Pharming Group announces first patient dosed in Phase II clinical trial of leniolisib for common variable immunodeficiency (CVID) with immune dysregulation

Core Insights - Pharming Group N.V. has initiated a Phase II clinical trial for leniolisib targeting common variable immunodeficiency (CVID) patients with immune dysregulation, marking a significant step in addressing unmet medical needs in this patient population [1][4]. Group 1: Clinical Trial Details - The Phase II trial is a single-arm, open-label study involving approximately 20 patients aged 12 and older, focusing on safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical efficacy of leniolisib in CVID patients [2]. - The trial specifically targets CVID patients exhibiting lymphoproliferation and at least one additional clinical manifestation of immune dysregulation, such as interstitial lung disease or autoimmune cytopenias [2][3]. - The lead investigator is Dr. Jocelyn Farmer, with clinical sites located in the US, UK, and EU [2]. Group 2: Disease Context and Unmet Needs - CVID is the largest group of symptomatic primary immunodeficiency patients, with about 50% experiencing autoimmune and lymphoproliferative complications due to immune dysregulation [3]. - Patients with CVID and immune dysregulation have an 11-fold increased mortality rate compared to those with infectious manifestations alone, highlighting the critical need for effective therapies [3]. - The global prevalence of CVID with immune dysregulation is estimated at approximately 39 patients per million [3]. Group 3: Leniolisib Overview - Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, currently approved in the U.S. for treating activated phosphoinositide 3-kinase delta syndrome (APDS) [5][6]. - The drug has shown statistically significant improvement in clinical trials for APDS, and its safety and tolerability for long-term use have been supported by interim data [6]. - Leniolisib is under regulatory review in multiple regions, including the European Economic Area, Canada, and Australia, with plans for further approvals in Japan and South Korea [6]. Group 4: Company Background - Pharming Group N.V. is a global biopharmaceutical company focused on developing therapies for rare and life-threatening diseases, with a portfolio that includes protein replacement therapies and precision medicines [7]. - The company is headquartered in Leiden, the Netherlands, and operates in over 30 markets worldwide [7].