Rett syndrome drug pipeline
- ICD-11 LD90.4
- MeSH D015518
1 compound from 1 company is in active clinical development for rett syndrome.
Mechanisms: Natural product (1)
Pipeline data compiled October 2026 · every fact sourced.
Upcoming readouts & recent results
ClinicalTrials.gov estimated primary completion of the Phase II/III placebo-controlled crossover study of NTI164 in Rett syndrome: October 2028 (not yet recruiting; estimated start July 2027). ↗
Open-label Phase I/II study of NTI164 in 11 girls with Rett syndrome published: improvement on CGI-I (p=0.028), CGI-S, RSBQ total score and caregiver burden after 12 weeks; well tolerated. ↗
Compounds in development (1)
| Compound | Company | Phase | Status | Mechanism |
|---|---|---|---|---|
| NTI164 | Neurotech International Limited | Phase 1/2 | Active | Natural product |
Trials recruiting now (7)
7 drug-interventional rett syndrome trials recruiting now, of 46 registered interventional trials. Browse all on ClinicalTrials.gov →
Bionetide, Placebo
fenfluramine hydrochloride
Lamivudine
Rett-T
mirtazapine
ION440
Vorinostat (SAHA), Placebo
Frequently asked
- How many drugs are in development for rett syndrome?
- 1 compound from 1 company is in active clinical development for rett syndrome.
- What are the next rett syndrome trial readouts?
- Upcoming catalysts include NTI164 (October 2028).
- How many clinical trials are recruiting for rett syndrome?
- 7 drug-interventional rett syndrome trials are currently recruiting participants, out of 46 registered interventional trials.