This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Start Date01 Mar 2025

Sponsor / Collaborator

NovelMed Therapeutics, Inc.

NCT05726916 / RecruitingPhase 3

Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic Syndrome: a Randomized Multicenter Controlled Trial

Hemolytic and uremic syndrome (HUS) is a clinic-biological syndrome related to thrombotic microangiopathy affecting predominantly the kidney. Atypical HUS (aHUS) has been historically defined as HUS occurring in the absence of infectious event. The role of complement dysregulation in aHUS pathophysiology has been largely demonstrated, since C genetic rare variants are present in 60-70% aHUS patients. In line with the frequency of C dysregulation in aHUS, Eculizumab, an anti-C5 monoclonal antibody, has dramatically improved aHUS patients prognosis.
Numerous conditions have been associated with aHUS, including hypertensive emergency (HE), a syndrome of acute blood pressure flare associated with end-organ damage. In cases of HE-aHUS, whether primary aHUS is complicated by secondary HE, or primary HE leads to secondary aHUS is still debated.
The investigators recently demonstrated that C genetic variants frequency was similar in patients with HE-aHUS and patients with aHUS without HE, suggesting a major role for C dysregulation in HE-aHUS. Consequently, the investigators propose to evaluate, in HE-aHUS patients, the benefit of a strategy with early Eculizumab therapy (used within its marketing authorization and its conditions of refunding by the health insurance in usual care), compared to standard of care including tight blood pressure control.
The hypothesis suggests that C dysregulation may impact renal prognosis of HE-aHUS patients. The investigator's aim to demonstrate that early Eculizumab therapy improves prognosis of HE-aHUS patients.
Method
The HYPERSHU study is a randomized, controlled, open-labelled study including HE-aHUS patients with severe AKI and no evidence of other conditions associated with HUS (infections, autoimmunity, drugs, pregnancy). The investigators plan to include 62 patients. Patients will be randomized in 2 arms:
Early Eculizumab therapy (for 3 months) added to standard of care (tight blood pressure control).
Standard of care alone with tight blood pressure control. Renal function after 6 months is the primary evaluation criterium.
HE is a frequently associated with aHUS, and strongly impacts patient renal prognosis. Efficient therapeutic strategies are still lacking for this condition. The HYPERSHU study will allow to evaluate the benefit of early Eculizumab therapy in patients with HE-aHUS and severe renal dysfunction.

Start Date09 Nov 2023

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT05876351 / RecruitingPhase 3

Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China

Start Date14 Jul 2023

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

[+1]

100 Clinical Results associated with Eculizumab biosimilar(Samsung Bioepis)

100 Translational Medicine associated with Eculizumab biosimilar(Samsung Bioepis)

100 Patents (Medical) associated with Eculizumab biosimilar(Samsung Bioepis)

Literatures (Medical) associated with Eculizumab biosimilar(Samsung Bioepis)

01 Dec 2023·Drugs in R&D

In-Use Stability of SB12 (Eculizumab, Soliris Biosimilar) Diluted in Saline and Dextrose Infusion Solution after an Extended Storage Period

Article

Author: Lee, Yoonsook ; Kim, Soyeon ; Tak, Minji ; Kim, Jihyun ; Jeong, Hawon ; Park, Su Jin ; Yun, Jihoon

INTRODUCTION:

SB12 is a biosimilar to eculizumab reference product [Soliris^TM (Soliris is a trademark of Alexion Pharmaceuticals, Inc.)] that acts as a C5 complement protein inhibitor. The infusion stability of in-use (diluted) SB12 outside the conditions stated in the reference product's label is unknown.

OBJECTIVE:

The objective of this study was to assess the stability of SB12 after extended storage in conditions not claimed in the originator label.

