SCH-39166 · program

Ecopipam for Tourette syndrome

Filed (NDA)ActiveTeva Pharmaceutical Industries Ltd. (TEVA)
  • 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'.

Data current as of

Development timeline

Filed (NDA)Jun 2026
  1. Teva submitted the U.S. NDA for ecopipam in pediatric Tourette syndrome (guided for 2H 2026; submitted 2026-06-18).↗
Phase 3Jan 2023 – May 2026
  1. 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.↗
  2. D1AMOND Phase 3 completed (primary completion 2025-01-13; study completion 2025-02-04); met primary and secondary endpoints.
  3. Pivotal Phase 3 randomized-withdrawal study D1AMOND (NCT05615220) started (first patient dosed).
Phase 2Sept 2021
  1. Phase 2b in children/adolescents with Tourette syndrome (NCT04007991, n=153) completed (primary completion 2021-09-23).

Readouts

  • 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. ↗

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)

Started Jan 2023· Primary completion Jan 2025· 📍 95 sites across 12 countries (United States, Spain, Italy, Poland)

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

Started Jun 2019· Primary completion Sept 2021· 📍 70 sites across 5 countries (United States, Poland, Germany, Canada)

Formulations

FormulationRouteRegimenPharmacokinetics
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.
OralOnce nightlyt½ 15.8 h · Tmax 1.51 h

Mechanism of action (compound-wide)

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.

TargetActionAffinity
D1 receptorprimaryDRD1AntagonistpKi 8.3ⓘ
D5 receptorDRD5AntagonistpKi 8.3ⓘ

← Full SCH-39166 compound page (identity, identifiers, all indications)

Sources

  1. Ecopipam Ligand Activity Charts (D1/D5 affinity) - IUPHAR/BPS GtoPdb — IUPHAR/BPS Guide to PHARMACOLOGY
  2. Ecopipam Meets Primary and Secondary End Points in Phase 3 Study for Tourette Syndrome — NeurologyLive
  3. Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents (Phase 2b, NCT04007991) — ClinicalTrials.gov
  4. Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults (D1AMOND) — ClinicalTrials.gov
  5. JAMA Neurology Publishes Phase 3 Data on D1 Receptor Antagonist Ecopipam in Tourette Syndrome — Businesswire (Emalex/Teva)
  6. Teva Submits NDA for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Tourette Syndrome — Teva Pharmaceutical Industries (IR)