A Phase 1, Randomized, 2-way Crossover, Food-effect Study of Ecopipam Pharmacokinetics After Administration of the to Be Marketed Tablet in Healthy Subjects

Standard food-effect study to identify the effect of food on ecopipam pharmacokinetics after administration of a to-be-marketed ecopipam tablet in healthy volunteers.

Start Date05 Nov 2024

Sponsor / Collaborator

Emalex Biosciences, Inc.

NCT06194864

/ CompletedPhase 1

A Phase 1, Sequential Drug Interaction Study to Evaluate the Effect of CYP3A4 and P-glycoprotein Inhibition by Itraconazole or Induction by Rifampicin on the Pharmacokinetics of Ecopipam and Its Metabolites in Healthy Subjects

This is a single center, 2-Part, Phase 1, open-label, fixed-sequence, drug-drug interaction study designed to compare the PK of ecopipam when administered alone and in combination with itraconazole (Part 1) or rifampicin (Part 2) in healthy subjects.

Start Date18 Jan 2024

Sponsor / Collaborator

Emalex Biosciences, Inc.

[+2]

NCT06021522

/ Active, not recruitingPhase 3

A Multicenter, Open-Label, Study to Evaluate the Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of age), and adults (>=18 years of age) with Tourette's Syndrome (TS).

Start Date16 Aug 2023

Sponsor / Collaborator

Emalex Biosciences, Inc.

100 Clinical Results associated with Ecopipam

100 Translational Medicine associated with Ecopipam

100 Patents (Medical) associated with Ecopipam

228

Literatures (Medical) associated with Ecopipam

01 Feb 2026·CTS-Clinical and Translational Science

The Effect of Ecopipam on the Pharmacokinetics of Concomitant Medications

Article

Author: Schmith, Virginia D. ; Lohmer, Lauren L. R. ; Graden, Danielle ; Schleyer, Joy ; Wanaski, Stephen P.

ABSTRACT:

Ecopipam is a first‐in‐class dopamine D1 receptor antagonist under investigation for the treatment of Tourette syndrome. This open‐label, fixed‐sequence, three‐cohort study evaluated whether ecopipam induces or inhibits cytochrome P450 (CYP) 1A2, CYP2B6, CYP2C19, CYP2D6, CYP3A4, organic anion‐transporting polypeptide (OATP) 1B1, and p‐glycoprotein (P‐gp). Probe substrates were administered alone and after single or steady‐state dosing of ecopipam (1.8 mg/kg/day). Cohort 1 received standard doses of omeprazole (CYP2C19), caffeine (CYP1A2), and dextromethorphan (CYP2D6) orally, and a microdose of midazolam (CYP3A4) intravenously. Cohort 2 received a standard dose of bupropion (CYP2B6) orally. Cohort 3 received an oral solution containing microdoses of dabigatran (P‐gp), pitavastatin (OATP1B1), rosuvastatin (BCRP, OATP, and P‐gp), atorvastatin (BCRP, OATP, P‐gp, and CYP3A4), and midazolam. A total of 56 healthy individuals (median [range] age, 36.5 [19–54] years; 85.7% male) were enrolled, and 48 completed the study. Ecopipam was well tolerated in the presence and absence of probe substrates. Steady‐state ecopipam administration increased dextromethorphan exposure (> 100‐fold); decreased the AUC inf of midazolam, omeprazole, and dabigatran by 44.2%, 44.1%, and 37.9%, respectively; and decreased unconjugated bilirubin (UGT1A1) by 19.5%. Single‐dose ecopipam increased atorvastatin Cmax by 95% and rosuvastatin Cmax by 11%, whereas steady‐state ecopipam decreased the AUC inf of atorvastatin by 26.8% and rosuvastatin by 16.4%. There were no changes in caffeine, bupropion, or pitavastatin exposure. Ecopipam is a strong inhibitor of CYP2D6 and weak inducer of CYP3A4, CYP2C19, P‐gp, and UGT1A1. Ecopipam did not inhibit CYP3A4, CYP2C19, CYP2B6, CYP1A2, UGT1A1, P‐gp, or OATP1B1 and did not induce OATP1B1 or CYP2B6.