METHODS:

Infusion stability was assessed in SB12 samples (diluted in 0.9% NaCl, 0.45% NaCl, and 5% dextrose, final concentration of 5 mg/mL per clinical trial protocol and the reference product's label) kept at 5 ± 3 °C for up to 3 months, then 30 ± 2 °C/65 ± 5% relative humidity (RH) for 72 h. The product was stored in different containers [polyolefin (PO) bags, glass bottles and syringes], and the protocol followed International Conference on Harmonisation (ICH) and European Medicines Agency (EMA) requirements for stability evaluation of biological products. Stability was evaluated using complementary assays, including pH, protein concentration (A₂₈₀), purity (size exclusion-high-performance liquid chromatography, capillary electrophoresis-sodium dodecyl sulfate, and imaged capillary isoelectric focusing), biological activity (C5 binding and inhibition), and safety (subvisible particles).

RESULTS:

Except for charge variants in SB12 diluted in 5% dextrose, all results met the stability acceptance criteria. There were no major changes in terms of physicochemical stability, biological activity, and subvisible particles.

CONCLUSIONS:

The infusion stability of SB12 after extended storage (5 ± 3 °C for up to 3 months, then 30 ± 2 °C/65 ± 5% RH for 72 h) was demonstrated for longer periods and at higher temperatures than what is stated in the EU and US labels of the reference product. The physicochemical properties, biological activity, and subvisible particles of in-use SB12 diluted in 0.9% NaCl and 0.45% NaCl were maintained under the described conditions and for all tested containers. However, instability was observed for the diluted SB12 in 5% dextrose. These results may reduce the workload of clinical staff and minimize drug waste from treatment delays without any loss in product quality and biological activity.

12 Aug 2023·Molecules (Basel, Switzerland)

Scaffold Morphing and In Silico Design of Potential BACE-1 (β-Secretase) Inhibitors: A Hope for a Newer Dawn in Anti-Alzheimer Therapeutics.

Article

Author: Singh, Thakur Gurjeet ; Sharma, Pratibha ; Singh, Tanveer ; Singh, Varinder ; Mishra, Krishna Kumar ; Mujwar, Somdutt ; Bhatia, Shiveena ; Singh, Manjinder ; Ahmad, Sheikh Fayaz

Alzheimer's disease (AD) is the prime cause of 65-80% of dementia cases and is caused by plaque and tangle deposition in the brain neurons leading to brain cell degeneration. β-secretase (BACE-1) is a key enzyme responsible for depositing extracellular plaques made of β-amyloid protein. Therefore, efforts are being applied to develop novel BACE-1 enzyme inhibitors to halt plaque build-up. In our study, we analyzed some Elenbecestat analogues (a BACE-1 inhibitor currently in clinical trials) using a structure-based drug design and scaffold morphing approach to achieve a superior therapeutic profile, followed by in silico studies, including molecular docking and pharmacokinetics methodologies. Among all the designed compounds, SB306 and SB12 showed good interactions with the catalytic dyad motifs (Asp228 and Asp32) of the BACE-1 enzyme with drug-likeliness properties and a high degree of thermodynamic stability confirmed by the molecular dynamic and stability of the simulated system indicating the inhibitory nature of the SB306 and SB12 on BACE 1.

01 Jul 2023·BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy

Characterization for the Similarity Assessment between Proposed Biosimilar SB12 and Eculizumab Reference Product Using a State-of-the-Art Analytical Method.

Article

Author: Kim, Yikwon ; Lee, Jungmin ; Cho, Miju ; Yoo, Taekyung ; Hong, Eunkyoung ; Kim, Hyunsoo ; Kim, Jihye ; Hong, Seokku

BACKGROUND:

SB12 is being developed as a proposed biosimilar to eculizumab reference product (RP), a humanized monoclonal antibody (IgG2/4 kappa immunoglobulin) that binds to the human C5 complement protein. Binding to this protein inhibits complement-mediated intravascular hemolysis by blocking its cleavage into C5a and C5b. Eculizumab RP is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive, and neuromyelitis optica spectrum disorder in adult patients who are anti-aquaporin-4 antibody-positive.

OBJECTIVE:

The objective of this study was to demonstrate structural, physicochemical, and biological similarity between eculizumab RP and SB12 using various state-of-the-art analytical methods.

