French pharma company Sanofi has announced that it has halted the clinical programme of venglustat in autosomal dominant polycystic kidney disease (ADPKD).
Although the safety profile of venglustat remains consistent with previously reported results, a Phase II/III trial of the drug did not meet its futility criteria.
In addition, the study confirmed venglustat effectively inhibits the glycosphingolipid (GSL) pathway by demonstration a reduction in the lip GL-1.
The STAGED-PKD study was stopped for futility after an independent analysis of the annualised rate change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
In a statement, Sanofi said that trends from this analysis showed venglustat did not provide meaningful reduction in TKV growth rate – the primary endpoint of stage one of the study.
The company added that the analysis suggests the reduction of GSLs may not play a significant role in the prevention of kidney cyst growth, meaning it may not be a primary pathway associated with the progression of ADKPD.
“The venglustat development programme started with our confidence in the promise of a potential breakthrough treatment to address the unmet needs of people living with lysosomal storage disorders,” said John Reed, global head of research and development at Sanofi.
“In parallel, we set out to evaluate venglustat in autosomal dominant polycystic kidney disease, a leading cause of kidney transplant. This outcome is not what we hoped for, especially for these patients. However, our research has furthered the scientific understanding of ADPKD by demonstrating that modulating the GSL pathway is insufficient to restore kidney function in adults affected by this disease,” he added.
Sanofi has completed studies and also has additional active studies currently evaluating venglustat in Gaucher disease type 3, Fabry disease and GM2 Gangliosidosis, which are all lysosomal storage disease caused by inherited genetic abnormalities.
