An oral investigational treatment, venglustat is designed to potentially reduce the progression of some diseases by hindering abnormal glycosphingolipid (GSL) build-up.
The Phase II/III trial was suspended for futility based on an independent evaluation of the annualised rate of change in total kidney volume (TKV) in subjects who received venglustat versus placebo.
Analysis trends demonstrated that venglustat did not offer a meaningful decrease in the growth rate of TKV, the primary goal of the study’s first stage. The company noted that the safety profile of venglustat in this trial is in line with earlier reported data with more than 500 subjects treated for up to four years in all clinical programmes so far.
Furthermore, biomarker data from the trial validated that venglustat could efficiently hinder the GSL pathway by lowering GL-1, a lipid that amasses in some cells.
Sanofi concluded that the interim analysis signifies GSL reduction may not have a vital role in preventing kidney cyst growth, indicating that it may not be a key pathway linked to ADPKD progression.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below formBy GlobalData
The investigational research of venglustat in ADPKD was intended to investigate a new biological role for GSLs apart from their known role in lysosomal storage diseases (LSDs), the company noted.
Sanofi Research and Development global head John Reed said: “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.
“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.”
Sanofi has so far completed trials and is also conducting studies assessing venglustat in Gaucher disease type 3, Fabry disease and GM2 Gangliosidosis, which are LSDs caused by inherited genetic abnormalities.
Last month, Sanofi and GlaxoSmithKline commenced participant enrolment in a Phase III clinical trial of their adjuvanted recombinant-protein Covid-19 vaccine candidate.