Pharming Reports Positive Phase 2 Data for Leniolisib in Genetic Immune Disorders

04:42 AM EDT, 10/14/2026 (MT Newswires) -- Pharming (PHAR) presented positive topline data from its Phase 2 trial of leniolisib for the treatment of certain genetic white blood cell deficiencies, to the European Society for Immunodeficiencies, the company said Wednesday. Leniolisib is treating patients with genetically identifiable primary immunodeficiencies with immune dysregulation, linked to altered PI3K-delta signaling, Pharming said. The phase II trial is a single-arm, open-label, intra-patient dose-escalation study that enrolled 13 participants with genetically identifiable primary immunodeficiencies, Pharming said. Patients received oral leniolisib twice daily for 20 weeks, escalating from 10 milligrams for the first four weeks, to 30 milligrams for four weeks, to 70 milligrams for 12 weeks, Pharming said. Leniolisib was generally well-tolerated by patients, resulted in improvement of patient spleens, lymph nodes and livers, and improved pulmonary function, Pharming said. Infections were the most common adverse events and no new safety signals were identified, it added. All 12 participants who completed the phase II study enrolled in a three-year extension study, Pharming said.