Pharming Says Leniolisib Reduced Spleen Volume In 10 Of 11 Evaluable Participants And Lymph Node Size In All 4 In Phase II Genetic PID Trial, With No New Safety Signals

Benzinga · 2d ago
  • First clinical data for leniolisib in a larger group of PID participants beyond APDS, supporting the potential of targeted PI3Kδ inhibition to address immune dysregulation
  • Leniolisib was well-tolerated, with benefits observed across multiple measures of immune dysregulation, including improvement in lymphoproliferation and positive trends in lung and liver disease markers
  • All participants who completed the study elected to continue receiving treatment in a long-term extension study