METHODS:

Comprehensive analytical characterization was conducted with side-by-side comparison of SB12 with European Union (EU) and United States (US) eculizumab RPs using various analytical methods (more than 40 state-of-the-art assays). Comparisons included purity, product-related impurity, charge heterogeneity, primary structure, post-translational modification, higher-order structure, quantity, Fab-related biological activities (potency and binding activity), and Fc-related biological activities.

RESULTS:

Based on the analytical similarity assessment, the structural, physicochemical, and biological characterization results demonstrated that SB12 is highly similar to the EU and US eculizumab RP. In the structural aspects, it was confirmed that there is no difference between post-translational modification profiles and higher-order structures of SB12 compared with the eculizumab RP. Product-related impurities in the form of aggregates and charge variants were also confirmed to be similar. Mechanism of action (MoA)-related biological activities showed that SB12 is highly similar to the EU and US eculizumab RP with respect to overall critical and non-critical quality attributes analyzed. Moreover, similarity of comparative binding tendency of SB12 and eculizumab RP to Fc gamma receptors and C1q was confirmed through additional characterization methods. Based on these results, SB12 is expected to have highly similar safety and efficacy compared with eculizumab RP.

CONCLUSION:

In summary, the overall analytical characterization and similarity assessment results show that SB12 is highly similar to the EU and US eculizumab RP in terms of structural, physicochemical, biophysical, and biological attributes.

News (Medical) associated with Eculizumab biosimilar(Samsung Bioepis)

14 Jun 2024

·www.globenewswire.com

Samsung Bioepis Presents Post-hoc Analysis of Phase 3 Study for EPYSQLI™ (SB12; Eculizumab Biosimilar), at the European Hematology Association (EHA) Congress 2024

