Pharming Announces Positive Phase II Data for Leniolisib in Rare Disease Trial

Key Facts

1Pharming announced positive Phase II topline data for leniolisib in genetic primary immunodeficiencies (PIDs) linked to altered PI3Kδ signaling.

2The drug showed improvements in lung and liver disease markers, with all participants electing to continue in a long-term extension study.

Pharming announced positive Phase II topline data for its drug leniolisib in treating genetic primary immunodeficiencies (PIDs) linked to altered PI3Kδ signaling. According to a company statement via GlobeNewswire, the drug showed improvements in lung and liver disease markers, leading all participants to elect to continue in a long-term extension study.

The clinical trial evaluated the efficacy and safety of targeted PI3Kδ inhibition in patients with genetically identifiable PIDs beyond the previously studied APDS condition. PHAR closed at $9.44 on October 13, 2026, prior to the presentation of these results at the 2026 European Society for Immunodeficiencies (ESID) meeting.