Pharming Group reports 26.4% spleen reduction in Phase 2 leniolisib trial
The 13-person study had no control group and a safety primary endpoint, with a separate CVID readout expected in November.
Theo Marchetti
In Maastricht, the Netherlands, Phase 2 results presented on Wednesday showed a mean 26.4 per cent reduction in spleen volume in patients with genetic primary immunodeficiencies. In a study of 13 participants, Pharming Group N.V. AMSPHARM NASDAQPHAR tested leniolisib for potential expansion beyond its approved rare immune disorder indication.
Pharming's Nasdaq depositary shares closed at US$9.44 at 4 p.m. EDT on Tuesday, before the release, according to Google Finance's NASDAQPHAR page.
The registered primary endpoint for NCT06549114 counts participants with adverse events, including serious events, through 20 weeks. The open-label, single-arm study compares results with baseline, without a control group.
The company says leniolisib's safety and efficacy remain unestablished for these additional uses. It expects a separate Phase 2 readout in common variable immunodeficiency, or CVID, in November to inform plans for a potential registrational study.
Spleen Measurements Improved In Most Participants
Participants had variants in the CTLA4, SOCS1, NFKB1 or FAS genes. Nine of the 13 also had CVID, showing overlap between the populations Pharming is studying.
Doses rose in stages, reaching 70 milligrams twice daily for the final 12 weeks. Spleen measurements were evaluable in 11 participants, and 10 had reductions.
Lymph-node index lesion size fell by a mean 33.7 per cent in four evaluable participants. Pharming reported a mean 8.0 per cent increase in percent-predicted lung diffusing capacity among seven participants with impaired gas exchange at baseline.
Lung CT scores improved in four of five evaluable participants. Liver stiffness improved in two of three participants with elevated stiffness at baseline.
"The consistent reductions in spleen volume in nearly all evaluable participants are particularly encouraging," Pharming chief medical officer Anurag Relan said in the release.
Pharming said all treatment-related adverse events were Grade 1, and infections were the most common adverse events. Two participants had serious adverse events deemed unrelated to leniolisib.
One participant with a history of portal hypertension stopped treatment early because of acute hepatitis. All 12 participants who completed the study enrolled in a three-year extension study, NCT06990529.
Joenja Already Generates Commercial Revenue
Joenja is approved for activated phosphoinositide 3-kinase delta syndrome, or APDS, while the additional immune disorders remain investigational uses.
Pharming's July 30 results presentation reported Joenja revenue of US$17.9 million for the second quarter of 2026, up 40 per cent from a year earlier. At June 30, 132 U.S. patients were on paid therapy.
Mirum Pharmaceuticals, Inc. NASDAQMIRM is also studying expansion of a marketed rare disease medicine. On Oct. 12, it reported that LIVMARLI met its Phase 3 primary itch endpoint against placebo in ultra-rare cholestatic liver diseases.
Mirum plans a supplemental U.S. drug application in the first half of 2027.
Read more: Mirum Pharmaceuticals reports LIVMARLI met Phase 3 EXPAND endpoint
Pharming expects the separate CVID trial's topline results in November 2026, which it says will inform plans for a potential registrational study in the broader CVID population.