Transfusion avoidance results were comparable between SB12 and reference eculizumab-treated groups, supporting clinical efficacy between SB12 and reference eculizumab INCHEON, Korea, June 14, 2024 (GLOBE NEWSWIRE) -- Samsung Bioepis Co., Ltd. today presented the post-hoc analysis of the Phase 3 clinical study results for EPYSQLI™ (SB12), a biosimilar to Soliris1 (eculizumab), at the 29th European Hematology Association 2024 (EHA2024) Hybrid Congress held in Madrid, Spain and virtually, from June 13 to 16, 2024. The post-hoc analysis of SB12 pivotal Phase 3 study compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or reference eculizumab (ECU), respectively. Eculizumab is known to significantly reduce hemolysis resulting in improvements in anemia as indicated by increased hemoglobin stabilization and reduced need for red blood cell (RBC) transfusions.2 For this study, 50 adults with PNH who were complement-inhibitor naïve were randomized (1:1) to treatment sequence I (TS1: SB12 to ECU, n=25) or II (TS2: ECU to SB12, n=25), to receive 600 mg of SB12 or ECU intravenously every week for first 4 weeks and 900 mg for the fifth week, followed by 900 mg every 2 weeks thereafter. At Week 26, patients were switched to ECU or SB12, respectively, and received infusion until Week 50 (i.e., Period 1 followed by Period 2 from Week 26). This efficacy analysis was conducted in pooled patients treated with SB12 or ECU in either Periods 1 or 2. Transfusion avoidance was 68.1% for patients who received SB12 versus 72.9% for patients who received reference eculizumab, with a difference of -5.3% [95% confidence interval (CI), (-18.9%, 8.4%), p-value 0.4492], resulting in non-significant difference between the two treatment groups. “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,” said Hyejin Kim, Vice President and Medical & Lifecycle Safety Team Leader at Samsung Bioepis. “Our dedication to make a meaningful difference to our patients remain steadfast. We will continue to work collaboratively with clinicians and physicians to further expand the treatment options for patients with orphan diseases.” Details of the SB12 abstract are as follows: Abstract title: Transfusion Avoidance with EPYSQLI™(SB12), A Biosimilar to Reference Eculizumab: A Post-hoc Analysis from the Pivotal Phase 3 StudyAbstract number: P1916Program type: e-Poster PresentationAuthors: Jun Ho Jang, Jihye Park, Younsoo Kim, Jihyun Han and Paola Russo In May 2023, the European Commission (EC) approved SB12 as a biosimilar to Soliris (eculizumab) under the brand name EPYSQLI for the treatment of adults and children with PNH. In March 2024, EPYSQLI was granted an indication expansion for the treatment of adults and children with atypical haemolytic uremic syndrome (aHUS).3 About EPYSQLI (Eculizumab Biosimilar) in the European Union (EU) EPYSQLI is indicated in adults and children for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). EPYSQLI is not approved for and should not be used for the treatment of generalised myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). EPYSQLI must be administered by a healthcare professional and under the supervision of a physician experienced in the management of patients with hematological disorders or renal disorders. EPYSQLI EU Important Safety Information The EU Summary of Product Characteristics for EPYSQLI includes the following Special warning and Precautions: Meningococcal Infection EPYSQLI increases patient's susceptibility to Meningococcal infection (Neisseria meningitidis). Meningococcal disease due to any serogroup may occur. To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving eculizumab unless the risk of delaying eculizumab therapy outweighs the risks of developing a meningococcal infection. Patients who initiate eculizumab treatment less than 2 weeks after receiving a tetravalent meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Vaccines against serogroups A, C, Y and W 135 are recommended in preventing the commonly pathogenic meningococcal serogroups. Vaccines against serogroup B where available are also recommended. Patients must receive vaccination according to current national vaccination guidelines for vaccination use. Vaccination may further activate complement. As a result, patients with complement-mediated diseases, including PNH and aHUS, may experience increased signs and symptoms of their underlying disease, such as haemolysis (PNH) or TMA (aHUS). Therefore, patients should be closely monitored for disease symptoms after recommended vaccination. Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official local guidance on the appropriate use of antibacterial agents. Cases of serious or fatal meningococcal infections have been reported in eculizumab-treated patients. All patients should be monitored for early signs of meningococcal infection, evaluated immediately if infection is suspected, and treated with appropriate antibiotics if necessary. Patients should be informed of warning signs and symptoms and steps taken to seek medical care immediately. Other Systemic Infections Patients may have increased susceptibility to other type of serious infections, especially with Neisseria and encapsulated bacteria. Infusion Reactions Administration of eculizumab may result in infusion reactions or immunogenicity that could cause allergic or hypersensitivity reactions (including anaphylaxis). Eculizumab administration should be interrupted in all patients experiencing severe infusion reactions and appropriate medical therapy administered. Anticoagulant therapy Treatment with EPYSQLI should not alter anticoagulant management. PNH Laboratory Monitoring PNH patients should be monitored for signs and symptoms of intravascular haemolysis, including serum lactate dehydrogenase (LDH) levels, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days). aHUS Laboratory Monitoring aHUS patients receiving eculizumab therapy should be monitored for thrombotic microangiopathy by measuring platelet counts, serum LDH and serum creatinine, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days). Treatment Discontinuation for PNH If patients discontinue treatment with eculizumab they should be closely monitored for signs and symptoms of serious intravascular haemolysis. Treatment Discontinuation for aHUS If aHUS patients discontinue treatment with eculizumab, they should be monitored closely for signs and symptoms of severe thrombotic microangiopathy complications. The most common adverse reaction observed with eculizumab treatment in clinical trials was found to be headache, whereas the most serious adverse reaction was found to be meningococcal infection. Refer to the Summary of Product Characteristics for EPYSQLI’s full safety information. About Samsung Bioepis Co., Ltd. Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world's leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, endocrinology, hematology and nephrology. For more information, please visit: www.samsungbioepis.com and follow us on social media – follow us on social media – X, LinkedIn. MEDIA CONTACT Yoon Kim, yoon1.kim@samsung.com References: 1 Soliris is a trademark of Alexion Pharmaceuticals 2 European Medicines Agency. Soliris Product Information. https://www.ema.europa.eu/en/documents/product-information/soliris-epar-product-information_en.pdf. Accessed May 2024 3 European Medicines Agency. Epysqli Product Information. https://www.ema.europa.eu/en/documents/product-information/epysqli-epar-product-information_en.pdf. Accessed May 2024

