Gain Therapeutics to Present Positive Preclinical Data on its GBA1 Program at the AD/PD 2023 Meeting

BETHESDA, Md., March 29, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company leading the discovery and development of allosteric small molecule therapies, today announced the presentation of new pre-clinical data supporting the potential of its structurally targeted allosteric regulators (STARs) of glucocerebrosidase (GCase) to prevent neurodegeneration in Alzheimer’s disease (AD). The data will be shown in a poster presentation at the 2023 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD) being held March 28 – April 1, 2023 in Gothenburg, Sweden.

“We are pleased to expand upon the extensive package of preclinical data generated with our STAR compounds, which continue to demonstrate their potential advantages across a range of disorders associated with GCase protein misfolding,” said Matthias Alder, Chief Executive Officer. “We have shown previously that STAR-mediated augmentation of lysosomal GCase induces a neuroprotective effect in cellular and in vivo models of GBA1 Parkinson’s disease and Gaucher disease. In the presentation at AD/PD, we showcase the neuroprotective properties of these compounds in cellular models of Alzheimer’s disease, which support their potential for the treatment of Alzheimer disease and other tauopathies.”

Dr. Joanne Taylor, SVP, Head of Research, added: “Augmenting and restoring GCase function appears to constitute a fundamental mechanism of action that can result in beneficial effects across a range of neurodegenerative disease pathologies and indications. We look forward to providing further updates on our allosteric regulator programs throughout the year.”

The data generated in two cell-based assays of Alzheimer’s disease showed that Gain’s orally bioavailable, brain-penetrant structurally targeted allosteric regulators of GCase show promising activity against Amyloid Beta 1-42 (Aβ-1-42) and oligomeric Tau toxicity, which are thought to underlie neurodegeneration and cognitive impairment in Alzheimer's disease.

The neuroprotective properties of STAR compounds were evaluated in cellular models of AD with primary rat cortical and hippocampal neurons challenged with Aβ-1-42 and human Tau oligomers (hTauO), respectively. The data presented in the poster titled “Small-Molecule Structurally Targeted Allosteric Regulators of Glucocerebrosidase Show Neuroprotective Properties in Cell-Based Models of Alzheimer’s Disease,” demonstrate in vitro evidence that a STAR compound improves neuronal survival and neurite network and reduces hyperphosphorylated tau in primary rat cortical neurons after Aβ-1-42 injury. Additionally, STAR compounds were shown to reduce hTauO-induced neurotoxicity in primary rat hippocampal neurons. The data therefore demonstrate that structurally targeted allosteric regulators are a potential disease-modifying, novel pharmacological option for the treatment of AD and other tauopathies that warrants further investigation.

A PDF of the poster presented at the AD/PD 2023 Annual Meeting is available on the Science & Technology section of the Company’s website at .

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a biotechnology company leading the discovery and development of allosteric small molecule therapies. With its proprietary computational discovery platform SEE-Tx®, Gain Therapeutics is transforming drug discovery by identifying novel allosteric targets on proteins involved in diseases across the full spectrum of therapeutic areas. By binding to allosteric binding sites, the small molecules discovered with SEE-Tx provide opportunities for a range of drug-protein interactions, including protein stabilization, protein destabilization, targeted protein degradation, allosteric inhibition, and allosteric activation. Gain’s pipeline spans neurodegenerative diseases, lysosomal storage disorders (LSDs), metabolic disorders, as well as other diseases that can be targeted through protein degradation, such as oncology. 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. For more information, please visit

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are “forward-looking statements”. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding timing for reporting data from ongoing preclinical studies or the initiation of future clinical trials, including the timing for completion of IND-enabling toxicology studies, submission of the dossier requirement and commencement of a Phase 1 clinical program for GT-02287 for GBA1 Parkinson’s disease; the potential therapeutic and clinical benefits of the Company’s product candidates; the selection and development, and timing thereof, of future programs including the advancement of lead series identification for Krabbe disease and the identification of a lead candidate for its pre-clinical oncology program; the Company’s financial position and ability to execute on the next phase of its strategy; and the Company’s anticipated cash runway guidance, including the ability for the Company’s current and projected cash to allow the Company to meet value inflection points. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the COVID-19 pandemic and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors,” in the Company’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission on March 23, 2023 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Investor & Media Contact:



Argot Partners

(212) 600-1902