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For U.S. Healthcare Professionals Only
JOIN US AT THE ASH ANNUAL MEETING FOR A PRESENTATION ON ENJAYMO | MONDAY 12/12 @ 8 am
Not an official event of the 64th ASH Annual Meeting and Exposition. This presentation is not sponsored or endorsed by ASH. Not CME-accredited. MAT-US-2208046-v1.0-11/2022
VISIT US AT ASH | DECEMBER 10-13 | BOOTH #643
Not an official event of the 64th ASH Annual Meeting and Exposition. This presentation is not sponsored or endorsed by ASH. Not CME-accredited. MAT-US-2208046-v1.0-11/2022
ENJAYMO is an FDA-designated breakthrough therapy for the treatment of hemolysis in Cold Agglutinin Disease (CAD)1
Efficacy and safety of ENJAYMO were assessed in CARDINAL2,3
CARDINAL, a phase 3, open-label, single-arm, 6-month trial in 24 patients with Cold Agglutinin Disease, demonstrated efficacy and safety of ENJAYMO.
Study design of CARDINAL
CARDINAL is a phase 3, global, multicenter, open-label, single-arm, 6-month trial in 24 patients with Cold Agglutinin Disease (CAD) who received ≥1 transfusion during the preceding 6 months.* Following completion of the 6-month treatment period (Part A), patients continued to receive ENJAYMO in a long-term safety and durability of response extension phase (Part B) over 2 years.2,3
ENDPOINTS STUDIED2
PART A COMPOSITE PRIMARY ENDPOINTS
-
Increase in Hb to ≥12 g/dL OR ≥2 g/dL from baseline at TAT
-
Transfusion independence from baseline through Weeks 5 to 26
-
No use of protocol-prohibited CAD treatment from Weeks 5 to 26
PART A SECONDARY ENDPOINTS
-
Effect on Hb (mean change from baseline at TAT)†
-
Change in fatigue (mean change from baseline at TAT in FACIT-Fatigue score)‡
-
Laboratory measures of hemolysis (mean change from baseline at TAT in bilirubin and LDH)
- *Patients with confirmed Cold Agglutinin Disease and history of transfusion within 6 months prior to enrollment (n=24). Patients with CAS secondary to infection, rheumatologic disease, SLE, or overt hematologic malignancy were excluded, whereas patients with a history of or concomitant low-grade lymphoproliferative disease (bone marrow involvement <10%) were not excluded.2
- †Two patients discontinued prior to Week 23 and their status was considered unknown for the purposes of this analysis.3
- ‡Treatment assessment time point (TAT) was the mean value from Weeks 23, 25, and 26.2,3
| Parameter | Statistic |
ENJAYMO n=24
|
|---|---|---|
| Age | Mean (SD) (min, max) |
71.3 (8.2) (55, 85) |
|
Sex
Male
Female
|
n (%) |
9 (38)
15 (63)
|
| Body weight | Mean (SD) (min, max) |
67.8 (15.8) (40, 112 kg) |
| Hemoglobin | Mean (SD), g/dL (min, max) |
8.6 (1.16) [4.9, 11.1] |
| Bilirubin (total)§ | Mean (SD), mg/dL | 3.1 (1.41) (2.6 x ULN) |
| LDH | Mean (SD), U/L | 438 (484.60) |
|
History of blood transfusion
Within last 6 months
Within last 12 months
|
Median (range) |
2.0 (1, 19)
2.0 (1, 23)
|
- §n=21 for bilirubin data excluding patients with Gilbert’s syndrome.
Clinically meaningful improvement across key efficacy measures2,3
A majority of patients achieved an improvement of hemoglobin and transfusion independence with ENJAYMO and required no additional Cold Agglutinin Disease treatment (13/24)
A MAJORITY OF PATIENTS ACHIEVED
ALL 3 COMPOSITE ENDPOINT MEASURES AT TAT
54%
(13/24)
- Significant hemoglobin increase*
- Transfusion independence†
- No additional Cold Agglutinin Disease treatment 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).2
- †From Weeks 5 to 26.2
- ‡Prohibited therapies included rituximab alone or in combination with cytotoxic agents.2
- §Two patients discontinued prior to Week 23, and their status was considered unknown for the purposes of this analysis.3
“I'd been on ENJAYMO about 9 months, and I’m walking with my family to this restaurant, and all of a sudden I'm by myself. I turn around and they’re several feet behind me. My son goes, ‘Did you think we were speed walking or something? You're just booking it, Mom.’ He was really excited and happy for me that I had so much energy.”
