Pharming shares early evidence for wider use of leniolisib
October’s presentation adds detail to a small study in immune disorders. With 13 participants and no control group, the findings remain early.
Pharming is testing whether leniolisib, already used for the rare immune disorder APDS, could help people with other genetic immune disorders. These conditions can leave the immune system both unable to fight infections properly and prone to attacking healthy tissue.
On 14 October, the company presented more detail from a Phase II study first reported in September. The study enrolled 13 participants and had no control group. The previously reported average reduction in spleen volume was 26.4% among the 11 participants with evaluable measurements. October’s update added findings on lung and liver measures in smaller groups.
Those results give Pharming reasons to keep investigating, but they cannot establish how the medicine compares with existing care. The study was small and open-label, meaning participants and investigators knew which treatment was being given. One participant stopped treatment after acute hepatitis. Pharming reported no new safety signals, but a study this size cannot establish safety or effectiveness for wider use.
A separate leniolisib study in common variable immunodeficiency is expected to report initial results in November. It should add evidence from a different patient group, while the uses discussed here remain investigational.