Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (NASDAQ:PHAR) announced on Thursday that the first patient has been dosed in a Phase II clinical trial evaluating leniolisib for common variable immunodeficiency (CVID) with immune dysregulation.

The trial is a multi-centre, open-label, dose range-finding study being conducted at sites in the US, UK and EU, enrolling approximately 20 patients aged 12 and older.

The study aims to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of leniolisib, while exploring its clinical efficacy in CVID patients with immune dysregulation. Results will inform a subsequent Phase III programme. The lead investigator is Jocelyn Farmer, M.D./PhD, director of the Clinical Immunodeficiency Program at Beth Israel Lahey Health.

CVID is the largest group of symptomatic primary immunodeficiency (PID) patients, with 50% exhibiting immune dysregulation-related conditions such as interstitial lung disease, autoimmune cytopenias and enteropathy. This patient population faces an 11-fold higher mortality rate compared to CVID patients with infectious manifestations alone.

Leniolisib, marketed as Joenja in the US, is currently approved for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 years and older. The global prevalence of CVID with immune dysregulation is estimated at approximately 39 patients per million.