Gain Therapeutics Presents Positive Data on its Structurally Targeted Allosteric Regulators in both Parkinson’s and Alzheimer’s Disease at the AD/PD 2022 Conference

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BETHESDA, Md., March 15, 2022 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”), a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators (STARs) identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Parkinson’s and Alzheimer’s Disease programs. These results were highlighted in a poster presentation at the International Conference on Alzheimer’s (AD) and Parkinson’s Diseases (PD) being held March 15 – 20, 2022 virtually and in Barcelona, Spain. The data shows that improvement of the lysosomal function through the enhancement of glucocerebrosidase (GCase) levels and activity can improve key pathological features in α-synucleinopathies and AD.

Gain Therapeutics Outlines SEE-Tx Platform and Computational Biology Approach to Drug Design at R&D Day

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Bethesda-based Gain presented preclinical data from its Gaucher disease program at the 18th Annual WORLDSymposium on lysosomal disease research that showed the compounds the company is developing increase the levels of GCase protein, deplete phosphorylated alpha-synuclein, and increase autophagic flux. Gain Chief Executive Officer Eric Richman said the data presented at the symposium validates the company’s SEE-Tx platform, as well as the potential of the Gaucher-aimed candidate, GT-02329, that is demonstrating an ability to restore key lysosomal functions and showing promise as a first-in-class therapy for the treatment of neuronopathic Gaucher disease. Richman said the company’s Gaucher program could be in the clinic next year.

Gain Therapeutics Announces R&D Day Event and Participation in Upcoming Conferences

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Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”), a biotechnology company focused on identifying and optimizing allosteric binding sites that have never been targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that it will be hosting an R&D Day Event and participating in upcoming scientific conferences. Please see additional details below:

Gain Therapeutics, Inc. Announces Positive Topline Data in Patient-derived iPSC Study Evaluating STAR Compounds as a Treatment for Gaucher and Parkinson’s Disease

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Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced topline data from the Company’s study, conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds – GT-02287 and GT-02329 – for the treatment of Gaucher and GBA1 Parkinson’s Disease, demonstrated positive effects on all tested phenotypes.

Gain Therapeutics Announces Issuance of Two New PCT Patents Covering GBA and GALC Modulator Compounds for Treatment of CNS Disorders

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Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”), a biotechnology company focused on redefining drug discovery by identifying and optimizing allosteric binding sites that have never before been targeted, today announced the publication of two PCT patents. PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-​glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinson’s Disease, Lewy body dementia and Alzheimer’s.

Bethesda’s Gain Therapeutics Presents New GBA1 Parkinson’s Disease Program Pre-clinical Data

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Compounds reverse the neurodegenerative process observed in a Parkinson’s disease in vivo model

Compounds are novel allosteric regulators that bind, stabilize and restore activity of mutated GCase

Compounds decrease both phosphorylated and aggregated α-synuclein levels in vitro and in vivo

Bethesda’s Gain Therapeutics Announces Multi-Target Drug Discovery Collaboration Agreement with Zentalis Pharmaceuticals

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Collaboration to use Gain’s proprietary Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) computational platform technology to identify new and previously difficult-to-drug oncology targets

Gain Raises $40 Million in IPO, Funds Will Advance Rare Disease Pipeline

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Gain Therapeutics made its debut on the Nasdaq Thursday with a $40 million initial public offering. The company will use the funds to advance the development of its pipeline of therapeutics for rare genetic diseases characterized by protein misfolding.

Gain Therapeutics and UMSOM Partner to Investigate Small Molecule Drugs

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Gain Therapeutics and the University of Maryland School of Medicine forged a research collaboration to investigate Gain’s structurally targeted allosteric regulators (STARs) in cellular models of neuronopathic Gaucher disease (nGD) and Parkinson’s disease (PD).