Pharming Group NV of the Netherlands has achieve positive results from a Phase 2/3 registration-enabling study of the small molecule drug leniolisib, a candidate treatment for activated phosphoinositide 3-kinase delta (PI3K delta) syndrome, an ultra-rare primary immunodeficiency disease. The placebo-controlled study consisted of two sequential parts, the first of which was a successful dose escalation study which reported results in 2018. Results from the second part of the study, reported on 2 February, showed clinical efficacy, including a normalisation of immune dysfunction.
