DAYBUE® (trofinetide)
Rett Syndrome
Key Facts
About Neuren Pharmaceuticals
Neuren Pharmaceuticals is a clinical-stage biotech focused on developing first-in-class treatments for severe neurodevelopmental disorders. Its core achievement is the FDA approval and commercialization of DAYBUE® for Rett syndrome, executed through a strategic partnership with Acadia Pharmaceuticals. The company's strategy leverages a proprietary neurotrophic factor platform to advance a pipeline led by NNZ-2591 into multiple high-need orphan indications, aiming to replicate its initial success and build a sustainable neurology franchise.
View full company profileAbout Neuren Pharmaceuticals
Neuren Pharmaceuticals is a clinical-stage biotech focused on developing first-in-class treatments for severe neurodevelopmental disorders. Its core achievement is the FDA approval and commercialization of DAYBUE® for Rett syndrome, executed through a strategic partnership with Acadia Pharmaceuticals. The company's strategy leverages a proprietary neurotrophic factor platform to advance a pipeline led by NNZ-2591 into multiple high-need orphan indications, aiming to replicate its initial success and build a sustainable neurology franchise.
View full company profileTherapeutic Areas
Other Rett Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| NTI164 | Neurotech International | Phase 2 |
| RTT1 | Grann Pharmaceuticals | Pre-clinical |
| NEX-1 / NEX-2 | Nexien BioPharma | Pre-clinical / Phase 1 |
| KIT-13 | NeuroCores | Pre-clinical |
| DPM-1003 | DepYmed | Phase 1 |
| ExoEdit® for Rett Syndrome | Evox Therapeutics | Pre-clinical |
| RVL-001 (vorinostat) | Unravel Biosciences | Clinical Trial Initiated |
| Rett Syndrome Program | Herophilus | Discovery |
| Glutamate Modulator for Rett Syndrome | Numedicus | Licensed/Orphan Designation |
| TTI-0102 | Thiogenesis Therapeutics | Phase 1 |
| TSHA-102 | Taysha Gene Therapies | Phase 1/2 |
| Sarizotan | Newron Pharmaceuticals | Phase III |