—Gloria, living with
Cold Agglutinin Disease
RAPID improvement in anemia and normalization of bilirubin SUSTAINED over 2.5 years with continuous biweekly infusions2,4*
Mean Hb and bilirubin levels through CARDINAL Part B trial end (2.5 years) (n=19)†‡§
Data at TAT was tested for significance.
Other time points represent observed mean values and are descriptive only. Interpret these findings with caution, particularly given the small sample size.2,3
- *The recommended dosing regimen for adults with Cold Agglutinin Disease consists of an initial dose and a dose 1 week later, followed by 1 dose every 2 weeks.2
- †Trial end refers to last available on-treatment value.
- ‡Mean baseline values: Hb was 8.6 g/dL (SD: 1.16); bilirubin was 3.1 mg/dL (SD: 1.41). Improvement at TAT: LS mean change in Hb was 2.60 g/dL; LS mean change in bilirubin was -2.23 mg/dL (95% CI: -2.49 to -1.98).2
- §Mean Hb level of 12.23 g/dL and mean bilirubin level of 0.96 mg/dL were observed at the last on-treatment visit.2
A well-tolerated safety profile studied over 2.5 years in CARDINAL2
ENJAYMO efficacy and safety were evaluated in CARDINAL,* an open-label, single-arm study, 143 weeks (n=24)
In CARDINAL: adverse reactions (≥15%) in patients receiving ENJAYMO
| Adverse Reactions | n (%) (n=24) |
|---|---|
| Urinary tract infection | 9 (38%) |
| Respiratory tract infection | 6 (25%) |
| Bacterial infection | 6 (25%) |
| Nasopharyngitis | 5 (21%) |
| Viral infection | 5 (21%) |
| Dizziness | 7 (29%) |
| Headache | 5 (21%) |
| Fatigue | 8 (33%) |
| Peripheral edema | 6 (25%) |
| Pyrexia | 5 (21%) |
| Arthralgia | 6 (25%) |
| Hypertension | 6 (25%) |
| Acrocyanosis | 5 (21%) |
| Nausea | 6 (25%) |
| Abdominal pain | 5 (21%) |
| Cough | 6 (25%) |
| Infusion-related reaction | 4 (17%) |
Serious infections (bacterial and viral) were reported in 15% (10/66) of patients receiving ENJAYMO in the two phase 3 trials
- ENJAYMO increases susceptibility to serious infections, including infections caused by encapsulated bacteria, eg Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type B.
- Serious infections included urinary tract infection with sepsis, respiratory tract infection, pneumonia, otomastoiditis, and skin infections. One patient (1.5%) died due to Klebsiella pneumoniae. No meningococcal infections were reported.3
Infusion-related reactions occurred in 29% (19/66) of patients treated with ENJAYMO in the two phase 3 trials. One patient permanently discontinued ENJAYMO due to an infusion-related reaction.
Serious adverse reactions (ARs) in patients who received ENJAYMO
- In CARDINAL: 10/24 (42%) reported serious ARs with the most common (>5%) being acrocyanosis (n=2). One patient died due to Klebsiella pneumoniae.
Permanent discontinuation of ENJAYMO due to an adverse reaction occurred in 2/24 (8%) patients
- In CARDINAL: Klebsiella pneumoniae (n=1) and acrocyanosis (n=2).
Dosage interruption of ENJAYMO due to adverse reaction was reported in 7/24 (29%) patients in CARDINAL trial.
- *CARDINAL included confirmed Cold Agglutinin Disease patients with no history of transfusion within 6 months, or >1 in 12 months prior to enrollment.
Consider prescribing ENJAYMO for your next appropriate patient with Cold Agglutinin Disease
ENJAYMO HCP Indication and Important Safety Information
INDICATION
ENJAYMO® (sutimlimab-jome) is indicated for the treatment of hemolysis in adults with cold agglutinin disease (CAD).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
ENJAYMO is contraindicated in patients with known hypersensitivity to sutimlimab-jome or any of the inactive ingredients.
