RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (Canada), Orphan Drug (Japan)

Structure/Sequence

Molecular FormulaC13H21N3O6

InChIKeyBUSXWGRAOZQTEY-SDBXPKJASA-N

CAS Registry853400-76-7

Clinical Trials associated with Trofinetide

NCT04988867

/ TerminatedPhase 2/3

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

Start Date22 Sep 2021

Sponsor / Collaborator

ACADIA Pharmaceuticals, Inc.

NCT04776746

/ TerminatedPhase 3

An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome

To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

Start Date08 Nov 2020

Sponsor / Collaborator

ACADIA Pharmaceuticals, Inc.

NCT04279314

/ CompletedPhase 3

A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome

Start Date29 Jan 2020

Sponsor / Collaborator

ACADIA Pharmaceuticals, Inc.

100 Clinical Results associated with Trofinetide

100 Translational Medicine associated with Trofinetide

100 Patents (Medical) associated with Trofinetide

Literatures (Medical) associated with Trofinetide

01 Dec 2025·Current Neurology and Neuroscience Reports

Drug Treatments for Neurodevelopmental Disorders: Targeting Signaling Pathways and Homeostasis

Review

Author: Luu, Skylar ; Budimirovic, Dejan B ; Kaufmann, Walter E

PURPOSE OF THE REVIEW:

Preclinical and clinical evidence support the notion that neurodevelopmental disorders (NDDs) are synaptic disorders, characterized by excitatory-inhibitory imbalance. Despite this, NDD drug development programs targeting glutamate or gamma-aminobutyric acid (GABA) receptors have been largely unsuccessful. Nonetheless, recent drug trials in Rett syndrome (RTT), fragile X syndrome (FXS), and other NDDs targeting other mechanisms have met their endpoints. The purpose of this review is to identify the basis of these successful studies.

RECENT FINDINGS:

Despite increasing evidence of disruption in synaptic homeostasis, most genetic variants associated with NDDs implicate proteins involved in cell regulation and not in neurotransmission. Metabolic processes, in particular mitochondrial function, appear to play a role in NDD pathophysiology. NDDs are also characterized by distinctive cell signaling abnormalities, which link cellular and synaptic homeostasis. Recent successful trials in NDDs, including those of trofinetide, the first drug specifically approved for one of these disorders (i.e., RTT), implicate the targeting of downstream processes (i.e., signaling pathways) rather than neurotransmitter receptors. Recent positive drug studies in NDDs and their underlying mechanisms, in conjunction with new knowledge on the pathophysiology of these disorders, support the concept that targeting signaling and cellular and synaptic homeostasis may be a preferred approach for ameliorating synaptic abnormalities in many NDDs.

01 Oct 2025·CLINICAL THERAPEUTICS

Safety Profiles of Trofinetide in Pediatric Rett Syndrome Population: A Real-World Postmarketing Pharmacovigilance Analysis

Article

Author: Zhang, Zhiyue ; Yan, Tao ; Li, Jingjing

PURPOSE:

Trofinetide, a synthetic analog of glycine-proline-glutamate, is the only approved therapy for Rett syndrome. This study evaluated the safety profile of trofinetide in pediatric patients with Rett syndrome using real-world pharmacovigilance data.

METHODS:

Adverse event (AE) reports were extracted from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) from Q1 2023 to Q4 2024. Disproportionality analyses were conducted using ROR, PRR, BCPNN, and MGPS to detect AE signals associated with trofinetide use in individuals under 18 years. A Weibull distribution model was applied to assess time-to-onset patterns. Subgroup analyses by age and dose, as well as sensitivity analyses excluding common co-medications, were conducted to evaluate signal consistency.

FINDINGS:

Most patients were female (95.4%), and all reports originated from the United States. Common labeled AEs were confirmed, including diarrhoea (n = 1,528; ROR = 38.1), vomiting (n = 434; ROR = 5.96), and seizures (n = 251; ROR = 5.30). Off-label signals included weight decreased (n = 135; ROR = 7.21), tremor (n = 39; ROR = 3.37), dystonia (n = 18; ROR = 4.39), and dyskinesia (n = 24; ROR = 4.53). Over 70% of AEs occurred within the first month. Hypersomnia was more frequently reported in younger children (ROR = 12.11), and higher doses were associated with psychiatric symptoms. Subgroup and sensitivity analyses confirmed the robustness of key signals.

