SCH-39166 · program
Ecopipam for Tourette syndrome
- Orphan Drug
- Fast Track
Pediatric Tourette syndrome is the lead indication for ecopipam. The pivotal Phase 3 randomized-withdrawal study (D1AMOND, NCT05615220) met its primary endpoint - delayed time to relapse on the YGTSS Total Tic Score - in pediatric patients (HR=0.5, p=0.0084) and in the combined pediatric+adult population (HR=0.5, p=0.0050); results were published in JAMA Neurology on 2026-05-26. Supported by an earlier Phase 2b study (NCT04007991). FDA Orphan Drug and Fast Track designations. The U.S. NDA (for pediatric Tourette syndrome) was submitted by Teva on 2026-06-18, moving the program to 'filed'.
Development timeline
- metD1AMOND Phase 3 met primary and secondary endpoints: ecopipam delayed time to relapse vs placebo in pediatric (HR=0.5, p=0.0084) and combined (HR=0.5, p=0.0050) Tourette populations; published in JAMA Neurology.↗
- metPhase 3 randomized-withdrawal full results published in JAMA Neurology: ecopipam halved relapse risk (pediatric HR 0.47, p=.008)↗
- D1AMOND Phase 3 completed (primary completion 2025-01-13; study completion 2025-02-04); met primary and secondary endpoints.
- Pivotal Phase 3 randomized-withdrawal study D1AMOND (NCT05615220) started (first patient dosed).
- Phase 2b in children/adolescents with Tourette syndrome (NCT04007991, n=153) completed (primary completion 2021-09-23).
Readouts
- Late Q1 2027AnticipatedRegulatory
FDA PDUFA action on ecopipam NDA targeted late Q1 2027 (Priority Review) ↗
- 2026-06-18ReportedRegulatory
Teva submitted the U.S. NDA for ecopipam in pediatric Tourette syndrome (guided for 2H 2026; submitted 2026-06-18). ↗
- 2026-05-26ReportedTopline datametNCT05615220
D1AMOND Phase 3 met primary and secondary endpoints: ecopipam delayed time to relapse vs placebo in pediatric (HR=0.5, p=0.0084) and combined (HR=0.5, p=0.0050) Tourette populations; published in JAMA Neurology. ↗
- 2026-05-26ReportedFull resultsmet
Phase 3 randomized-withdrawal full results published in JAMA Neurology: ecopipam halved relapse risk (pediatric HR 0.47, p=.008) ↗
Clinical trials in Tourette syndrome
NCT05615220D1AMONDPhase 3Completedn=216
A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder (D1AMOND)
metprimaryTime from randomization to relapse during the 12-week double-blind withdrawal period (pediatric, ages 6-18; relapse = >=50% loss of YGTSS-TTS improvement, additional TD medication, or hospitalization) — HR=0.5 (0.0084)
Ecopipam significantly delayed time to relapse vs placebo in the pediatric population (HR=0.5, p=0.0084) - a ~50% relapse-risk reduction. All 216 participants (167 pediatric + 49 adult) first received 12 weeks open-label ecopipam; 104 responders (90 pediatric) were then randomized to continue ecopipam or switch to placebo.
metsecondaryTime to relapse during double-blind withdrawal (combined pediatric + adult population) — HR=0.5 (0.0050)
Ecopipam significantly delayed time to relapse vs placebo in the combined pediatric+adult population (HR=0.5, p=0.0050). Common adverse events: somnolence, insomnia, anxiety, fatigue, headache.
NCT04007991Phase 2Completedn=153
Multicenter, Placebo-Controlled, Double-Blind, Randomized, Parallel-Group, Phase 2b Study to Evaluate the Efficacy and Safety of Ecopipam in Children and Adolescents With Tourette's Syndrome
Formulations
| Formulation | Route | Regimen | Pharmacokinetics |
|---|---|---|---|
| Ecopipam oral tablet (ecopipam HCl) Immediate-release oral tablets of ecopipam hydrochloride, taken once daily in the evening, titrated by dose (Phase 3 tablet strengths 11.2, 22.4, 33.6, 44.8, 67.2, 89.6 mg base = 12.5, 25, 37.5, 50, 75, 100 mg ecopipam HCl); pediatric target ~2 mg/kg/day. | Oral | Once nightly | t½ 15.8 h · Tmax 1.51 h |
Mechanism of action
Selective antagonist of the dopamine D1-like receptor family (D1 and D5), with markedly lower affinity for D2-like (D2, D4) and serotonergic receptors. This D1/D5-selective mechanism is distinct from the D2-receptor blockade of antipsychotic-based tic therapies and is the basis for its first-in-class designation in Tourette syndrome.
| Target | Action | Affinity |
|---|---|---|
| D1primaryDRD1 | Antagonist | pKi 8.3ⓘ |
| D5DRD5 | Antagonist | pKi 8.3ⓘ |
← Full SCH-39166 compound page (identity, identifiers, all indications)
Sources
- Ecopipam Ligand Activity Charts (D1/D5 affinity) - IUPHAR/BPS GtoPdb — IUPHAR/BPS Guide to PHARMACOLOGY
- Ecopipam Meets Primary and Secondary End Points in Phase 3 Study for Tourette Syndrome — NeurologyLive
- Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents (Phase 2b, NCT04007991) — ClinicalTrials.gov
- Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults (D1AMOND) — ClinicalTrials.gov
- Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial — JAMA Neurology
- Efficacy and Safety of Ecopipam for Tourette Syndrome: Results from a Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Trial — American Academy of Neurology
- Emalex Biosciences Acquires Psyadon Pharmaceuticals to Develop Novel Treatment for Pediatric Tourette Syndrome — Emalex Biosciences / PR Newswire
- JAMA Neurology Publishes Phase 3 Data on D1 Receptor Antagonist Ecopipam in Tourette Syndrome — Businesswire (Emalex/Teva)
- Teva Closes Acquisition of Emalex Biosciences, Strengthening Late-Stage Neuroscience Pipeline and Advancing Pivot to Growth Strategy — Teva Pharmaceutical Industries
- Teva Submits NDA for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Tourette Syndrome — Teva Pharmaceutical Industries
- Teva to Acquire Emalex Biosciences, Adding NDA-Ready, First-in-Class Therapy to Neuroscience Pipeline and Accelerating Teva's Pivot to Growth Strategy — Teva Pharmaceutical Industries
- U.S. FDA Accepts Teva's New Drug Application (NDA) and Grants Priority Review for Ecopipam — Teva Pharmaceutical Industries