Wilson Disease - Pipeline Insight, 2024

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

Wilson Disease: Understanding

Wilson Disease: Overview

Wilson’s Disease is characterized by excess copper stored in various body tissues, particularly the liver, brain, and corneas of the eyes. Wilson’s disease (WD) is a rare genetic disorder beginning with liver dysfunction where damage begins by 6 years of age, but it is usually present clinically in the teenage years or early 20s. Wilson disease is inherited as an autosomal recessive trait. A mutation in the ATP7B gene, which codes for copper transportation, causes WD.

Common signs of associated liver disease include a yellow discoloration (jaundice) of the skin, mucous membranes and the membranes (sclera) that line the eye, swelling (edema) of the legs and abdomen (ascites) due to abnormal retention of fluid, presence of abnormal blood vessels in the esophagus that may bleed (esophageal varices), a tendency for bruising and prolonged bleeding, and excessive tiredness (fatigue). Some individuals with Wilson disease may have only abnormalities of liver function test and may show no other symptoms until many years later.

The genetic defect in Wilson disease impairs copper transport. The impaired transport decreases copper secretion into the bile, thus causing the copper overload and resultant accumulation in the liver, which begins at birth. The impaired transport also interferes with incorporation of copper into the copper protein ceruloplasmin, thus decreasing serum levels of ceruloplasmin.

Hepatic fibrosis develops, ultimately causing cirrhosis. Copper diffuses out of the liver into the blood, then into other tissues. It is most destructive to the brain but also damages the kidneys and reproductive organs and causes hemolytic anemia. Some copper is deposited around the rim of the cornea and edge of the iris, causing Kayser-Fleischer rings. The rings appear to encircle the iris.

Wilson disease may be diagnosed based upon a thorough clinical evaluation, a complete patient history, and specialized tests. Such tests may include slit-lamp examination of the eyes that reveals the presence of Kayser-Fleischer rings; tests of the fluid portion of the blood (serum) that demonstrate low levels of ceruloplasmin, a copper protein; and tests that reveal abnormally high levels of copper excreted in the urine. Besides imaging and biochemical markers, genotyping is also a potential tool for selecting treatment for WD patients.

Recent mapping studies have shown that the ATP7B mutation shows geographical variance. Treatment may be divided into three parts: first, treatment of symptomatic patients, second, maintenance therapy after copper has been reduced in affected tissues, and third, in asymptomatic patients, maintenance therapy may be used from the beginning. Treatment for Wilson disease includes three types of medications. First those that remove (chelate) copper from the body by urinary excretion such as penicillamine (Cuprimine) and trientine dihydrochloride (Syprine), second, zinc salts to prevent the gut from absorbing copper from the diet, and third, tetrathiomolybdate which both prevents absorbing copper and binds up toxic copper in the blood making it nontoxic.

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

Wilson Disease Emerging Drugs Chapters

This segment of the Wilson Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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.

Wilson Disease Emerging Drugs

VTX 801: Vivet Therapeutics

VTX-801 is a novel, investigational rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson Disease. VTX-801’s rAAV serotype was selected based on its demonstrated tropism for transducing human liver cells. VTX-801 is a novel investigational gene therapy for Wilson Disease, which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC) and Fast Track designation by the FDA. Currently, the drug is in the Phase I/II stage of its development for the treatment of Wilson Disease

Wilson Disease: Therapeutic Assessment

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

Major Players in Wilson Disease

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

Phases

The 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

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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

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

Wilson Disease: Pipeline Development Activities

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

Wilson Disease Report Insights

• Wilson Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Vivet therapeutics
• Depymed Inc
• Prime Medicine
• Deep genomics

Key Products

• VTX-801
• DPM 1001
• Research programme hepatolenticular degeneration gene therapies
• DG 12P1

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