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), a rare lysosomal storage disorder.
Data
Venglustat performed as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume, and hemoglobin levels, three key secondary endpoints of the study.
Sanofi will pursue global regulatory filings for venglustat in GD3.
Venglustat is also being studied for the treatment of Fabry disease, another rare lysosomal storage disorder.
Data from the phase 3 PERIDOT study show that a reduction in neuropathic and abdominal pain was observed in both study arms, and the primary endpoint was not met. Additional analyses of the data are ongoing.
A second phase 3 study, the CARAT study, evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease, is ongoing.
Venglustat was well tolerated overall with no new safety signals compared with previous studies.
Why It Matters
Sanofi currently markets Fabrazyme, an ERT for Fabry disease, and Cerezyme and Cerdelga for Gaucher disease, an ERT and oral therapy, respectively, in markets around the world.
In January 2026, the FDA approved an expanded label for Cerezyme to include non-central nervous system (CNS) manifestations of GD3, building on its approval in the US for GD1.
The LEAP2MONO study is ongoing, and results from its open-label phase will be presented in the future when available.
Price Action: SNY stock is up 1.28% at $47.64 during the premarket session at the last check on Monday, according to Benzinga Pro data.
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