Pharming reports leniolisib Phase II data
- Pharming (NASDAQ:PHAR) reported Phase II topline data for leniolisib showing improvements in genetically identifiable primary immunodeficiencies with immune dysregulation beyond APDS.
- The single-arm study enrolled 13 participants, with treatment twice daily over 20 weeks and improvements observed across several disease measures.
- Pharming said all 12 participants who completed the study entered a three-year extension period.
Pharming (NASDAQ:PHAR) presented Phase II topline results from a study evaluating leniolisib in patients with genetically identifiable primary immunodeficiencies involving immune dysregulation beyond activated PI3K delta syndrome (APDS).
The open-label study included 13 participants treated twice daily for 20 weeks, with 11 evaluable participants showing a mean spleen volume reduction of 26.4% from baseline.
The company reported additional improvements, including a 33.7% reduction in lymph-node index lesion size among four participants, an 8% average increase in lung gas-exchange capacity among seven participants with baseline impairment, and reduced lung disease scores in four of five evaluable participants.
Pharming reported that all treatment-related adverse events were Grade 1, while two serious adverse events were considered unrelated to leniolisib and one participant discontinued treatment due to acute hepatitis.
Pharming develops treatments for rare diseases, including immune system disorders, and is also preparing separate clinical trial results for leniolisib in common variable immunodeficiency (CVID).
