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Rett Syndrome Clinical Trials Review: An Updated Status Report on Emerging Drugs by DelveInsight

DelveInsight Business Research LLP
·7-min read

The report offers the most up-to-date information on Rett Syndrome pipeline products being developed by notable companies such as Anavex Life Sciences Corp., GW Research Ltd, Neuren Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Edison Pharmaceuticals, Novartis, Neurolixis, Echo Pharmaceuticals, Relmada Therapeutics, and others.

Los Angeles, USA , April 19, 2021 (GLOBE NEWSWIRE) -- Rett Syndrome Clinical Trials Review: An Updated Status Report on Emerging Drugs by DelveInsight

The report offers the most up-to-date information on Rett Syndrome pipeline products being developed by notable companies such as Anavex Life Sciences Corp., GW Research Ltd, Neuren Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Edison Pharmaceuticals, Novartis, Neurolixis, Echo Pharmaceuticals, Relmada Therapeutics, and others.

DelveInsight’s “Rett Syndrome Pipeline Insight” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in the Rett Syndrome pipeline landscapes. It comprises Rett Syndrome pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Rett Syndrome therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Rett Syndrome pipeline products.

Some of the key takeaways of the Rett Syndrome Pipeline Report

  • There is no cure for Rett syndrome. Around 8+ key companies are developing Rett Syndrome therapies. These key players are Anavex Life Sciences Corp., GW Research Ltd, Neuren Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Edison Pharmaceuticals, Novartis, Neurolixis, Echo Pharmaceuticals, Relmada Therapeutics, and others have their Rett Syndrome drug candidates in the different phases of clinical development.

  • Trofinetide is a novel synthetic analogue of the amino‐terminal tripeptide of IGF-1 designed to treat the Rett Syndrome symptoms.

  • Acadia has an exclusive license for developing and commercialising trofinetide in North America from Neuren Pharmaceuticals. The drug is in Phase III clinical evaluation for the treatment of Rett Syndrome.

  • The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan Drug designation to Neuren for trofinetide in Rett Syndrome. The FDA has also accorded Fast Track designation.

  • In February 2020, GW Research initiated a phase III trial to investigate the long-term safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in patients with Rett Syndrome. The trial is anticipated to be finished by June 2022.

  • NLX-101 (previously known as F15599) is a Phase I drug intended to treat Rett's syndrome. It is a novel compound that activates serotonin 5-HT1A receptors with exceptional selectivity, having over 1000-fold higher affinity for this target over other receptors. Also, NLX-101 is a 'biased agonist' at 5-HT1A receptors, preferentially activating 5-HT1A receptors in those brain regions that control mood and cognition. The FDA and the EMA have awarded NLX-101 Orphan Drug Status.

Get an overview of pipeline landscape @ Rett Syndrome Clinical Trials Analysis

Rett Syndrome is a neurodevelopmental disorder, which affects girls almost exclusively. It is characterised by average early growth and development followed by a decrease of development, loss of purposeful use of the hands, distinctive hand movements, decreased brain and head growth, walking, seizures, and intellectual disability.

There is no cure for the disease. Rett Syndrome treatment is symptomatic, focusing on managing symptoms and supportive, needing a multidisciplinary approach. Medication may be required for breathing irregularities and motor difficulties, and anticonvulsant drugs may be utilised to control seizures. Occupational therapy can benefit children develop skills required for performing self-directed activities, while physical therapy and hydrotherapy may prolong mobility.

Rett Syndrome Emerging Drugs

  • Trofinetide by Neuren Pharmaceuticals

Trofinetide is a novel synthetic analogue of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett Syndrome by reducing neuroinflammation and supporting synaptic function.

Research and Development

Phase III

NCT04181723: ACADIA Pharmaceuticals, in November 2019, initiated a Phase III study titled “Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women with Rett Syndrome”. With an estimated 184 participants, the trial is currently in recruiting stage and expected to be completed by October 2021.

Neuren's North American partner for trofinetide, ACADIA Pharmaceuticals, has commenced a Phase 3 trial ("Lavender") in Rett syndrome. Neuren previously conducted a Phase 2 double-blind placebo-controlled clinical trial in girls aged 5 to 15 years with Rett syndrome. This followed positive trends observed in Neuren's earlier Phase 2 trial in adolescents and adults aged 16 to 45 years.

For further information, refer to the detailed report @ Rett Syndrome Pipeline Therapeutics

Scope of Rett Syndrome Pipeline Drug Insight

  • Coverage: Global

  • Major Players: 8+ Key Players

  • Prominent Players: Anavex Life Sciences Corp., GW Research Ltd, Neuren Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Edison Pharmaceuticals, Novartis, Neurolixis, Echo Pharmaceuticals, Relmada Therapeutics, and many others.

  • Key Drugs Profiles: 8+ Products

    • Phases:

      • Rett Syndrome Therapies Late-stage (Phase III)

      • Rett Syndrome Therapies Mid-stage (Phase II)

      • Rett Syndrome Therapies Early-stage (Phase I)

      • Rett Syndrome Pre-clinical stage and Discovery candidates

      • Discontinued and Inactive candidates

    • Mechanism of Action:

      • Sigma-1 receptor agonists

      • Cannabinoid receptor CB1, CB2 inverse agonists

      • Cytokine inhibitors

    • Molecule Types:

      • Stem cell therapy

      • Small molecule

      • Gene therapy

    • Route of Administration:

      • Inhalation

      • Intravenous

      • Oral

      • Subcutaneous

    • Product Types:

      • Monotherapy

      • Combination

      • Mono/Combination

Key Questions regarding Current Rett Syndrome Treatment Landscape and Emerging Therapies Answered in the Pipeline Report

  • What are the current options for Rett Syndrome treatment?

  • How many companies are developing therapies for the treatment of Rett Syndrome?

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

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

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

  • What are the critical designations that have been granted for the emerging therapies for Rett Syndrome?

  • How many patents are granted and pending for the emerging therapies to treat Rett Syndrome?

Table of Contents

1

Rett Syndrome Introduction

2

Rett Syndrome Executive Summary

3

Rett Syndrome Overview

4

Rett Syndrome- Analytical Perspective In-depth Commercial Assessment

5

Rett Syndrome Pipeline Therapeutics

6

Rett Syndrome Late Stage Products (Registered)

7

Rett Syndrome Late Stage Products (Phase III)

7.1

Trofinetide: Neuren Pharmaceuticals

7.2

GWP42003-P: GW Research Ltd.

8

Rett Syndrome Mid Stage Products (Phase II/III)

8.1

ANAVEX2-73 oral liquid: Anavex Life Sciences Corp.

9

Rett Syndrome Mid Stage Products (Phase II)

9.1

Triheptanoin: Ultragenyx Pharmaceutical Inc.

10

Rett Syndrome Early Stage Products (Phase I)

10.1

NLX 101: Neurolixis

11

Rett Syndrome Pre-clinical and Discovery Stage Products

12

Rett Syndrome Therapeutic Assessment

13

Rett Syndrome Inactive Products

14

Company-University Collaborations (Licensing/Partnering) Analysis

15

Rett Syndrome Key Companies

16

Rett Syndrome Key Products

17

Rett Syndrome Unmet Needs

18

Rett Syndrome Market Drivers and Barriers

19

Rett Syndrome Future Perspectives and Conclusion

20

Rett Syndrome Analyst Views

21

Appendix

22

About DelveInsight

Get a customised pipeline report @ Rett Syndrome Drugs Pipeline Report

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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.

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