Pharming Group doses first patient in phase 2 trial of leniolisib in CVID
Pharming Group has announced that the first patient has been dosed in a phase 2 clinical trial evaluating leniolisib (sold under the brand name Joenja) in common variable immunodeficiency (CVID) patients with immune dysregulation.
The company, based in Leiden, the Netherlands, specialises in developing and commercialising medicines for “patients with rare, debilitating and life threatening diseases”.
The trial will assess leniolisib’s safety, tolerability, pharmacokinetics, pharmacodynamics and clinical efficacy. It takes place internationally, with sites located in the US, UK and EU. Around 20 patients aged 12 years and older with both a CVID diagnosis and at least one additional manifestation of immune dysregulation are scheduled to join the phase 2 trial.
CVID with immune dysregulation affects approximately 39 people per million globally, and has similar clinical phenotypes to activated PI3K delta syndrome (APDS). An early mortality rate from CVID has been well-documented due to a lack of effective therapies for CVID patients.
The trial’s lead investigator, Jocelyn Farmer, is optimistic about this development, stating that “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,” she said.