01 Aug 2025·Movement Disorders Clinical Practice

Safety and Effect of 12‐Month Ecopipam Treatment in Pediatric Patients with Tourette Syndrome

Article

Author: Miller, Meredith M. ; Kim, David J.B. ; Munschauer, Frederick E. ; Cunniff, Timothy M. ; Gilbert, Donald L. ; Karkanias, George B. ; Wanaski, Stephen P. ; Atkinson, Sarah D.

Abstract:

Background:

Tourette syndrome (TS) is a chronic neurodevelopmental tic disorder with a considerable quality of life (QOL) burden.

Objectives:

The goal was to determine the long‐term safety, tolerability, and clinical effects of ecopipam, a first‐in‐class dopamine D1 receptor antagonist, for TS.

Methods:

This 12‐month, open‐label extension (OLE) study enrolled patients age ≥6 to ≤18 years with confirmed TS who completed a phase 2b randomized, placebo‐controlled, 12‐week trial. Ecopipam was titrated over 4 weeks to achieve a target oral dose of 1.8 mg/kg/day. Study visits occurred at baseline, monthly for 12 months, and 7 and 14 days after last dose.

Results:

A total of 121 patients were included (74% male; 68% age 12–18 years), and 80 (66%) completed the study. Ecopipam was well tolerated. The most common adverse events were nasopharyngitis (14.0%) and anxiety (9.1%). At month 12, there were no significant changes from baseline in body mass index Z‐score (mean [standard deviation] change, 0.05 [0.43]; P = 0.35), glycated hemoglobin (0.03% [0.31]; P = 0.60), or total cholesterol (0.2 mmol/L [0.7]; P = 0.14). No notable changes in scales assessing akathisia, movement disorders, anxiety, or depression occurred. At all time points, significant improvements (P < 0.001 vs. baseline) in both the Yale Global Tic Severity Scale Total Tic Score and the Gilles de la Tourette Syndrome Quality of Life Scale for Children and Adolescents total score were observed.

Conclusions:

Twelve months of ecopipam dosing was well tolerated during this study and no new adverse events were detected. Compared to baseline, significantly reduced TS symptom severity and improved QOL were observed in children and adolescents.

01 Feb 2025·CNS DRUGS

Safety and Efficacy of Ecopipam in Patients with Tourette Syndrome: A Systematic Review and Meta-analysis

Review

Author: Panda, Prateek Kumar ; Sharawat, Indar Kumar ; Mishra, Anand Santosh ; Dawman, Lesa ; Kumar, Vinod ; Panda, Pragnya

BACKGROUND AND OBJECTIVES:

Ecopipam is a selective antagonist of the dopamine D1 receptor, and its efficacy and safety have recently been explored in several clinical trials involving patients with Tourette syndrome (TS). The objectives of this systematic review were to determine the pooled estimate for efficacy [in terms of reduction in tic Yale Global Tic Severity Scale (YGTSS) scores] and safety of oral ecopipam in subjects with TS.

METHODS:

All clinical trials that explored the efficacy and/or safety of ecopipam in patients with TS were included to determine the pooled estimate for change in YGTSS, Clinical Global Impression (CGI)-TS, and the severity of comorbid attention-deficit hyperactive disorder (ADHD), obsessive compulsion disorder (OCD), and depressive symptoms, as well as the nature and frequency of adverse effects. Case-series, retrospective studies, and case reports were excluded. Databases, such as PUBMED, EMBASE, Cochrane Central Register of Controlled Trials, and SCOPUS were searched to identify these trials using suitable combination of MESH terms/keywords on 15 June 2024. ROB 2.0 and ROBINS-I tool were used to assess the risk of bias in included randomized-controlled trials (RCTs) and non-randomized intervention studies, respectively, and the GRADE system to determine the certainty of the collated evidence.

RESULTS:

A total of 96 records were identified in the database search and 31 records were screened after removing duplicates. After excluding 23 irrelevant records, the full-text review included 8 records. Finally, six publications from three completed clinical trials (two RCTs, with one having an open-label extension) and one ongoing clinical trial were included. A total of 251 participants were included. The pooled estimate for mean change in YGTSS-TTS from baseline to the completion of the randomization period was statistically better in the ecopipam group compared with the placebo group [mean difference: - 3.0, 95% (confidence interval (CI) - 4.2 to - 1.9, I² = 55%, p < 0.0001]. The ecopipam group also fared statistically better in terms of YGTSS-motor tic score, phonic tic score, as well as CGI-TS-S (p < 0.0001). Changes in depressive and obsessive-compulsive symptoms were comparable in both groups, as well as the incidence of adverse effects.

