Pharming Highlights Interim Data From Rare Immune Deficiency Study

Pharming Group N.V. PHAR announced data from an interim analysis of its open-label extension study of leniolisib for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, characterized by recurrent infections.
The data were shared at the 2022 Annual Meeting of the American Society of Hematology (ASH).
The results demonstrate the long-term tolerability of leniolisib, with a median duration on study therapy of just over two years (102 weeks) and five subjects being treated for five years or more.
Leniolisib treatment demonstrated a significant reduction in the annualized infection rate, while 37% of study patients on Immunoglobulin Replacement Therapy (IRT) could either reduce or stop their IRT regimens altogether.
It also indicated the durability of the efficacy results seen in the randomized, controlled trial, which showed significant improvement over placebo in the co-primary endpoints of reduction in lymph node size and increase in naïve B cells.
Most adverse events (AEs) reported were mild or moderate, including upper respiratory tract infection, headache, and pyrexia.
Among study participants, some experienced reductions in APDS disease markers.
Based on the Phase 2/3 trial results and the long-term, open-label extension data, the FDA is conducting a priority review of the leniolisib marketing application for APDS, with an action date of March 29, 2023.
Marketing authorization for leniolisib in the European Union is anticipated in H1 2023.
Price Action: PHAR shares closed at $11.81 on Wednesday.

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