Business
Gain Therapeutics Announces Dosing of First Two Subjects in Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease
First small molecule allosteric binder discovered with Gain’s computational drug discovery platform enters clinical development BETHESDA, Md., Oct. 04, 2023
About this update from Gain Therapeutics, Inc.
[{"type":"text","content":"First small molecule allosteric binder discovered with Gain’s computational drug discovery platform enters clinical development\nBETHESDA, Md., Oct. 04, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced dosing of the first two subjects in a Phase 1 clinical trial of GT-02287, Gain’s lead drug candidate for the treatment of GBA1 Parkinson’s disease. The Company expects to complete this trial in the first half of 2024. “Initiating first-in-human dosing with GT-02287 is an important milestone for Gain as we enter a new era as a clinical-stage company,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics. “I am very proud of the work accomplished by the entire Gain team to get us to this stage today, and we are eager to advance our understanding of the safety, tolerability and effect of GT-02287 in humans. This represents another major step forward toward providing a treatment for Parkinson’s patients and their families impacted by this devastating disease.” Compelling preclinical data demonstrated that GT-02287 can restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to a GBA1 gene mutation, the most common genetic risk factor for the development of Parkinson’s disease. Restoring GCase function with GT-02287 was shown to have profound effect in animal models of Parkinson’s disease on the entire disease cascade, including a neuroprotective effect on dopaminergic neurons and improvement of motor deficiencies. Based on these data, GT-02287 has the potential to slow or even stop the progression of Parkinson’s disease. “Today marks an important step for Gain in the journey to bring a novel, potentially disease-modifying therapy to patients for whom only symptom-focused therapeutics exist,” said Dr. Robin Ely MD, Director, Integrative and Regenerative Medicine; Founder, National Gaucher Foundation; and Clinical-Scientific Consultant to NGF Global Diagnostic and Treatment Initiative. “If GT-02287 proves successful in disrupting the disease process in GBA1 Parkinson’s, its fundamental mechanism of action could play a crucial role in addressing various neurodegenerati...