Hemochromatosis - Pipeline Insight, 2024

This “Hemochromatosis- Pipeline Insight, 2024” report provides comprehensive insights about 4+ companies and 4+ 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

Hereditary hemochromatosis is a genetic disorder that can cause severe liver disease and other health problems. Hemochromatosis is a disorder in which the body can build up too much iron in the skin, heart, liver, pancreas, pituitary gland, and joints. Too much iron is toxic to the body and over time the high levels of iron can damage tissues and organs and lead to:

• Cirrhosis (liver damage),
• Hepatocellular carcinoma (liver cancer),
• Heart problems,
• Arthritis (joint pain), and
• Diabetes.

Causes
Hereditary hemochromatosis is most commonly caused by certain variants in the HFE gene. If you inherit two of these variants, one from each parent, you have hereditary hemochromatosis and are at risk for developing high iron levels. If you have a family member, especially a sibling, who is known to have hereditary hemochromatosis, talk to your doctor about genetic testing.

In the United States, about 1 in 300 non-Hispanic White people has hereditary hemochromatosis, with lower rates among people of other races and ethnicities. Many people with hereditary hemochromatosis don’t know they have it. Early symptoms of hemochromatosis, such as feeling tired or weak, are common and can cause it to be confused with a variety of other diseases. Men with hereditary hemochromatosis are more likely to develop complications and often at an earlier age. An estimated 9% (about 1 in 10) of men with hereditary hemochromatosis will develop severe liver disease. However, most people with hereditary hemochromatosis never develop symptoms or complications.

Symptoms
Symptoms of hemochromatosis include:
? Feeling of tiredness or weakness,
? Weight loss,
? Joint pain,
? Bronze or grey skin color,
? Abdominal pain, and
? Loss of sex drive.

Diagnosis
A blood test can be used to screen people who may have hemochromatosis by measuring how much iron is in their blood. Affected people with or without a known family history of hemochromatosis can be diagnosed through blood tests for iron followed by genetic testing if they are symptomatic or have complications.

Treatment
In most cases, doctors treat hemochromatosis with phlebotomy, or drawing about a pint of blood at a time, on a regular schedule. This is the most direct and safe way to lower body stores of iron.

People who receive blood transfusions to treat certain types of anemia and develop secondary hemochromatosis cannot have phlebotomy to lower their iron levels. To treat secondary hemochromatosis in these people, doctors prescribe medicines, called chelating agents, which bind to iron and allow it to pass from the body in urine. Chelating agents may be pills taken by mouth or intravenous (IV) medicines, and they do not remove iron as effectively as phlebotomy.

Hemochromatosis- Pipeline Insight, 2024 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 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

Rusfertide (PTG-300): Protagonist Therapeutics, Inc.

Rusfertide (PTG-300) is a novel injectable synthetic mimetic of the natural hormone hepcidin. Rusfertide has the potential to provide substantial benefit to patients with erythrocytosis or abnormal tissue iron overload by managing hematocrit rapidly, sustainably, and durably.

Ongoing clinical studies suggest that rusfertide has a unique iron regulatory mechanism which allows for persistent control of hematocrit without causing iron deficiency. It was also evaluated in a small open-label Phase II study in hereditary hemochromatosis (HH), a blood disorder causing abnormal iron levels in tissues and organs arising primarily from absence or deficiency of the hepcidin pathway in these patients which got completed in February 2022.

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. 4+ 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, Protagonist Therapeutics.

Phases

This report covers around 4+ 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• 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.

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

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, Inc.
• Sirnaomics
• Bond Biosciences

Key Products

• Rusfertide (PTG-300)
• STP251G
• BBI-001

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