Sanofi provides update on venglustat clinical program.

The safety profile of venglustat remains consistent with previously reported results with more than 500 patients treated to date over a period of up to four years across all clinical programs. Biomarker data from the study confirmed venglustat effectively inhibits the glycosphingolipid (GSL) pathway by demonstrating a reduction in GL-1, a lipid that accumulates in certain cells. The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo. Trends from the analysis showed venglustat did not provide a meaningful reduction in TKV growth rate, the primary endpoint of stage 1 of the Phase II/II study. This interim analysis suggests the reduction of GSLs may not play a significant role in the prevention of kidney cyst growth, and as such, may not be a primary pathway associated with the progression of ADPKD. The investigational research of venglustat in ADPKD was an attempt to explore a novel biological role for GSLs beyond the established role of these lipids in lysosomal storage diseases (LSDs).