Rett Syndrome - Pipeline Insight, 2022

Rett Syndrome - Pipeline Insight, 2022

DelveInsight’s, “Rett Syndrome - Pipeline Insight, 2022,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Rett Syndrome 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

Rett Syndrome Understanding

Rett Syndrome: Overview

Rett syndrome is a rare genetic neurological disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements. Rett syndrome is usually recognized in children between 6 to 18 months as they begin to miss developmental milestones or lose abilities they had gained. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 900 different mutations found on the MECP2 gene. Most of these mutations are found in eight different “hot spots.” Rett syndrome is not a degenerative disorder with individuals living to middle age or beyond. Rett syndrome occurs worldwide in 1 of every 10,000 female births, and is even rarer in boys. Rett syndrome can present with a wide range of disability ranging from mild to severe. The course and severity of Rett syndrome is determined by the location, type and severity of the mutation and X-inactivation.

""Rett Syndrome - Pipeline Insight, 2022"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Rett Syndrome pipeline landscape is provided which includes the disease overview and Rett Syndrome treatment guidelines. The assessment part of the report embraces, in depth Rett Syndrome 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, Rett Syndrome 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 Rett Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Rett Syndrome.

Rett Syndrome Emerging Drugs Chapters

This segment of the Rett Syndrome 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.

Rett Syndrome Emerging Drugs

Trofinetide: Neuren Pharmaceuticals

Trofinetide (previously designated by Neuren as NNZ-2566) and NNZ-2591 are patented synthetic analogs of molecules which are derived from IGF-1 and occur naturally in the brain. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease. . The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted Orphan Drug designation to Neuren for trofinetide in Rett syndrome. The FDA has also granted Fast Track designation. The drug also has a Rare Pediatric Disease designation. Neuren's North American partner for trofinetide, ACADIA Pharmaceuticals, has announced positive top-line results from a Phase III trial in Rett syndrome.Neuren Pharmaceuticals reported that the US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) of trofinetide for the treatment of Rett syndrome, that was submitted by Neuren’s US partner

Acadia Pharmaceuticals (Nasdaq: ACAD). The FDA had granted a Priority Review and assigned a

Prescription Drug User Fee Act (PDUFA) action date of 12 March 2023.

ANAVEX2-73 (blarcamesine): Anavex Life Sciences

ANAVEX2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. The drug is in phase III stage of clinical trial evaluation for the treatment of patients with Rett syndrome.

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

Rett Syndrome: Therapeutic Assessment

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

Major Players in Rett Syndrome

There are approx. 20+ key companies which are developing the therapies for Rett Syndrome. The companies which have their Rett Syndrome drug candidates in the most advanced stage, i.e. Preregistration include,Neuren Pharmaceuticals.


DelveInsight’s report covers around 20+ 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

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






Molecule Type

Products have been categorized under various Molecule types such as

Monoclonal Antibody



Small molecule

Gene therapy

Product Type

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

Rett Syndrome: Pipeline Development Activities

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

Rett Syndrome Report Insights

Rett Syndrome Pipeline Analysis

Therapeutic Assessment

Unmet Needs

Impact of Drugs

Rett Syndrome 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 Rett Syndrome drugs?

How many Rett Syndrome drugs are developed by each company?

How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Rett Syndrome?

What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Rett Syndrome 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 Rett Syndrome and their status?

What are the key designations that have been granted to the emerging drugs?

Key Players

Neuren Pharmaceuticals

Anavex Life Sciences

Edison Pharmaceuticals


Ultragenyx Pharmaceutical

GW Pharmaceuticals

Q-State Biosciences


AMO Pharma


Biohaven Pharmaceuticals

Echo Pharmaceuticals

Vyant Bio

Prilenia Therapeutics


Relmada Therapeutics

VICO Therapeutics

Taysha Gene Therapies

Sarepta therapeutics

Key Products





REL 1017



STRX 230

Research programme: Rett syndrome therapeutics

AMO 04


NLX 101

BHV 5000

Executive Summary
Rett Syndrome: Overview
Mechanism of Action
Signs and Symptoms
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
Rett Syndrome – DelveInsight’s Analytical Perspective
Late Stage Products (Preregistration)
Comparative Analysis
Trofinetide: Neuren Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Late Stage Products (Phase III)
Comparative Analysis
Blarcamesine: Anavex Life Sciences
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Comparative Analysis
Drug Name: Company Name
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
Comparative Analysis
BHV 5000: Biohaven Pharmaceuticals
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Preclinical and Discovery 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
Rett Syndrome Key Companies
Rett Syndrome Key Products
Rett Syndrome- Unmet Needs
Rett Syndrome- Market Drivers and Barriers
Rett Syndrome- Future Perspectives and Conclusion
Rett Syndrome Analyst Views
Rett Syndrome Key Companies

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