I Just came across this - I don't know if it been already posted...

I Just came across this - I don't know if it been already posted or is relevent

June 1, 2021 06:57 AM EDTR&DPharmaSanofi slashes another program for key molecule, while keeping hopes in handful of rare diseasesJason MastEditorSeveral years ago, when Sanofi unveiled a molecule to prevent the accumulation of a certain type of fat in cells, they figured it would work in a handful of rare diseases where the body can’t properly clear toxic chemicals. But they also had hopes and some biological intuition that it might provide a new way of treating a couple of larger disorders.Those hopes have now come up empty. Sanofi announced Tuesday that it scrapped a pivotal trial for the molecule, venglustat, in autosomal polycystic dominant kidney disease — the most common form of inherited kidney disease — after an independent review showed little difference between patients on the drug arm and patients on the placebo.It’s the second nail in the coffin for Sanofi’s ambitions to move venglustat outside of a cluster of similar rare diseases, called lysosomal disorders, after they quietly scrapped a Phase II study in Parkinson’s at their year-end earnings in February.Sanofi CMO John Reed didn’t mince words in a statement. The French pharma had hoped that by blocking a key enzyme in one of the cell’s lipid-making assembly line, called glucosylceramide synthase (GCS), they could prevent the growth of cysts in patients with this common kidney disorder. It turned out interfering with these lipid assembly lines — also known as the GSL pathway — wasn’t enough, Reed said.“This outcome is not what we hoped for, especially for these patients,” he said. “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.”Paul HudsonThe study’s primary endpoint had been the percent change in a patient’s total kidney volume, as measured by MRI. The review found there wasn’t a “meaningful reduction” in the drug arm compared to placebo. Although Sanofi did not say how many patients were ultimately dosed, the study had been slated to enroll 640 patients.The failure deprives Sanofi of what could have been a key win in a major indication, as new CEO Paul Hudson continues to try and revitalize their pipeline and recover from early setbacks in their Covid-19 vaccine program. But the company still has hopes for venglustat in lysosomal disorders, where Sanofi has a well-established franchise and where they believe the drug has far better odds of success.Three trials are underway in these diseases: A Phase III study in Tay-Sachs disease and Phase II trials in Gaucher disease type 3 and Fabry disease.AUTHORJason [email protected]