Pharming Group has dosed the first patient in a Phase III clinical trial assessing its investigational drug leniolisib’s new paediatric formulation in children aged between one and six years to treat activated phosphoinositide 3-kinase delta syndrome (APDS).

The rare primary immunodeficiency APDS is caused by variants in either one of two identified genes known as PIK3CD or PIK3R1. These genes play a key role in the development and function of immune cells in the body.

Leniolisib is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor.

The single-arm, open-label, multinational clinical trial, conducted in the US, the European Union (EU), and Japan, will assess the tolerability, safety, and efficacy of this therapy in 15 children aged one to six years with a confirmed APDS diagnosis.

A specific, paediatric granulated formulation of leniolisib will be administered to these patients.

The primary efficacy endpoints and secondary endpoints of the trial are identical to those used in assessing the therapy’s clinical outcomes in previous Phase II/III APDS trials involving patients aged 12 and older.

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By GlobalData

Pharming chief medical officer Anurag Relan said: “With the first patient dosed in this paediatric trial of leniolisib, using a new, granulated formulation, we are continuing to demonstrate Pharming’s ongoing commitment to providing new treatment options for younger paediatric 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.”

The company’s first paediatric trial, revealed in February this year, is currently underway. It is designed to assess the tablet formulation of leniolisib as an investigational treatment for APDS in 15 children aged four to 11 years.

Leniolisib, marketed as Joenja in the US, obtained approval from the US Food and Drug Administration in March 2023 to treat APDS in adults and paediatric subjects aged 12 years and above.