Phase 3Clinical ResultDrug Approval

30 May 2024

·www.globenewswire.com

Samsung Bioepis to Present Post-hoc Analysis of Phase 3 Study for EPYSQLI™ (SB12; Eculizumab Biosimilar), at the European Hematology Association (EHA) Congress 2024

Post-hoc analysis of Phase 3 study to highlight transfusion avoidance results of SB12 compared to reference eculizumab-treated groups INCHEON, Korea, May 30, 2024 (GLOBE NEWSWIRE) -- Samsung Bioepis Co., Ltd. today announced that a post-hoc analysis of the Phase 3 clinical study results for EPYSQLI™ (SB12), a biosimilar to Soliris1 (eculizumab), will be presented at the 29th European Hematology Association 2024 (EHA2024) Hybrid Congress held in Madrid, Spain and virtually, from June 13 to 16, 2024. SB12 has shown equivalent efficacy with reference eculizumab (ECU) by lactate dehydrogenase (LDH) and comparable safety, pharmacokinetics, pharmacodynamics, and immunogenicity profiles in paroxysmal nocturnal hemoglobinuria (PNH) through the pivotal Phase 3 study.2 The post-hoc analysis of SB12 pivotal Phase 3 study presented at EHA2024 compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or ECU, respectively. Eculizumab is known to significantly reduce hemolysis resulting in improvements in anemia as indicated by increased hemoglobin stabilization and reduced need for red blood cell (RBC) transfusions.3 “We are excited to present this data at EHA2024, that adds to the growing body of evidence that supports comparable clinical efficacy of SB12 with reference eculizumab in treating PNH patients,” said Hyejin Kim, Vice President and Medical & Lifecycle Safety Team Leader at Samsung Bioepis. “We will continue to advance with our scientific research, publication and educational activities to bring more awareness of biosimilars for rare disease treatment and hope our study results serve as a guidance for clinicians.” Details of the SB12 abstract are as follows: Abstract title: Transfusion Avoidance with EPYSQLI™(SB12), A Biosimilar to Reference Eculizumab: A Post-hoc Analysis from the Pivotal Phase 3 StudyAbstract number: P1916Program type: e-Poster PresentationAuthors: Jun Ho Jang, Jihye Park, Younsoo Kim, Jihyun Han and Paola Russo In May 2023, European Commission (EC) approved SB12 as a biosimilar to Soliris (eculizumab) under the brand name EPYSQLI for the treatment of adults and children with PNH. In March 2024, EPYSQLI was granted an indication expansion for the treatment of adults and children with atypical haemolytic uremic syndrome (aHUS).4 About EPYSQLI (Eculizumab Biosimilar) in the European Union (EU) EPYSQLI is indicated in adults and children for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). EPYSQLI must be administered by a healthcare professional and under the supervision of a physician experienced in the management of patients with hematological disorders or renal disorders. EPYSQLI EU Important Safety Information The EU Summary of Product Characteristics for EPYSQLI includes the following Special warning and Precautions: Meningococcal Infection EPYSQLI increases patient's susceptibility to Meningococcal infection (Neisseria meningitidis). Meningococcal disease due to any serogroup may occur. To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving eculizumab unless the risk of delaying eculizumab therapy outweighs the risks of developing a meningococcal infection. Patients who initiate eculizumab treatment less than 2 weeks after receiving a tetravalent meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Vaccines against serogroups A, C, Y and W 135 are recommended in preventing the commonly pathogenic meningococcal serogroups. Vaccines against serogroup B where available are also recommended. Patients must receive vaccination according to current national vaccination guidelines for vaccination use. Vaccination may further activate complement. As a result, patients with complement-mediated diseases, including PNH and aHUS, may experience increased signs and symptoms of their underlying disease, such as haemolysis (PNH) or TMA (aHUS). Therefore, patients should be closely monitored for disease symptoms after recommended vaccination. Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official local guidance on the appropriate use of antibacterial agents. Cases of serious or fatal meningococcal infections have been reported in eculizumab-treated patients. All patients should be monitored for early signs of meningococcal infection, evaluated immediately if infection is suspected, and treated with appropriate antibiotics if necessary. Patients should be informed of warning signs and symptoms and steps taken to seek medical care immediately. Other Systemic Infections Patients may have increased susceptibility to other type of serious infections, especially with Neisseria and encapsulated bacteria. Infusion Reactions Administration of eculizumab may result in infusion reactions or immunogenicity that could cause allergic or hypersensitivity reactions (including anaphylaxis). Eculizumab administration should be interrupted in all patients experiencing severe infusion reactions and appropriate medical therapy administered. Anticoagulant therapy Treatment with EPYSQLI should not alter anticoagulant management. PNH Laboratory Monitoring PNH patients should be monitored for signs and symptoms of intravascular haemolysis, including serum lactate dehydrogenase (LDH) levels, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days). aHUS Laboratory Monitoring aHUS patients receiving eculizumab therapy should be monitored for thrombotic microangiopathy by measuring platelet counts, serum LDH and serum creatinine, and may require dose adjustment within the recommended 14±2 day dosing schedule during the maintenance phase (up to every 12 days). Treatment Discontinuation for PNH If patients discontinue treatment with eculizumab they should be closely monitored for signs and symptoms of serious intravascular haemolysis. Treatment Discontinuation for aHUS If aHUS patients discontinue treatment with eculizumab, they should be monitored closely for signs and symptoms of severe thrombotic microangiopathy complications. The most common adverse reaction observed with eculizumab treatment in clinical trials was found to be headache, whereas the most serious adverse reaction was found to be meningococcal infection. Refer to the Summary of Product Characteristics (SmPC) for EPYSQLI’s full safety information. About Samsung Bioepis Co., Ltd. Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world's leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, endocrinology, hematology and nephrology. For more information, please visit: www.samsungbioepis.com and follow us on social media – follow us on social media – X, LinkedIn. References: _____________________1 Soliris is a trademark of Alexion Pharmaceuticals 2 Jang JH, Gomez RD, Bumbea H, Nogaieva L, Wong LLL, Lim SM, Kim Y, Park J. 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 Dec 20;4(1):26-36. doi: 10.1002/jha2.632. PMID: 36819188; PMCID: PMC9928655. 3 European Medicines Agency. Soliris Product Information. https://www.ema.europa.eu/en/documents/product-information/soliris-epar-product-information_en.pdf. Accessed May 2024 4 European Medicines Agency. Epysqli Product Information. https://www.ema.europa.eu/en/documents/product-information/epysqli-epar-product-information_en.pdf. Accessed May 2024

