Pharming reports positive Phase II results for leniolisib in rare immune disorders

• Pharming (NASDAQ:PHAR) reported positive Phase II topline results for leniolisib in genetically identifiable primary immunodeficiencies linked to PI3Kδ signaling.
• The study showed reductions in lymphoproliferative disease measures, including a mean 26.4% reduction in spleen volume.
• Additional data will be presented at the ESID 2026 meeting.

Pharming Group (NASDAQ:PHAR) announced positive topline results from a Phase II study evaluating leniolisib in patients with genetically identifiable primary immunodeficiencies (PIDs) associated with immune dysregulation caused by PI3Kδ signaling abnormalities.

The study was accepted as a late-breaking abstract for presentation at the 2026 European Society for Immunodeficiencies (ESID) meeting.

The single-arm, open-label, intra-patient dose-escalation study evaluated 13 patients and demonstrated clinical improvements in lymphoproliferative disease, including reductions in spleen size and index lesion measurements.

Patients experienced an average spleen volume reduction of 26.4%, according to the company.

Leniolisib was generally well tolerated in the study.

Infections were the most commonly reported adverse events, and Pharming reported no new safety signals beyond the established safety profile of the treatment.

Additional study findings are expected to be presented at the ESID 2026 conference in October in the Netherlands.

Nine of the 13 participants also had common variable immunodeficiency (CVID). Pharming said it expects to report separate Phase II topline results evaluating leniolisib in CVID patients with immune dysregulation during the fourth quarter of 2026.

The company said these results will help guide potential future registrational development plans for additional patient populations.