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Gain Therapeutics Presents Positive Data on its Small Molecule Lead Compound in GBA1 Parkinson’s Disease at the 2022 Synuclein Meeting

Lead candidate GT-02287 is bioavailable, increases GCase levels, depletes alpha-synuclein, increases lysosomal health, and improves neuronal network and survival in relevant neuronal cell models

BETHESDA, Md., April 12, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”), a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Parkinson’s Disease (PD) program. The results were highlighted in a poster presentation at the 2022 Synuclein Meeting being held April 12 - 15, 2022, in Leuven, Belgium. The data show that the the lead compound GT-02287 increases the levels and activity of the beta-glucocerebrosidase (GCase) protein, improves lysosomal health, corrects abnormal phenotypes of PD including alpha-synuclein, and most importantly improves neuronal network connections and neuron survival.

Eric Richman, Chief Executive Officer of Gain comments, “The data we presented at the Synuclein Meeting demonstrates further encouraging disease modifying potential for patients with Parkinson’s disease who currently have no effective treatment options.”

“Our lead candidate, identified with the proprietary SEE-Tx drug discovery platform, can allosterically bind and stabilize GCase, thus avoiding its degradation and allowing its transport to the lysosomes where the enzyme can carry out its biological function,” said Manolo Bellotto, Ph.D., President and General Manager of Gain. “The new data builds on a body of evidence showing a beneficial effect of our lead candidate on enzyme levels, substrate depletion, lysosomal health, and most importantly for disease pathology, neuronal health.”

The data was generated in two different in vitro models covering both WT and the two most pathogenic GBA1 mutations L444P and N370S. The models used are the BE2-M17 dopaminergic neuron model which is implicated in Parkinson’s disease pathology, and a CBE model of rat mescencephalic neurons which induces the aggregation of alpha-synuclein as well as neuronal cell death.

The presentation titled “Targeting Glucocerebrosidase with Structurally Targeted Allosteric Regulators Corrects Abnormal Phenotypes in Models of Parkinson’s Disease” showed that the orally bioavailable and brain-penetrant lead molecule had a promising effect, including:

  • Increase of GCase levels and activity in WT, L444P, and N370S BE2-M17 dopaminergic neurons
  • Reduction of aggregated and phosphorylated alpha-synuclein levels in WT, L444P and N370S BE2-M17 dopaminergic neurons
  • Reduction of aggregated alpha-synuclein in TH+ neurons and improved lysosomal health in mescencephalic neurons treated with CBE
  • Increase in neuronal connections and neuron survival in mescencephalic neurons treated with CBE

About Parkinson’s Disease
Mutations in the GBA1 gene, encoding the lysosomal enzyme GCase, represent the most common genetic risk factor for PD. Impaired GCase function has garnered attention due to its association with α-synuclein pathology in GBA-associated PD patients, but also in sporadic PD, as well as in related α-synucleopathies. Although less investigated, decreased GCase levels and activity is also involved with the pathophysiology of AD. Enhancing the activity of mutant and wild-type GCase may represent a therapeutic strategy for the treatment of neurodegenerative diseases.

About Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform SEE-Tx®. The ability to identify never-seen-before allosteric targets on proteins involved in diseases across the full spectrum of therapeutic areas provides 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, metabolic diseases and 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 .

Forward-Looking Statements

Any statements in this release that are not historical facts may be considered to be “forward-looking statements.” Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, the expected progress of research and development programs and the effect of structurally targeted allosteric regulators for the treatment of Gaucher disease. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain’s ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

Media Contact:
Joleen Schultz
Joleen Schultz & Associates
+1 760-271-8150