CONCLUSIONS:

Ecopipam is effective in reducing the severity of tics in subjects with TS and has a good safety profile. However, only a limited number of studies were included in the review, with some having small sample sizes and short duration of follow-up.

News (Medical) associated with Ecopipam

08 Oct 2025

·www.businesswire.com

Emalex Biosciences’ Expanded Access Program for Investigational Tourette Syndrome Drug Receives FDA Authorization

CHICAGO--(BUSINESS WIRE)--Emalex Biosciences today announced that the U.S. Food and Drug Administration has authorized the company’s Expanded Access Program (EAP) for ecopipam, an investigational therapy in development for the treatment of Tourette syndrome. “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. The program (ClinicalTrials.gov Identifier: NCT07093541) allows physicians to request access to ecopipam for eligible patients who have been treated with an FDA-approved treatment for Tourette syndrome (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist and have experienced treatment failure, tolerability or safety issues or do not have access to any of these approved medications. Emalex plans to submit a New Drug Application (NDA) to the FDA in late 2025. “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. “This pathway offers a potential option for patients and their physicians while development continues.” Expanded Access Programs provide a regulatory pathway for patients with serious conditions to access investigational medicines outside of clinical trials. Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics. The condition is associated with increased mortality and significant morbidity. In the majority of individuals, Tourette syndrome substantially impacts day-to-day physical and social function. Ecopipam is an investigational, first-in-class small-molecule drug that selectively blocks dopamine-1 (D1) receptors. It is not an antipsychotic. Investigators are evaluating ecopipam as a potential therapy for Tourette syndrome. This mechanism represents a novel approach, distinct from currently approved therapies that primarily target dopamine-2 (D2) receptors. Participation in the Expanded Access Program requires physician oversight, FDA authorization, and Institutional Review Board approval. Enrollment is limited, and eligibility is determined on a case-by-case basis. Ecopipam is an investigational drug and has not been approved by the FDA for any indication. In clinical studies in pediatric patients with Tourette syndrome, ecopipam was generally well tolerated and the most common (≥ 5%) adverse events were headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness. About Ecopipam Ecopipam is a first-in-class investigational compound that is being studied as a potential treatment for certain central nervous system (CNS) disorders. It blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors. Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and rarely, suicidal ideation. About Emalex Biosciences Emalex Biosciences was created by Paragon Biosciences to develop new treatments for CNS disorders. Emalex is in late-stage development of a new class of drug for patients with Tourette syndrome and other conditions that have limited treatment options. Visit https://emalexbiosciences.com to learn more. About Paragon Biosciences Paragon Biosciences, founded by Jeff Aronin, is a global life science leader that creates, builds and funds innovative biology-based companies. Our portfolio companies accelerate scientific breakthroughs to solve some of society’s most challenging problems. Learn more at https://www.paragonbiosci.com.

