Pharming Doses First Patient in Phase II Trial of Leniolisib for CVID

Pharming Group NV, a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases, announced that the first patient has been dosed in a phase II 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, 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, Allergist and Immunologist, Lahey Hospital & Medical Center, Burlington, MA; Director, Clinical Immunodeficiency Programme, 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. PI3K? 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. CVID patients with immune dysregulation have an unmet medical need with an eleven-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, CMO 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 US for the treatment of APDS in adult and pediatric patients 12 years of age and older.

Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor approved in the US and several other countries as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Results from a randomized, placebo-controlled phase III clinical trial demonstrated statistically significant improvement in the coprimary endpoints, reflecting a favorable impact on the immune dysregulation and deficiency seen in these patients, and interim open label extension data has supported the safety and tolerability of long-term leniolisib administration. Leniolisib is currently under regulatory review in the European Economic Area, Canada and Australia for APDS, with plans to pursue further regulatory approvals in Japan and South Korea. Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS, and in a phase II clinical trial in primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3K? signaling in lymphocytes. The safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS.

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