Pharming Group N.V. has initiated a Phase II clinical trial for leniolisib, targeting common variable immunodeficiency (CVID) patients with immune dysregulation. The multi-center trial will enroll approximately 20 patients aged 12 and older, focusing on safety, tolerability, and efficacy of leniolisib. This study aims to establish a foundation for a subsequent Phase III program.
This trial is crucial because CVID represents a significant portion of symptomatic primary immunodeficiency cases, with roughly half experiencing complications like autoimmunity and lymphoproliferation driven by immune dysregulation. These patients currently lack effective treatment options, resulting in a substantially higher mortality rate compared to CVID patients without immune dysregulation. Addressing this unmet medical need could significantly improve patient outcomes and survival rates.
The Phase II trial is a single-arm, open-label study designed to assess various aspects of leniolisib’s performance, including its pharmacokinetics and pharmacodynamics. This trial follows the successful U.S. approval of leniolisib (Joenja®) for treating activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 and older. The expansion into CVID, a diagnosis based on clinical findings rather than genetics, represents a significant increase in the patient population potentially benefiting from leniolisib. Epidemiological data suggest the global prevalence of the targeted CVID population with immune dysregulation is approximately 39 patients per million.
This Phase II trial’s initiation signifies a pivotal step in expanding leniolisib’s therapeutic potential beyond APDS. Positive results could pave the way for a Phase III trial and eventual approval, offering a much-needed treatment option for this sizable and underserved CVID patient population. This progress could reshape the treatment landscape for CVID with immune dysregulation and significantly improve long-term patient prognosis.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
