Pharming（PHAR）leniolisib 治疗 APDS 之外罕见免疫缺陷的 II 期阳性数据，实为 9 月 22 日已披露数据在 ESID 会议的正式展示，无新增事实

Pharming Group N.V. (PHAR) reported positive topline results from a Phase II study evaluating Leniolisib in patients with genetically identifiable primary immunodeficiencies associated with altered PI3Kdelta signaling.
The findings were presented at the 2026 meeting of the European Society for Immunodeficiencies (ESID).
Primary immunodeficiencies (PIDs) are a group of inherited disorders in which part of the immune system does not function properly. In some patients, genetic defects can lead not only to recurrent infections but also to immune dysregulation, causing enlarged lymph nodes, enlarged spleen, lung complications, liver disease, and other serious health problems.
Study Design
The open-label Phase II study enrolled 13 patients carrying disease-causing variants in the CTLA4, SOCS1, NFKB1 or FAS genes. Nine participants also had common variable immunodeficiency (CVID), one of the most common forms of primary immunodeficiency.
Patietns received leniolisib twice daily for 20weeks, with doses increased gradually from 10 mg to 30 mg and then to 70 mg during the study period.
Improvements Across Multiple Disease Measures
According to the company, leniolisib demonstrated benefits across several indicators of immune dysregulation.
Among evaluable participants, mean spleen volume decreased by 26.4%, with reductions observed in 10 of 11 patients. Enlarged spleen is considered a key sign of abnormal immune cell growth and activity.
Lymph node measurements also improved, with a mean reduction of 33.7% in index lesion size among four evaluable participants. All four patients experienced reductions.
The study also showed encouraging findings in lung disease. Among patients with impaired lung gas exchange at baseline, lung function improved by an average of 8% as measured by diffusing capacity of the lungs for carbon monoxide (DLCO). Additionally, imaging-based scores for interstitial lung disease improved in four of five evaluable patients.
Researchers also reported improvement in liver stiffness in two of three participants who had elevated liver stiffness at study entry.
Safety Profile
Leniolisib was generally well tolerated across all genetic subgroups included in the study. The most commonly reported adverse events were infections, and no new safety concerns were identified. All treatment-related adverse events were classified as Grade 1 in severity.
Two patients experienced serious adverse events that investigators determined were unrelated to leniolisib. One participant discontinued treatment early due to acute hepatitis and had a pre-existing history of portal hypertension.
Long-Term Follow-Up
All 12 participants who completed the Phase II study chose to enroll in an ongoing three-year extension trial designed to further evaluate the long-term safety and effectiveness of leniolisib in these rare immune disorders.
The results represent the first clinical evidence suggesting that the targeted PI3K delta inhibition with leniolisib may benefit patients with immune dysregulation caused by genetic conditions beyond APDS, potentially expanding the drug's future role in rare immunological diseases.
PHAR has traded between $9.32 and $21.34 over the past year. The stock closed Tuesday's trading at $9.44, down 1.77%
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