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Gain Therapeutics Receives Approval to Commence Phase 1 Clinical Study of GT-02287 from the Human Research Ethics Committee (HREC) in Australia
First drug candidate identified with Gain’s proprietary computational drug discovery platform SEE-Tx® to enter clinical development phase BETHESDA, Md.,
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":" First drug candidate identified with Gain’s proprietary computational drug discovery platform SEE-Tx® to enter clinical development phase BETHESDA, Md., Sept. 12, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced that the Company has received approval from the Bellberry Human Research Ethics Committee (HREC) in Australia to initiate a Phase 1 clinical study of its lead drug candidate GT-02287 in development for GBA1-Parkinson’s disease (GBA1-PD). Dosing in healthy volunteers to assess the safety and pharmacokinetics of GT-02287 is expected to begin in the near term. “Following the recent presentation of encouraging preclinical data at the International Congress of Parkinson's Disease and Movement Disorders® showing the robust effect of GT-02287 against PD-associated pathology and its ability to significantly decrease plasma NfL levels, an emerging biomarker for neurodegeneration, we are pleased to receive HREC’s approval to enable the advancement of GT-02287 into the clinic,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics. “This approval is a key step for Gain and further validates our differentiated allosteric approach and our computational drug discovery platform as we endeavor to bring a novel, potentially disease-modifying therapeutic to patients with GBA1-Parkinson’s disease.” About GT-02287 Gain Therapeutics’ lead drug candidate, GT-02287, is in development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally available, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration. The program has been awarded funding support from The Michael J. Fox Foundation for Parkinson...