Orphan DrugFast TrackNDADrug Approval

26 Feb 2025

·www.pharmaceutical-technology.com

Emalex eyes FDA approval for Tourette syndrome therapy after Phase III success

Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with Ecopipam experienced some form of relapse. Credit: Shutterstock / Studio Romantic Emalex Biosciences is eyeing approval for its Tourette syndrome therapy after it achieved its primary and secondary endpoints in a Phase III study. The US company’s central nervous system (CNS) disorder drug ecopipam, a dopamine-1 receptor antagonist treatment, met both primary and secondary endpoints in both paediatric and adult patients living with the condition that can lead, in some cases, to involuntary tics and vocalisations. The drug achieved a 41.9% of paediatric patients relapse against the placebo group’s 68.1%. Following the positive data, Emalex Biosciences said it will be meeting with the US Food and Drug Administration (FDA) and other global health authorities to discuss the submission of a New Drug Application (NDA) later in 2025. The DIAMOND registrational study (NCT05615220) examined a total of 167 paediatric subjects and 49 adult subjects with Tourette syndrome. The trial saw a clinically meaningful reduction in vocal and motor tics in paediatric patients receiving ecopipam during a 12-week open-label period. Patients were then randomised to remain on the study drug or be moved to a placebo for a 12-week double-blind withdrawal period. Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with ecopipam experienced some form of relapse against the placebo arm’s 67.9% of subjects. Frederick Munschauer, chief medical officer for Emalex Biosciences, said: “These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome. “The topline data from our large, multi-national, randomised withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for paediatric subjects with Tourette syndrome as compared to placebo.” Research by GlobalData uncovered that in 2024 the global market for Tourette syndrome therapies stood at approximately $248m, with that figure expected to plummet down to just $84m by the end of 2030. Whilst there are many marketed drugs, Otsuka Holdings’ Abilify (aripiprazole) is the most commonly used antipsychotic therapy and is expected to bring in around $192m by the end of 2025. GlobalData is the parent company of Clinical Trials Arena. The US Centers for Disease Control (CDC) estimates that across the US one out of every 333 (0.3%) children between the ages of three and 17 has been diagnosed with the condition. Additionally, 44% of those children have been diagnosed with moderate or severe Tourette syndrome, with boys three times more likely to live with the condition than girls. Previously in results taken from a Phase IIb trial , ecopipam was able to reduce the number of tics experienced by a paediatric patient by 30% compared with placebo. Eric Messner, Emalex Biosciences’ CEO, added: “The Emalex team worked closely with physician investigators and patient advocates throughout the drug development process and we are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome. We’re entering a new era of progress for people with central nervous system conditions with limited or no treatment options.” Elsewhere in the market of Tourette therapies, Israeli pharmaceutical company SciSparc has received approval from the US Food and Drug Administration (FDA) to begin a Phase IIb clinical trial of SCI-110 in adults living with the condition.

Phase 2Clinical ResultPhase 3NDA

26 Feb 2025

·www.biospace.com

Emalex Heads to FDA After Phase III Tourette Syndrome Win

iStock, Cristina Gaidau Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year. Emalex Biosciences’ experimental dopamine blocker ecopipam cleared late-stage studies on Tuesday, paving the way for the Chicago-based biotech to submissions with the FDA. In the Phase III trial, 41.9% of pediatric Tourette syndrome patients relapsed after ecopipam treatment, as compared with 68.1% of placebo comparators—resulting in a 50% relapse reduction, a statistically significant result, as per Emalex’s news release. These results remained true when analysis was expanded to cover both adults and children. Emalex reported that ecopipam was well-tolerated and its common side effects were excessive sleepiness, insomnia, anxiety, fatigue and headaches. Emalex Chief Medical Officer Frederick Munschauer in a statement said these results highlight the efficacy of ecopipam to elicit and maintain “clinically meaningful reductions in vocal and motor tics” in children with Tourette syndrome—and “strengthen our confidence in ecopipam as a potential first-in-class treatment” in this indication. Emalex plans to meet with the FDA and other international health authorities to discuss the potential regulatory path for ecopipam. A New Drug Application is planned for “later this year,” as per Tuesday’s announcement. Tourette syndrome is a neurological disorder characterized by tics, or sudden and brief uncontrollable movements. In the most severe cases, tics can disrupt a patient’s communication, quality of life and daily functioning. The exact causes of Tourette syndrome are not known and currently the disease is managed through certain neuroleptic medications or with behavioral therapy. Designed to be taken orally, ecopipam differentiates itself from other Tourette treatments by acting on the D1 subtype of dopamine receptors, instead of on the D2 subtype. Dopamine is a critical neurotransmitter involved in controlling motion, and dysfunction in its signaling could lead to movement-related disorders. With ecopipam’s Phase III win on Tuesday—and impending regulatory filings—Emalex pulls ahead of many other biotechs advancing Tourette treatments. One of these is Relmada Therapeutics, which earlier this month acquired sepranolone from Asarina Pharma for €3 million. The asset, a first-in-class GABAA modulating steroid agonist, is currently ready to enter Phase IIb development. Also in the Tourette space are SciSparc , testing a synthetic form of tetrahydrocannabinol, and Noema Pharma , advancing a PDE10A inhibitor, likewise assessing their respective assets in mid-stage studies.

