- GBA1 Parkinson's disease
Announcement
Gain Therapeutics, Inc. announced the Company has received approval from the Bellberry Human Research Ethics Committee and Alfred Hospital Ethics Committee to extend dosing by an additional nine months in its ongoing Phase 1b clinical study of GT-022887, beyond the 90-day period currently allowed in protocol.
Read Announcement- 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
- Target Action Date:
- Q4 2024
- Estimated Target Date Range:
- October 1, 2024 - December 31, 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.
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GT-02287 + GT-02329 FDA Regulatory 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.
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