Data from phase 3 study of leniolisib in APDS published

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Results of a phase 3 clinical trial published in the journal Blood demonstrated the efficacy of the investigational drug leniolisib in patients with activated phosphoinositide 3-kinase delta syndrome (APDS). Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor.

In the study, 31 patients with APDS were randomly assigned 2:1 to leniolisib (70 mg) or placebo twice daily for 12 weeks.

Co-primary outcomes were differences from baseline in index lymph node size and in the percentage of naïve B cells in peripheral blood, assessed as proxies for immune dysregulation and deficiency.

Both primary outcomes were met. The adjusted mean change (95% CI) between leniolisib and placebo for lymph node size was -0.25 (-0.38, -0.12; P = 0.0006; n = 26) and for the percentage of naïve B cells it was 37.30 (24.06, 50.54; P = 0.0002; n = 13).

Leniolisib was found to be well tolerated.

“As we continue to seek a better understanding of APDS as a recently characterized rare disease, we remain committed to sharing our findings with researchers and doctors around the world. With this commitment in mind, we are pleased the results of this phase 3 clinical trial in leniolisib have been published in the flagship journal of the American Society of Hematology,” said Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, in a press release. “The APDS patient population, and their families, have lived with unmet needs and without targeted therapies, and the publishing of this study is an integral step in improving the patient journey for this community. We are proud to share these results which demonstrated leniolisib to be a well-tolerated, targeted therapy for APDS. We thank all of our study participants and investigators for their efforts and the essential role they played in the development of leniolisib.”

Rao VK, Webster S, Šedivá A, et al. Randomized, Placebo-Controlled, Phase 3 Trial of PI3Kδ Inhibitor Leniolisib for Activated PI3Kδ Syndrome. Blood. 2022;blood.2022018546.