PHAR - Pharming reports publishing of phase 3 data of rare immunodeficiency disorder drug

Pharming ( NASDAQ: PHAR ) said data from a phase 3 trial of its drug to treat adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS) were published in the medical journal Blood .

The data from this study were previously announced in February 2022.

APDS is a rare immunodeficiency disease that affects 1 to 2 people per million and is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells, according to the company.

Pharming said the trial showed that leniolisib was well tolerated and significant improvement over placebo was notable in the co-primary goals, reflecting a favorable impact on patients' immune dysregulation and deficiency.

In the trial, which enrolled 31 patients with APDS aged 12 years or older, participants were randomized in a 2:1 ratio to receive 70 mg leniolisib or placebo twice daily for 12 weeks.

The company said improvement over placebo was significant in the co-primary goals which evaluated reduction in lymph node size and increase in naïve B cells, reflecting the impact on immune dysregulation and correction of immunodeficiency in these patients, respectively.

Leniolisib is currently under review in the U.S. and EU .

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