Pharming Group N.V. announced the successful completion of patient enrollment in the pivotal Phase II/III triple-blind, randomized, placebo-controlled study of leniolisib for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) on June 23.
Leniolisib is a small molecule PI3Kδ inhibitor that was developed by Novartis1 and was licensed to Pharming in 2019.8 The study, sponsored by Novartis, is a phase II/III potentially registration enabling study composed of two sequential parts, the first part including 6 patients was an open-label dose escalation study that was designed to assess the safety, tolerability, pharmacodynamics and pharmacokinetics of leniolisib2. The second part is a randomized, blinded, placebo-controlled study that includes approximately 30 additional patients and is designed to assess the efficacy of leniolisib in APDS patients. The co-primary endpoints of the second part of the study are (i) change in the size of lesions from baseline and (ii) change in baseline percentage of naive B cells out of total B cells. Pharming anticipates launch of leniolisib in Q4 2022, subject to regulatory approval.
APDS is an ultra-rare primary immunodeficiency disease caused by a genetic mutation affecting approximately 1-2 people per million. Current treatment is generally limited to supportive therapies, such as antibiotics and the use of immunoglobulin replacement therapy, as there is no approved therapy for the treatment of the disease.
Patients with APDS are often misdiagnosed with other immunodeficiencies or autoimmune disorders and often have a protracted journey to obtain a correct diagnosis. A definitive diagnosis can be made only by a genetic test. In March 2021, Pharming, in collaboration with Invitae Corporation, announced the launch of a genetic testing program, navigateAPDS, which is designed to assist clinicians in identifying patients with APDS and may lead to earlier diagnosis.
"With the completion of enrollment, we move one step closer to making this medicine available for APDS patients around the world", states Anurag Relan, Chief Medical Officer of Pharming.
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- Gene Therapy