Wilson Disease Pipeline Insight

DelveInsight’s, “Wilson Disease – Pipeline Insight, 2022,” 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 disease is a rare genetic disorder characterized by excess copper stored in various body tissues, particularly the liver, brain, and corneas of the eyes. The disease is progressive and, if left untreated, it may cause liver (hepatic) disease, central nervous system dysfunction, and death. 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). Wilson disease is inherited as an autosomal recessive trait. Wilson disease is caused by disruption or changes (mutations) of the ATP7B gene, which plays an important role in the movement of excess copper from the liver to the bile to eventually be excreted from the body through the intestines. Treatment for Wilson disease is life-long and aimed at lowering copper levels to nontoxic levels, and at preventing the progression of the disease and trying to reverse any signs and symptoms that have appeared because of copper accumulation in the body.


"Wilson Disease - Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Wilson’s 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 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.


Wilson Disease Emerging Drugs


ALXN1840: Alexion Pharmaceuticals

ALXN1840 (bis-choline tetrathiomolybdate) is a novel oral copper-protein binding agent with a unique mechanism of action, under investigation for Wilson disease, a rare, chronic, genetic, and potentially life-threatening liver disorder of impaired copper transport. Enrollment of participants have been completed in a Phase 3 study of ALXN1840 in Wilson disease. The study results are expected in the first half of 2022.


TETA 4HCL: Orphalan

Triethylenetatramine (TETA) is a highly selective divalent Cu(II) chelator and a drug that revereses copper overload in tissues. Trientine is a copper chelator used in the treatment of Wilson's disease as an alternative to D-penicillamine. Clinical trials for TETA 4HCL are being carried out in phase III stage of development for the treatment of Wilsons disease.

Further product details are provided in the report……..

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 III include, Alexion Pharmaceuticals.

Phases

DelveInsight’s 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

  • Oral
  • Subcutaneous
  • Intravenous
  • Intramuscular
  • Molecule Type


Products have been categorized under various Molecule types such as

  • Bispecific Antibody
  • Peptides
  • Small molecule
  • Gene therapy
  • 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?

Introduction

Executive Summary

Wilson Disease: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Wilson Disease – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

  • Wilson Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Wilson Disease Collaboration Deals

  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Preregistration)

  • Comparative Analysis

ALXN1840: Alexion Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

Bis-Choline Tetrathiomolybdate: Alexion Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

VTX-801: Vivet Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical Stage Products

  • Comparative Analysis

Drug Name: Company name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Wilson Disease Key Companies

Wilson Disease Key Products

Wilson Disease- Unmet Needs

Wilson Disease- Market Drivers and Barriers

Wilson Disease- Future Perspectives and Conclusion

Wilson Disease Analyst Views

Wilson Disease Key Companies

Appendix

List of Table

Table 1: Total Products for Wilson Disease

Table 2: Late Stage Products

Table 3: Mid Stage Products

Table 4: Early Stage Products

Table 5: Pre-clinical & Discovery Stage Products

Table 6: Assessment by Product Type

Table 7: Assessment by Stage and Product Type

Table 8: Assessment by Route of Administration

Table 9: Assessment by Stage and Route of Administration

Table 10: Assessment by Molecule Type

Table 11: Assessment by Stage and Molecule Type

Table 12: Inactive Products

List of Figures

Figure 1: Total Products for Wilson Disease

Figure 2: Late Stage Products                              

Figure 3: Mid Stage Products

Figure 4: Early Stage Products

Figure 5: Preclinical and Discovery Stage Products

Figure 6: Assessment by Product Type

Figure 7: Assessment by Stage and Product Type

Figure 8: Assessment by Route of Administration

Figure 9: Assessment by Stage and Route of Administration

Figure 10: Assessment by Molecule Type

Figure 11: Assessment by Stage and Molecule Type

Figure 12: Inactive Products    

•  Alexion Pharmaceuticals

•  Vivet Therapeutics

•    Orphalan

•  Ultragenyx Pharmaceutical

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