Enjaymo

CARDINAL Study

The open-label, single-arm phase 3 CARDINAL study (ClinicalTrials Identifier: NCT03347396) evaluated the efficacy and safety of sutimlimab in 24 patients with primary CAD who had a recent blood transfusion. Patients received an intravenous infusion of sutimlimab through week 26.

The primary endpoint was the proportion of patients with a response, defined by an increase in hemoglobin (Hgb) of at least 2g/dL from baseline or reaching a Hgb level of at least 12g/dL at the 26-week treatment assessment timepoint, as well as the absence of blood transfusions from weeks 5 to 26 or any other CAD-related treatments.

Results showed that 54% (n=13) of patients achieved the composite endpoint. Sixty-three percent (n=15) of patients had an increase in Hgb of at least 2g/dL or reached an Hgb of at least 12g/dL, while 71% (n=17) did not receive RBC transfusion after week 5 and 92% (n=22) did not use other CAD-related treatments.

Treatment with sutimlimab was also associated with improvements in total bilirubin (mean reduction in bilirubin levels [n=14]: -2.23mg/dL [95% CI, -2.49, -1.98]) and lactate dehydrogenase (least squared mean change: -126 [95% CI, -218, -35]). At week 3, the mean increase in Hgb level was observed to be 2.29g/dL (SE: 0.308). At the 26-week treatment assessment timepoint, the mean increase in Hgb was 3.18g/dL (SE: 0.476).

CADENZA Study

The expanded approval was based on data from the CADENZA study (ClinicalTrials.gov Identifier: NCT03347422). Enjaymo had originally been approved based on data from Part A of the CARDINAL study.

The phase 3, double-blind, placebo-controlled CADENZA study included patients with CAD without a recent history of blood transfusion (N=42). Results showed a statistically significant treatment effect for Enjaymo over placebo. The responder rate difference between Enjaymo and placebo was reported to be 58.78% (95% CI, 34.6-82.96; P =.0004.)

A participant was considered a responder if they did not receive a blood transfusion from week 5 through week 26 and did not receive treatment for CAD beyond what was permitted per protocol. Additionally, hemoglobin level must have increased to at least 1.5g/dL from baseline at the treatment assessment timepoint (defined as average of values from the week 23, 25, and 26 visits).

A statistically significant improvement of symptoms and impact on fatigue, as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue, were also observed with Enjaymo vs placebo (LS mean difference of 8.93; 95% CI, 4.0-13.85; P <.001).

In both trials, the signs and symptoms of recurrent hemolysis were observed 9 weeks after the last dose of Enjaymo.