Pharming Group (PHARM), on Thursday, announced that the first patient has been dosed in its Phase II clinical trial of Leniolisib for the treatment of Common Variable Immunodeficiency or CVID with immune dysregulation.
This multi-center, open-label, dose-ranging study will evaluate Leniolisib in approximately 20 patients aged 12 and older who have CVID and show evidence of immune dysregulation, including conditions like autoimmune cytopenias, interstitial lung disease, and enteropathy.
The trial will assess safety, pharmacokinetics, pharmacodynamics, and clinical efficacy in this population, with the goal of informing future Phase III studies.
Leniolisib is already approved in the U.S. under the brand name Joenja for the treatment of Activated Phosphoinositide 3-Kinase Delta Syndrome or APDS, and this trial represents an important step in exploring its potential for other primary immunodeficiencies or PIDs with immune dysregulation.
The trial is being led by Dr. Jocelyn Farmer, a prominent allergist and immunologist from Lahey Hospital & Medical Center, and includes clinical sites in the U.S., UK, and EU.
Given the high mortality rate among CVID patients with immune dysregulation, leniolisib could address a significant unmet need in this patient group.
Pharming is expanding the use of leniolisib to a larger patient population, beyond just APDS, with the goal of providing a much-needed therapeutic option for CVID patients who currently have no approved treatments.
The company anticipates continued patient enrollment in the coming months and is optimistic about the trial's potential to address a significant healthcare gap in the field of immunodeficiencies.
Currently, PHAR is trading at $8.93 up by 0.73 percent on the Nasdaq.
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