ENJAYMO is a breakthrough in the treatment of Cold Agglutinin Disease (CAD)1
ENJAYMO is indicated to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD.
The CARDINAL trial established the efficacy and safety of ENJAYMO for approval in CAD.1,2
CARDINAL was a phase 3, global, multicenter, open-label, single-arm, 6-month trial in 24 patients with CAD.1-3*
Secondary endpoints assessed: Effect on Hb and laboratory measures of hemolysis (mean change from baseline in bilirubin and LDH) at the TAT.2†
A majority of patients continued on ENJAYMO, entering an extension phase.
- Following completion of the 6-month treatment period, patients continued to receive ENJAYMO in a long-term safety and durability of response extension phase for an additional 24 months1
- Apart from those who discontinued, all patients, including the 3 without objective evidence of a response, continued to the extension phase2
- *Patients with confirmed CAD and a recent blood transfusion in 6 months prior to enrollment received ENJAYMO (N=24). Patients with CAS secondary to infection, rheumatologic disease, SLE, or overt hematologic malignancy were excluded, whereas patients with a history of concomitant low-grade lymphoproliferative disease were not excluded.
- †The treatment assessment time point (TAT) was defined as the mean value from Weeks 23, 25, and 26.
|Age||Mean (SD) Range||71.3 (8.2)
55 to 85 years
|n (%)||15 (63)
|Body weight||Mean (SD) Range||67.8 (15.8)
40 to 112 kg
|Hemoglobin||Mean (SD), g/dL||8.6 (1.16)|
|Bilirubin (total)‡||Mean (SD), mg/dL||3.1 (1.41) (2.6 × ULN)|
|LDH||Mean (SD), U/L||438 (484.60)|
|History of transfusion
Within last 6 months
Within last 12 months
|Median Number of Transfusions (Range)||2.0 (1, 19)
2.0 (1, 23)
- ‡N=21 for bilirubin data excluding patients with Gilbert’s syndrome.
Stop the unpredictability of C1-activated hemolysis in CAD1-3
The majority of patients achieved an improvement in hemoglobin and transfusion independence with ENJAYMO and required no additional CAD treatment (N=24)
A MAJORITY OF
ALL 3 COMPOSITE ENDPOINT MEASURES
- Substantial hemoglobin increase§
- Transfusion independence||
- No additional CAD treatments used||¶#
Treatment assessment time point (TAT) was defined as the mean value from Weeks 23, 25, and 26.
§Hb level ≥12 g/dL achieved in 38% of patients (9/24); increase in Hb level of ≥2 g/dL achieved in 63% of patients (15/24).
||From Weeks 5 to 26.
¶Prohibited therapies included rituximab alone or in combination with cytotoxic agents.
#Two patients discontinued prior to Week 23 and their status was considered unknown for the purposes of this analysis.
Controlling C1-activated hemolysis in CAD starts with the first dose of ENJAYMO1,2
Mean Hb and bilirubin levels with ENJAYMO (N=24)
- Hb data at each time point between baseline and Week 26 is the observed mean. Interpret these data with discretion given small sample size and descriptive statistics.
- Hb and bilirubin data at each time point between baseline and Week 26 is the observed mean. Interpret these data with discretion given small sample size and descriptive statistics.
- Bilirubin data at each time point between baseline and Week 26 is the observed mean. Interpret these data with discretion given small sample size and descriptive statistics.
Patients also experienced a mean improvement from baseline (424 U/L) in LDH, achieving levels that decreased to 1.2 × ULN at TAT (301 U/L)**
#Mean Hb at baseline: 8.6 g/dL (SD: 1.16);
#Mean Hb at baseline: 8.6 g/dL (SD: 1.16); mean bilirubin at baseline: 3.1 mg/dL (SD: 1.41).
#Mean bilirubin at baseline: 3.1 mg/dL (SD: 1.41).
**Among 17 patients with baseline and follow-up LDH values.
ENJAYMO provides rapid and sustained control of anemia and C1-activated hemolysis1,2
RAPID RESULTS BY WEEK 3
Rapidly improved anemia
2.29 g/dL increase in mean Hb levels††
Rapidly inhibited chronic hemolysis
Achieved normalization of bilirubin levels‡‡
SUSTAINED RESULTS THROUGH TAT
Sustained improvement in anemia
3.18 g/dL improvement in mean Hb levels††
Sustained inhibition of chronic hemolysis
Maintained normalization of bilirubin levels‡‡
††Observed mean model change in Hb was 2.60 g/dL (95% CI: 0.74-4.46) at TAT from baseline (mean Hb 8.6 g/dL [SD: 1.16]).
‡‡In CARDINAL, normal range of bilirubin defined as <1.2 mg/dL. Normalization maintained with LS mean decrease of 2.2 mg/dL (95% CI: 2.0-2.5).
The safety of ENJAYMO was demonstrated in CARDINAL1
The most common adverse reactions occurring in ≥10% of patients were respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and peripheral edema.
Adverse reactions (≥5%) in patients receiving ENJAYMO in CARDINAL (N=24)
|ADVERSE REACTION||PATIENTS n (%)|
|Respiratory tract infection§§||6 (25)|
|Viral infection§§||3 (13)|
|Urinary tract infection§§||2 (8)|
|Bacterial infection§§||2 (8)|
|Systemic hypertension||2 (8)|
|Abdominal pain||2 (8)|
|Arthralgia, arthritis§§||3 (13)|
|Peripheral edema||3 (13)|
|Infusion reaction||2 (8)|
§§Events may be counted in more than 1 grouped term (eg, viral upper respiratory tract infection is counted in viral infection and respiratory tract infection).
Only 3 patients experienced serious adverse reactions with ENJAYMO
- Serious adverse reactions were streptococcal sepsis and staphylococcal wound infection (n=1), arthralgia (n=1), and respiratory tract infection (n=1)
No patients discontinued ENJAYMO due to an adverse reaction
- Dosage interruptions due to an adverse reaction occurred in 17% (4/24) of patients who received ENJAYMO
No meningococcal infections were reported with ENJAYMO
- ENJAYMO may increase susceptibility to serious infections, including infections caused by encapsulated bacteria such as Neisseria meningitides (any serogroup), Streptococcus pneumoniae, and Haemophilus influenzae