Homozygous Familial Hypercholesterolemia - Pipeline Insight, 2024

This “ Homozygous Familial Hypercholesterolemia - Pipeline Insight, 2024,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Homozygous Familial Hypercholesterolemia 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

Homozygous Familial Hypercholesterolemia Understanding

Homozygous Familial Hypercholesterolemia: Overview

Homozygous familial hypercholesterolaemia (HoFH) is a rare and life-threatening disease originally characterized clinically by plasma cholesterol levels >13 mmol/L (>500 mg/dL), extensive xanthomas, and marked premature and progressive atherosclerotic cardiovascular disease (ACVD). Studies in cultured fibroblasts from these patients showed a severe defect in the ability to bind and internalize LDL particles, subsequently shown to be caused by mutations in both alleles of the gene encoding the LDL receptor (LDLR). Recent genetic insights, however, indicate that mutations in alleles of other genes, including APOB, PCSK9, and LDLRAP1, may be present in some individuals with HoFH. Untreated, most patients with markedly elevated LDL-C levels develop overt atherosclerosis before the age of 20 years, and generally do not survive past 30 years. Thus, the primary goals of management are prevention of ACVD by early and comprehensive control of hypercholesterolaemia, and early detection of complications, with specific focus on ostial occlusion and aortic stenosis. Unfortunately, HoFH is typically diagnosed when considerable coronary atherosclerosis has already developed, emphasizing the need for optimization of treatment in childhood.

Homozygous Familial Hypercholesterolemia - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Homozygous Familial Hypercholesterolemia pipeline landscape is provided which includes the disease overview and Homozygous Familial Hypercholesterolemia treatment guidelines. The assessment part of the report embraces, in depth Homozygous Familial Hypercholesterolemia 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, Homozygous Familial Hypercholesterolemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Homozygous Familial Hypercholesterolemia.
• In the coming years, the Homozygous Familial Hypercholesterolemia market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics that are working to assess challenges and seek opportunities that could influence Homozygous Familial Hypercholesterolemia R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• A detailed portfolio of major pharma players who are involved in fueling the Homozygous Familial Hypercholesterolemia treatment market. Several potential therapies for Homozygous Familial Hypercholesterolemia are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Homozygous Familial Hypercholesterolemia market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Homozygous Familial Hypercholesterolemia) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Homozygous Familial Hypercholesterolemia Emerging Drugs Chapters

This segment of the Homozygous Familial Hypercholesterolemia 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.

Homozygous Familial Hypercholesterolemia Emerging Drugs

ARO-ANG 3: Arrowhead PharmaceuticalsARO-ANG3 is designed to reduce production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase. ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular disease. Currently, it is in Phase II stage of clinical trial evaluation to treat Homozygous Familial Hypercholesterolemia.

Tafolecimab: Innovent BiologicsTafolecimab injection, developed by Innovent, is an lgG2 fully human monoclonal antibody that can specifically bind to PCSK-9 and reduce LDL-C level by inhibiting PCSK-9-mediated low-density lipoprotein receptor (LDLR) endocytosis, subsequently enhancing the clearance of LDL-C, resulting in a reduction in LDL-C level.

Homozygous Familial Hypercholesterolemia: Therapeutic Assessment

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

Major Players in Homozygous Familial Hypercholesterolemia

There are approx. 3+ key companies which are developing the therapies for Homozygous Familial Hypercholesterolemia. The companies which have their Homozygous Familial Hypercholesterolemia drug candidates in the most advanced stage, i.e. phase II include, Arrowhead Pharmaceuticals.

Phases

This report covers around 3+ 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

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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

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

Homozygous Familial Hypercholesterolemia: Pipeline Development Activities

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

Homozygous Familial Hypercholesterolemia Report Insights

• Homozygous Familial Hypercholesterolemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Arrowhead Pharmaceuticals
• Innovent Biologics
• Akeso Biopharma
• LIB Therapeutics LLC

Key Products

• ARO-ANG 3
• Tafolecimab
• AK102
• Lerodalcibep