Los Angeles, USA , May 17, 2021 (GLOBE NEWSWIRE) -- Revolutionary Treatment for Hemophilia B Shows Promise in Clinical Trials
Novel Hemophilia B pipeline therapeutics include SIG-003, CB 2679d-GT, SerpinPC, AAV5-hFIX, Dalcinonacog alfa, Concizumab, Fitusiran, AMT-061, and others to bolster treatment options for patients.
DelveInsight’s “Hemophilia B Pipeline Insight” report provides comprehensive insights about 25+ companies and 20+ pipeline drugs in the Hemophilia B pipeline landscapes. It comprises Hemophilia B pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Hemophilia B therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Hemophilia B pipeline products.
Some of the key takeaways of the Hemophilia B Pipeline Report
Get an overview of pipeline landscape @ Hemophilia B Clinical Trials Analysis
Hemophilia B is a rare genetic bleeding disorder in which affected individuals have insufficient blood protein levels called factor IX. The disease is also known as factor IX deficiency or Christmas disease.
The Hemophilia B treatment is to replace factor IX to achieve adequate blood clotting and to prevent complications associated with the disorder. Currently, the replacement of factor IX to achieve a sufficient level is commonly done utilizing recombinant products or products derived from human blood or plasma. The ultimate goal of Hemophilia B therapy is to confer a treatment, which allows for long-term expression of the missing or lacking factor in a patient’s blood without continuous medical intervention, and Gene therapy has the potential to fulfil this objective.
Hemophilia B Emerging Drugs
Concizumab is an antibody targeting a natural anticoagulant protein known as a tissue factor pathway inhibitor. It is currently in the phase III stage of development and is being developed by Novo Nordisk.
Research and Development Phase IIINCT04083781: In October 2019, Novo Nordisk initiated a trial titled “Efficacy and Safety of Concizumab Prophylaxis in Patients with Hemophilia A or B with Inhibitors”. This study will test how well a new medicine called concizumab works in the body of people with hemophilia A or B with inhibitors. The study is expected to get completed by June 2024, with an expected 136 enrolled participants.
Fitusiran is subcutaneously administered investigational therapy that harnesses the natural RNA interference mechanism to regulate antithrombin (AT), enhancing thrombin generation (TG) and promoting hemostasis in people with hemophilia A or B, with or without inhibitors. The mechanism of action of fitusiran is independent of intrinsic and extrinsic coagulation pathways. The drug is curently in phase III stage of development for the treatment of patients with hemophilia A or B.
Research and Development Phase IIINCT03417102: In February 2018, Sanofi Genzyme initiated a phase 3 Study to evaluate the efficacy and safety of fitusiran in patients with Hemophilia A or B, with inhibitory antibodies to Factor VIII or IX. The purpose of this study is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK). The study is expected to get completed by June 2021.
MarzAA is a subcutaneously (SQ) administered next-generation engineered coagulation Factor VIIa (FVIIa) for the treatment of episodic bleeding in subjects with hemophilia A or B with inhibitors. The drug is currently in phase III stage of development.
Research and Development Phase IIINCT04489537: In May 2021, Catalyst Biosciences initiated a phase 3 study to evaluate the efficacy and safety of subcutaneous marzeptacog alfa (Activated) for on demand treatment and control of bleeding episodes in subjects with hemophilia A or hemophilia B, with inhibitors. The study will enroll approximately 60 subjects to treat 244 eligible bleeding episodes with each treatment. The primary endpoint is hemostatic efficacy using a standard 4-point assessment scale at the 24-hour timepoint. The study will assess the effectiveness of SQ MarzAA, using up to three doses to treat a bleeding episode, compared with the Standard of Care. The study is expected to get completed by March 2022.
For further information, refer to the detailed report @ Hemophilia B Pipeline Therapeutics
Scope of Hemophilia B Pipeline Drug Insight
Key Questions regarding Current Hemophilia B Treatment Landscape and Emerging Therapies Answered in the Pipeline Report
Table of Contents
Get a customised pipeline report @ Hemophilia B Drugs Pipeline Report
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