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IN THE BIOSIMILARITY STUDY

There were no clinically
meaningful differences between EPYSQLI® and Soliris®1

Study design1,2*

Graphic depicting the study design for the EPYSQLI biosimilarity study
  • Randomized, double-blind, multinational, crossover, phase 3 study evaluating the efficacy, pharmacokinetics, pharmacodynamics, immunogenicity, and safety of EPYSQLI and Soliris in adult patients with paroxysmal nocturnal hemoglobinuria (PNH)
  • At Week 26, patients who initially received Soliris were switched to EPYSQLI, and patients who initially received EPYSQLI were switched to Soliris
  • Patients who benefitted from study treatment could opt for extended, 2-year, open-label EPYSQLI treatment

Primary endpoint1

  • Reduction of hemolysis assessed by lactose dehydrogenase (LDH) levels at Week 26 and time-adjusted AUEC of LDH from Weeks
    14-26 and 40-52

Secondary endpoints1

  • Time-course of LDH
  • Number of transfused packed red blood cell units

Additional endpoints1

  • Comparisons of pharmacokinetics, pharmacodynamics, safety, and immunogenicity

AUEC=area under the effect curve; IV=intravenous.

EPYSQLI demonstrated highly similar improvements in lactose dehydrogenase
(LDH) levels to Soliris1

LDH is a sensitive biochemical marker of intravascular hemolysis, as LDH levels can increase far above normal during periods of severe hemolysis

  • Accordingly, LDH at a single time point was selected as a primary endpoint to detect clinically meaningful differences by directly measuring intravascular hemolysis
  • In addition, an integrated measure of LDH over time (time-adjusted AUEC of LDH) was defined as a coprimary endpoint to increase sensitivity for detecting potential differences between EPYSQLI and Soliris
  • LDH levels at Week 26 were equivalent between EPYSQLI and Soliris
    • Least squares mean (LSM)* of LDH at Week 26 was 284.20 U/l in patients treated with EPYSQLI and 249.72 U/l in patients treated with Soliris
    • The estimated LSM difference (34.48; 95% CI, -47.66‒116.62 U/l) was within the predefined equivalence margin,† confirming equivalent efficacy of EPYSQLI and Soliris
  • Geometric LSM‡ of time-adjusted AUEC of LDH was 279.65 with EPYSQLI and 258.73 U/l with Soliris
    • The estimated ratio of geometric LSM (1.08; 90% CI, 0.95‒1.23) was within the predefined equivalence margin,§ confirming equivalent efficacy of EPYSQLI and Soliris

*Estimated from a linear model with loge-transformed LDH at Week 26 as a dependent variable, and treatment and gender as fixed effects.

†The equivalence margin for the 2-sided 95% CI of the mean difference of LDH levels at Week 26 was -1.2x ULN to 1.2x ULN (-337.2 to 337.2 U/l).

‡Estimated from the linear mixed model with loge-transformed AUEC of LDH as a dependent variable, treatment, sequence, period, and gender as fixed effects and patient nested within sequence as a random effect.

§The equivalence margin of the 2-sided 90% CI of the mean ratio of time-adjusted AUEC of LDH was 0.77 to 1.29.



LDH profiles were comparable between treatment sequences throughout the study1

  • LDH levels decreased significantly after the first dose and remained within or just above the normal range during the entire study
    • Mean LDH stayed below 2x ULN of LDH from Week 3 onwards


LDH PROFILE OVER TIME

Chart depicting LDH levels over time from the EPYSQLI biosimilarity study

Reductions in transfused packed red blood cell (pRBC) units were consistent between both treatment sequences1

  • The number of transfused pRBC units decreased in both treatment sequences after the first dose
  • No statistically significant difference in number of transfused pRBC units between treatment groups within periods