IMPLICATIONS:

This study provides critical real-world safety data on trofinetide use in pediatric Rett syndrome, identifying both expected and emerging AEs. Findings highlight the importance of monitoring dose-related and age-specific adverse events in clinical settings.

DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Real‐world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study

Article

Author: Abler, Victor ; Cosand, Louise ; Downs, Jenny ; Mayman, Haya

Abstract:

Aim:

To describe the real‐world effects of trofinetide in individuals with Rett syndrome (RTT) using the 18‐month follow‐up analysis of the LOTUS study.

Method:

Caregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate. Assessments included the Behavioral Improvement Questionnaire (BIQ), the Quality of Life Inventory‐Disability (QI‐Disability) questionnaire, the Gastrointestinal Health Questionnaire, and safety monitoring. Data are reported to 12 months since the initiation of trofinetide.

Results:

Overall, 227 caregivers participated in this follow‐up. The median trofinetide dose at week 1 was 36.0% of the target dose and increased to over 80% by week 10 onwards; 55.2% of patients took the full trofinetide recommended daily dose. Caregivers (71%–90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non‐verbal communication (48%–71%), alertness (44%–70%), and social interaction and connectedness (33%–58%). The median QI‐Disability total score change from baseline ranged from 4.7 to 4.6 in months 1 to 12. The incidence of diarrhea varied from weeks 1 to 12 (23%–50%) and months 4 to 12 (26%–38%); most reports of diarrhea were contained inside the patient's diaper. Safety reports were consistent with previous trofinetide clinical trials.

Interpretation:

Results from the LOTUS study show improvements in the symptoms of RTT, with early insights into managing gastrointestinal symptoms in the context of real‐world patient care.

News (Medical) associated with Trofinetide

06 Oct 2025

·www.einpresswire.com

Biomed Industries, Inc. to Present Promising NA-921 Phase 2/3 Results for Rett Syndrome Treatment at EAPS 2025

