On Monday, Sanofi SA (NASDAQ:SNY) shared topline data from results from the LEAP2MONO phase 3 study of once daily oral venglustat versus intravenous enzyme replacement therapy (ERT) every two weeks in adults and pediatric patients aged 12 and older with type 3 Gaucher disease (GD3).

Venglustat, which works by reducing the abnormal accumulation of sugar-and fat molecules in cells and organs, is an investigational glucosylceramide synthase inhibitor that crosses the blood-brain barrier.

Forty-three (43) patients were randomized to receive venglustat and placebo infusion or ERT and placebo tablet.

The trial demonstrated that venglustat met the primary and three out of four key secondary endpoints in adults and pediatric patients (12 years and older) with neurological manifestations of type 3 Gaucher disease (GD3), ...