Pharming Group N.V. announced that the first patient has been enrolled in its Phase III clinical trial in Japan evaluating leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years of age and older. Pharming's single-arm, open-label clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in three patients 12 years of age and years who have a confirmed APDS diagnosis. Each patient will receive weight-based dosing up to 70mg of leniolisib twice daily for 12 weeks.

With patients currently supportive on supportive treatments, the prospect of a disease-modifying treatment for this rare primary immunodeficiency could represent an exciting new treatment option for patients, their families, caregivers and their doctors in Japan. In May 2023, leniolisib was granted orphan drug designation (ODD) by the Ministry of Health, Labour and Welfare of Japan (MHLW) for the treatment of APDS. Leniolisib received regulatory approval from the United States Food and Drug Administration (FDA) for the treatment of AP DS in patients 12 years of age or older in March 2023 and was commercially launched under the brand name Joenja(R) in the U.S. in April 2023.

Results from a randomized, placebo-controlled Phase II/III clinical trial demonstrated clinical efficacy of Joenja(R) In the coprimary endpoints; demonstrating statistically significant impact on immune dysregulation and normalization of immunophenotype within these patients, and interim open label extension data has supported the safety and tolerability of long-term Joenja(R) administration.(8) Leniolisib is currently under regulatory review by the European Medicines Agency, with plans to pursue further regulatory approvals in the UK, Canada, Australia and Japan. Leniolisib is also being evaluated in a Phase III clinical trial in children aged 4 to 11 with APDS, with a further trial planned in children aged 1 to 6 years with APDS. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments.