GBA1

Data highlight potential of FLT201 to set new standard of care for Gaucher disease Dramatic and durable reductions observed in lyso-Gb1, an established biomarker of clinical response, in patients with persistently high levels despite years of prior treatment on currently approved therapies Substantial improvements in bone marrow burden demonstrate FLT201 penetrating deeper tissues that currently approved therapies poorly address Favorable safety and tolerability continue to be seen in all patients dosed On track to initiate Phase 3 trial of FLT201 in 2025 Oct. 23, 2024 -- Spur Therapeutics today announced positive new data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1, showing durable reductions in glucosylsphingosine (lyso-Gb1), one of the best predictors of clinical response in Gaucher disease, and bone marrow burden, as well as improvements or maintenance of blood counts and organ volume in patients treated with a single infusion of FLT201. FLT201 continues to demonstrate a favorable safety and tolerability profile in all patients treated in the study. These data are being showcased in an oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress taking place in Rome, Italy. “Gaucher disease is a debilitating chronic disorder, and despite treatment with currently approved therapies, many patients continue to have serious symptoms,” said Pamela Foulds, M.D., Spur’s Chief Medical Officer. “Consistent with what is seen in real-world practice, patients entered our trial with different unmet needs. All of them had bone disease, which current therapies poorly address. Most had high levels of harmful substrate accumulation. Some also had an enlarged spleen, low platelets, pain or fatigue. What is exciting is that after a single infusion of FLT201, patients improved in areas their disease was poorly controlled and remained well-controlled in areas their disease was controlled. These data underscore FLT201’s potential to set a new standard of care for Gaucher disease type 1.” Today’s oral presentation at ESGCT during the session titled “AAV Vectors as Tools in Gene Therapy of Rare Diseases - Recent Development to Improve Efficacy and Safety” will include updated data on safety and tolerability, as well as biomarker and efficacy endpoints from the ongoing Phase 1/2 GALILEO-1 study, a first-in-human, international, multicenter dose-finding study in adults with Gaucher disease Type 1. Dosing has been completed in the trial. A total of six patients were treated with a single infusion of FLT201 at a dose of 4.5e11 vg/kg and have been followed for between seven and 15 months after dosing. All six patients are included in the safety analysis; one patient with detectable pre-existing neutralizing antibodies (NAbs) to the AAVS3 capsid below the protocol cut-off has been excluded from the efficacy analysis. The data as of September 27, 2024 demonstrated: Favorable safety and tolerability, with no infusion reactions or dose limiting toxicities. All treatment-related adverse events were mild to moderate in nature Durable reductions, ranging from 42% to 96%, in lyso-Gb1 levels in patients who entered the trial with persistently high levels despite years of treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). Low lyso-Gb1 levels maintained for more than a year as of the cut-off date after the withdrawal of prior therapy in the one patient who entered the trial with well-controlled levels. Lyso-Gb1 levels in the blood are highly correlated with substrate levels in disease-affected tissues and one of the best predictors of clinical response in Gaucher disease. Substantial improvements in bone marrow burden (BMB) seen in all five patients, including those who entered the trial with severe bone involvement. Notably, BMB is correlated with increased fractures, necrosis, bone pain and joint replacements. Improvement or maintenance of spleen and liver volume. Maintenance of hemoglobin levels, an established endpoint for Gaucher disease clinical trials, was observed beyond a year post withdrawal of treatment with ERT or SRT. Improvement or maintenance of platelet counts was also seen post withdrawal of treatment with ERT or SRT. “FLT201 exemplifies our focus on developing the next generation of gene therapies, optimizing each component of our gene therapy candidates with the aim of changing the course of disease and changing people’s lives,” said Michael Parini, Spur’s Chief Executive Officer. “Our more stable GCase85 enzyme, combined with our potent and proprietary capsid, is leading to improvements in bone, pain and fatigue, all of which are key areas of ongoing need in Gaucher disease. These data highlight FLT201’s potential to provide better outcomes than current standard of care while dramatically reducing the treatment burden for patients, and we are actively engaging with regulators to advance FLT201 into a Phase 3 trial.” Spur expects to report additional data from the Phase 1/2 GALILEO-1 trial in the first half of 2025 and initiate a Phase 3 trial for FLT201 in adults with Gaucher disease type 1 in 2025. FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the Phase 1/2 GALILEO-1 clinical trial in adults with Gaucher disease. FLT201 leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the enzyme deficient in people with Gaucher disease, with the goal of stopping disease progression, reducing or eliminating symptoms, and allowing patients to come off current lifelong treatments. Preclinical and clinical data for FLT201 have shown robust and durable expression and a substantial reduction in the toxic buildup of substrate that results from the enzyme deficiency. For more information about the GALILEO-1 trial, please visit clinicaltrials.gov (NCT05324943). Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells, which then accumulate in tissues and organs throughout the body, causing inflammation and dysfunction. Despite treatment with currently approved therapies, many people with Gaucher disease continue to experience debilitating symptoms, including enlarged organs, fatigue, bone pain and reduced lung function. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, France, Germany, Spain, Italy and Israel. Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine. The content above comes from the network. if any infringement, please contact us to modify.

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