Gain Therapeutics, Inc.

GT-03842, Identified by the Company’s Magellan Drug Discovery Platform, Effectively Hinders Phosphorylation and Activation of DDR2 GT-03842 May Offer Potential Favourable Therapeutic Attributes for Oncology Compared to Traditional Kinase Inhibitors BETHESDA, Md., Oct. 23, 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 the presentation of a poster at the 36th EORTC-NCI-AACR (ENA) Symposium on molecular targets and cancer therapeutics that details the use of the Company’s Magellan drug discovery platform to identify allosteric inhibitors targeting discoidin domain receptor 2 (DDR2). ENA 2024 is being held October 23-25 in Barcelona, Spain. “We believe the identification of allosteric small molecule inhibitors targeting DDR2 validates the Company’s Magellan platform. Our belief is strengthened that this innovative approach can one day result in much-needed novel therapeutics for people with DDR2-driven malignancies,” commented Joanne Taylor, Ph.D., Senior Vice President of Research at Gain. The poster, “Identification of allosteric inhibitors targeting Discoidin Domain Receptor 2 (DDR2),” which was presented on-site on October 23 by Sara Cano-Crespo, Ph.D., Senior Scientist of Biology at Gain, detailed how the 3D structure of DDR2 guided a high-throughput virtual screening and subsequent experimental validation that resulted in GT-03842 and additional analogue compounds. Upon binding to collagen, DDR2 undergoes autophosphorylation, triggering its activation and initiating downstream signaling cascades. After conducting a phosphorylation assay based on AlphaLISA with HEK293 cells overexpressing DDR2, GT-03842 and its analogues were found to inhibit DDR2 phosphorylation in a dose-dependent manner. Aberrant DDR2 phosphorylation is associated with various cancer types and is involved in critical processes in tumor progression, including proliferation, migration, invasion, metastasis, epithelial-mesenchymal transition, and immunotherapy resistance. In a metastatic breast cancer model, GT-03842 also reduced DDR2 phosphorylation. Additionally, GT-03842 inhibits DDR2 activity without targeting the kinase domain, showing the potential to overcome resistance and offer enhanced selectivity and safety compared to traditional kinase inhibitors. A PDF of the poster presented at the 36th ENA Symposium on molecular targets and cancer therapeutics is available on the Science and Technology section of the Company’s website at https://gaintherapeutics.com/science-and-technology/posters. 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 StatementsThis 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 Padalaajammu@gaintherapeutics.comchuck@lifesciadvisors.com Media Contacts:Russo PartnersNic Johnson and Elio Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(760) 846-9256

BETHESDA, Md., Oct. 15, 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 that a poster detailing the use of the Company’s Magellan platform to discover novel allosteric discoidin domain receptor 2 (DDR2) inhibitors will be presented at the 36th EORTC-NCI-AACR (ENA) Symposium on molecular targets and cancer therapeutics being held October 23-25 in Barcelona, Spain. DDR2 displays aberrant expression and mutations across various cancer types and is involved in critical processes in tumor progression, including proliferation, migration, invasion, metastasis, epithelial-mesenchymal transition, and chemotherapy resistance. Allosteric small molecule inhibitors have the potential to overcome resistance and offer enhanced selectivity and safety compared to traditional kinase inhibitors. ENA 2024 stands as the drug development and translational research meeting, giving the spotlight to preclinical and phase I studies, ensuring in-depth scientific discussions on the latest developments in targets and drugs. Title: Identification of allosteric inhibitors targeting Discoidin Domain Receptor 2 (DDR2) Presenting Author: Sara Cano-Crespo, Ph.D., Gain TherapeuticsPoster Session: Molecular Targeted AgentsDate and Time: Wednesday, October 23, 2024, 11am CEST 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 StatementsThis 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 Padalaajammu@gaintherapeutics.comchuck@lifesciadvisors.com Media Contacts:Russo PartnersNic Johnson and Elio Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(760) 846-9256

BETHESDA, Md., Oct. 09, 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 that Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain, will give an oral presentation on the Company’s clinical stage lead drug candidate GT-02287 at the Michael J. Fox Foundation’s (MJFF) 16th Annual Parkinson’s Disease Therapeutics Conference on October 17, 2024, in New York City. The oral presentation titled, “Brain-penetrant small molecule GT-02287 is well-tolerated in healthy volunteers, shows GCase target engagement, and achieves therapeutic exposures shown to modulate PD pathobiology in preclinical models,” will be presented as part of the conference’s first session, “Progress in the Therapeutics Pipeline”. “We look forward to presenting Phase 1 data for GT-02287, in development for the treatment of Parkinson’s disease, at the upcoming MJFF conference and appreciate the opportunity to contextualize these data with exciting preclinical data that have been generated recently for GT-02287. The Michael J. Fox Foundation shares our mission of bringing potentially life-changing treatments to people living with Parkinson’s disease,” commented Jonas Hannestad. The Parkinson's Disease Therapeutics Conference (PDTC) is The Michael J. Fox Foundation's annual scientific conference and the only one in the world focused exclusively on Parkinson's disease drug development. Each year, the PD Therapeutics Conference brings together 300 research and clinical development professionals from both academia and industry to showcase the most exciting and innovative research from MJFF's research portfolio. Novel advances in basic, translational, and clinical research from both academic and industry labs are highlighted through speaker presentations and a poster session. The event is a platform for field leaders to share new and unpublished results and for fostering new relationships and collaborations. About GT-02287Gain 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 StatementsThis 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 Padalaajammu@gaintherapeutics.comchuck@lifesciadvisors.com Media Contacts:Russo PartnersNic Johnson and Elio Ambrosionic.johnson@russopartnersllc.com elio.ambrosio@russopartnersllc.com (760) 846-9256