Gain Therapeutics Announces Oral Presentation at AD/PD 2025

Rhea-AI Summary

Gain Therapeutics (GANX) announced that Dr. Joanne Taylor, Senior Vice President of Research, will present their clinical stage lead drug candidate GT-02287 at the AD/PD™ 2025 International Conference on Alzheimer's and Parkinson's Diseases in Vienna, Austria.

The presentation, titled 'Clinical Stage Glucocerebrosidase Modulator, GT-02287, Shows Disease Modifying Potential In Preclinical Models Of Both GBA1 And Idiopathic Parkinson's Disease', will take place on April 5, 2025, at 6:10 PM CET in Hall B during the session on Advances in PD, LBD and MSA Drug Development.

Positive

• Clinical-stage drug candidate GT-02287 shows disease-modifying potential in preclinical models

Negative

• None.

BETHESDA, Md., March 06, 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 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.

Details of the oral presentation are as follows:

Presentation Title: Clinical Stage Glucocerebrosidase Modulator, GT-02287, Shows Disease Modifying Potential In Preclinical Models Of Both GBA1 And Idiopathic Parkinson’s Disease
Presenter: Joanne Taylor
Authors: Joanne Taylor, Beatriz Guzman, Natalia Pérez-Carmona, Ana Maria García-Collazo
Session Name: 7550 – Advances in PD, LBD and MSA Drug Development
Session Type: Symposium
Date: April 5, 2025
Lecture Time: 6:10 – 6:25pm CET
Room: Hall B

The full abstract can be accessed at the AD/PD online interactive program.

The AD/PD™ Conference is an annual event attracting leading medical and scientific professionals from around the world, focusing on the advances of science and therapy of Alzheimer’s and Parkinson’s Diseases and related neurological disorders.

About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) 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 models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop 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 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement and modulation of GCase enzyme.

Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ 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, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates. 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 Form 10-K for the year ended December 31, 2023 and Form 10-Q for the quarter ended September 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
ajammu@gaintherapeutics.com
chuck@lifesciadvisors.com

Media Contacts:
Russo Partners
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(760) 846-9256

FAQ

What will be presented about GANX's GT-02287 drug candidate at AD/PD 2025?

Disease modifying potential in preclinical models of both GBA1 and idiopathic Parkinson's Disease will be presented by Dr. Joanne Taylor on April 5, 2025.

When and where is GANX presenting at the AD/PD 2025 conference?

GANX will present on April 5, 2025, at 6:10 PM CET in Hall B in Vienna, Austria.

What is the significance of GT-02287 in GANX's drug pipeline?

GT-02287 is GANX's clinical stage lead drug candidate, functioning as a Glucocerebrosidase Modulator for potential treatment of Parkinson's Disease.

Which GANX executive will present at AD/PD 2025?

Dr. Joanne Taylor, Senior Vice President of Research at Gain Therapeutics, will give the oral presentation.