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Hemochromatosis - Pipeline Insight, 2021

  • ID: 5306344
  • Drug Pipelines
  • March 2021
  • Region: Global
  • 60 pages
  • DelveInsight

FEATURED COMPANIES

  • Disc Medicine
  • Protagonist Therapeutics
  • Silence Therapeutics
This “Hemochromatosis - Pipeline Insight, 2021,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Hemochromatosis 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
Hemochromatosis Understanding

Hemochromatosis: Overview

Hemochromatosis is a disorder in which extra iron builds up in the body to harmful levels. Without treatment, hemochromatosis can cause iron overload, a buildup of iron that can damage many parts of the body, including your liver, heart, pancreas, endocrine glands, and joints. There are two forms of hemochromatosis: primary and secondary. Primary hemochromatosis, also known as hereditary hemochromatosis, usually results from genetic factors. Secondary hemochromatosis occurs when a buildup of iron stems from another medical condition, such as erythropoietic hemochromatosis. Diagnosis for hemochromatosis is based on blood tests to check levels of iron and certain proteins in the blood and to check for gene mutations that commonly cause hemochromatosis. Treatment of hemochromatosis is done with phlebotomy, or drawing about a pint of blood at a time, on a regular schedule. Another option is chelation. This is a developing therapy that can help manage iron levels, but it is expensive and not a first-line treatment option.

"Hemochromatosis - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemochromatosis pipeline landscape is provided which includes the disease overview and Hemochromatosis treatment guidelines. The assessment part of the report embraces, in depth Hemochromatosis 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, Hemochromatosis 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 Hemochromatosis R&D. The therapies under development are focused on novel approaches to treat/improve Hemochromatosis.

Hemochromatosis Emerging Drugs Chapters

This segment of the Hemochromatosis 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.

Hemochromatosis Emerging Drugs

PTG-300: Protagonist Therapeutics

PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin that regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body and is based on peptide technology platform. PTG-300 (Rusfertide) is currently being evaluated for various disorders associated with iron overload and/or excessive erythrocytosis (red blood cell production). Rusfertide is being evaluated in a small open-label Phase 2 study in hereditary hemochromatosis (HH).

DISC-A: Disc Medicine

DISC-A is an oral small molecule and potent inhibitor of matriptase-2. It increases hepcidin levels and have potential therapeutic utility in diseases such as beta thalassemia, myelodysplastic syndrome and hemochromatosis. Disc Medicine is conducting preclinical studies of a matriptase-2 inhibitor as a potential treatment for rare hematologic disease.

Hemochromatosis: Therapeutic Assessment

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

Major Players in Hemochromatosis

There are approx. 5+ key companies which are developing the therapies for Hemochromatosis. The companies which have their Hemochromatosis drug candidates in the most advanced stage, i.e. Phase III include, Sound Pharmaceuticals.

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
Hemochromatosis 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
  • Parenteral
  • Intravitreal
  • Subretinal
  • Topical
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hemochromatosis: Pipeline Development Activities

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

Hemochromatosis Report Insights
  • Hemochromatosis Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
Hemochromatosis Report Assessment
  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs
Key Questions Answered

Current Treatment Scenario and Emerging Therapies:
  • How many companies are developing Hemochromatosis drugs?
  • How many Hemochromatosis drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemochromatosis?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hemochromatosis 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 Hemochromatosis and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Players
  • Protagonist Therapeutics
  • Disc Medicine
  • Silence Therapeutics
  • La Jolla Pharmaceutical Company
Key Products
  • PTG-300
  • DISC-A
  • SLN-124
  • LJPC-401
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Disc Medicine
  • Protagonist Therapeutics
  • Silence Therapeutics
Introduction

Executive Summary

Hemochromatosis: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management
Pipeline Therapeutics
  • Comparative Analysis
Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type
Hemochromatosis - Analytical Perspective

In-depth Commercial Assessment
  • Hemochromatosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Hemochromatosis Collaboration Deals
  • Company-Company Collaborations (Licensing/Partnering) Analysis
  • Company-University Collaborations (Licensing/Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
Drug name: Company name
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
PTG-300: Protagonist Therapeutics
  • Product Description
  • Research and Development
  • Product Development Activities
Early Stage Products (Phase I)
  • Comparative Analysis
Drug name: Company name
  • Product Description
  • Research and Development
  • Product Development Activities
Preclinical and Discovery Stage Products
  • Comparative Analysis
Matriptase-2 inhibitor: Disc Medicine
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Hemochromatosis Key Companies

Hemochromatosis Key Products

Hemochromatosis- Unmet Needs

Hemochromatosis- Market Drivers and Barriers

Hemochromatosis- Future Perspectives and Conclusion

Hemochromatosis Analyst Views

Hemochromatosis Key Companies

Appendix

List of Tables
Table 1 Total Products for Hemochromatosis
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List of Figures
Figure 1 Total Products for Hemochromatosis
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Note: Product cover images may vary from those shown
  • Protagonist Therapeutics
  • Disc Medicine
  • Silence Therapeutics
  • La Jolla Pharmaceutical Company
Note: Product cover images may vary from those shown
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