FDA Clears IND for Gain Therapeutics' GT-02287, Enabling Phase 2 Development in Parkinson's Disease

Research evidence excerpt

FDA Clears IND for Gain Therapeutics' GT-02287, Enabling Phase 2 Development in Parkinson's Disease

■ ...benefit over the low baseline group, with a 4.8-point separation in combined MDS-UPDRS Part II and III scores at Day 150.

The results build on the previously reported 81% average reduction in GluSph after 90 days. Safety and tolerability remain

intact with all 16 participants still on study, and a Data Monitoring Committee review in March concluded no protocol changes

were needed.

■ Recent biomarker data. At AD/PD, new supportive biomarker data were unveiled. CSF levels of DOPA decarboxylase (DDC) —

an emerging PD biomarker reflective of dopaminergic neuron dysfunction — were elevated at baseline in high-GluSph patients

and decreased following 90 days of GT-02287 treatment (Exhibit 1). Gain highlighted a correlation between decreased GluSph,

decreased DDC, and improvements in MDS-UPDRS scores, a multi-biomarker signal suggesting that target engagement may

be driving clinical improvement. MDS-UPDRS scores remained stable through Day 150, with high-GluSph patients showing aBIOTECHNOLOGY

6.7-point advantage over low-GluSph patients. These more recent updates build on the previous data release and make one

thing clear: GT-02287 increases the activity of its target enzyme.

■ Prior KOL event worth another look. The event serves as excellent background for the company and detailed the differencesEQUITY between glucosylsphingosine and glucosylceramide both in disease progression and importantly contrasted the two in their

usefulness as a biomarker. Glucosylceramide exists at higher levels and is more variable than the levels of glucosylsphingosine,

making it less ideal as a marker of GCase activity. Glucosylsphingosine is also highly toxic, and its levels are more tightly