Haemochromatosis - Pipeline Insight, 2024

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

Haemochromatosis Understanding

Haemochromatosis: Overview

Hemochromatosis is a disorder associated with deposits of excess iron that causes multiple organ dysfunction. Normally, iron absorption is tightly regulated because the body is incapable of excreting excess iron. Hemochromatosis occurs when there are high pathologic levels of iron accumulation in the body. Hemochromatosis has been called “bronze diabetes” due to the discoloration of the skin and associated disease of the pancreas. Hereditary hemochromatosis is the most common autosomal recessive disorder in whites. Secondary hemochromatosis occurs because of erythropoiesis disorders and treatment of the diseases with blood transfusions. After the damage of transfused red blood cells by macrophages, iron freed from heme is accumulated in the body. Secondary hemochromatosis is mainly caused by thalassemia, sickle cell anemia, hereditary spherocytosis, X-linked sideroblastic anemia, and pyruvate kinase deficiency.

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

This segment of the Haemochromatosis 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.

Haemochromatosis Emerging Drugs

Rusfertide: Protagonist Therapeutics Rusfertide (PTG-300) is an investigational, injectable hepcidin mimetic that is currently being developed for various disorders associated with iron overload and/or excessive erythrocytosis (red blood cell production). Rusfertide regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Discovered through Protagonist's peptide technology platform, rusfertide is currently being investigated in the REVIVE Phase II proof-of-concept clinical trial for polycythemia vera (PV), a rare chronic blood disorder that affects about 160,000 patients in the U.S., the PACIFIC Phase 2 study in PV subjects with high hematocrit levels, and a recently completed Phase IIa study for hereditary hemochromatosis.

Haemochromatosis: Therapeutic Assessment

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

Major Players in Haemochromatosis

There are approx. 5+ key companies which are developing the therapies for Haemochromatosis. The companies which have their Haemochromatosis drug candidates in the most advanced stage, i.e. Phase II include, Protagonist Therapeutics.

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

Haemochromatosis 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
• Intravenous
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

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

Haemochromatosis : Pipeline Development Activities

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

Haemochromatosis Report Insights

• Haemochromatosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• La Jolla Pharmaceutical Company
• Protagonist Therapeutics
• Bond Biosciences
• Disc Medicine
• Sirnaomics

Key Products

• LJPC 401
• Rusfertide
• BBI 001
• DISC a
• STP 251G

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