DelveInsight’s ‘Rett Syndrome Pipeline Insight 2023‘ report provides comprehensive global coverage of available, marketed, and pipeline Rett Syndrome therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Rett Syndrome pipeline domain.
For Rett Syndrome emerging drugs, the Rett Syndrome pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Key Takeaways from the Rett Syndrome Pipeline Report
- DelveInsight’s Rett Syndrome Pipeline analysis depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Rett Syndrome treatment.
- The leading Rett Syndrome companies include 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, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others are evaluating their lead assets to improve the Rett Syndrome treatment landscape.
- Key Rett Syndrome pipeline therapies in various stages of development include Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.
- In March 2023, Neuren Pharmaceuticals, based in Melbourne, announced that its North American partner Acadia Pharmaceuticals had received US Food and Drug Administration (FDA) approval for Neuren’s lead candidate Daybue (trofinetide) to treat Rett syndrome in adults and children.
- In February 2023, Unravel Biosciences, Inc., a therapeutics company that advances drugs for complex diseases by leveraging a machine-learned model of human health, announced the successful completion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) for its RVL001 program in Rett Syndrome.
- In February 2023, PharmaTher Holdings Ltd., a leader in specialty ketamine pharmaceuticals, announced that the United States Food and Drug Administration had designated KETARX (racemic ketamine) as an orphan drug for the treatment of Rett Syndrome, a rare genetic neurological disorder.
- In February 2023, Anavex Life Sciences Corp. announced the enrollment of 92 Rett syndrome patients in the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 study in Rett syndrome patients aged 5 to 17 years (inclusive).
- In January 2023, Neurogene Inc., a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced that the FDA has approved the Company’s Investigational New Drug (IND) application for NGN-401 to treat Rett syndrome.
Request a sample and discover the recent breakthroughs happening in the Rett Syndrome pipeline landscape @ Rett Syndrome Pipeline Outlook
Rett Syndrome Overview
Rett syndrome is a rare genetic neurological disorder that primarily affects girls and causes severe impairments in nearly every aspect of the child’s life, including their ability to speak, walk, eat, and even breathe easily. Near-constant repetitive hand movements distinguish Rett syndrome. Rett syndrome is typically diagnosed in children aged 6 to 18 months when they begin to miss developmental milestones or lose previously acquired abilities. Mutations on the X chromosome in a gene called MECP2 cause Rett syndrome. On the MECP2 gene, there are over 900 different mutations. Most of these mutations are found in eight distinct “hot spots.” Rett syndrome is not a degenerative disorder, with most people living into their forties or fifties. Rett syndrome affects 1 out of every 10,000 female births worldwide, and it is even rarer in boys. Rett syndrome can cause a wide range of disabilities, from mild to severe. The location, type, and severity of the mutation and X-inactivation determine the course and severity of Rett syndrome.
Find out more about Rett Syndrome medication @ New Drug for Rett Syndrome
Rett Syndrome Treatment Analysis: Drug Profile
Trofinetide: Neuren Pharmaceuticals
Trofinetide (previously known as NNZ-2566) and NNZ-2591 are patented synthetic analogs of molecules derived from IGF-1 that naturally occur in the brain. IGF-1 is a growth hormone-stimulated growth factor. IGF-1 is produced in the central nervous system by both types of brain cells, neurons, and glia. IGF-1 is essential in the brain for both normal development and responding to injury and disease. Neuren has received Orphan Drug designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for trofinetide in Rett syndrome. The FDA has also granted fast Track status. On 10 March 2023, it was approved by the FDA.
ANAVEX2-73 (blarcamesine): Anavex Life Sciences
ANAVEX2-73 (blarcamesine) activates the S1R protein, which functions as a molecular chaperone and functional modulator in the restoration of cellular homeostasis. The drug is currently in phase III clinical trials for the treatment of Rett syndrome patients.
Key Rett Syndrome Therapies and Companies
- Trofinetide: Neuren Pharmaceuticals
- ANAVEX2-73 (blarcamesine): Anavex Life Sciences
- Vatiquinone: PTC Therapeutics
- Fingolimod: Novartis
- Triheptanoin: Ultragenyx Pharmaceutical
- Cannabidivarin: GW Pharmaceuticals
- STRX 230: StrideBio
- AMO 04: AMO Pharma
- Pridopidine: Prilenia Therapeutics
- NLX 101: Neurolixis
- BHV 5000: Biohaven Pharmaceuticals
Learn more about the novel and emerging Rett Syndrome pipeline therapies @ Rett Syndrome Clinical Trials
Rett Syndrome Therapeutics Assessment
- By Product Type
- Monotherapy
- Combination Therapy
- By Stage
- Discovery
- Pre-Clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
- By Route of Administration
-
- Oral
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- By Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
Scope of the Rett Syndrome Pipeline Report
- Coverage: Global
- Key Rett Syndrome 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, VICO Therapeutics, Taysha Gene Therapies, Sarepta therapeutics, and others.
- Key Rett Syndrome Pipeline Therapies: Blarcamesine, EPI-743, Fingolimod, REL 1017, Triheptanoin, Cannabidivarin, STRX 230, Research programme: Rett syndrome therapeutics, AVXS 201, AMO 04, Pridopidine, NLX 101, BHV 5000, and others.
Dive deep into rich insights for drugs used for Rett Syndrome treatment; visit @ Rett Syndrome Drugs
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Rett Syndrome Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Rett Syndrome Pipeline Therapeutics |
| 6. | Rett Syndrome Pipeline: Late Stage Products (Phase III) |
| 7. | Rett Syndrome Pipeline: Late Stage Products (Phase III) |
| 8. | Rett Syndrome Pipeline: Mid Stage Products (Phase II) |
| 9. | Rett Syndrome Pipeline: Early Stage Products (Phase I) |
| 10. | Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the Rett Syndrome therapy in the pipeline, reach out @ Rett Syndrome Medication
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