Pharming Presented Leniolisib Phase II Clinical Results
The drug manufacturer released data showing reductions in spleen and lymph node size during a 20-week trial.
Updated on Oct. 14, 2026 in Asthma
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Pharming has presented Phase II clinical trial data for its treatment leniolisib, which was administered to 13 patients with immune dysregulation. The study measured physical improvements in organ and node volume over a 20-week period.
Why it matters
These findings help characterize the efficacy of leniolisib in treating complex primary immunodeficiencies. The data provides a clinical baseline for the drug's impact on patient physiological markers.
Spleen volume decreased by a mean of 26.4 percent, and index lymph node size fell by 33.7 percent across evaluable participants. Additionally, pulmonary diffusing capacity improved by a mean of 8.0 percent during the 20-week trial.
The players
Pharming
This is a biopharmaceutical company based in Leiden, Netherlands, that focuses on developing treatments for rare diseases.
The details
The trial employed an intra-patient dose-escalation design, with participants receiving twice-daily oral doses of 10 mg, 30 mg, or 70 mg. One participant discontinued the study after developing acute hepatitis.
Timeline
The data was presented at the ESID meeting in Maastricht from October 14-17, 2026.
Pharming expects to release Phase II results for CVID in November 2026.
Health Landscape
This study aligns with the broader medical shift toward precision therapies for rare immunological disorders. It marks a departure from legacy symptomatic treatments by focusing on underlying physiological markers like spleen volume.
The study offers new insights into how leniolisib may affect pulmonary capacity and organ size in specific patient groups. Patients should consult their medical providers regarding these results as they do not yet constitute a standardized treatment protocol.
The takeaway
The study highlights the potential of dose-escalation treatments to manage complex immune dysregulations. Ongoing extension research will be critical for determining the durability of these therapeutic benefits.
What happens next
Pharming is scheduled to report topline results from a separate Phase II trial focusing on CVID in November 2026.
Further reading
Learn more about the latest clinical developments in Asthma.
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