Phase 3Drug ApprovalClinical ResultVaccine

08 Jun 2023

·pipelinereview.com

Samsung Bioepis Presents Population Pharmacokinetic (PK), Pharmacodynamic (PD), Efficacy Modeling of SB12 (Eculizumab), A Biosimilar to Soliris, at the European Hematology Association (EHA) Congress

• PK, PD, and efficacy similarities between SB12 and compared to reference eculizumab pooled population of healthy subjects and paroxysmal nocturnal hemoglobinuria (PNH) patients • Approved by European Commission (EC) in late May as a biosimilar to Soliris 1 (eculizumab) under the brand name EPYSQLI™ • Abstract available at EHA2023 website INCHEON, Korea I June 8 2023 I Samsung Bioepis Co., Ltd. today announced results from the population PK/PD/efficacy modeling of SB12, a biosimilar to Soliris (eculizumab) 2 , and reference eculizumab at the 28th European Hematology Association 2023 (EHA2023) Hybrid Congress held in Frankfurt, Germany and virtually, from June 8 to 11, 2023. The modeling analysis showed similarities of PK, PD, and efficacy profiles between SB12 and reference eculizumab in pooled population of healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH). SB12 was granted marketing authorization by the European Commission (EC) on May 26, 2023 under the brand name EPYSQLI™, as a biosimilar to Soliris for treatment of adult and children patients with PNH. 3 The purpose of the modeling was to describe and compare PK, PD, and efficacy of SB12 and reference eculizumab, and find clinically significant covariate relationships. The PK, PD and efficacy data of SB12 and reference eculizumab were obtained from Phase 1 study and Phase 3 study. The population modeling showed similarities in PK, PD and efficacy between SB12 and reference eculizumab in pooled population of healthy subjects and PNH patients, supporting the totality of evidence on biosimilarity for SB12. Details of the SB12 abstract are as follows: • Abstract title: Population Pharmacokinetic/Pharmacodynamic/Efficacy Modeling of SB12 (proposed eculizumab biosimilar) and Reference Eculizumab • Abstract number: PB2051 • Program type: Publication Only • Authors: Hyuna Lee, et al. About EPYSQLI™ (eculizumab biosimilar) in the EU EPYSQLI™ is indicated in adults and children for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with hemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history. EPYSQLI must be administered by a healthcare professional and under the supervision of a physician experienced in the management of patients with hematological disorders. EPYSQLI EU Important Safety Information The EU Summary of Product Characteristics for EPYSQLI includes the following Special warning and Precautions: EPYSQLI increases patient's susceptibility to Meningococcal infection (Neisseria meningitidis). Meningococcal disease due to any serogroup may occur. To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving eculizumab unless the risk of delaying eculizumab therapy outweighs the risks of developing a meningococcal infection. Patients who initiate eculizumab treatment less than 2 weeks after receiving a tetravalent meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Vaccines against serogroups A, C, Y, W 135 and B where available, are recommended in preventing the commonly pathogenic meningococcal serogroups. Patients must receive vaccination according to current national vaccination guidelines for vaccination use. Vaccination may not be sufficient to prevent meningococcal infection. Consideration should be given to official guidance on the appropriate use of antibacterial agents. Cases of serious or fatal meningococcal infections have been reported in eculizumab-treated patients. All patients should be monitored for early signs of meningococcal infection, evaluated immediately if infection is suspected, and treated with appropriate antibiotics if necessary. Patients should be informed of these signs and symptoms and steps taken to seek medical care immediately. Patients may have increased susceptibility to other type of serious infections, especially with Neisseria and encapsulated bacteria. Treatment with EPYSQLI should not alter anticoagulant management. Administration of EPYSQLI may result in infusion reactions or immunogenicity that could cause allergic or hypersensitivity reactions (including anaphylaxis). The most common adverse reaction observed with eculizumab treatment in clinical trials was headache, and the most serious adverse reaction was meningococcal sepsis. About Samsung Bioepis Co., Ltd. Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world's leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, hematology, endocrinology and gastroenterology. For more information, please visit: www.samsungbioepis.com and follow us on social media – Twitter , LinkedIn . References: i Soliris is a trademark of Alexion Pharmaceuticals, Inc. ii European Medicines Agency. Soliris Product Information. https:// www.ema.europa.eu/en/documents/product-information/soliris-epar-product-information_en.pdf iii European Medicines Agency. EPYSQLI Product Information. https:// www.ema.europa.eu/en/medicines/human/EPAR/epysqli SOURCE: Samsung Bioepis

