Rett Syndrome - Pipeline Insight, 2024

This “Rett Syndrome - Pipeline Insight, 2024” 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 neurodevelopmental disorder characterized by a regression of previously acquired skills following a period of typical development. This syndrome presents with a variety of symptoms, including deceleration in head growth, gait abnormalities, loss of purposeful hand movements often replaced by repetitive stereotypical movements like hand-wringing, as well as loss of speech and breathing abnormalities. Rett syndrome is associated with a complex phenotype and is classified into typical, atypical, and variant presentations. Around 90% of reported cases of Rett syndrome inherit mutations in the methyl-CpG-binding protein 2 (MECP2) gene, while some atypical cases may result from mutations in cyclin-dependent kinase-like 5 (CDKL5). Mutations in MECP2 have been linked to impacting the development of neurons and axodendritic connections, contributing to the diverse symptoms observed in individuals with Rett syndrome.

Rett syndrome is an X-linked dominant disorder caused primarily by mutations in the MECP2 gene, which encodes a protein responsible for transcriptional regulation. While most cases are associated with MECP2 mutations, some atypical cases result from mutations in CDKL5 or FOXG1. The severity of Rett syndrome symptoms varies depending on the type and location of the MECP2 mutation, with early truncating mutations and those affecting the nuclear localization signal (NLS) region leading to a more severe phenotype compared to missense mutations. The R133C mutation is typically linked to a milder variant with preserved speech. MECP2 mutations disrupt the protein's methyl-binding domain and transcription repression domain, altering gene expression and contributing to the complex Rett syndrome phenotype.

Rett syndrome is characterized by a range of signs and symptoms that typically emerge after a period of apparently normal development in the first 6-18 months of life. The most common symptoms include deceleration of head growth, loss of purposeful hand movements often replaced by repetitive stereotypical hand movements like hand-wringing, loss of speech, and gait abnormalities. Other symptoms may include breathing irregularities such as apnea, hyperventilation, and breath-holding, seizures, intellectual disability, scoliosis, and cardiac dysfunction. Unusual eye movements, irritability, anxiety, and sleep disturbances are also frequently observed in individuals with Rett syndrome.

Treatment for Rett syndrome involves a multidisciplinary approach, including physical, occupational, and speech therapy to improve mobility, hand function, and communication. Medications may be prescribed to manage seizures, breathing problems, and other symptoms. Assistive devices, nutritional support, and regular monitoring for complications are crucial.

"Rett Syndrome - Pipeline Insight, 2024" report 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 III, II/III, 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

ANAVEX2-73: Anavex Life Sciences

Blarcamesine (also known as ANAVEX2-73) is a small molecule, orally administered drug that restores cellular homeostasis by targeting SIMGAR1 and muscarinic receptors that is pivotal to restoring cellular homeostasis and promoting neuroplasticity. The drug candidate has received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA. The drug is currently being evaluated under Phase III clinical trial for the treatment of patients with Rett Syndrome.

NGN-401: Neurogene

NGN-401 is an investigational AAV9 gene therapy being developed as a one line treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT technology. The drug is currently being evaluated under Phase I/II clinical trial for the treatment of patients with Rett Syndrome.

GXV-001: GEXVal

GXV-001 is being developed by GEXVal. The drug candidate is used for the treatment of several neurological disorders, including rare diseases. The drug is currently being evaluated under Phase I clinical trial for the treatment of patients with Rett Syndrome.

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. Phase III include, Anavex Life Sciences.

Phases

The 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:

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as:

• Monoclonal antibody
• Small molecule
• Peptide

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

• ANAVEX2-73
• GEXVal
• Anavex Life Sciences
• AMO Pharma
• Unravel Biosciences
• Taysha Gene Therapies
• Alcyone Lifesciences
• Stoke Therapeutics
• Lucy Therapeutics
• TTI-0102

Key Products

• NGN-401
• GXV-001
• ANAVEX2-73
• AMO-04
• RVL-001
• TSHA-102
• ACTX-101
• Research programme
• Research programme
• Thiogenesis Therapeutics

This product will be delivered within 1-3 business days.