Gain Therapeutics has dosed the first two subjects in a Phase I clinical trial of GT-02287 to treat GBA1 Parkinson’s disease (PD).
The single and multiple ascending doses (SAD/MAD), single-centre, placebo-controlled, double-blind, randomised study of the company’s lead drug candidate is anticipated to be completed in the first half of next year.
It will assess the tolerability and safety of the allosteric protein modulator GT-02287 in healthy adults who will receive GT-02287 orally once a day.
The secondary objective of the study is to assess the pharmacokinetics of SAD and MAD dose levels, determine a maximum tolerated dose and identify recommended doses.
These doses will be further used by the company in clinical development in the setting of GBA1 Parkinson’s disease.
The trial will also study the lysosomal protein enzyme glucocerebrosidase (GCase) target engagement and activity in blood as an exploratory endpoint.
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In preclinical PD models, GT-02287 restored the enzymatic function of GCase, improved motor function and increased dopamine levels, as well as reduced neuronal death, neuroinflammation, aggregated α-synuclein and plasma neurofilament light chain levels.
Gain Therapeutics CEO Matthias Alder said: “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.
“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.”
The programme received funds from InnoSuisse, The Silverstein Foundation for Parkinson’s with GBA and The Michael J. Fox Foundation for Parkinson’s Research.
The company is also engaged in developing new disease-modifying treatments for oncology, rare genetic disorders, and neurodegenerative diseases.