Positive leniolisib top-line data in pediatric clinical trial from Pharming

Netherlands-based Pharming Group (Euronext: PHARM) today released positive top-line results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).

The news lifted Pharming’s shares more than 9% to 0.91 euros by late morning.

Leniolisib, marketed under the brand name Joenja in the USA, received approval from the US Food and Drug Administration (FDA) for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023. Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.