Pharming Group N.V. announces a new phase II clinical trial of Leniolisib for Primary Immunodeficiencies with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes. The clinical trial is open for enrollment and will include PI patients with ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency, among others.
Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
“The initiation of this study is an important milestone for Pharming as it represents the second primary immunodeficiency (PID) clinical program for leniolisib. Based on our experience in APDS, and the significant role of PI3Kd in regulating lymphocytes, leniolisib has the potential to address the underlying immune dysregulation and deficiency in a number of rare PID disorders with significant unmet medical needs, including ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency. We are excited to be leading this important scientific effort and to sharing the results of the study with the medical community.”