Sobi(TM) will present new data at the 63rd Annual Meeting of the
For the first time, the
"We look forward to presenting data from the
In addition to the
In haemophilia A, new data on efanesoctocog alfa, previously known as BIVV001, will be presented. Efanesoctocog alfa, an investigational once-weekly factor therapy for people with haemophilia A, has the potential to provide high sustained factor VIII activity and near-normal factor levels for the majority of the week. Efanesoctocog alfa represents a potential new class of factor replacement treatment. Currently in phase 3 development in partnership with
Sobi will also present new data from the pilot study of Gamifant® (emapalumab), a fully human, anti-interferon gamma (IFNγ) monoclonal antibody, in patients with systemic juvenile idiopathic arthritis (sJIA) developing MAS. These data demonstrate the pathogenic role of IFNγ in MAS/sJIA and the therapeutic value of IFNγ neutralisation in patients with MAS who have failed standard therapy with high-dose glucocorticoids. Additionally, new exposure-safety analyses of data obtained from the pivotal trial of Gamifant in patients with primary HLH will be presented.
Haemophilia | ||
Alprolix® (eftrenonacog alfa) | Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial. |
#498. Oral presentationSession: 322. Sunday, 12 December, Session Time: |
Efanesoctocog alfa (investigational) | Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies. |
#1035. Poster presentationSaturday, 11 December, |
Elocta®/Eloctate® (efmoroctocog alfa) | Retrospective Observational Descriptive Study on the Effectiveness and Usage of Emicizumab and Antihemophilic Factor (recombinant), Fc Fusion Protein in Patients with Hemophilia A in the US. |
#3031. Poster presentationSession: 904. Sunday, 12 December, |
Paroxysmal nocturnal haemoglobinuria | ||
Aspaveli®/Empaveli(TM) (pegcetacoplan) |
Efficacy and Safety of Pegcetacoplan Treatment in Complement-Inhibitor Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the Phase 3 |
#606. Oral presentationMonday, 13 December, |
Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network. |
#1112. Poster presentationSaturday, 11 December, | |
Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter. |
#2194. Poster presentationSunday, 12 December, | |
Evaluation of the Long-Term Safety and Efficacy of Pegcetacoplan Treatment for Paroxysmal Nocturnal Hemoglobinuria Patients: An Extension Study. |
#2175. Poster presentationSunday, 12 December, | |
Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials. |
#1104. Poster presentationSaturday, 11 December, | |
Chronic immune thrombocytopenia | ||
Doptelet® (avatrombopag) | Durability of Platelet Response When Switching from Eltrombopag or Romiplostim to Avatrombopag in Immune Thrombocytopenia (ITP): A Multicenter Study. |
#1015. Poster presentationSession: 311. Saturday, 11 December, |
Further Characterization of |
#2086. Poster presentationSession: 311. Sunday, 12 December, | |
Rationale and Design of an Observational Multicenter Study to Evaluate the Use and Effectiveness of Avatrombopag in Adult Patients with Immune Thrombocytopenia: The Adopt Study. | #4210. Online publication in November issue of Blood. | |
Rationale and Design of a Phase 3b Multicenter, Randomized, Double-Blind Placebo-Controlled, Parallel-Group Trial with an Open-Label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Pediatric Patients with Immune Thrombocytopenia. | #4211. Online publication in November issue of Blood. | |
Hemophagocytic lymphohistiocytosis | ||
Gamifant® (emapalumab) | Safety of Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis (HLH): Relationship to Treatment Exposure. |
#2061. Poster presentation.Session: 201Sunday, 12 December, |
Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and Other Clinical Conditions Treated with Emapalumab: The Real-HLH Study Design | #4991. Online publication in November issue of Blood. | |
Macrophage activation syndrome | ||
Gamifant® (emapalumab) | Macrophage Activation Syndrome (MAS) in Systemic Juvenile Idiopathic Arthritis (sJIA): Treatment with Emapalumab, an Anti-Interferon Gamma (IFNγ) Monoclonal Antibody. |
#2058. Poster presentation.Session: 201 Sunday, 12 December, |
Trials in Progress: A Two-Cohort, Open-Label, Single-Arm Study of Emapalumab, an Anti-Interferon Gamma (IFNγ) Monoclonal Antibody, in Patients with Macrophage Activation Syndrome (MAS) in Rheumatic Diseases | #4195. Online publication in November issue of Blood. |
All abstracts can be accessed via the official ASH website.
About Elocta®/Eloctate®
Elocta®/Eloctate® (efmoroctocog alfa) is a recombinant clotting factor therapy developed for haemophilia A using Fc fusion technology (rFVIIIFc) to prolong circulation in the body. It is engineered by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Elocta/Eloctate to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Elocta/Eloctate is approved and marketed by Sobi for the treatment of haemophilia A in the EU including
About Alprolix®
Alprolix® (eftrenonacog alfa) is a recombinant clotting factor therapy developed for haemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Alprolix to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Sobi and
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa (rFVIIIFc-VWF-XTEN) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. Efanesoctocog alfa was granted orphan drug designation by the
About Aspaveli®/Empaveli(TM)
Aspaveli®/Empaveli(TM) (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Empaveli is approved in
About the
The
About the Sobi and Apellis Collaboration
Sobi and Apellis collaborate to develop and commercialize systemic pegcetacoplan. Sobi has exclusive ex-
About Doptelet®
Doptelet® is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved by the
About Gamifant®
Gamifant® (emapalumab) is a monoclonal antibody that binds to and neutralises interferon gamma (IFNγ). In the US, emapalumab is indicated for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). In
About Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.
References
- Lambert et al. Blood 2017.
XTEN® is a registered trademark of Amu
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