Small Molecule · SCH-39166

Ecopipam

Filed (NDA)Emalex Biosciences Inc.
  • Orphan Drug
  • Fast Track

First-in-class, orally active, brain-penetrant selective dopamine D1/D5 receptor antagonist (a benzazepine, historically SCH-39166). Originally discovered in the CNS preclinical labs at Schering-Plough Corporation (now Merck) and previously studied in schizophrenia, substance use and obesity; redeveloped by Emalex Biosciences (development codes EBS-101 / PSYRX-101) for Tourette syndrome. Unlike marketed antipsychotic-based tic therapies that block D2 receptors, ecopipam selectively antagonizes the D1-like (D1/D5) receptor family. If approved it would be the first new mechanistic class for Tourette syndrome in decades. Emalex was acquired by Teva Pharmaceutical Industries (acquisition closed 2026-06-10); Teva submitted the U.S. NDA for pediatric Tourette syndrome on 2026-06-18.

Also known as: SCH-39166, EBS-101, PSYRX-101, Ecopipam, 112108-01-7

Data current as of

Key facts

Modality
Small molecule
Chemical class
benzazepine
Chemistry
Single enantiomer
Mechanism
D1 receptor antagonist
Highest phase
Filed (NDA)
Lead indication
Tourette syndrome
Developer
Emalex Biosciences Inc.
Designations
Orphan Drug, Fast Track
Trials
2 tracked · 165 sites
Next catalyst
Late Q1 2027 — Regulatory (Tourette syndrome)

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.

TargetActionAffinity
D1 receptorprimaryDRD1AntagonistpKi 8.3ⓘ
D5 receptorDRD5AntagonistpKi 8.3ⓘ

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

Development timeline#

2020202220242026TodayFiled (NDA)1 Jan 2027 — upcoming — FDA PDUFA action on ecopipam NDA targeted late Q1 2027 (Priority Review)19 Aug 2026 — FDA Priority Review granted — FDA grants Priority Review for the ecopipam NDA in pediatric Tourette syndrome19 Aug 2026 — PDUFA date set — FDA accepts ecopipam NDA with Priority Review; PDUFA target late Q1 202718 Jun 2026 — readout — Teva submitted the U.S. NDA for ecopipam in pediatric Tourette syndrome (guided for 2H 2026; submitted 2026-06-18).18 Jun 2026 — NDA/BLA filed — Teva submits U.S. NDA for ecopipam in pediatric Tourette syndrome14 Aug 2018 — Acquisition — Emalex Biosciences acquires Psyadon Pharmaceuticals, gaining ecopipam rightsPhase 223 Sept 2021 — phase change — Phase 2b in children/adolescents with Tourette syndrome (NCT04007991, n=153) completed (primary completion 2021-09-23).Phase 310 Jun 2026 — Acquisition — Teva closes acquisition of Emalex Biosciences26 May 2026 — readout (met) — 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.26 May 2026 — readout (met) — Phase 3 randomized-withdrawal full results published in JAMA Neurology: ecopipam halved relapse risk (pediatric HR 0.47, p=.008)29 Apr 2026 — Acquisition — Teva to acquire Emalex Biosciences for $700M upfront, adding NDA-ready ecopipam1 Apr 2026 — Conference presentation — Ecopipam Phase 3 randomized-withdrawal results presented at AAN 20264 Feb 2025 — phase change — D1AMOND Phase 3 completed (primary completion 2025-01-13; study completion 2025-02-04); met primary and secondary endpoints.31 Jan 2023 — phase change — Pivotal Phase 3 randomized-withdrawal study D1AMOND (NCT05615220) started (first patient dosed).
Phase change Readout Event UpcomingHover a marker for details.
Filed (NDA)Aug 2018 – Jan 2027
  1. UpcomingFDA PDUFA action on ecopipam NDA targeted late Q1 2027 (Priority Review)↗
  2. FDA Priority Review grantedFDA grants Priority Review for the ecopipam NDA in pediatric Tourette syndrome↗
  3. PDUFA date setFDA accepts ecopipam NDA with Priority Review; PDUFA target late Q1 2027↗
  4. Teva submitted the U.S. NDA for ecopipam in pediatric Tourette syndrome (guided for 2H 2026; submitted 2026-06-18).↗
  5. NDA/BLA filedTeva submits U.S. NDA for ecopipam in pediatric Tourette syndrome↗
  6. AcquisitionEmalex Biosciences acquires Psyadon Pharmaceuticals, gaining ecopipam rights↗
Phase 3Jan 2023 – Jun 2026
  1. AcquisitionTeva closes acquisition of Emalex Biosciences↗
  2. 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.↗
  3. metPhase 3 randomized-withdrawal full results published in JAMA Neurology: ecopipam halved relapse risk (pediatric HR 0.47, p=.008)↗
  4. AcquisitionTeva to acquire Emalex Biosciences for $700M upfront, adding NDA-ready ecopipam↗
  5. Conference presentationEcopipam Phase 3 randomized-withdrawal results presented at AAN 2026↗
  6. D1AMOND Phase 3 completed (primary completion 2025-01-13; study completion 2025-02-04); met primary and secondary endpoints.
  7. 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).

Ecopipam for Tourette syndrome#

Filed (NDA)ActiveTourette syndrome indication →

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'.

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#

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 StatesSpainItalyPoland

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 StatesPolandGermanyCanada

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. Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial — JAMA Neurology
  6. Efficacy and Safety of Ecopipam for Tourette Syndrome: Results from a Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Trial — American Academy of Neurology
  7. Emalex Biosciences Acquires Psyadon Pharmaceuticals to Develop Novel Treatment for Pediatric Tourette Syndrome — Emalex Biosciences / PR Newswire
  8. JAMA Neurology Publishes Phase 3 Data on D1 Receptor Antagonist Ecopipam in Tourette Syndrome — Businesswire (Emalex/Teva)
  9. Teva Closes Acquisition of Emalex Biosciences, Strengthening Late-Stage Neuroscience Pipeline and Advancing Pivot to Growth Strategy — Teva Pharmaceutical Industries
  10. Teva Submits NDA for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Tourette Syndrome — Teva Pharmaceutical Industries
Show all 12 sources
  1. 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
  2. U.S. FDA Accepts Teva's New Drug Application (NDA) and Grants Priority Review for Ecopipam — Teva Pharmaceutical Industries