Policy & Regulation
Gain Therapeutics reports first participant dosed in Phase 1b trial of GT-02287 for Parkinson's disease
14 March 2025 -

Clinical-stage biotechnology company Gain Therapeutics Inc (Nasdaq:GANX) on Friday announced the dosing of the first participant in its Phase 1b clinical trial of GT-02287, an allosteric small molecule in development for Parkinson's disease.

The trial will evaluate the safety and tolerability of GT-02287 in patients with and without GBA1 mutations.

The open-label, multi-centre study will enrol up to 20 participants, each receiving daily doses for three months. Secondary endpoints include pharmacokinetics, glucocerebrosidase (GCase) modulation, and biomarker analysis in plasma and cerebrospinal fluid.

Gain expects interim data from the trial by the end of the second quarter of 2025.

This Phase 1b study follows a successful Phase 1 trial in healthy volunteers, completed in the third quarter of 2024.

In the Phase 1 study, GT-02287 demonstrated a favourable safety and tolerability profile along with therapeutic-range plasma and CNS exposures. The trial also showed significant target engagement, with GCase activity increasing by more than 50%.

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