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A few days ago, Pharming announced the latest data from the pivotal Phase 2/3 clinical trial of the PI3Kδ inhibitor leniolisib in patients with PI3Kδ activation syndrome
PI3Kδ activation syndrome is a rare primary immunodeficiency caused by mutations in PIK3CD or PIK3R1, which regulate leukocyte maturation
Leniolisib is a small molecule inhibitor of PI3Kδ
From less than 48% at baseline, the proportion of naive B cells was increased by 34.
▲ Leliolisib molecular structure (Image source: PubChem)
In terms of safety, the drug was well tolerated, with no discontinuation or death due to adverse events.
"It is good news that Leniolisib has received such positive results in part of the Phase 3 clinical trial for the treatment of PI3Kδ activation syndrome
References:
[1] Pharming Announces Positive Data from Phase II/III Leniolisib Trial Presented at Clinical Immunology Society 2022 Annual Meeting.