Pharming Group announces first patient dosed in Phase II clinical trial of leniolisib for common variable immunodeficiency (CVID) with immune dysregulation
March 20, 2025 – Biotechnology, Clinical Trials, Drug Discovery, Pharmaceutical – Pharming Group NV, clinical trials, common variable immunodeficiency, immune dysregulation
- Multi-center clinical trial includes sites located in the US, UK and EU
- Second Phase II clinical trial studying leniolisib for additional primary immunodeficiencies (PIDs)
- CVID patients demonstrate clinical phenotypes similar to APDS, with global prevalence estimated at approximately 39 per million.
20 March 2025 — Leiden, the Netherlands — Pharming Group announces that the first patient has been dosed in a Phase II, proof of concept, clinical trial evaluating leniolisib in common variable immunodeficiency (CVID) patients with immune dysregulation.
The Phase II clinical trial is a single arm, open-label, dose range-finding, multi-center study to be conducted in approximately 20 patients 12 years of age and older. The trial will include patients with a CVID diagnosis, a requirement for evidence of lymphoproliferation, and at least one additional clinical manifestation of immune dysregulation, including interstitial lung disease, autoimmune cytopenias, or enteropathy. The objectives for the trial are to assess safety and tolerability, pharmacokinetics, pharmacodynamics, and explore clinical efficacy of leniolisib in the targeted CVID with immune dysregulation population. The trial has been designed to inform a subsequent Phase III program. The lead investigator for the Phase II study is Jocelyn Farmer, M.D./PhD, Director of the Clinical Immunodeficiency Program of Beth Israel Lahey Health (Lahey Hospital & Medical Center in Burlington, MA), with additional clinical sites in the US, UK and EU.
Jocelyn Farmer, MD, PhD; Allergist and Immunologist, Lahey Hospital & Medical Center, Burlington, MA; Director, Clinical Immunodeficiency Program, Beth Israel Lahey Health, MA; Associate Professor, UMass Chan-Lahey Medical School, Burlington, MA commented: “As a physician with clinical responsibility for a large group of common variable immunodeficiency (CVID) patients, I understand the significant disease burden they face. This includes autoimmune and end-organ lympho-infiltrative clinical complications resulting from their immune dysregulation. Due to the absence of effective therapies for these CVID patients, the disease manifestations can easily progress, leading to the well-documented early mortality in this patient group. PI3Kẟ is a multi-faceted regulator of lymphocytes, functioning to control their proliferation, differentiation, antibody production and migration, and hence leniolisib has significant potential to treat the immune dysregulation seen in these CVID patients. Therefore, I am very excited that we have dosed our first patient with leniolisib in this phase 2 proof of concept study in CVID patients with immune dysregulation, where leniolisib provides an opportunity to help these patients with a large, unmet medical need.”
CVID represents the largest group of symptomatic primary immunodeficiency (PID) patients, where approximately 50% display autoimmune, lymphoproliferative and/or end-organ lympho-infiltrative clinical manifestations driven by immune dysregulation.1,2,3 CVID patients with immune dysregulation have an unmet medical need with an 11-fold enhanced rate of mortality as compared to CVID patients with infectious manifestations alone, and the majority exhibit a spectrum of clinical manifestations with similarities to activated phosphoinositide 3-kinase delta syndrome (APDS) patients.
Based on available epidemiological data, it is estimated that the global prevalence of the targeted CVID with immune dysregulation population is approximately 39 patients per million.
Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented: “The initiation of this second Phase II clinical study outside the APDS indication is a substantial expansion of our work in primary immunodeficiency disorders. Unlike the initial APDS indication and our ongoing Phase II study in PIDs with immune dysregulation with specific genetic drivers, CVID patients are diagnosed based on standard clinical findings, independently of genetics. CVID patients with immune dysregulation have significant clinical unmet need, with no approved therapies, and represent a significantly larger patient population. We are therefore very excited about the potential leniolisib holds for treating CVID patients with immune dysregulation and look forward to enrolling more patients over the coming months.”
Leniolisib is marketed under the brand name Joenja® in the U.S. for the treatment of APDS in adult and pediatric patients 12 years of age and older.
About Pharming Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com
