Rett Syndrome - Pipeline Insight, 2022

DelveInsight’s, “Rett Syndrome - Pipeline Insight, 2022,” report provides comprehensive insights about 15+ companies and 15+ 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.

• ANAVEX2-73 (blarcamesine): Anavex Life Sciences
ANAVEX®2-73 (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 II/III 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. 15+ 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, Neuren Pharmaceuticals.

• Phases
DelveInsight’s report covers around 15+ 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
• Oral
• Parenteral
• intravenous
• Subcutaneous
• Topical.
• Molecule Type

Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• 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
• Novartis
• Ultragenyx Pharmaceutical
• GW Pharmaceuticals
• Q-State Biosciences
• AveXis
• AMO Pharma
• Neurolixis
• Biohaven Pharmaceuticals
• Echo Pharmaceuticals
• Vyant Bio
• Prilenia Therapeutics
• StrideBio
• Relmada Therapeutics

Key Products
• Trofinetide
• Blarcamesine
• EPI-743
• Fingolimod
• REL 1017
• Triheptanoin
• Cannabidivarin
• STRX 230
• Research programme: Rett syndrome therapeutics
• AVXS 201
• AMO 04
• Pridopidine
• NLX 101
• BHV 5000

Companies

• Neuren Pharmaceuticals
• Anavex Life Sciences
• Edison Pharmaceuticals
• Novartis
• Ultragenyx Pharmaceutical
• GW Pharmaceuticals
• Q-State Biosciences
• AveXis
• AMO Pharma
• Neurolixis
• Biohaven Pharmaceuticals
• Echo Pharmaceuticals
• Vyant Bio
• Prilenia Therapeutics
• StrideBio
• Relmada Therapeutics