Gain declares positive Phase I results for Parkinson’s therapy

In February, Gain Therapeutics revealed preclinical data indicating that GT-02287 restored motor function and significantly reduced plasma neurofilament light chain (NfL) levels, a burgeoning biomarker for neurodegeneration, in mouse models. GT-02287 demonstrated safety and tolerability in a Phase I trial involving healthy volunteers.

GT-02287, an allosteric protein modulator, is being developed to treat Parkinson's disease linked to GBA1 gene mutation, affecting up to 15% of Parkinson's patients. GBA1 mutation results in dysfunctional Glucocerebrosidase (GCase) lysosomal protein enzyme production. GT-02287 aims to restore GCase function, potentially slowing disease progression.

The Phase I trial, conducted at a single centre and placebo-controlled, evaluated GT-02287's safety and tolerability in healthy volunteers. Initially, the trial assessed the therapy in a single ascending dose (SAD) cohort and later progressed to a multiple ascending dose (MAD) phase in February, approved by the Bellberry Human Research Ethics Committee (HREC) in Australia.

The SAD cohort, comprising 40 participants, examined five therapy doses. Gain reported a "favourable safety and tolerability profile," with no serious adverse events noted up to the highest planned dose level. The MAD phase is slated for completion in the second quarter of this year.