You are now
leaving this site

If you'd like to stay, click CANCEL.
X
CO-PRIMARY END POINTS DEMONSTRATED
CO-PRIMARY END POINTS DEMONSTRATED

Significant reductions in lymphadenopathy1

Significant increase in naïve B cells1-3

Log10-transformed SPD of index lesions (excluding patients with
0 lesions at baseline) at week 121,*

*The LS mean change from baseline, difference in LS mean change from baseline between Joenja and placebo and its P value, were obtained from an ANCOVA model with treatment, glucocorticoid use, and immunoglobulin replacement therapy at baseline, and baseline measurement as covariates.

The analysis excluded 2 patients from each treatment group due to protocol deviations and 1 Joenja patient having complete resolution of the index lesion identified at baseline.

Significantly improved immunophenotype vs placebo at week 12

  • In patients with <48% of naïve B cells at baseline,* the adjusted mean difference between Joenja (n=8) and placebo (n=5) in the percentage of naïve B cells out of total B cells was 37.30 (95% CI: 24.06, 50.54), P=0.0002

At week 12, patients saw a
significant reduction in
lymphadenopathy with Joenja vs placebo1

Absolute percentage of naïve B
cells over time

%

REDUCTION WITH joenja
vs 5% with placebo2
MEAN naïve B CELL LEVELS WITHIN NORMAL RANGE
by week 4 and maintained
through week 12 with Joenja
Not an actual patient.

Joenja effectively helped normalize immune balance in patients with APDS1,3

SECONDARY AND EXPLORATORY END POINTS

Joenja significantly reduced splenomegaly3

Not actual patients.

Significant reductions in spleen by 2D and 3D analysis compared
to placebo

  • The adjusted mean difference in bidimensional spleen size between Joenja (n=19) and placebo (n=9) was -13.5 cm2 (95% CI: -24.1, -2.91), P=0.0148
  • The adjusted mean difference in 3D spleen volume between Joenja (n=19) and placebo (n=9) was -186 cm3 (95% CI: -297, -76.2), P=0.0020
at week 12

%

REDUCTION in 3d spleen volume
  • The adjusted mean difference in bidimensional spleen size between Joenja (n=19) and placebo (n=9) was -13.5 cm2 (95% CI: -24.1, -2.91), P=0.0148
  • The adjusted mean difference in 3D spleen volume between Joenja (n=19) and placebo (n=9) was -186 cm3 (95% CI: -297, -76.2), P=0.0020
Not actual patients.

Secondary measure: Spleen volume scan results of actual patient illustrate average improvement documented for patients taking Joenja

Actual images of a 29-year-old female’s response of spleen size reduction, representing the median response in the study. As individual results vary, images may not be representative of all patients.
Scan results
prior to treatment (spleen volume 491 mL)
Scan results
after 12 weeks (spleen volume 314 mL)
Slide the orange dot across to see the difference.

In the PD analysis set, the mean (SD) percentage change from baseline to week 12 in 3D spleen volume (mm3) was -26.68% (12.137) with Joenja (n=19) and -1.37% (24.238) with placebo (n=9). The ANCOVA model was used with treatment as a fixed effect and log10-transformed baseline as a covariate for index and non-index lesions. The use of both glucocorticoids and IV Ig at baseline was included as
categorical (yes/no) covariates.

This analysis excluded 2 patients in each treatment group. In the Joenja group, 1 patient with a complete index lesion response was excluded, and 3 patients were excluded for no non-index lesion at baseline.

Ig, immunoglobulin; IV, intravenous; PD, pharmacodynamics.

Other secondary end points included patient-reported benefits with Joenja that were assessed using the
SF-36 (Short Form 36) Survey and WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), visual analogue scales for Physician’s Global Assessment (PGA), and Patient’s Global Assessment (PtGA), and patient narratives by investigator. Clinical relevance from these assessments was not established.
SECONDARY AND EXPLORATORY END POINTS

An exploratory end point showed Joenja reduced IgM levels3

  • In the Joenja arm, IgM was elevated above normal limits in 6 patients at baseline, and by week 12 was reduced in all, with 50% returning to within normal limits
  • In contrast, IgM was elevated above normal limits at baseline in 4 patients in the placebo arm, and by day 85 levels remained stable
    or elevated, with 0% returning to within
    normal limits

Mean serum IgM reduced to within normal limits

Soluble biomarkers, including IgM, were prespecified exploratory end points in the protocol. Although an observational decrease in IgM was noted in some patients, no statistical significance can be made from this analysis, and no conclusions should be drawn.

About APDS

APDS is a rare, primary immunodeficiency.
Hyperactivity along the Pl3Kδ signaling
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,3,4,7
learn more

Additional
Safety

The long-term safety and tolerability
of Joenja is under investigation in
the open-label extension study with
some patients receiving treatment
for as long as 5 years.1,6
See More Data
References: 1. Joenja (leniolisib). Prescribing information. Pharming Healthcare, Inc; 2023. 2. Data on file. Pharming Healthcare, Inc. 3. Rao VK, Webster S, Šedivá A, et al. A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome. Blood. 2023;141(9):971-983. doi:10.1182/blood.2022018546 4. Maccari ME, Abolhassani H, Aghamohammadi A,
et al. Disease evolution and response to rapamycin in activated phosphoinositide 3-kinase δ syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry. Front Immunol. 2018;9:543. doi:10.3389/fimmu.2018.00543 5. Rao VK, Webster S, Dalm VASH, et al. Effective “activated PI3Kδ syndrome”—targeted therapy with the PI3Kδ inhibitor leniolisib. Blood. 2017;130(21):2307-2316. doi:10.1182/blood-2017-08-801191 6. US National Library of Medicine. ClinicalTrials.gov/NCT02859727. 7. Angulo I, Vadas O, Garçon F, et al. Phosphoinositide 3-kinase δ gene mutation predisposes to respiratory infection and airway damage. Science. 2013;342(6160):866-871. doi:10.1126/science.1243292

Indications and Usage

JOENJA® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Important Safety Information

Verify pregnancy status in females of reproductive potential prior to initiating treatment with JOENJA.

Indications and Usage

JOENJA® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Important Safety Information

Verify pregnancy status in females of reproductive potential prior to initiating treatment with JOENJA.

JOENJA may cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus and to use highly effective methods of contraception during treatment with JOENJA and for 1 week after the last dose of JOENJA.

Live, attenuated vaccinations may be less effective if administered during JOENJA treatment.

Use of JOENJA in patients with moderate to severe hepatic impairment is not recommended. There is no recommended dosage for patients weighing less than 45 kg.

The most common adverse reactions (incidence >10%) seen in clinical trials were headache, sinusitis, and atopic dermatitis.

Seven (33%) patients receiving JOENJA developed an absolute neutrophil count (ANC) between 500 and 1500 cells/microL. No patients developed an ANC <500 cells/ microL and there were no reports of infection associated with neutropenia.

Before prescribing JOENJA, please read the full Prescribing Information.