Encouraging Clinical Trials of Wilson Disease Make the Pipeline Robust With Several Pipeline Drugs, States DelveInsight

·5 min read

Several novel therapies are being developed for the Wilson disease treatment. Among them, ALXN1840 and UX701 are in their most advanced stage (Phase III).

Los Angeles, USA, Oct. 26, 2021 (GLOBE NEWSWIRE) -- Encouraging Clinical Trials of Wilson Disease Make the Pipeline Robust With Several Pipeline Drugs, States DelveInsight

Several novel therapies are being developed for the Wilson disease treatment. Among them, ALXN1840 and UX701 are in their most advanced stage (Phase III).

DelveInsight’s “Wilson Disease Pipeline Insight” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in the Wilson Disease pipeline landscapes. It comprises Wilson Disease pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Wilson Disease therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Wilson Disease pipeline products.

Some of the key takeaways from the Wilson Disease Pipeline Report

  • Major companies such as Alexion Pharmaceuticals, Vivet Therapeutics, Ultragenyx Pharmaceutical, Deep Genomics, Generation Bio, DepYmed, IncNobelpharma, Orphalan, and others are developing potential drug candidates to improve the Wilson Disease treatment scenario.

  • In September 2021, the US FDA accepted for review the Orphalan’s New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the first-line treatment of Wilson’s Disease. TETA 4-HCl has also been granted Orphan Drug Designation for first-line treatment of Wilson’s Disease.

  • ALXN1840 is a potential new once-daily oral medicine in development for the treatment of Wilson disease. It has been granted Orphan Drug Designation in the US and EU for Wilson disease.

  • In August 2021, Vivet Therapeutics and Pfizer announced the US FDA had granted Fast Track designation to VTX-801 to treat Wilson disease.

  • UX701 is an investigational AAV type 9 gene therapy designed to deliver a stable ATP7B copper transporter expression following a single intravenous infusion. In December 2020, the US FDA granted Orphan Drug Designation to UX701 for the treatment of Wilson disease

  • In January 2021, US FDA cleared the Investigational New Drug (IND) application for UX701, an investigational AAV9 gene therapy being evaluated for the Wilson Disease treatment. It is currently being evaluated in the Phase III stage of development.

  • In April 2018, Alexion struck a deal to buy Stockholm-based Wilson Therapeutics for $855 million in cash. This deal added a Phase III drug WTX101 for a rare disease to its pipeline — marking the new executive crew’s “first step” in reorganizing the pipeline.

  • In September 2021, Vivet Therapeutics entered into a manufacturing agreement. Pfizer will offer clinical supply for a Phase I/II clinical trial assessing Vivet’s proprietary, investigational gene therapy, VTX-801, for the Wilson disease treatment.

Get an overview of pipeline landscape @ Wilson Disease Clinical Trials Analysis

Wilson Disease is a rare genetic disorder characterized by excess copper stored in various body tissues, particularly the liver, brain, and eyes’ corneas.

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 has been completed in a Phase III study of ALXN1840 in Wilson disease.

  • UX701: Ultragenyx Pharmaceutical

UX701 is an investigational adeno-associated virus type 9 gene therapy that works by stabilizing the expression of the ATP7B copper transporter after a single intravenous infusion. In preclinical studies, UX701 has been shown to normalize copper trafficking and excretion from the body. UX701 was granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States. A randomized, double-blind, placebo-controlled Phase III study has been initiated to treat patients with Wilson disease.

For further information, refer to the detailed report @ Wilson Disease Pipeline Therapeutics

Scope of Wilson Disease Pipeline Drug Insight

  • Coverage: Global

  • Major Players: 6+ Key Players

  • Prominent Players: Alexion Pharmaceuticals, Vivet Therapeutics, Ultragenyx Pharmaceutical, Deep Genomics, Generation Bio, DepYmed, IncNobelpharma, Orphalan, and many others.

  • Key Drugs Profiles: 6+ Products

  • Phases:

· Wilson Disease Therapies Late-stage (Phase III)
· Wilson Disease Therapies Mid-stage (Phase II)
· Wilson Disease Therapies Early-stage (Phase I)
· Wilson Disease Preclinical stage and Discovery candidates
· Discontinued and Inactive candidates

  • Mechanism of Action:

· Copper-transporting ATPase replacements
· Chelating agents
· Gene transference
· Protein tyrosine phosphatase non-receptor type 1 antagonists

  • Molecule Types:

· Peptides
· Bispecific Antibody
· Small molecules
· Gene therapy

  • Route of Administration:

· Subcutaneous
· Intravenous
· Intramuscular
· Oral

  • Product Types:

· Monotherapy
· Combination
· Mono/Combination

Key Questions regarding Current Wilson Disease Treatment Landscape and Emerging Therapies Answered in the Pipeline Report

  • What are the current options for Wilson Disease treatment?

  • How many companies are developing therapies for the treatment of Wilson Disease?

  • How many are Wilson Disease emerging therapies in the early-stage, mid-stage, and late development stages to treat Wilson Disease?

  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Wilson Disease market?

  • Which are the dormant and discontinued products and the reasons for the same?

  • What is the unmet need for current therapies for the treatment of Wilson Disease?

  • What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Wilson Disease therapies?

  • What are the critical designations that have been granted for the emerging therapies for Wilson Disease?

  • How many patents are granted and pending for the emerging therapies to treat Wilson Disease?

Table of Contents

1

Wilson Disease Report Introduction

2

Wilson Disease Executive Summary

3

Wilson Disease Overview

4

Wilson Disease Pipeline Therapeutics

5

Wilson Disease Therapeutic Assessment

6

Wilson Disease – DelveInsight’s Analytical Perspective In-depth Commercial Assessment

7

Wilson Disease Late Stage Products (Phase III)

7.1

UX701: Ultragenyx Pharmaceutical

8

Wilson Disease Mid Stage Products (Phase II)

8.1

Drug Name: Company Name

9

Wilson Disease Early Stage Products (Phase I/II)

9.1

VTX-801: Vivet therapeutics

10

Wilson Disease Preclinical and Discovery Stage Products

11

Wilson Disease Inactive Products

12

Wilson Disease Key Companies

13

Wilson Disease Key Products

14

Wilson Disease Unmet Needs

15

Wilson Disease Market Drivers and Barriers

16

Wilson Disease Future Perspectives and Conclusion

17

Wilson Disease Analyst Views

18

Appendix

19

About DelveInsight

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About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.

CONTACT: Contact Us: Shruti Thakur info@delveinsight.com +1(919)321-6187 www.delveinsight.com


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