Although the safety profile of venglustat remains consistent with previously reported results, the Phase 2/3 STAGED-PKD study of the drug did not meet its futility criteria.
In addition, the study confirmed venglustat effectively inhibits the glycosphingolipid (GSL) pathway by demonstrating a reduction in the lip GL-1.
The study was stopped for futility after an independent analysis of the annualized rate change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
Analysis showed venglustat did not provide a meaningful reduction in TKV growth rate – the primary endpoint of stage one of the study.
The company added that the analysis suggests the decrease of GSLs may not play a significant role in preventing kidney cyst growth, meaning it may not be a primary pathway associated with the progression of ADKPD.
Sanofi has completed studies and has additional active studies currently evaluating venglustat in Gaucher disease type 3, Fabry disease, and GM2 Gangliosidosis, all of which are lysosomal storage disease caused by inherited genetic abnormalities.
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