Joenja works to help restore balance to your immune system
How Joenja was studied
Joenja was studied in a clinical trial for a period of 12 weeks, involving 31 participants. Each participant took a fixed 70 mg dose of Joenja or placebo, twice a day. The participants, sponsor staff, and trial staff did not know what treatment each participant took. This prevented participants from influencing some of the results.Patients with APDS taking Joenja saw improvement in 2 important ways:
The size of the most swollen lymph nodes was reduced
The size of the trial participants’ most swollen lymph nodes was studied before and after treatment. These lymph nodes shrank more in participants who took Joenja than in those who took placebo. This was a meaningful difference.
*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.
*Change in index lesion size was measured using the log10-transformed SPD of the largest lymph nodes (maximum of 6) identified as per the Cheson criteria on CT/MRI.
APDS, activated PI3K delta syndrome; CT, computed tomography; MRI, magnetic resonance imaging; SPD, sum of product diameters.
At week 12, patients saw a significant reduction in lymphadenopathy with Joenja vs placebo*
Reduction computed based on estimates for the adjusted mean changes.
Reduction computed based on estimates for the adjusted mean changes.
*Change in index lesion size was measured using the log10– transformed SPD of the largest lymph nodes (maximum of 6) identified as per the Cheson criteria on CT/MRI.
APDS, activated PI3K delta syndrome; CT, computed tomography; MRI, magnetic resonance imaging; SPD, sum of product diameters.
2 The number of functioning B cells increased to show efficacy
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‡
The graph below shows the average change in the percentage of functional B cells in these participants’ blood from before treatment to after 12 weeks of treatment.‡ Safety data set represents all patients in the trial.
†Cell surface markers used to distinguish naïve B cells on flow cytometry were CD19+, CD27-, and CD10-. Baseline is defined as the arithmetic mean of the baseline and day 1 values when both were available, and if either was missing, the existing value was used.
‡The analysis excluded 2 patients from each treatment group due to protocol deviations, 5 Joenja patients and 3 placebo patients with ≥48% naïve B cells at baseline, 5 Joenja patients with no day 85 measurement, and 1 Joenja patient with no baseline measurement.
Possible side effects
The table to the right shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
The table below shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
Joenja is generally well tolerated. The occurrence of side effects in patients taking Joenja was less frequent than in patients taking placebo, and no patients discontinued Joenja due to a side effect.
If you are concerned about side effects related to taking Joenja, talk with your healthcare provider.§Dermatitis atopic: including dermatitis atopic and eczema.
||Tachycardia: including tachycardia and sinus tachycardia.
Side effects reported by ≥2 Joenja-treated patients
| Joenja (21 participants) | Placebo (10 participants) | |
|---|---|---|
| Headache | 5 (24%) | 2 (20%) |
| Sinusitis (sinus infection) | 4 (19%) | 0 (0%) |
| Dermatitis atopic§ (skin rash) | 3 (14%) | 0 (0%) |
| Tachycardia ||(elevated heart rate) | 2 (10%) | 0 (0%) |
| Diarrhea | 2 (10%) | 0 (0%) |
| Fatigue | 2 (10%) | 1 (10%) |
| Pyrexia (fever) | 2 (10%) | 0 (0%) |
| Back pain | 2 (10%) | 0 (0%) |
| Neck pain | 2 (10%) | 0 (0%) |
| Alopecia (hair loss) | 2 (10%) | 0 (0%) |
The table to the right shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
The table below shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
Joenja is generally well tolerated. The occurrence of side effects in patients taking Joenja was less frequent than in patients taking placebo, and no patients discontinued Joenja due to a side effect.
If you are concerned about side effects related to taking Joenja, talk with your healthcare provider.§Dermatitis atopic: including dermatitis atopic and eczema.
||Tachycardia: including tachycardia and sinus tachycardia.
Spleen size also shrank with treatment
Prior to treatment: 491 mL
At week 12: 314 mL
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.
At week 12, patients with an enlarged spleen saw an average 27% decrease in the size of their spleen.¶
¶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.
PD, pharmacodynamics; SD, standard deviation.
2 The number of functioning B cells increased to show efficacy
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‡
The graph below shows the average change in the percentage of functional B cells in these participants’ blood from before treatment to after 12 weeks of treatment.‡ Safety data set represents all patients in the trial.
†Cell surface markers used to distinguish naïve B cells on flow cytometry were CD19+, CD27-, and CD10-. Baseline is defined as the arithmetic mean of the baseline and day 1 values when both were available, and if either was missing, the existing value was used.
‡The analysis excluded 2 patients from each treatment group due to protocol deviations, 5 Joenja patients and 3 placebo patients with ≥48% naïve B cells at baseline, 5 Joenja patients with no day 85 measurement, and 1 Joenja patient with no baseline measurement.
Possible side effects
The table to the right shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
The table below shows the side effects that happened in 2 or more participants. Other side effects were reported by fewer participants.
Joenja is generally well tolerated. The occurrence of side effects in patients taking Joenja was less frequent than in patients taking placebo, and no patients discontinued Joenja due to a side effect.
If you are concerned about side effects related to taking Joenja, talk with your healthcare provider.§Dermatitis atopic: including dermatitis atopic and eczema.
||Tachycardia: including tachycardia and sinus tachycardia.
Side effects reported by ≥2 Joenja-treated patients
| Joenja (21 participants) | Placebo (10 participants) | |
|---|---|---|
| Headache | 5 (24%) | 2 (20%) |
| Sinusitis (sinus infection) | 4 (19%) | 0 (0%) |
| Dermatitis atopic§ (skin rash) | 3 (14%) | 0 (0%) |
| Tachycardia ||(elevated heart rate) | 2 (10%) | 0 (0%) |
| Diarrhea | 2 (10%) | 0 (0%) |
| Fatigue | 2 (10%) | 1 (10%) |
| Pyrexia (fever) | 2 (10%) | 0 (0%) |
| Back pain | 2 (10%) | 0 (0%) |
| Neck pain | 2 (10%) | 0 (0%) |
| Alopecia (hair loss) | 2 (10%) | 0 (0%) |
Spleen size also shrank with treatment
Prior to treatment: 491 mL
At week 12: 314 mL
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.
At week 12, patients with an enlarged spleen saw an average 27% decrease in the size of their spleen.¶
¶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.
PD, pharmacodynamics; SD, standard deviation.
Focus on the things you love
Select Safety Information
Tell your healthcare provider if you are pregnant or plan to become pregnant. JOENJA may harm your unborn baby. Your healthcare provider will do a pregnancy test before you start receiving JOENJA.
Focus on the things you love
Select Safety Information
Tell your healthcare provider if you are pregnant or plan to become pregnant. JOENJA may harm your unborn baby. Your healthcare provider will do a pregnancy test before you start receiving JOENJA.
Starting and Staying on Joenja
Find information about starting Joenja and helpful tips for getting into a routine.
Support for You
Learn more about the APDS Assist program.
