Down Syndrome drug pipeline
1 compound from 1 company is in active clinical development for down syndrome.
1Compound
1Company
0Phase 3+
1Upcoming readout
1Trials recruiting
- Phase 2 1
Mechanisms: Small molecule (1)
Pipeline data compiled July 2026 · every fact sourced.
Upcoming readouts & recent results
Phase 2b dose-finding trial of AEF0217 in Down syndrome (NCT07334912): preliminary results anticipated in 2027. ↗
Positive Phase 1/2 in young adults with Down syndrome: safety primary met; AEF0217 0.2 mg improved several VABS adaptive-behavior domains and modified a CB1-dependent EEG target-engagement marker vs placebo. ↗
Compounds in development (1)
| Compound | Company | Phase | Status | Mechanism |
|---|---|---|---|---|
| AEF0217 | Aelis Farma S.A. | Phase 2 | Recruiting | CB1 NAM |
Trials recruiting now
2 registered interventional down syndrome trials — none recruiting at present. Browse all on ClinicalTrials.gov →
Frequently asked
- How many drugs are in development for down syndrome?
- 1 compound from 1 company is in active clinical development for down syndrome.
- What are the next down syndrome trial readouts?
- Upcoming catalysts include AEF0217 (2027).