Sanofi Stops Trial Assessing Venglustat for Kidney Disease

Sanofi announced that it has halted its trial of drug candidate venglustat for the treatment of autosomal dominant polycystic kidney disease, or ADPKD, following disappointing results.

The study was stopped after an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients getting venglustat compared to placebo showed that venglustat didn’t provide a meaningful reduction in the TKV growth rate, the primary endpoint of Stage 1 of the phase 2/3 study, Sanofi said.

The study confirmed that venglustat effectively inhibits the glycosphingolipid (GSL) pathway by demonstrating a reduction in GL-1, a lipid that accumulates in certain cells. But the interim analysis showed 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 company said.