Pharming Reports Positive Phase II Data For Leniolisib In Rare Genetic Immune Disorders

(RTTNews) - On Wednesday, Pharming Group N.V. (PHAR) announced positive topline results from a Phase II study evaluating Leniolisib in patients with genetically identifiable primary immunodeficiencies (PIDs) associated with immune dysregulation linked to altered PI3Kdelta signaling.

Leniolisib is an approved PI3Kdelta inhibitor for activated PI3K delta syndrome (APDS), a rare genetic immune disorder, and is being explored for broader use in other genetically driven immune diseases.

The Phase II trial enrolled 13 patients with mutations in the CTLA4, SOCS1, NFKB1 or FAS genes and evaluated the drug over a 20-week treatment period.

According to the company, Leniolisib was generally well tolerated and demonstrated improvements across several measures of immune dysregulation, including a mean 26.4% reductions in spleen volume and reductions in the size of index lesions, alongside positive trends in lung and liver disease markers.

All 12 participants who completed the study elected to continue treatment in a three-year extension study.

PHAR closed Tuesday's trading at $9.44, down 1.77%.