On Wednesday, Pharming Group N.V. ( PHAR ) revealed topline results of data from its Phase 3 trial evaluating leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).

APDS is a genetic disorder that impairs the immune system. Individuals with this condition often have low numbers of white blood cells, particularly certain types of B and T cells. These cells are necessary to recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection.

Leniolisib, marketed under Joenja in the U.S., received FDA approval for APDS in adult and pediatric patients 12 years and older in March 2023.

Pharming ( PHAR ) plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.

The study enrolled 21 children with APDS ages 4 to 11 years.

The study’s primary efficacy endpoints are a reduction in index lymph node size and an increased proportion of naïve B cells out of total B cells from baseline at 12 weeks.

Secondary endpoints include assessing the ability of leniolisib to modify health-related quality of life.

All 21 patients enrolled completed the 12-week treatment period.

Lymphoproliferation improved as measured by a mean reduction in index lesion size, and immunophenotype correction was demonstrated by an increase in the percent of naïve B cells.

The improvement in lymphoproliferation and immunophenotype correction was seen across the four dose levels being investigated and was consistent with the improvements previously reported in adolescent and adult patients.

All treatment-emergent adverse events were reported to be mild to moderate in nature. There were no drug-related serious adverse events, and all patients completed the 12-week treatment period.

In October, Pharming ( PHAR ) started a Phase 2 proof-of-concept trial evaluating leniolisib in primary immunodeficiencies with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes.

Price Action: PHAR stock is up 11.6% at $9.75 at last check Wednesday.

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