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Revolutionary Treatment for Hemophilia B Shows Promise in Clinical Trials

·8 min read

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.

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

  • Prominent companies such as Sigilon Therapeutics, Catalyst Biosciences, Centessa Pharmaceuticals, UniQure Biopharma, Novo Nordisk, Sanofi Genzyme, and others are developing potential drug candidates to boost the Hemophilia B treatment scenario.

  • In August 2020, Novo Nordisk announced that it had resumed its phase 3 clinical trials exploring concizumab as a potential treatment for Hemophilia A and B.

  • Recombinant factor IX therapy is the recommended treatment for Hemophilia B individuals. In the U.S., the currently available recombinant factor IX products are BeneFIX, Rixubis, Ixinity, and Rebinyn.

  • In December 2020, Catalyst Biosciences announced that the FDA had granted Fast Track designation for MarzAA. MarzAA, a next-generation SQ FVIIa is currently in Phase 3 trial (MAA-304) in patients with Hemophilia A or B with inhibitors.

  • Fidanacogene elaparvovec (previously SPK-9001 or PF-06838435) is a novel, investigational bio-engineered AAV vector under Phase III (NCT03861273) stage of clinical development by Pfizer/Spark Therapeutics. FLT180a is a next-generation AAV gene therapy for people with hemophilia B being developed by Freeline Therapeutics.

  • uniQure is advancing a promising clinical program focused on hemophilia B, a severe orphan blood clotting disorder. The gene therapy product candidate etranacogene dezaparvovec (AMT-061) consists of an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX (FIX-Padua). uniQure is now conducting the Phase III HOPE-B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B.

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: Novo Nordisk

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 III
NCT04083781: 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: Sanofi Genzyme

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 III
NCT03417102: 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.

  • Marzeptacog alfa (MarzAA): Catalyst Biosciences

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 III
NCT04489537: 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

  • Coverage: Global

  • Major Players: 25+ Key Players

  • Prominent Players: Sigilon Therapeutics, Catalyst Biosciences, Centessa Pharmaceuticals, UniQure Biopharma, Novo Nordisk, Sanofi Genzyme, ASC Therapeutics, Freeline Therapeutics, OPKO Health, Spark Therapeutics, Sangamo BioSciences, Bayer, Pfizer, Chia Tai Tianqing Pharmaceutical Group, Staidson Beijing BioPharmaceuticals, Expression Therapeutics, GC Pharma, Amarna Therapeutics, Intellia Therapeutics, Regeneron, LogicBio Therapeutics, ISU ABXIS, Alnylam Pharmaceuticals, and many others.

  • Key Drugs Profiles: 20+ Products

    • Phases:

    • Hemophilia B Therapies Late-stage (Phase III)

    • Hemophilia B Therapies Mid-stage (Phase II)

    • Hemophilia B Therapies Early-stage (Phase I)

    • Hemophilia B Pre-clinical stage and Discovery candidates

    • Discontinued and Inactive candidates

    • Mechanism of Action:

    • Antithrombin III inhibitors

    • Lipoprotein-associated coagulation inhibitor inhibitors

    • Factor VIIa replacements

    • Factor IX replacements

    • Factor IX stimulants

    • Molecule Types:

    • Small Molecule

    • Gene Therapy

    • Stem Cell Therapy

    • Route of Administration:

    • Intravenous

    • Inhalation

    • Subcutaneous

    • Oral

    • Product Types:

    • Monotherapy

    • Combination

Key Questions regarding Current Hemophilia B Treatment Landscape and Emerging Therapies Answered in the Pipeline Report

  • What are the current options for Hemophilia B treatment?

  • How many companies are developing therapies for the treatment of Hemophilia B?

  • How many are Hemophilia B emerging therapies in the early-stage, mid-stage, and late development stages to treat Hemophilia B?

  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Hemophilia B market?

  • Which are the dormant and discontinued products and the reasons for the same?

  • What is the unmet need for current therapies for the treatment of Hemophilia B?

  • What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Hemophilia B therapies?

  • What are the critical designations that have been granted for the emerging therapies for Hemophilia B?

  • How many patents are granted and pending for the emerging therapies to treat Hemophilia B?

Table of Contents

1

Hemophilia B Report Introduction

2

Hemophilia B Executive Summary

3

Hemophilia B Overview

4

Hemophilia B – DelveInsight’s Analytical Perspective In-depth Commercial Assessment

5

Hemophilia B Pipeline Therapeutics

6

Hemophilia B Therapeutic Assessment

7

Hemophilia B Late Stage Products (Phase III)

7.1

Concizumab: Novo Nordisk

8

Hemophilia B Mid Stage Products (Phase II)

8.1

Drug name: Company name

9

Hemophilia B Early Stage Products (Phase I/II)

9.1

SerpinPC: Centessa Pharmaceuticals

10

Hemophilia B Early Stage Products (Preclinical)

10.1

CB 2679d-GT: Catalyst Biosciences

11

Hemophilia B Inactive Products

12

Company-University Collaborations (Licensing/Partnering) Analysis

13

Hemophilia B Key Companies

14

Hemophilia B Key Products

15

Hemophilia B Unmet Needs

16

Hemophilia B Market Drivers and Barriers

17

Hemophilia B Future Perspectives and Conclusion

18

Hemophilia B Analyst Views

19

Appendix

20

About DelveInsight

Get a customised pipeline report @ Hemophilia B Drugs Pipeline Report

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About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.

CONTACT: Contact Us: Shruti Thakur info@delveinsight.com +1(919)321-6187 www.delveinsight.com