Phase 3Phase 2Acquisition

100 Deals associated with Ecopipam

External Link

KEGG	Wiki	ATC	Drug Bank
D03937	Ecopipam	-	DB12273

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Tourette Syndrome	Phase 3	United States	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Bulgaria	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Canada	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Denmark	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	France	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Germany	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Hungary	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Italy	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Poland	Emalex Biosciences, Inc.	31 Jan 2023
Tourette Syndrome	Phase 3	Romania	Emalex Biosciences, Inc.	31 Jan 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05615220 (D1AMOND, CTgov)	Phase 3	Tourette Syndrome	216	Ecopipam (Double- Blind R/WD Period: Ecopipam 1.8 mg/kg/Day)	ypbhfwaidv(reksarmvdz) = ruxvlmmhdz juxkapboyp (uevnnxhcre, frfgwpelcj - jwxpvcdlnh) View more	-	25 Nov 2025
				placebo+ecopipam HCl (Double- Blind R/WD Period: Placebo)	ypbhfwaidv(reksarmvdz) = sqahlldpvu juxkapboyp (uevnnxhcre, ghanjegmhz - ddxpwbhmqt) View more
MDS2025	Phase 3	Tourette Syndrome	104	Ecopipam 1.8 mg/kg/day	bzzuyzfraj(hntekuxlxq) = 9.7% hwqhcmcbee (vhntmctpji ) View more	Positive	05 Oct 2025
				Placebo
P11-6.002 (AAN2025)	Phase 2	Tourette Syndrome	-	Ecopipam	ilxirbggum(gadtndubbc) = kvuhhfkwdd tecetnlgsk (umzpsrptwh ) View more	Positive	07 Apr 2025
NCT05615220 (D1AMOND, Company_Website) Manual	Phase 3	Tourette Syndrome	216	ecopipam (pediatric)	ynthvfhrkt(uelyxuumxf) = eoyvrtmztc jwjnspujub (mgliwkpnvk ) Met	Positive	25 Feb 2025
				Placebo (pediatric)	ynthvfhrkt(uelyxuumxf) = idvralheyk jwjnspujub (mgliwkpnvk ) Met
NCT04114539 (CTgov)	Phase 2	Tourette Syndrome	124	Ecopipam HCI	hrqxlxxxas = pyyghsmzwk jbyhkueill (likszhcqhh, nbyvyuawma - ordhpxzvzd) View more	-	01 Dec 2023
NCT04114539 (Corporate Publications) Manual	Phase 2	Tourette Syndrome	-	Ecopipam	yohvaeqqql(gtvqsvdglt) = hqvwfrgcnj cnljzpxsqm (gdtmibvfne ) View more	Positive	05 Oct 2023
NCT04007991 (D1AMOND, CTgov)	Phase 2	Tourette Syndrome	153	Ecopipam (Ecopipam HCI 2 mg/kg/Day)	fjofwnlyzf(ztyhbckgxn) = rmxjepzvos kbbblmoand (cnpjbfngjn, 1.062) View more	-	04 Oct 2023
				Placebo (Placebo)	fjofwnlyzf(ztyhbckgxn) = jwmurwedey kbbblmoand (cnpjbfngjn, 1.006) View more
MDS2022	Phase 2	Tourette Syndrome	153	Ecopipam	iarwllfxqu(hgobcwfcxt): LS mean [SE] difference = -3.4 (95% CI, -6.1 to -0.8), P-Value = 0.011 View more	Positive	15 Sep 2022
				Placebo
MDS2021	Phase 2	Tourette Syndrome	26	Ecopipam	cbuilxxgqx(icrzxgelaz) = zupfbhwcgt stqvmzhpfl (xppkrccknz )	Positive	17 Sep 2021
NCT01065558 (Pubmed \| Mol Genet Metab) Manual	Phase 1	Lesch-Nyhan Syndrome	5	ecopipam	adijtyhzon(tvcbnrelha) = sedation being the most common dose-limiting event ghfqkagihf (znbdttdrdb )	Positive	01 Apr 2016

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