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

Hemochromatosis is a genetic disorder characterized by excessive absorption and accumulation of iron in the body, leading to potential damage to vital organs such as the liver, heart, and pancreas. This condition is primarily hereditary, with the most common form linked to mutations in the HFE gene. There are different types of hemochromatosis, each associated with distinct genetic mutations. The most prevalent form is Type 1, resulting from mutations in the HFE gene, particularly C282Y and H63D mutations. Other types include Type 2 (juvenile hemochromatosis), Type 3, and Type 4 (ferroportin disease), each associated with different genetic mutations.

Hereditary hemochromatosis typically manifests later in life, with symptoms often appearing between the ages of 40 and 60 in men and post-menopause in women. Early symptoms are often nonspecific, making diagnosis challenging. These initial symptoms can include fatigue, joint pain, abdominal pain, weakness, loss of libido, and weight loss. As the disease progresses without treatment, more severe symptoms can develop, such as diabetes, liver disorders like cirrhosis and liver cancer, heart problems including heart failure and arrhythmias, skin pigmentation changes (bronze or gray skin tone), and hypogonadism (decreased function of the gonads).

Secondary hemochromatosis, while less common, occurs due to factors such as excessive iron intake, multiple blood transfusions, or chronic liver diseases like hepatitis or alcoholic liver disease. Unlike hereditary hemochromatosis, secondary hemochromatosis is acquired and can affect individuals without genetic predisposition. Both forms, however, result in similar pathological iron overload and require medical intervention to prevent organ damage and associated complications.

Diagnosis of hemochromatosis involves a combination of clinical evaluation, blood tests, and genetic testing. Blood tests typically measure serum ferritin levels and transferrin saturation, which indicate iron overload. If these tests suggest iron overload, genetic testing is conducted to identify mutations in the HFE gene or other related genes. Early detection is crucial for preventing irreversible organ damage, and family screening is often recommended for relatives of affected individuals to identify asymptomatic carriers of the genetic mutations.

Treatment for hemochromatosis primarily involves therapeutic phlebotomy, a procedure that removes blood from the body to reduce iron levels. In some cases, chelation therapy, which involves medications that bind to iron and facilitate its excretion, may be used. Lifestyle modifications, such as avoiding iron supplements and limiting dietary iron intake, are also recommended. With early diagnosis and proper management, individuals with hemochromatosis can lead normal, healthy lives, minimizing the risk of severe complications.

"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. The clinical studies suggest that rusfertide has a unique iron regulatory mechanism which allows for persistent control of hematocrit without causing iron deficiency. In a Phase II open-label study, the hepcidin mimetic rusfertide prevented iron reaccumulation in the absence of therapeutic phlebotomy, demonstrating the potential utility of using rusfertide in the setting of hemochromatosis. These findings were published in the journal Lancet Gastroenterology and Hepatology.

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

The 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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