Pharming reports positive Phase II data for leniolisib in rare primary immunodeficiencies
Pharming Group presented topline results from a single‑arm, open‑label Phase II study of leniolisib in 13 genetically identified primary immunodeficiency patients. The drug reduced spleen volume by a mean 26.4% in 10 of 11 evaluable participants and cut lymph‑node size by 33.7% in all four patients assessed. Lung diffusing capacity improved by an average of 8.0% among those with baseline impairment, and liver stiffness fell in two of three affected participants. All 12 participants who completed the trial enrolled in a three‑year extension study.
Why it matters
The company says the data demonstrate that PI3Kδ inhibition can benefit immune‑dysregulation beyond APDS, expanding leniolisib’s potential market. A CVID Phase II topline readout is expected in November, which could inform a registrational study and act as a near‑term catalyst.
Key facts
- 1The Phase II study enrolled 13 patients with CTLA4, SOCS1, NFKB1 or FAS variants. stocktitan.net
- 2Mean spleen‑volume reduction was 26.4% (n=11) with reductions observed in 10 of 11 evaluable participants. stocktitan.net
- 3Mean lymph‑node size reduction was 33.7% (n=4), observed in all four evaluable participants. stocktitan.net
- 4Among participants with impaired gas exchange (n=7), DLCO increased by a mean 8.0%. stocktitan.net
- 5Liver stiffness improved in 2 of 3 participants who had elevated stiffness at baseline. stocktitan.net
- 6All 12 participants who completed the study enrolled in a three‑year extension trial. stocktitan.net
Summary written by AlphAI AI Desk from 4 of 4 sources. Not investment advice. Figures are as stated by the linked sources.