Hemophilia A - Pipeline Insight, 2024

This “Hemophilia A - Pipeline Insight, 2024” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in Hemophilia A pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Hemophilia A: Understanding

Hemophilia A: Overview

Hemophilia, which means love (philia) of blood (hemo), manifests with prolonged and excessive bleeding either spontaneously or after insignificant trauma. Hemophilia encompasses a group of inherited ailments that alter the body's normal blood coagulation. A hereditary hemorrhagic disorder resulting from a congenital deficit or scarcity of factor VIII, hemophilia A, which is known as classical hemophilia, manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. An X-linked, recessive hemorrhagic trait or gene induces Hemophilia A. Hemophilia A's X-linked trait manifests as a congenital absence or decrease in plasma clotting Factor VIII, a pro-Coagulation cofactor and robust initiator of thrombin that is essential for the generation of adequate amounts of fibrin to form a platelet-fibrin plug at sites of endothelial disruption. When the vascular endothelium sustains an injury, the hemostatic process initiates the coagulation cascade to restore vascular integrity and prevent further bleeding. Platelet activation occurs at the site of vascular rupture, initiating promulgation of clotting factors and fibrin formation, resulting in a platelet-fibrin plug to inhibit further bleeding. Factor VIII, the deficit of which causes hemophilia A, provides essential enhancement of thrombin generation and promulgation of fibrin formation to inhibit further bleeding.

Diagnostic evaluation for hemophilia occurs in the setting of a known family history, excessive bleeding out of proportion to the traumatic injury, or abnormally activated partial thromboplastin time. Normal hemogram and prothrombin time in the setting of elevated activated partial thromboplastin time heightens the suspicion of hemophilia and should prompt factor VIII and IX determination. Diagnosis for suspected hemophilia include tests like Complete blood cell count, Screening coagulation studies, FVIII assay and FVIII inhibitor assay. Treatment goal for hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. Medication to treat hemophilia A is concentrated FVIII product. Several new treatments for hemophilia A are under development, from gene therapy to new non-factor replacement therapies.

"Hemophilia A - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia A pipeline landscape is provided which includes the disease overview and Hemophilia A treatment guidelines. The assessment part of the report embraces, in depth Hemophilia A commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hemophilia A collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia A

Hemophilia A Emerging Drugs Chapters

This segment of the Hemophilia A report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hemophilia A Emerging Drugs

Fitusiran: Sanofi

Fitusiran is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors. Fitusiran is designed to lower antithrombin, a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Fitusiran utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability. Currently, the drug is in phase III stage of its clinical trial for the treatment of Hemophilia A.

STSP-0601: Staidson Beijing Biopharmaceuticals

STSP-0601 is under development for the treatment of unspecified indication, hemophilia A and hemophilia B. It is administered through intravenous route. The drug candidate acts by targeting coagulation factor X. Currently, the drug is in phase II stage of its clinical trial for the treatment of hemophilia A disease.

TU7710: TiumBio

TU7710 is a bypassing agent treatment for patients with neutralizing antibodies and is an innovative drug candidate with a half-life approximately 7 times greater than conventional treatments through our transferrin fusion gene recombination technology. TU7710 is expected to dramatically improve the convenience and quality of life of hemophilia patients with neutralizing antibodies. Currently, the drug is in phase I stage of its clinical trial for the treatment of Hemophilia A.

Hemophilia A Therapeutic Assessment

This segment of the report provides insights about the different Hemophilia A drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hemophilia A

There are approx. 25+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Sanofi.

Phases

This report covers around 30+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hemophilia A Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Hemophilia A therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hemophilia A drugs.

Hemophilia A Report Insights

• Hemophilia A Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hemophilia A Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hemophilia A drugs?
• How many Hemophilia A drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia A?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hemophilia A and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• TiumBio Co.
• Jiangsu Gensciences lnc
• Novo Nordisk
• Genzyme
• Spark Therapeutics
• G & P Bioscience
• Gritgen Therapeutics
• Biocad
• Belief Biomed
• Chugai Pharmaceutical
• Staidson Beijing BioPharmaceuticals
• Jiangsu Gensciences
• Poseida Therapeutics
• Chia Tai Tianqing Pharmaceutical Group
• Takeda

Key Products

• TU7710
• SS109
• Mim8
• Fitusiran
• SPK-8011
• GB 106
• GS-1191
• ANB 010
• BBM-H803
• NXT 007
• STSP 0601
• FRSW 117
• P-FVIII-101
• TQG 203
• TAK-754

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