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

Study results provide additional validation that SEE-Tx platform identifies brain-penetrant small molecules ideally suited for CNS diseases caused by protein misfolding

Demonstration of in vitro and in vivo functional efficacy in neurodegenerative disease with
GCase causality

First demonstration of an allosteric regulator with functional activity in both Parkinson’s and Alzheimer’s disease models

BETHESDA, MD, USA I March 15, 2022 I 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.

“We are excited to present this data that provides further evidence of the significant therapeutic potential of our STAR small molecules in neurodegenerative diseases, including Parkinson’s and Alzheimer’s Disease,” said Eric Richman, Chief Executive Officer of Gain. “It also continues to validate our computational target and drug discovery platform that is uniquely suited to identify novel allosteric binding sites and small molecule that regulate protein function.”

“The new data presented today show that our STARs molecules, which stabilize wild-type GCase protein in a dose dependent-manner, promote depletion of α-synuclein toxic forms as well as hyperphosphorylated tau protein, thus improving key pathophysiological features in PD and AD, respectively,” said Manolo Bellotto, Ph.D., President and General Manager of Gain. “The new data corroborate similar results we have recently published in our PD program, and represent the first data showing a beneficial effect of our STAR compounds in an AD model.”

The effect of STARs treatment on lysosomal function was presented from two models – CBE-induced PD mouse model and Aβ 1-42-induced AD neuronal model. The presentation titled “Brain-penetrant Structurally targeted Allosteric Regulators for Glucocerebrosidase (GCase) Show Promising Pharmacological Activity in Models of Parkinson Disease” demonstrated the following results:

  • Orally bioavailable and brain-penetrant lead STARs reduce neurotoxicity derived from the accumulation of toxic oligomeric species of Aβ and α-synuclein.
  • In the CBE-induced PD mouse model, treatment with the STAR small molecule resulted in:
    • Decrease of CBE-induced neurotoxicity and motor deficit
    • Decrease of toxic forms of alpha-synuclein
    • Decrease of neuroinflammation
  • In an Aβ 1-42-induced AD neuronal model, treatment with the STAR small molecule resulted in:
    • Decrease of Aβ 1-42-induced neurotoxicity
    • Decrease of hyperphosphorylated tau
    • Increase of neuronal survival
    • Improvement of the neurite network

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 https://www.gaintherapeutics.com

SOURCE: Gain Therapeutics

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