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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

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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

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CADENZA Study Design
Rapid and Sustained Efficacy
Long-term Efficacy
Safety

ENJAYMO is a breakthrough in the treatment of Cold Agglutinin Disease (CAD)¹

ENJAYMO is indicated for the treatment of hemolysis in adults with CAD.

CADENZA, the first placebo-controlled trial in CAD, demonstrated a significant benefit for patients treated with ENJAYMO^1,2

CADENZA, a phase 3, placebo-controlled, global, multicenter, randomized, double-blind trial, demonstrated efficacy and safety of ENJAYMO in 42 patients with CAD and no transfusion history.^1-3*

Download the CADENZA Blood publication

Study design of CADENZA

CADENZA, a phase 3, placebo-controlled, global, multicenter, randomized, double-blind trial, demonstrated efficacy and safety of ENJAYMO in 42 patients with CAD and no transfusion history*

ENDPOINTS STUDIED

COMPOSITE PRIMARY ENDPOINT

≥1.5 g/dL Hb increase from baseline at TAT^†
Transfusion independence from Weeks 5 to 26
No use of protocol-prohibited CAD treatment from Weeks 5 to 26

KEY 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 CAD and no history of transfusion within 6 months or >1 in 12 months prior to enrollment (N=42). 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.
^†In CADENZA Part A, 3 patients discontinued treatment in the ENJAYMO arm and did not complete the trial. All 39 patients who completed Part A continued to Part B.
^‡Treatment assessment time point (TAT) was defined as the mean value from Weeks 23, 25, and 26.
FACIT-Fatigue is a patient-reported outcome instrument with scores ranging from 0 to 52, with higher scores indicating less fatigue.

Baseline characteristics of patients included in CADENZA¹

Parameter	Statistic	Placebo n=20	ENJAYMO n=22
Age	Mean (min, max)	68.2 (51, 83)	65.3 (46, 88)
Sex Male Female	n (%)	4 (20.0) 16 (80.0)	5 (22.7) 17 (77.3)
Body weight	Mean, kg (min, max)	64.9 (48, 95)	66.8 (39, 100)
Hemoglobin	Mean, g/dL	9.33	9.15
Bilirubin (total)^§	Mean, mg/dL	2.09 (1.75 × ULN)	2.41 (2 × ULN)
LDH	U/L	380.8	421.5
History of transfusion Within last 6 months Within last 12 months	Median (range)	0 0	0 0.14 (0, 1)
FACIT-Fatigue scale^‖	Mean	32.99	31.67

^§Bilirubin data excluding patients with either a positive or no available test for Gilbert’s syndrome (n=18 for placebo, n=20 for ENJAYMO).
^‖Fatigue is measured on a scale of 0 (worst fatigue) to 52 (no fatigue).

ENJAYMO is the first and only treatment for CAD, with a proven and significant benefit vs placebo across key efficacy measures^1,2

The majority of patients achieved an improvement in hemoglobin and transfusion independence with ENJAYMO and required no additional CAD treatment (16/22)

73%

(16/22)

achieved all 3
composite endpoint measures^¶

Significant hemoglobin increase
Transfusion independence
No additional treatment used^#

vs 15% (3/20) with placebo

(Responder rate difference: 58.8%,
95% CI: 34.6%-83.0%, P=0.0004)

73%

(16/22)

ACHIEVED A CLINICALLY MEANINGFUL Hb INCREASE of ≥1.5 g/dL at TAT from baseline (9.15 g/dL) vs 15% with placebo (3/20)

82%

(18/22)

MAINTAINED TRANSFUSION INDEPENDENCE
from baseline through Weeks 5 to 26 vs 80% with placebo (16/20)

86%

(19/22)

RECEIVED NO ADDITIONAL TREATMENT
from Weeks 5 to 26 vs 100% with placebo (20/20)^#

Treatment assessment time point (TAT) was defined as the mean value from Weeks 23, 25, and 26.

^¶Two patients discontinued prior to Week 23, and their status was considered unknown for the purposes of this analysis.

^#Prohibited therapies included rituximab alone or in combination with cytotoxic agents.

Controlling complement-mediated hemolysis in CAD starts with the first dose of ENJAYMO^1,2

Mean Hb and bilirubin levels through Week 26 with ENJAYMO vs placebo (N=42)**

Bilirubin

Data at TAT (mean values from Weeks 23, 25, and 26) were tested for significance. Between baseline and Week 26, data at each time point were the observed mean. Interpret these data with discretion given small sample.

Between baseline and Week 26, data at each time point were the observed mean. Interpret these data with discretion given the small sample and descriptive statistics.

^**Mean baseline values: Hb was 9.15 g/dL for ENJAYMO and 9.33 g/dL for placebo; bilirubin was 2.41 mg/dL for ENJAYMO and 2.09 mg/dL for placebo. Improvement at TAT: LS mean change in Hb was 2.66 g/dL for ENJAYMO and 0.09 g/dL for placebo; mean change in bilirubin was –1.29 mg/dL for ENJAYMO and –0.11 mg/dL for placebo.

