Gain Therapeutics

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

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

Company To Discuss Positive Results Presented at the International MDS Congress and the Design of Phase 1b Trial in Parkinson’s Disease Patients

Event Will Be Held on Monday, September 30, 2024, at 8:30 am ET

BETHESDA, Md., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, 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.

Webinar Details

Date: Monday, September 30, 2024

Time: 8:30 am ET

Register for the event or join the conference call through the News and Events section of the Company website at .

A live question and answer session will follow the formal presentations. A replay of the call will be available in the News and Events section of the Company website after the event.

About GT-02287

Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.

Compelling preclinical data in mouse models of GBA1-PD, including that presented at FENS Forum 2024 in June describing improvement in cognitive performance in addition to motor performance after administration of GT-02287, suggests that GT-02287 may have the potential to slow the progression of Parkinson’s disease.

Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 for the treatment of Parkinson’s disease with or without a GBA1 mutation, is currently being evaluated in a Phase 1 clinical trial.

Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Its AI and machine-learning tools and virtual screening capabilities leverage the emerging on-demand compound libraries covering vast chemical spaces of over five trillion compounds to identify and select suitable small molecule hits for experimental validation.

Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology.

Forward-Looking Statements

This release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, risks associated with market conditions and the satisfaction of customary closing conditions related to the offering and uncertainties related to the offerings and the use of proceeds from the offerings. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s prospectus supplement to be filed with the SEC, and the documents incorporated by reference therein, including the Company’s Form 10-K for the year ended December 31, 2023 and Form 10-Q for the quarter ended June 30, 2024.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contacts:

Apaar Jammu and Chuck Padala



Media Contacts:

Russo Partners

Nic Johnson and Elio Ambrosio





(760) 846-9256



EN
26/09/2024

Reports on Gain Therapeutics

 PRESS RELEASE

Gain Therapeutics Announces Announces Conference Participation During ...

Gain Therapeutics Announces Announces Conference Participation During J.P. Morgan Healthcare Conference Week BETHESDA, Md., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced its attendance at conferences during the week of the 44th Annual J.P. Morgan Healthcare Conference (“JPM week”) including at the Sachs Neuroscience Innovation Forum, the Demy-Colton/Informa Biotech Showcase™, and t...

 PRESS RELEASE

Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Mod...

Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287 Results to be discussed in KOL event today at 10 a.m. ET BETHESDA, Md., Jan. 06, 2026 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, 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 furth...

 PRESS RELEASE

Gain Therapeutics to Host Virtual KOL Event on GT-02287 for Parkinson’...

Gain Therapeutics to Host Virtual KOL Event on GT-02287 for Parkinson’s Disease Event to be held January 6th, 2026, at 10 a.m. EST BETHESDA, Md., Dec. 18, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today 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 emerg...

 PRESS RELEASE

Gain Therapeutics Announces Positive Results in Key Exploratory Endpoi...

Gain Therapeutics Announces Positive Results in Key Exploratory Endpoint from its Phase 1b Clinical Study of GT-02287 in People with Parkinson’s Disease Results showed CNS target engagement with reduction in GCase substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), a prespecified endpoint 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) KOL event planned for early January to discus...

 PRESS RELEASE

Gain Therapeutics Presents Preclinical GT-02287 Data at Neuroscience 2...

Gain Therapeutics Presents Preclinical GT-02287 Data at Neuroscience 2025 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 GT-02287 improves mitochondrial function by facilitating GCase trafficking to the mitochondria in patient-derived fibroblasts harboring GBA1 mutation BETHESDA, Md., Nov. 20, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotec...

ResearchPool Subscriptions

Get the most out of your insights

Get in touch