Pharming Reports Positive Phase 2 Leniolisib Results in Genetic Immunodeficiencies
In a 13-patient uncontrolled study, mean spleen volume fell 26.4%, lymph-node lesions shrank 33.7%, and lung gas-exchange capacity improved 8.0%. No new safety signals were reported; all treatment-related adverse events were Grade 1. All 12 study completers entered a three-year extension. Separate Phase 2 CVID topline data are expected in November.