Gain Therapeutics Sees Stable MDS-UPDRS Scores and 6.7-Point Improvement After 90-Day GT-02287 Dosing
Gain Therapeutics reported that in its Phase 1b study of GT-02287, cerebrospinal fluid GluSph decreased substantially after 90 days and MDS-UPDRS scores remained stable at Day 150 among 14 extension participants, with high-GluSph subgroup showing a 6.7-point improvement. The company also advanced novel GCase modulator GT-04686 to IND-enabling studies.
1. Phase 1b Enrollment and Extension
Of 19 participants completing initial GT-02287 dosing in Part 1, 16 elected to continue into the nine-month extension, following a Data Monitoring Committee decision on March 5 to proceed without modifications.
2. Biomarker and Clinical Outcomes
In individuals with elevated baseline CSF glucosylsphingosine, GT-02287 reduced GluSph levels markedly after 90 days and lowered DOPA decarboxylase concentrations, while MDS-UPDRS Parts II and III scores held steady at Day 150, with the high-GluSph subgroup outperforming others by 6.7 points.
3. Novel Chemical Series Progress
A structurally distinct allosteric GCase modulator series led by GT-04686 has been advanced into IND-enabling studies, demonstrating brain penetration and restoration of key Parkinson’s disease–related biological activities in preclinical models.