Biomed Industries, Inc. to Present Promising NA-921 Phase 2/3 Results for Rett Syndrome Treatment at EAPS Conference, October 18-20, 2025 in Lisbon, Portugal. The Phase 2/3 results of NA-921 show strong potential as an effective, well-tolerated treatment with a favorable safety profile, promising better patient adherence and improved long-term outcomes” — Dr. Lloyd L. Tran, CEO of Biomed SAN JOSE, CA, UNITED STATES, October 6, 2025 / EINPresswire.com / -- - Biomed Industries, Inc . (Biomed) today announced that its Chief Executive Officer, Dr. Lloyd L. Tran, will present the topline results of Phase 2/3 clinical trials of NA-921, a novel investigational treatment for Rett syndrome at the European Academy of Paediatric Societies (EAPS), October 18-20, 2025 in Lisbon, Portugal. The presentation, titled “PHASE 2 CLINICAL TRIALS RESULTS OF NA-921 FOR THE TREATMENT OF RETT SYNDROME ", summarizes findings from a randomized, double-blind, placebo-controlled Phase 2/3 study conducted in girls and young women aged 5 to 20 years diagnosed with Rett syndrome (ClinicalTrials.gov Identifier: NCT06849973). KEY FINDINGS: The topline results from the study demonstrate compelling proof of safety and efficacy for NA-921, an orally administered small molecule. Biomed conducted a comparative analysis of adverse reactions observed in the clinical trials, showing a significantly improved safety and tolerability profile for NA-921. COMPARISON WITH CURRENT TREATMENTS: While FDA-approved treatments such as DAYBUE™ have shown clinical efficacy, they come with significant safety concerns, including high rates of severe diarrhea, vomiting, and weight loss (Treatment Management Guide for Healthcare Professionals by Acadia Pharmaceuticals, Inc. https://www.daybuehcp.com/treatment-management-guide.pdf ). A comparison of clinical trial data between NA-921 and DAYBUE revealed the following: • Lower incidences of common side effects: Diarrhea (14% for NA-921 vs. 82% for DAYBUE) and vomiting (8% for NA-921 vs. 29% for DAYBUE). • Improved treatment retention: Unlike the DAYBUE trial, where 35.7% of patients discontinued treatment due to adverse events, no patients withdrew from the NA-921 trial. The details of the findings will be presented at the EAPS Conference. These results underscore Biomed Industries’ commitment to developing safer, more effective therapies that can significantly improve patient outcomes. “Our latest clinical data reinforce NA-921’s potential to provide a more effective and well-tolerated treatment option,” said Dr. Lloyd L. Tran, CEO of Biomed Industries, Inc. “Unlike existing therapies, which are often associated with severe gastrointestinal side effects and high discontinuation rates, NA-921 has demonstrated a remarkably low incidence of adverse events. With a favorable safety profile, NA-921 represents a promising treatment that could enhance patient adherence and long-term outcomes.” Biomed Industries is advancing NA-921 into the next phase of clinical trials to further assess its efficacy and long-term safety. AN UNMET NEED IN RETT SYNDROME Rett syndrome is a rare and severe neuro-developmental disorder that primarily affects females and is often caused by mutations in the MECP2 gene on the X chromosome. Approximately 16,000 individuals in the United States and 100,000 worldwide are affected by the condition (Rett Syndrome Research Trust). There are currently no curative treatments for Rett syndrome. Management strategies focus on alleviating symptoms and improving quality of life. The disorder progresses through four stages, beginning with developmental delays and slowed growth between 6 and 18 months of age. As it advances, individuals experience motor impairments, loss of speech, seizures, breathing difficulties, and profound cognitive and physical disabilities, requiring lifelong care. ABOUT BIOMED INDUSTRIES, INC. Biomed Industries, Inc. is a pioneering biopharmaceutical company dedicated to developing and commercializing novel drug therapeutics to address unmet medical needs. The company's research team has developed a new platform of drugs targeting Alzheimer’s disease, ALS, Major Depressive Disorder (MDD), Diabetes, Obesity, Metabolic dysfunction-associated Steatohepatitis (MASH), and rare diseases, including Rett Syndrome and Fragile X. Michael Willis Biomed Industries, Inc. +1 800-824-5135 email us here Visit us on social media: LinkedIn X Biomed introduction video Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Clinical ResultPhase 2

24 Sep 2025

·www.businesswire.com

Acadia Pharmaceuticals Announces Phase 3 COMPASS PWS Trial of Intranasal Carbetocin (ACP-101) for Hyperphagia in Prader-Willi Syndrome Did Not Meet Primary Endpoint

