Pharming : Positive Data From Phase 3 Study Of Leniolisib In Rare Primary Immunodeficiency Published

Pharming Group N.V. (PHAR) said that the positive results of a Phase 3 clinical trial of the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, in adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, have been published in Blood, the peer-reviewed international medical journal of the American Society of Hematology.

The company noted that Leniolisib was well tolerated and significant improvement over placebo was notable in the co-primary endpoints, reflecting a favorable impact on patients' immune dysregulation and deficiency.

Activated Phosphoinositide 3-Kinase d Syndrome is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. It is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kd (phosphoinositide 3-kinase delta) pathway.
Balanced signaling in the PI3Kd pathway is essential for physiological immune function.

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