Sobi will present new data at the 64th Annual Meeting of the
Sobi's commitment to developing innovative treatments for people living with rare diseases is highlighted in studies spanning several rare disorders, including haemophilia, paroxysmal nocturnal haemoglobinuria (PNH), cold agglutinin disease (CAD), immune thrombocytopenia (ITP) and primary haemophagocytic lymphohistiocytosis (pHLH).
Results from the long-term extension study of Aspaveli/Empaveli (pegcetacoplan) in adults with PNH will be presented, demonstrating robust and sustained improvements for approximately two years in key markers of disease across a broad population of PNH patients.
In haemophilia A, new data on efanesoctocog alfa will be presented. Efanesoctocog alfa, an investigational new class of factor therapy for people with haemophilia A, has the potential to elevate standards of care by providing high sustained factor VIII activity into the near-normal range for the majority of the week with once-weekly prophylaxis.
New data also will be presented from the REAL-HLH study, which is the first study describing the clinical characteristics, treatment patterns and treatment outcomes of a larger cohort of patients who received Gamifant (emapalumab) in a real-world clinical setting.
New information will also be presented supporting the role of Doptelet (avatrombopag) in the treatment of ITP and describing the design of a study with Aspaveli/Empaveli for the treatment of CAD.
'The breadth of data being presented at ASH this year showcases our ongoing commitment to providing innovative treatments for those affected by rare, ultra-rare and life-threatening conditions,' said
Sobi collaboration partner
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa 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 is currently under clinical investigation and its safety and efficacy have not been reviewed by any regulatory authority. Efanesoctocog alfa was granted orphan drug designation by the
About the Sobi and
Sobi and
About Aspaveli/Empaveli
Aspaveli/Empaveli (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. It is approved as Aspaveli for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months in the
About the Sobi and Apellis Collaboration
Sobi and Apellis collaborate to develop and commercialize systemic Aspaveli/Empaveli. Sobi has exclusive ex-US commercialisation rights for systemic Aspaveli/Empaveli. Apellis has exclusive US commercialisation rights for systemic Empaveli. The companies have global co-development rights for systemic Aspaveli/Empaveli.
About Doptelet
Doptelet (avatrombopag) 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 an anti-interferon gamma (IFN?) monoclonal antibody that binds to and neutralises IFN?. In the
About loncastuximab tesirine
Loncastuximab tesirine (US brand name ZYNLONTA) is a CD19-directed antibody-drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalised by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumour cell death.
The
ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.
Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across
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