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced top-line results from the Phase 3 COMPASS PWS trial evaluating the efficacy and safety of intranasal carbetocin (ACP-101) in patients with hyperphagia in Prader-Willi syndrome (PWS). Intranasal carbetocin did not demonstrate a statistically significant improvement over placebo on the study’s primary endpoint, change from baseline to Week 12 on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), nor was there separation from placebo on any secondary endpoint. The safety and tolerability profile of intranasal carbetocin was consistent with previous clinical trials, showing a low rate of adverse events. “We are disappointed by these findings, especially for Prader-Willi syndrome patients, their families and the entire community,” said Elizabeth H.Z. Thompson, Ph.D., Acadia’s Head of Research and Development. “I want to thank the many patients, families, study site personnel, and physicians who participated in the COMPASS PWS study as well as the intranasal carbetocin clinical development program, for their dedication and contributions in this important study. We are committed to sharing a summary of the data in the future to ensure learning for the PWS community; however, given these results, we do not intend to investigate intranasal carbetocin any further.” COMPASS PWS was a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial evaluating the efficacy and safety of intranasal carbetocin 3.2 mg three times daily (TID) in 175 enrolled children and adults aged five to 30 years with PWS. “Despite this disappointment, Acadia is well-positioned to deliver long-term, sustainable growth supported by two approved products projected to generate over $1 billion in net sales in 2025, and a robust pipeline that includes eight disclosed and multiple undisclosed programs,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “Looking ahead, we anticipate seven Phase 2 or 3 study starts through 2026 and four data readouts by the end of 2027. For more details on our programs, I encourage you to revisit our recent R&D Day presentation on our website.” About Acadia Pharmaceuticals Acadia is advancing breakthroughs in neurological and rare diseases to elevate life. Since our founding we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only approved drug in the United States and Canada for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Alzheimer’s disease psychosis, Lewy Body Demetia psychosis and multiple other programs targeting neuroscience and neuro-rare diseases. For more information, visit us at acadia.com and follow us on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “intends,” “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: our clinical development plans related to intranasal carbetocin (ACP-101); our plans to share a summary of the data in the future; the safety and tolerability of intranasal carbetocin; our estimates regarding our future financial performance, including our full year 2025 financial guidance; and our business strategy, objectives and opportunities, including the timing for and clinical and market potential of our pipeline assets, and potential for enhanced shareholder value. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to: our dependency on the continued successful commercialization of NUPLAZID and DAYBUE and our ability to maintain or increase sales of NUPLAZID or DAYBUE; our ability to generate or obtain the necessary capital to fund our clinical development plans; the risks associated with clinical trials and their outcomes, including risks of negative or inconsistent results; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ, please refer to our quarterly report on Form 10-Q for the quarter ended June 30, 2025 filed with the Securities and Exchange Commission on August 7, 2025, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.

Phase 3Clinical ResultDrug Approval

24 Sep 2025

·firstwordpharma.com

Acadia pulls plug on Prader-Willi drug after trial flop

Acadia Pharmaceuticals is scrapping development of intranasal carbetocin after the experimental Prader-Willi syndrome (PWS) drug failed a Phase III trial, sending company shares down nearly 12% on Wednesday.The decision scuttles Acadia's effort to bring a second treatment option to patients struggling with the rare genetic disorder's hallmark symptom of insatiable hunger. Soleno Therapeutics' Vykat XR (diazoxide choline) became the first drug approved by the FDA specifically for hyperphagia in PWS this past March.Intranasal carbetocin, also known as ACP-101, is a synthetic analogue of oxytocin, a neuropeptide involved in regulating eating behaviour that is deficient in PWS patients.The 12-week COMPASS PWS trial tested a 3.2-mg three-times-daily dose in 175 children and adults ages 5 through 30 years old, measuring changes from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) as the main outcome. Acadia reported no statistically significant improvement over placebo on the primary or any secondary measures.The outcome dashes hopes raised by an earlier Phase III study, CARE PWS, which had hinted at benefit with the same low dose, but not a higher one. Ahead of Wednesday's readout, analysts at Citizens JMP said investor expectations were "muted" for COMPASS PWS given the inverse dose response seen in the previous Phase III trial. Acadia has suggested that the lack of efficacy at the high dose may have been due to activity on vasopressin receptors, which offsets the beneficial activity driven through oxytocin receptors.Still, the analysts noted that learnings from that earlier study were used to optimise COMPASS PWS, including focusing solely on the lower dose and extending the duration of treatment from 8 to 12 weeks, in hopes of replicating the efficacy signal. They were also projecting a "highly conservative" $250 000/year price at launch, compared to the roughly $500,000 expected average annual cost of Vykat XR."Despite this disappointment, Acadia is well-positioned to deliver long-term, sustainable growth supported by two approved products projected to generate over $1 billion in net sales in 2025, and a robust pipeline," remarked CEO Catherine Owen Adams. "Looking ahead, we anticipate seven Phase II or III study starts through 2026 and four data readouts by the end of 2027."Acadia's two marketed products are Nuplazid (pimavanserin) for Parkinson's disease psychosis and the Rett syndrome therapy Daybue (trofinetide).