WARNINGS AND PRECAUTIONS
Serious Infections Including Those Caused by Encapsulated Bacteria
- ENJAYMO, a proximal classical complement C1s inhibitor, increases susceptibility to serious infections, including those caused by encapsulated bacteria e.g. Neisseria meningitidis (any serogroup, including non-groupable strains), Streptococcus pneumoniae, and Haemophilus influenzae type B.
- Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.
- Serious infections (bacterial and viral) were reported in 15% (10/66) of patients receiving ENJAYMO in the two phase 3 trials. These infections included urinary tract infection with sepsis, respiratory tract infection, pneumonia, otomastoiditis, and skin infections. One patient (1.5%) died due to Klebsiella pneumoniae.
- Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of ENJAYMO, according to the most current ACIP recommendations for patients receiving a complement inhibitor.
- If urgent ENJAYMO therapy is indicated in a patient who is not up to date on their vaccine(s), administer as soon as possible.
- Vaccination does not eliminate the risk of serious encapsulated bacterial infections. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected.
-
If ENJAYMO treatment is administered to patients with active systemic infections, monitor closely for signs and symptoms of worsening infection. Some infections may become rapidly life-threatening or fatal if not recognized and treated promptly. Inform patients of these signs and symptoms and steps to be taken to seek immediate medical care.
- Consider interruption of ENJAYMO treatment in patients who are undergoing treatment for serious infection.
- Consider patients’ immune status when initiating treatment with ENJAYMO.
Infusion-Related Reactions
- Administration of ENJAYMO may result in infusion-related reactions. In the two phase 3 trials, 29% (19/66) of patients treated with ENJAYMO experienced infusion-related reactions. One patient permanently discontinued ENJAYMO due to an infusion-related reaction.
- Monitor patients for infusion-related reactions and interrupt if a reaction occurs.
- Discontinue ENJAYMO infusion and institute appropriate supportive measures if signs of hypersensitivity reactions, such as cardiovascular instability or respiratory compromise, occur.
Risk of Autoimmune Disease
- Based on its mechanism of action, ENJAYMO may potentially increase the risk for developing autoimmune diseases such as systemic lupus erythematosus (SLE). Development of SLE has been associated with inherited classical complement deficiency.
- In clinical trials, 4.5% (3/66) of patients developed a relapse or worsening of previously diagnosed autoimmune disease.
- Monitor ENJAYMO patients for signs and symptoms and manage medically.
Recurrent Hemolysis After ENJAYMO Discontinuation
- If treatment with ENJAYMO is interrupted, closely monitor patients for signs and symptoms of recurrent hemolysis, eg, elevated levels of total bilirubin or lactate dehydrogenase (LDH) accompanied by a decrease in hemoglobin, or reappearance of symptoms such as fatigue, dyspnea, palpitations, or hemoglobinuria. Consider restarting ENJAYMO if signs and symptoms of hemolysis occur after discontinuation.
ADVERSE REACTIONS
- The most common adverse reactions in the CADENZA trial (Part A) (incidence ≥18%) are rhinitis, headache, hypertension, acrocyanosis, and Raynaud’s phenomenon. The most common adverse reactions in the CARDINAL trial (incidence ≥25%) are urinary tract infection, respiratory tract infection, bacterial infection, dizziness, fatigue, peripheral edema, arthralgia, cough, hypertension, and nausea.
CAS=cold agglutinin syndrome; CI=confidence interval; FACIT=Functional Assessment of Chronic Illness Therapy; Hb=hemoglobin; LDH=lactate dehydrogenase; LS=least squares; SD=standard deviation; SLE=systemic lupus erythematosus; TAT=treatment assessment time point; ULN=upper limit of normal.
References:
- FDA: Food and Drug Administration. FDA approves treatment for adults with rare type of anemia. FDA. February 4, 2022. Accessed October 28, 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-adults-rare-type-anemia
- ENJAYMO. Prescribing information.
- Röth A, Barcellini W, D'Sa S, et al. Sutimlimab in Cold Agglutinin Disease. N Engl J Med. 2021;384(14):1323-1334.
- Röth A, Barcellini W, D'Sa S, et al. Sustained inhibition of complement C1s with sutimlimab over 2 years in patients with cold agglutinin disease. Am J Hematol. 2023;98(8):1246-1253.
