Efficacy results
For children ages 4-11
Pediatric patients aged 4+ saw meaningful improvements with Joenja
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Clinical trial design
Co-primary end points demonstrated
Secondary & exploratory end points
CLINICAL TRIAL DESIGN
How Joenja was studied in children 4-11 years of age
Duration
12 weeks
Population
21 participants
Age
Patients 4-11
Conditions
- Phase 3, 12-week, open-label study of pediatric patients ages 4-11 (N=21) who weighed ≥28 lb and <100 lb at baseline with pathogenic variants in PIK3CD or PIK3R1; dose range was 20-50 mg followed by ≤70 mg based on weight in the 1-year extension
- Co-primary endpoints:
- Change from baseline in the log10-transformed SPD of index lesions
- Change from baseline in percentage of naïve B cells out of total B cells
- Additional outcomes:
- Changes in B- and T-cell subsets, Ig levels, and spleen volume
- Safety assessment
Baseline demographic and disease characteristics in pediatric patients with APDS
Patient Characteristic
Joenja
(n=21)
Age, median (range), y
7.0 (4-11)
Sex, male-to-female ratio, n
13:8
Self-identified race, n (%)
White
Asian
Other
Not reported/unknown/missing
9 (42.9)
4 (19.0)
1 (4.8)
7 (33.3)
Self-identified ethnicity, n (%)
Hispanic
Non-Hispanic
Not reported
3 (14.3)
12 (57.1)
6 (28.6)
Weight at baseline, median (range), kg
23.9 (15.4-44.5)
Time since APDS diagnosis, median (range), mo
32.9 (2.2-142.2)
APDS variant classification, n (%)
PIK3CD
PIK3R1
17 (81.0)
4 (19.0)
Treatment compliance, %
91.0
Medical History
System Organ Class & Preferred Term
No. of Patients
(%)
Infections & Infestations
Otis media
Pneumonia
Sinusitis
15 (71.4)
4 (19.0)
3 (14.3)
3 (14.3)
Respiratory, thoracic, and mediastinal disorders
Bronchiectasis
Asthma
14 (66.7)
3 (14.3)
3 (14.3)
Gastrointestinal disorders
Abdominal pain
9 (42.9)
3 (14.3)
Blood and lymphatic system disorders
Lymphadenopathy
Splenomegaly
8 (38.1)
4 (19.0)
3 (14.3)
Surgical & medical procedures
Tonsillectomy
Ear tube insertion
Adenoidectomy
8 (38.1)
6 (28.6)
5 (23.8)
4 (19.0)
Skin and subcutaneous tissue disorders*
6 (28.6)
Investigations†
3 (14.3)
APDS, activated PI3Kδ syndrome; Ig, immunoglobulin; SPD, sum of product diameters.
*
Most common disorder within this category was dry skin in 2 patients (9.5%).
†
Bone marrow biopsy, cardiac murmur, and tuberculin test positive were reported in 1 patient each (4.8%).
CO-PRIMARY END POINTS DEMONSTRATED
Joenja helped normalize the immune system in children with APDS
Improvements with Joenja were seen across all age groups at week 12 in both lymphadenopathy and B-cell function
For lymphadenopathy, results were consistent with those seen in patients 12+ years of age
Reduction in lymphadenopathy
n=19
33.8%
REDUCTION IN LYMPHADENOPATHY
Increased naïve B cells
n=11
8.6%
MEAN INCREASE IN NAÏVE B CELLS
Joenja is the only FDA-approved treatment designed to help normalize the immune system in children 4-11 by targeting the underlying immune defect
APDS, activated PI3Kδ syndrome; CFB, change from baseline; FDA, Food and Drug Administration; SPD, sum of product diameters.
SECONDARY & EXPLORATORY ENDPOINTS
Joenja significantly reduced splenomegaly in pediatric patients ages 4-11
During the clinical trial, patients saw a reduction in spleen volume consistent with that seen in patients 12+ years of age
- At week 12, mean spleen volume decreased to 176.60 cm³ from 236.30 cm³ at baseline (n=21)
Reduction in splenomegaly
n=21
25%
REDUCTION IN SPLEEN VOLUME AT WEEK 12
Joenja safety profile
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APDS, activated PI3K delta syndrome.
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