Phase 3Clinical ResultDrug Approval

100 Deals associated with Trofinetide

External Link

KEGG	Wiki	ATC	Drug Bank
-	Trofinetide	-	DB06045

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Rett Syndrome	United States	ACADIA Pharmaceuticals, Inc.	10 Mar 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Brain Concussion	Phase 2	United States	Neuren Pharmaceuticals Ltd.	01 Sep 2014
Fragile X Syndrome	Phase 2	United States	Neuren Pharmaceuticals Ltd.	01 Jan 2014
Brain Injuries, Traumatic	Phase 1	Australia	Neuren Pharmaceuticals Ltd.	01 Mar 2010

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04988867 (DAFFODIL, Pubmed \| Med)	Phase 2/3	Rett Syndrome	-	Trofinetide	pghgmhknbp(cprkfxihnt) = 80.0% lxcsthdeyu (zndnbkyont ) View more	Positive	01 Jun 2025
NCT04776746 (LILAC-2, CTgov)	Phase 3	Rett Syndrome	77	Trofinetide	vbbtjnuygt = mrlmzcdnkp uutsaszwhq (xtfxinvcmw, nfpcwjjmko - osjkyvqqgu) View more	-	27 Sep 2024
NCT04988867 (DAFFODIL \| DAFFODIL™, CTgov)	Phase 2/3	Rett Syndrome	15	Trofinetide	npiwjzoshd = ayycokxxfj xylnkldqxi (fhpuujgvyj, ndnyeghkie - fjkmvooxnj) View more	-	24 Sep 2024
NCT04776746 (LILAC-2, BusinessWire) Manual	Not Applicable	Rett Syndrome	-	DAYBUE (LILAC-1)	zqrtqvwufc(ofukkgxkig) = pmywjlevqy zzxzbzgsxa (tsasupprqt, 1.62) View more	Positive	18 Jul 2024
NCT04776746 (LILAC-2, BusinessWire) Manual	Not Applicable	Rett Syndrome	-	Placebo (LILAC-1)	zqrtqvwufc(ofukkgxkig) = xkedpfapdl zzxzbzgsxa (tsasupprqt, 1.61) View more	Positive	18 Jul 2024
NCT04776746 (LILAC-2, Pubmed)	Phase 3	Rett Syndrome	77	Trofinetide	njngsidmzl(buwnxktanc) = 27.3% adoevrylwa (oimmrzbkup ) View more	Positive	16 Jul 2024
AAN2024 Manual	Not Applicable	Rett Syndrome	325	Trofinetide	sefohsysfg(xxkskhptzr) = pklbyjkojd npaoqdxsrq (jcppguafyo )	Positive	13 Apr 2024
NCT04279314 (LILAC™, CTgov)	Phase 3	Rett Syndrome	154	Trofinetide	sjxnnjzjqt = fmozfeqxds tlhtowlwnf (xlkbnmsata, zpansfkmih - ldxpndvfyx) View more	-	11 Apr 2024
S37.008 (AAN2024)	Phase 3	Rett Syndrome	33	Trofinetide (LILAC/LILAC-2)	piigmbbgnl(ntxkzvhkrq) = bytsripgbl hlbdrxdxqq (yklinczwbv ) View more	Positive	09 Apr 2024
NCT04181723 (CTgov)	Phase 3	Rett Syndrome	187	Trofinetide (Drug - Trofinetide)	hscspwxabg(xrtvhqhuur) = lwjybogoxz bqzlqxgcji (orikudouky, 0.94) View more	-	08 Apr 2024
NCT04181723 (CTgov)	Phase 3	Rett Syndrome	187	Placebo (Placebo)	hscspwxabg(xrtvhqhuur) = gwjetnelkp bqzlqxgcji (orikudouky, 0.90) View more	-	08 Apr 2024
LAVENDER study (AAN2023)	Phase 3	Rett Syndrome	-	Trofinetide	dpleqbwsiq(neurhkzzox): P-Value = 0.0257 View more	Positive	25 Apr 2023
LAVENDER study (AAN2023)	Phase 3	Rett Syndrome	-	Placebo	dpleqbwsiq(neurhkzzox): P-Value = 0.0257 View more	Positive	25 Apr 2023

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