Pharming Group NV (NASDAQ:PHAR) has announced results from the Phase 2/3study of leniolisib for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), an ultra-rare primary immunodeficiency disease.
- Clinical hallmarks of the disease are significant lymphoproliferation and immune dysfunction and increased risk of malignant lymphoma.
- Leniolisib is a small molecule PI3Kδ inhibitor discovered and developed by Novartis AG (NYSE:NVS) and was licensed to Pharming in 2019.
- The primary efficacy results demonstrated clinical efficacy of leniolisib over placebo with a statistically significant reduction from baseline in the index lymphadenopathy lesions and normalization of immune dysfunction.
- In the study, leniolisib was generally well-tolerated. The majority of reported adverse events in both treatment groups were mild.
- There were no adverse events that led to the discontinuation of study treatment. The incidence of serious adverse events (SAEs) was lower in the leniolisib group than placebo. None of the SAEs were suspected to be related to study treatment.
- Full results will be presented at upcoming medical conferences and published in a peer-reviewed journal.
- Price Action: PHAR shares are up 13.10% at $9.97 during the market session on the last check Wednesday.
