Factor IX - Pipeline Insight, 2024

This “Factor IX - Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Factor IX 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

Factor IX : Understanding

Factor IX : Overview

Factor IX (FIX, Christmas factor) is a blood clotting factor, a zymogen of serine protease. Upon activation, FIX is converted into the active serine protease and, in the presence of Ca 2+ and membrane phospholipids, it hydrolyses one arginine-isoleucine bond in factor X to form the activated factor X (Xa). The catalytic efficiency of activated FIX (FIXa) is greatly increased by the cofactor, the activated factor VIII (FVIIIa). It is produced in the liver, and the inactive precursor protein is processed in the endoplasmic reticulum and Golgi, where it undergoes multiple post-translational modifications and is secreted into the bloodstream upon proteolytic cleavage of the propeptide. Deficiency of factor IX causes Christmas disease (hemophilia B). There are approximately 3000 variants of factor IX has been described. Some cause no symptoms, but may lead to a significant bleeding disorder. The original Christmas disease mutation was identified by sequencing of Christmas' DNA, revealing a mutation which changed a cysteine to a serine.

Factor IX is produced as a zymogen, an inactive precursor. It is processed to remove the signal peptide, glycosylated and then cleaved by factor XIa of the contact pathway or factor VIIa of the tissue factor pathway to produce a two chain form, where the chains are linked by a disulfide bridge. When activated into factor IXa, in the presence of ca 2+ membrane phospholipids and a factor VIII cofactor, it hydrolases one arginine-isoleucine bond in factor X to form factor Xa. Factor IX is inhibited by antithrombin.

“Factor IX - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Factor IX pipeline landscape is provided which includes the disease overview and Factor IX treatment guidelines. The assessment part of the report embraces, in depth Factor IX 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, Factor IX 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 Factor IX R&D. The therapies under development are focused on novel approaches to treat/improve Factor IX.

Factor IX Emerging Drugs Chapters

This segment of the Factor IX report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Factor IX Emerging Drugs

TNX-832: Tanox
TNX-832 is in a clinical trial for the treatment of acute lung injury/acute respiratory distress syndrome. Its mechanism of action include inhibition of factor IX and factor X. A humanized version of TNX-832 is in preclinical development as a cancer therapy. Currently, the drug is in Phase I/II stage of its clinical trial.

AMA 005: Amarna Therapeutics
AMA 005 is a gene therapy drug its target is factor IX coagulation factor. Its mechanism of action include Factor IX replacements and gene transference. Its therapeutic areas include congenital disorders, Hemic and lymphatic disease. Currently, the drug is in preclinical stage of its clinical trial Tanox.

Factor IX: Therapeutic Assessment

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

Major Players in Factor IX

There are approx. 5+ key companies which are developing the therapies for Factor IX. The companies which have their Factor IX drug candidates in the most advanced stage, i.e. Phase I/II include Tanox.

Phases

This report covers around 5+ 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

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Factor IX: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Factor IX 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 Factor IX drugs.

Factor IX Report Insights

• Factor IX Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Factor IX 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 Factor IX drugs?
• How many Factor IX drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Factor IX?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Factor IX 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 Factor IX and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Biocad
• Catalyst Biosciences
• Asklepios BioPharmaceutical
• Amarna Therapeutics
• UBI Pharma
• Tanox Inc.
• Pfizer
• Frontera Therapeutics

Key Products

• ANB 002
• Dalcinonacog alfa
• AskBio 009
• AMA 005
• UB-854 Recombinant Factor IX
• TNX-832
• Fidanacogene elaparvovec
• FT 004

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