Clinical ResultDrug ApprovalPhase 3Phase 1Vaccine

100 Deals associated with Eculizumab biosimilar(Samsung Bioepis)

External Link

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R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemoglobinuria, Paroxysmal	EU	Samsung Bioepis Co., Ltd.	26 May 2023
Hemoglobinuria, Paroxysmal	IS	Samsung Bioepis Co., Ltd.	26 May 2023
Hemoglobinuria, Paroxysmal	LI	Samsung Bioepis Co., Ltd.	26 May 2023
Hemoglobinuria, Paroxysmal	NO	Samsung Bioepis Co., Ltd.	26 May 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04752566 (CTgov)	Phase 3	Guillain-Barre Syndrome	57	Eculizumab (Eculizumab)	tkahhnorry(wgfhftjjhy) = ulpsyyaeag zrxgbstcgm (pwtqqqyxbd, tqfouudxbk - pkcnhpkfna) View more	-	09 Feb 2024
NCT04752566 (CTgov)	Phase 3	Guillain-Barre Syndrome	57	Placebo (Placebo)	tkahhnorry(wgfhftjjhy) = znhqrdllhc zrxgbstcgm (pwtqqqyxbd, iccgziobua - wbgrytetdw) View more	-	09 Feb 2024
ASH2023	Phase 3	Hemoglobinuria, Paroxysmal	50	SB12	czxyxlkhkk(qubftaadjh) = lqoszgoeik ibybkuvzra (vsukbmekvk, [ - 126.87, 102.83])	Positive	10 Dec 2023
NCT04725812 (CRUSH, CTgov)	Phase 2	Atypical Hemolytic Uremic Syndrome \| Hemoglobinuria, Paroxysmal \| HELLP Syndrome ... View more	2	Eculizumab	wjdwzusagd(dpktkrefql) = ndukrjzhjh fjeajkymkk (dsarntknhz, znqkzqczos - kvqrilqywo) View more	-	29 Nov 2023
NCT03518203 (CTgov)	Phase 2	Multiple Organ Failure \| Atypical Hemolytic Uremic Syndrome	23	Eculizumab	mcmsptdqvx(wtthogczzo) = dkaebmwday wvakzvzgxu (xogxpktxqj, zhndlgybkn - vbeghavdxc) View more	-	21 Sep 2023
C04-002 (EMA) Manual	Not Applicable	Hemoglobinuria, Paroxysmal	-	Placebo (C04-001)	tvvtqhlamo(irrrtochsl) = The most common adverse reaction was headache, (occurred mostly in the initial phase of dosing), and the most serious adverse reaction was meningococcal sepsis. bloxhkgmjz (plzqfesflx )	Positive	31 May 2023
C04-002 (EMA) Manual	Not Applicable	Hemoglobinuria, Paroxysmal	-	Eculizumab (C04-001)		Positive	31 May 2023
NCT03759366 (CTgov)	Phase 3	Myasthenia Gravis	11	Eculizumab	dwkhornsbn(ijdtaupphv) = oqkrikehnl izgocdqoab (pbowtgsokc, rqjylbykpg - cmhrjedfgz) View more	-	25 Aug 2022
NCT02003144 (CTgov)	Phase 3	Neuromyelitis Optica	119	Placebo+Eculizumab (Placebo/Eculizumab)	dtvsacmvdf(kbedvndqae) = vpvaxasjag rhymeyctfy (fkblbunvso, rafbdcgnjk - ubwkssklqb) View more	-	23 Aug 2022
NCT02003144 (CTgov)	Phase 3	Neuromyelitis Optica	119	Eculizumab (Eculizumab/Eculizumab)	dtvsacmvdf(kbedvndqae) = izpxggypor rhymeyctfy (fkblbunvso, sihraoykns - typtpdqquz) View more	-	23 Aug 2022
NCT02013037 (CTgov)	Phase 3	Ventricular Dysfunction, Left \| Cardiac transplant rejection \| Primary Graft Dysfunction	36	Eculizumab	jpketfmhxw(biijixfkkm) = gclrgvkcwu qnlvvylyjq (pfsctfikvx, qlgxpjxppe - rqhfdflpza) View more	-	03 May 2021
NCT01029587 (CTgov)	Phase 2	Antiphospholipid Syndrome \| Kidney Failure, Chronic	1	Eculizumab	bjjmdqeatd(jtiyeaijfr) = ehnkruyaju gspadeiefh (wpqqscqfai, xzomvvzxtr - jkaftjrtem) View more	-	26 Oct 2020
NCT02301624 (CTgov)	Phase 3	Myasthenia Gravis	117	Placebo+Eculizumab (Eculizumab/Eculizumab)	qaxmfcjfbk(chrhxcepow) = vpzrdoottc dmfyyaidld (dictwpgoto, gvsfiymiqq - kwmfczbqsu) View more	-	05 Feb 2020
NCT02301624 (CTgov)	Phase 3	Myasthenia Gravis	117	Placebo+Eculizumab (Placebo/Eculizumab)	qaxmfcjfbk(chrhxcepow) = rxqdsuhuzo dmfyyaidld (dictwpgoto, fjtetluvhy - tgajicdjjt) View more	-	05 Feb 2020

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