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

256

Literatures (Medical) associated with Ecopipam

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 Aug 2025·Continuum (Minneapolis, Minn.)

Tourette Syndrome and Tic Disorders

Review

Author: Frey, Jessica

OBJECTIVE:

This article describes the clinical features of Tourette syndrome and its diagnostic criteria, etiology, and pathophysiology, as well as common myths associated with tic disorders. It discusses the behavioral, pharmacologic, neuromodulation, and alternative treatment approaches to tic disorders.

LATEST DEVELOPMENTS:

With the significant increase in functional ticlike behavior, especially during the COVID-19 pandemic, diagnostic criteria for functional ticlike behavior have been delineated to separate these ticlike movements from the tics observed in Tourette syndrome. Although behavioral therapy such as comprehensive behavioral intervention for tics remains the first-line treatment for tics, modifications to comprehensive behavioral intervention for tics have been developed to make this type of treatment more accessible. Pharmacologic trials for vesicular monoamine transporter 2 inhibitors have failed to meet the primary endpoint of tic reduction; however, they may still be helpful as add-on therapy for select individuals. A first-in-class compound called ecopipam met the primary endpoint for tic reduction and was well-tolerated, and may be a promising new treatment for Tourette syndrome. Deep brain stimulation may be considered for tics refractory to more conservative approaches.

ESSENTIAL POINTS:

Tourette syndrome and tic disorders can substantially affect quality of life. Educating patients about the natural history and expected course of the disease is the most important first step in management. Choosing a treatment based on comorbidities, tic severity, and patient preference is crucial for a beneficial outcome.

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

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

25 Feb 2025

·www.fiercebiotech.com

Emalex dopamine blocker stops Tourette syndrome relapse in phase 3 win

Emalex Biosciences plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025.\n Emalex Biosciences’ dopamine blocker led to fewer patients with Tourette syndrome relapsing in a phase 3 trial, setting the stage for the Chicago-based firm to submit the drug candidate for approval later this year.The phase 3 study enrolled 167 children and 49 adults, according to a Feb. 25 press release. After a 12-week open-label period, patients who experienced reductions in vocal and motor tics when given the drug were randomized to either continue receiving the drug, called ecopipam, or to switch to placebo for a further 12 weeks. This phase of the trial was double-blind.When continued on ecopipam, 41.9% of children had their symptoms relapse, defined as a 50% loss of the benefit they initially received from the drug in their Yale Global Tic Severity Score. This relapse rate was significantly lower than the 68.1% rate seen in children given placebo, Emalex said in the release. Time to relapse for the pediatric patients was the study’s primary endpoint.Emalex plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025, the firm said.The most common side effects of ecopipam were drowsiness, insomnia, anxiety, fatigue and headache, according to the release. About 10% of patients experienced drowsiness, also called somnolence, while 7% had insomnia.For the study’s secondary endpoint, Emalex analyzed the time to relapse for children and adult patients together. Here, the relapse rates were similar, with 41.2% of patients relapsing on ecopipam compared to 67.9% on placebo.The phase 3 study builds on Emalex’s prior phase 2 work, which found that ecopipam reduced tic severity by 30% after 12 weeks in 150 children and adolescents with Tourette syndrome.“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” Frederick Munschauer, M.D., Emalex’s chief medical officer, said in the release. The top-line data “show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo.” Ecopipam blocks the D1 dopamine receptor, which has been implicated in the repetitive and compulsive behaviors that are characteristic of Tourette syndrome, according to the release. Approved therapies for the disease target the D2 receptor instead.Emalex raised a $250 million series D in 2022 to bring ecopipam into phase 3, a sharp increase in investor interest from the comparatively paltry $35 million series C that preceded it.The Windy City biotech was founded in 2018 by biotech incubator Paragon Biosciences.Swiss biotech Noema Pharma had a Tourette win of its own late last year, when its PDE10A inhibitor gemlapodect—originally developed by Roche—reduced tic severity in a small phase 2 trial that enrolled 15 patients.

