Encouraging clinical trials in Wilson’s disease



Los Angeles, United States, October 26, 2021 (GLOBE NEWSWIRE) – Encouraging Wilson Disease Clinical Trials Strengthen Pipeline With Multiple Drugs In Pipeline, Says DelveInsight

Several new therapies are under development for the treatment of Wilson’s disease. Among them, ALXN1840 and UX701 are at their most advanced stage (Phase III).

“DelveInsight”Wilson’s disease pipeline overview”Report provides comprehensive information on 6+ companies and 6+ pipeline drugs in Wilson’s disease pipeline landscapes. It includes drug profiles from the Wilson Disease pipeline, including clinical and non-clinical stage products. It also includes the therapeutic evaluation of Wilson’s disease by product type, stage, route of administration and molecule type and highlights inactive products in the Wilson disease pipeline.

Some of the main takeaways from the Wilson’s disease pipeline report

  • Large 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’s disease treatment scenario.
  • In September 2021, the US FDA accepted Orphalan’s New Drug Application (NDA) review for trientine tetrachlorhydrate (TETA 4HCl) for the first-line treatment of Wilson’s disease. TETA 4-HCl has also achieved orphan drug designation for the first-line treatment of Wilson’s disease.
  • ALXN1840 is a potential new oral once-daily drug in development for the treatment of Wilson’s disease. It has received orphan drug designation in the US and the EU for Wilson’s disease.
  • In August 2021, Vivet Therapeutics and Pfizer announced that the US FDA had granted Fast Track designation to VTX-801 to treat Wilson’s disease.
  • UX701 is an investigational AAV type 9 gene therapy designed to deliver stable expression of the copper transporter ATP7B after a single intravenous infusion. In December 2020, the US FDA granted orphan drug designation to UX701 for the treatment of Wilson’s disease
  • In January 2021, the US FDA cleared the Investigational New Drug Application (IND) for UX701, an investigational AAV9 gene therapy under evaluation for the treatment of Wilson’s disease. It is currently being evaluated in phase III of development.
  • In April 2018, Alexion struck a deal to buy Stockholm-based Wilson Therapeutics for $ 855 million in cash. The deal added a rare disease phase III drug WTX101 to its pipeline – marking the new leadership team’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 evaluating Vivet’s proprietary investigational gene therapy, VTX-801, for the treatment of Wilson’s disease.

Get an overview of the pipeline landscape @ Wilson’s disease clinical trial review

Wilson’s disease is a rare genetic disorder characterized by excess copper stored in various body tissues, particularly the liver, brain, and cornea of ​​the eyes.

Emerging drugs for Wilson’s disease

  • 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’s disease, a rare, chronic, genetic and life-threatening liver disease caused by impaired transport of copper. The registration of participants was carried out in a Phase III study of ALXN1840 in Wilson’s disease.

  • UX701: Ultragenyx Pharmaceutical

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

For more information, refer to the detailed report @ Wilson’s disease pipeline therapeutics

Scope Wilson’s disease pipeline drug overview

  • Blanket: Global
  • Major players: 6+ key players
  • Leading players: Alexion Pharmaceuticals, Vivet Therapeutics, Ultragenyx Pharmaceutical, Deep Genomics, Generation Bio, DepYmed, IncNobelpharma, Orphalan, and many more.
  • Key Drug Profiles: 6+ Products
  • Steps :

Advanced stage Wilson’s disease therapies (phase III)
Intermediate-stage Wilson’s disease therapies (phase II)
Early stage Wilson’s disease therapies (phase I)
Preclinical stage of Wilson’s disease and candidates for discovery
Abandoned and inactive candidates

ATPase replacements carrying copper
Chelating agents
Gene transfer
Non-receptor type 1 antagonists of protein tyrosine phosphatase

Bispecific antibody
Small molecules
· Genetical therapy

· Subcutaneous
· Intravenous
· Intramuscular

· Combination
Mono / Combination

Key questions about the current Wilson’s disease treatment landscape and emerging therapies addressed in the pipeline report

  • What are the current options for the treatment of Wilson’s disease?
  • How many companies are developing therapies for the treatment of Wilson’s disease?
  • How many emerging Wilson’s disease therapies in the early, middle and late stages of development are there to treat Wilson’s disease?
  • What are the key collaborations (industry-industry, industry-university), mergers & acquisitions, and significant licensing activities that will impact the Wilson Disease market?
  • What are dormant and abandoned products and the reasons for them?
  • What is the unmet need for current therapies for the treatment of Wilson’s disease?
  • What are the new treatments, targets, mechanisms of action and technologies currently being developed to overcome the limitations of existing treatments for Wilson disease?
  • What are the critical designations that have been given to emerging therapies for Wilson’s disease?
  • How many patents are granted and pending for emerging therapies to treat Wilson’s disease?


1 Presentation of the Wilson disease report
2 Summary of Wilson’s disease
3 Overview of Wilson’s disease
4 Wilson’s disease pipeline therapeutics
5 Therapeutic evaluation of Wilson’s disease
6 Wilson’s Disease – In-depth Business Assessment from DelveInsight’s Analytical Perspective
7 Advanced Wilson’s disease products (phase III)
7.1 UX701: Ultragenyx Pharmaceutical
8 Mid-stage Wilson’s disease products (phase II)
8.1 Drug name: company name
9 Early stage Wilson’s disease products (Phase I / II)
9.1 VTX-801: Therapeutic Vivet
ten Wilson’s disease preclinical and discovery stage products
11 Inactive products for Wilson’s disease
12 Wilson’s disease Key companies
13 Key Wilson’s disease products
14 Wilson’s disease Unmet needs
15 Wilson’s Disease Market Drivers and Obstacles
16 Future prospects and conclusion of Wilson’s disease
17 Wilson’s Disease Analysts Opinions
18 Annex
19 About DelveInsight

Get a Custom Pipeline Report @ Wilson’s Disease Drug Pipeline Report

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