Patients experienced significant improvement in the symptoms and impact of fatigue at TAT^1,2

Mean FACIT-Fatigue scores through Week 26 with ENJAYMO vs placebo (N=42)^††

Data at TAT (mean values from Weeks 23, 25, and 26) were tested for significance. Between baseline and Week 26, data at each time point were the observed mean. Interpret these data with discretion given small sample.

^††Mean baseline score for FACIT-Fatigue was 31.67 for ENJAYMO and 32.99 for placebo. LS mean improvement at TAT was 10.83 points for ENJAYMO and 1.91 points for placebo.

Patients experienced significant and sustained improvements in anemia and fatigue at TAT

ENJAYMO had sustained treatment effect in CAD with long-term use over 1.5 years^1,2

	BASELINE	MONTH 6 (TAT)	YEAR 1.5 (LAST VALUE AT TRIAL END)
ANEMIA IMPROVED	9.15 g/dL mean Hb¹	11.87 g/dL mean Hb (range 8.3-13.9)²	11.58 g/dL mean Hb (range 6.9-15.3)¹
CHRONIC HEMOLYSIS INHIBITED	2.41 mg/dL mean bilirubin¹	0.71 mg/dL mean bilirubin (range: 0.41-1.48)²	1.01 mg/dL mean bilirubin (range: 0.29-5.54)¹

	ANEMIA IMPROVED	CHRONIC HEMOLYSIS INHIBITED
BASELINE	9.15 g/dL mean Hb¹	2.41 mg/dL mean bilirubin¹
MONTH 6 (TAT)	11.87 g/dL mean Hb (range 8.3-13.9)²	0.71 mg/dL mean bilirubin (range: 0.41-1.48)²
YEAR 1.5 (LAST VALUE AT TRIAL END)	11.58 g/dL mean Hb (range 6.9-15.3)¹	1.01 mg/dL mean bilirubin (range: 0.29-5.54)¹

FACIT-Fatigue scores over time in CADENZA (mean)

Study Limitation: Patient-reported fatigue after TAT may be an under- or overestimation due to the open-label design and lack of a comparator arm during the extension period (6 months-1.5 years). Interpret these data with discretion given the small sample size and descriptive statistics.

Baseline: 31.67 | Month 6 (TAT): 43.15 | Year 1.5: 43.25

^‡‡LS mean improvement in FACIT-Fatigue Scores vs placebo at TAT was significant (treatment effect: 8.93, 95% CI: 4.0-13.9, P<0.001), with scores nearing the general population (43.6 mean).^1,4

A well-tolerated safety profile studied over 2.5 years^1,2

ENJAYMO safety was evaluated in CADENZA, a 6-month placebo-controlled study (Part A [n=42]), followed by a 1 year open-label, single-arm study (Part B [n=39]) and CARDINAL (an open-label, single-arm study [n=24])

Adverse reactions (≥10%) in patients receiving ENJAYMO with a difference >5% vs placebo (CADENZA Part A)

Adverse reactions	ENJAYMO (n=22)	Placebo (n=20)
Headache Hypertension Rhinitis Acrocyanosis Raynaud's phenomenon	5 (23%) 5 (23%) 4 (18%) 4 (18%) 4 (18%)	2 (10%) 0 0 0 0

No meningococcal infections were reported with ENJAYMO

ENJAYMO may increase susceptibility to serious infections, including infections caused by encapsulated bacteria such as Neisseria meningitidis (any serogroup), Streptococcus pneumoniae, and Haemophilus influenzae

Serious adverse reactions were reported in 2 patients

Serious adverse reactions were Raynaud's phenomenon (n=1) and febrile infection (n=1)

Only 2 patients discontinued due to adverse reaction

Adverse reactions leading to discontinuation were Raynaud's phenomenon (n=1), acrocyanosis (n=1), and infusion-related reactions (n=1)

Most common adverse reactions

The most common adverse reactions (≥18%) reported were rhinitis, headache, hypertension, acrocyanosis, and Raynaud’s phenomenon

Watch videos of real patients and their providers talking about results with ENJAYMO.

Prescribe ENJAYMO for your next appropriate patient with CAD to inhibit complement-mediated hemolysis

Patient portrayal.

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.

Please see full Prescribing Information.

See the full Medication Guide.

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CAD=Cold Agglutinin Disease; CAS=cold agglutinin syndrome; FACIT=Functional Assessment of Chronic Illness Therapy; Hb=hemoglobin; LDH=lactate dehydrogenase; LS=least squares; SLE=systemic lupus erythematosus; TAT=treatment assessment time point; ULN=upper limit of normal.

References:

ENJAYMO. Prescribing information. Genzyme Corporation.
Röth A, Berentsen S, Barcellini W, et al. Sutimlimab in patients with cold agglutinin disease: results of the randomized placebo-controlled phase 3 CADENZA trial. Blood. 2022;140(9):980-991. doi.org/10.1182/blood.2021014955
Data on file. Sanofi.
Cella D, Lai J-S, Chang C-H, Peterman A, Slavin M. Fatigue in cancer patients compared with fatigue in the general United States population. Cancer. 2002;94(2):528-538. doi:10.1002/cncr.10245

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