Sanofi's venglustat shows breakthrough results in GD3 trial

Sanofi today reported positive phase 3 LEAP2MONO results showing venglustat delivering meaningful neurological benefits for adults and children with type 3 Gaucher disease (GD3), a rare disorder with no approved treatments for brain-related symptoms. The LEAP2MONO results from Sanofi highlight progress for a long-neglected patient population.

LEAP2MONO enrolled adult and pediatric patients with neurological manifestations of GD3. Venglustat, a glucosylceramide synthase inhibitor, reduces toxic sugar-fat buildup and crosses the blood-brain barrier.

"These findings underscore Sanofi's commitment to rare disease research and the promise we aim to deliver for people living with these conditions," said Houman Ashrafian, Executive Vice President and Head of Research and Development at Sanofi.

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"What excites us most is the potential to address critical unmet medical needs. A daily pill could make a serious difference for Gaucher patients facing neurological challenges. Most importantly, none of this would be possible without the courage of the patients and families who participate in our studies, and for that we owe them a debt of gratitude,” he added.

By week 52, the venglustat patients experienced statistically significant neurological improvement scores based on modified SARA and RBANS scores compared to enzyme replacement therapy. Liver and spleen volume and hemoglobin were similar in terms of non-neurological measures.

The drug was generally well tolerated, with no new safety signals. Common adverse events included headache, nausea, spleen enlargement, and diarrhea. Venglustat is also being studied in Fabry disease, where the phase 3 PERIDOT trial missed its primary endpoint and CARAT is assessing cardiac outcomes. Sanofi plans global regulatory filings for GD3.