Pharming announces first patient dosed in pediatric clinical trial for children aged 1 to 6 years for leniolisib

The multinational Phase III study is evaluating a new pediatric granulated formulation of leniolisib in children aged 1 to 6 years with APDS, a rare primary immunodeficiency

Leiden, The Netherlands, November 21, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM)/(Nasdaq: PHAR) announces that the first patient has been dosed in its Phase III pediatric clinical trial evaluating a new pediatric formulation of the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children ages one to six years, with activated phosphoinositide 3-kinase delta syndrome (APDS).

At sites in the U.S., Japan, and the EU, the single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis. These patients will receive a specific, pediatric granulated formulation of leniolisib. The study’s primary efficacy endpoints and secondary endpoints mirror those used to evaluate the clinical outcomes in previous leniolisib Phase II/III APDS trials for patients aged 12 and older.

Dr. Eveline Wu, MD, MSCR, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, commented: 

“As a physician who has seen how significantly APDS can affect children’s lives, I’m excited that a targeted treatment may soon become available for even younger patients. While standard supportive therapies can ease some of the progressive, immune-related symptoms that characterize APDS, leniolisib is designed to help prevent those symptoms from arising. This could improve the standard of care for these children with the hope of a more full and enjoyable participation in the typical activities of childhood.”

Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented: 

“With the first patient dosed in this pediatric trial of leniolisib, using a new, granulated formulation, we are continuing to demonstrate Pharming’s ongoing commitment to providing new treatment options for younger pediatric patients with APDS. We look forward to generating the necessary supporting data to facilitate additional regulatory filings, thereby ensuring that patients from as broad an age range as possible can potentially benefit from this disease-modifying treatment.”