Pharming Group announces presentation of new leniolisib data at 2022 ASH Annual Meeting
Presentation to highlight data from the ongoing long-term open-label extension study of leniolisib, a PI3Kδ inhibitor under investigation for APDS, a rare primary immunodeficiency
Presentation title: Interim Analysis of Safety and Hematological Parameters of an Ongoing Long-Term Open-Label Extension Study of Investigational PI3Kδ Inhibitor Leniolisib for Patients with Activated PI3Kδ Delta Syndrome (APDS) through
Presentation type: oral
Session name: 203. Lymphocytes and Acquired or Congenital Immunodeficiency Disorders: Delineating Immunity from Mice to Humans
Abstract number: 608
Session date and time: Monday, December 12, 2022 from
Presentation date and time: Monday, December 12, 2022 at
About Activated Phosphoinositide 3-Kinase δ Syndrome (APDS)
APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.
Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K. PI3Kδ is expressed predominately in hematopoietic cells and is essential to normal immune system function through conversion of phosphatidylinositol-4-5-trisphosphate (PIP2) to phosphatidylinositol-3-4-5-trisphosphate (PIP3). Leniolisib inhibits the production of PIP3 and PIP3 serves as an important cellular messenger activating AKT (via PDK1) and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of hematopoietic origin. The central role of PI3Kẟ in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3Kẟ is a valid and potentially effective therapeutic target for immune diseases such as APDS. To date, leniolisib has been well tolerated during both the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration-enabling study in patients with APDS.
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1. Lucas CL, et al. Nat Immunol. 2014; 15:88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016; 138(1):210-218.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017; 139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018; 9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2019;
7. Condliffe AM, Chandra A. Front Immunol. 2018; 9:338.
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