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Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. ("Pharming" or "the Company") (NASDAQ:PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).
Pharming has engaged with the US Food and Drug Administration (FDA) and has received feedback on its plans to develop leniolisib for PID disorders with immune dysregulation. This includes the recent FDA review of a Phase 2, proof of concept, clinical trial protocol in PIDs with immune dysregulation linked to PI3Kẟ signaling submitted under the existing leniolisib IND.
The Phase 2 clinical trial will evaluate leniolisib in PIDs with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes to APDS. These PID disorders are defined by loss-of-function variants in the following genes: cytotoxic T-lymphocyte associated protein 4 (CTLA4), FAS (causing autoimmune lymphoproliferative syndrome or ALPS), and phosphatase and tensin homolog (PTEN), among others. The epidemiology of these targeted PID genetic disorders suggests a prevalence of approximately 5 patients per million.
Posted In: PHAR