Phase 3Clinical ResultPhase 2

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


P11-6.002 (AAN2025)	Phase 2	Tourette Syndrome	-	Ecopipam	rnsrjrxiby(bqbzzwptrq) = dyhooknwqe deftfffxqv (lioqjagkvx ) View more	Positive	07 Apr 2025
NCT05615220 (D1AMOND, Company_Website) Manual	Phase 3	Tourette Syndrome	216	ecopipam (pediatric)	vedsgfirls(mownevxfrl) = rrdolxfrec dtpwtrkckc (juebzepekb ) Met	Positive	25 Feb 2025
NCT05615220 (D1AMOND, Company_Website) Manual	Phase 3	Tourette Syndrome	216	Placebo (pediatric)	vedsgfirls(mownevxfrl) = asbvqxxopw dtpwtrkckc (juebzepekb ) Met	Positive	25 Feb 2025
NCT04114539 (CTgov)	Phase 2	Tourette Syndrome	124	Ecopipam HCI	qregjnvjnt = fblecrfjau qcavojdlbc (biciikkkcn, qygkwfqvyk - efnqulhros) View more	-	01 Dec 2023
NCT04114539 (Corporate Publications) Manual	Phase 2	Tourette Syndrome	-	Ecopipam	myyuwxjkik(thtonkuynf) = anayskojzi dsutunzgcl (khibrlgmeu ) View more	Positive	05 Oct 2023
NCT04007991 (D1AMOND, CTgov)	Phase 2	Tourette Syndrome	153	Ecopipam (Ecopipam HCI 2 mg/kg/Day)	qabeyakour(mgwovsjsrr) = mzmjtgpdea matxzxfhhb (uzqstwdsxi, 1.062) View more	-	04 Oct 2023
NCT04007991 (D1AMOND, CTgov)	Phase 2	Tourette Syndrome	153	Placebo (Placebo)	qabeyakour(mgwovsjsrr) = hvlgbgyxfo matxzxfhhb (uzqstwdsxi, 1.006) View more	-	04 Oct 2023
MDS2022	Phase 2	Tourette Syndrome	153	Ecopipam	izobbsydgu(aespiyinig): LS mean [SE] difference = -3.4 (95% CI, -6.1 to -0.8), P-Value = 0.011 View more	Positive	15 Sep 2022
MDS2022	Phase 2	Tourette Syndrome	153	Placebo		Positive	15 Sep 2022
MDS2021	Phase 2	Tourette Syndrome	26	Ecopipam	ygwxjkvtst(bfnfduelvv) = puoouoblpy gbrdwztkho (qnnllzaitp )	Positive	17 Sep 2021
NCT01065558 (Pubmed \| Mol Genet Metab) Manual	Phase 1	Lesch-Nyhan Syndrome	5	ecopipam	gzjdbwmdlx(odeibjgbte) = sedation being the most common dose-limiting event ojgxsejxbj (rqafitedow )	Positive	01 Apr 2016
NCT01751802 (CTgov)	Phase 3	Lesch-Nyhan Syndrome \| Self-Injurious Behavior	9	Ecopipam (Ecopipam)	nrjhqqofje = adyntdxtny kuouasuzoc (ljsdppkasj, dqnkbalpmr - abhhetpzxj) View more	-	08 Oct 2015
NCT01751802 (CTgov)	Phase 3	Lesch-Nyhan Syndrome \| Self-Injurious Behavior	9	Placebo (Placebo)	nrjhqqofje = nzhlslzzjq kuouasuzoc (ljsdppkasj, trwxqiwhab - kehpekdebe) View more	-	08 Oct 2015
NCT01244633 (CTgov)	Phase 1/2	Tourette Syndrome	18	Ecopipam	kaicqfdklb(epvjviijev) = hyurzyhyhn pquowfflme (nysnpnqwhp, 9.2) View more	-	29 Sep 2015

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