Epysqli, Soliris biosimilar, found to reduce need for blood transfusions

Researchers analyze Phase 3 study of the PNH treatment, approved in the EU

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by Patricia Inacio PhD |

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Epysqli (SB12), a biosimilar of Soliris (eculizumab), is as effective as the reference therapy in reducing the need for blood transfusions in patients with paroxysmal nocturnal hemoglobinuria (PNH).

That’s according to a post-hoc analysis of the pivotal Phase 3 SB12 study (NCT04058158), whose findings supported Epysqli’s approval in the European Union (EU) following a positive recommendation from the EU’s Committee for Medicinal Products for Human Use (CHMP).

Findings were presented in a poster titled “Transfusion avoidance with Epysqli (SB12), a biosimilar to reference eculizumab: a post-hoc analysis from the pivotal phase III study” at the 29th European Hematology Association 2024 (EHA2024) Hybrid Congress, recently held in Spain and virtually.

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“This post-hoc analysis supports the previously demonstrated comparable clinical efficacy of SB12 with reference eculizumab in treating PNH patients, with no significant difference in reducing transfusion burden compared to reference eculizumab,” Hyejin Kim, vice president and medical and lifecycle safety team leader at Samsung Bioepis, said in a company press release.

Soliris is an antibody-based therapy that is approved in Europe for adults and children with PNH, regardless of a history of blood transfusions. Epysqli, a biosimilar of Soliris, was approved for a similar indication in Europe in 2023. A biosimilar is a biological product that’s highly similar to an already approved biological medicine and developed to be equivalent in terms of quality, safety, and effectiveness.

Eculizumab, the active ingredient in both Soliris and Epysqli, binds to C5, a protein that is part of the complement pathway, which is overly active in PNH. By binding to C5 and preventing its cleavage, eculizumab is designed to block complement activation, which is the driver of hemolysis, or red blood cell destruction, in PNH.

The study analysis

The Phase 3 SB12 study compared the safety, efficacy, and pharmacological properties of Epysqli with those of Soliris. A total of 50 patients, ages 18 and older, who had never been treated with a complement inhibitor enrolled in the study.

Patients were randomly assigned to receive Epysqli or Soliris, delivered as an into-the-vein infusion, at a dose of 600 mg every week for the first four weeks, followed by 900 mg for the fifth week, and then 900 mg every two weeks as a maintenance regimen. After 26 weeks (about six months), patients were switched to the opposing regimen until week 50.

Previous data from the trial showed that the levels of lactate dehydrogenase, a marker of cell and tissue damage often used to assess PNH disease activity, were similar with both treatments, suggesting that Epysqli was clinically equivalent to Soliris.

Data from a post-hoc analysis of the trial now showed that 68.1% of the patients treated with Epysqli did not require blood transfusions, a milestone achieved by 72.9% of those treated with Soliris. The 5.3% difference between the two treatment groups was not considered statistically significant, indicating Epysqli is therapeutically equivalent to Soliris in its ability to reduce hemolysis and the need for blood transfusions.

“Transfusion avoidance by [Epysqli] versus [Soliris] treatments showed comparable results, although the small sample size represents a limit to data interpretation,” the researchers wrote, adding that nevertheless, the findings “support the previously demonstrated clinical efficacy of EPYSQLI.”