TRANSFUSION RECORDS

​ EPYSQLI/
Soliris		 Soliris/
EPYSQLI		  
Study overall Total
Number of patients 24 25 49
Mean (SD) 2.9 (8.05) 2.7 (4.85) 2.8 (6.55)
Median (min-max) 0.0 (0-39) 0.0 (0-22) 0.0 (0-39)
P value|| 0.47  
Pretreatment
Number of patients 24 25 49
Mean (SD) 0.7 (1.08) 0.8 (1.37) 0.8 (1.23)
Median (min-max) 0.0 (0-4) 0.0 (0-5) 0.0 (0-5)
P value|| >0.9999  
Study period 1
(prior to crossover)
Number of patients 24 25 49
Mean (SD) 1.1 (3.72) 0.9 (2.06) 1.0 (2.96)
Median (min-max) 0.0 (0-18) 0.0 (0-8) 0.0 (0-18)
P value|| 0.46  
Study period 2
(after crossover)
Number of patients 23 23 46
Mean (SD) 1.1 (4.05) 1.0 (2.61) 1.1 (3.37)
Median (min-max) 0.0 (0-19) 0.0 (0-12) 0.0 (0-19)
P value|| 0.43  

||P values based on Wilcoxon rank-sum test for difference between treatment sequences EPYSQLI/Soliris and Soliris/EPYSQLI.

"Pretreatment” period refers to the period of time from date of informed consent to first study drug administration.

AUEC=area under the effect curve; CI=confidence interval; SD=standard deviation; ULN=upper limit of normal.


Pharmacokinetic and pharmacodynamic equivalence was demonstrated out to Week 521

MEAN SERUM ECULIZUMAB CONCENTRATION–TIME PROFILES

Chart showing similar mean eculizumab serum concentrations for EPYSQLI and Soliris through Week 52 of the biosimilarity study

MEAN TERMINAL COMPLEMENT ACTIVITY–TIME PROFILES

Chart showing similar mean terminal complement activity-time profiles for EPYSQLI and Soliris through Week 52 of the biosimilarity study

EPYSQLI and Soliris had no clinically meaningful differences in safety1

SAFETY PROFILES

​ EPYSQLI (n=47)
n (%)		 Soliris (n=47)
n (%)
Adverse events
Patients with TEAEs 34 (72) 32 (68)
Any TEAE with incidence >5% of patients 18 (38) 11 (23)
Diarrhea 4 (9) 2 (4)
Coronavirus infection 8 (17) 3 (6)
Alanine aminotransferase increased 3 (6) 2 (4)
Headache 2 (4) 3 (6)
Hemoglobinuria 8 (17) 2 (4)
Hypertension 3 (6) 0 (0)
Patients with AESI 0 (0) 4 (9)
Infusion-site hypersensitivity 0 (0) 1 (2)
Cellulitis 0 (0) 1 (2)
Dyspnea 0 (0) 1 (2)
Rash 0 (0) 1 (2)
Urticaria 0 (0) 1 (2)
Patients with serious TEAEs 3 (6) 2 (4)
Patients with TEAEs leading to IP discontinuation 0 (0) 1 (2)
Death 0 (0) 1 (2)
  • Most TEAEs were grade 1 (66 events in 3 patients) or grade 2 (140 events in 30 patients) and not related to the study drug (198 events in 31 patients)
  • There were a total of 6 serious TEAEs observed in 5 patients
    • None of the 3 serious TEAEs reported in the EPYSQLI treatment group was considered related to the study drug
    • Two of the 3 serious TEAEs reported in the Soliris treatment group were considered related to the study drug
    • All serious TEAEs were resolved except for 1 event (cellulitis) in the Soliris treatment group, which remained at the time of death
  • One patient in the Soliris treatment group had a fatal TEAE of portal vein thrombosis that was considered not related to the study drug

AESI=adverse event of special interest; IP=investigational product; TEAE=treatment-emergent adverse event.

​

REFERENCES

1. Jang JH, Gomez RD, Bumbea H, et al. A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria. EJHaem. 2022;4(1):26-36. 2. Jang JH, Gomez RD, Bumbea H, et al. A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria (supplement). EJHaem. 2022;4(1):26-36.

REFERENCE

1. Jang JH, Gomez RD, Bumbea H, et al. A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria. EJHaem. 2022;4(1):26-36.

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EPYSQLI® coverage is expanding

As of October 1, 2025, EPYSQLI is now covered on:

  • Aetna

  • Anthem Blue Cross Blue Shield

  • Blue Cross Blue Shield of Florida

  • Blue Cross Blue Shield of Kansas

  • Blue Cross Blue Shield of Massachusetts

  • Blue Cross Blue Shield of Michigan

  • Cigna

  • Centene

  • Excellus Blue Cross Blue Shield

  • HCSC Blue Cross Blue Shield

  • UnitedHealthcare

This is not a complete list. Coverage varies by insurer. Check your patient’s health plan.

ECU-40086  August 2025

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