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

Structure/Sequence

Molecular FormulaC13H21N3O6

InChIKeyBUSXWGRAOZQTEY-SDBXPKJASA-N

CAS Registry853400-76-7

Clinical Trials associated with Trofinetide

JPRN-jRCT2031250113

/ RecruitingPhase 3

A Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome, Comprising a Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Period Followed by an Open-Label Treatment Period

Start Date10 Jun 2025

Sponsor / Collaborator

ACADIA Pharmaceuticals, Inc.

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.

100 Clinical Results associated with Trofinetide

100 Translational Medicine associated with Trofinetide

100 Patents (Medical) associated with Trofinetide

Literatures (Medical) associated with Trofinetide

31 Dec 2026·JOURNAL OF MEDICAL ECONOMICS

Rett syndrome and real-world treatment patterns of trofinetide in the United States

Article

Author: Rashid, Nazia ; Syed, Safiuddin Shoeb ; Yakkala, Vinod Kumar ; Rajagopalan, Krithika

BACKGROUND:

Trofinetide (TROF) remains the first and only FDA-approved pharmacologic treatment for Rett syndrome (RTT). There is limited real-world evidence on TROF use, restarts, dosing patterns and predictors of non-persistence. This study evaluated long-term treatment patterns and baseline predictors of non-persistence among RTT individuals initiating TROF.

METHODS:

A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed. RTT individuals initiating 1^st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent. Median (IQR) time on treatment was analyzed, and Kaplan-Meier assessed time to non-persistence. Restarts among non-persistent were examined. Dosing patterns such as mean dose in milligrams (mg) and percentage target daily dose (%TDD) at each RX were evaluated. Predictors of non-persistence were assessed using logistic regression.

RESULTS:

Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted. Mean ± SD age was 14.7 ± 10.8 vs. 16.5 ± 11.6 years and median ± IQR time on TROF was 14.3 ± 4.6 vs. 3.0 ± 3.8 months, respectively. Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up. Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2. Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time. Similar patterns were observed for %TDD. Older age and history of infectious disorders were significantly associated with non-persistence.

CONCLUSIONS:

In this real-world analysis, more than half of RTT individuals remained persistent on TROF for ≥14 months. Stable dosing patterns were observed among persistent individuals, while greater dose variability was observed among non-persistent individuals. Thus, these findings may provide guidance to clinicians about long-term TROF use in real-world practice.

01 Nov 2026·SEIZURE-EUROPEAN JOURNAL OF EPILEPSY

Remarkable seizure reduction with trofinetide treatment in an adult with Rett syndrome: A case report

Article

Author: Nei, Maromi ; Ardestani, Pooneh Memar ; Adegbite, Semihan

01 Oct 2026·ADVANCES IN THERAPY

Real-World Demographics, Clinical Characteristics, and Predictors of Persistence Among Males with Rett Syndrome Treated with Trofinetide

Article

Author: Rashid, Nazia ; Yakkala, Vinod ; Rajagopalan, Krithika

INTRODUCTION:

Rett syndrome (RTT) is a rare neurodevelopmental disease that primarily affects females but also occurs in males. Trofinetide (TROF) is approved in the USA and Canada for individuals with RTT regardless of sex aged ≥ 2 years; efficacy and safety of TROF was primarily studied among females, thus real-world data for males remain limited. This study evaluated real-world characteristics, treatment persistence, and predictors of non-persistence among males with RTT.

METHODS:

This retrospective cohort study using IQVIA database linked to TROF specialty pharmacy claims from 01/01/2021 to 09/30/2024 identified males with ≥ 1 medical claim for RTT (ICD-10 F84.2). Two groups were created based on treatment status and their respective index dates: treated and untreated. Continuous enrollment of ≥ 6 months pre-index and post-index was required. Pre-index demographics and clinical characteristics were compared descriptively. Among treated males, Kaplan-Meier analysis assessed time to non-persistence, and logistic regression evaluated predictors of non-persistence.

RESULTS:

Of 8047 individuals with RTT identified in the database, 1219 (15.1%) were males; of these, 75 (6.2%) were treated with TROF and 1144 (93.8%) were untreated; after final study criteria, 51 (10.9%) were treated and 415 (89.1%) were untreated. The mean (SD) age for treated vs untreated males was 18.8 (18.3) vs 17.7 (13.2) years. Neurologists managed most patients treated with TROF (64.7% vs 12.7%). Treated males had higher pre-index rates of epilepsy (52.9% vs 25.5%), gastrostomy (31.4% vs 16.1%), and respiratory failure (25.5% vs 13.3%) than untreated (all p < 0.05). Among treated males, 52.9% were persistent and 47.1% were non-persistent. Approximately 70% remained on TROF for ≥ 6 months and > 50% for ≥ 17 months. Pre-index neurologic disorders were significantly associated with non-persistence.

CONCLUSIONS:

Most males with RTT remained untreated during this study period, while TROF initiation appeared concentrated among those with greater pre-index concomitant comorbidities and neurology involvement. Persistence beyond 6 months was common, supporting continued long-term use of TROF in males with RTT in real-world practice.

105

News (Medical) associated with Trofinetide

08 Oct 2026

·www.biospace.com

Marvel Biosciences Reports MB-204 as Highly Effective at Treating Autism Behaviours in Shank3 Mouse Model

CALGARY, Alberta, Oct. 08, 2026 (GLOBE NEWSWIRE) -- Marvel Biosciences Corp. (TSXV: MRVL | OTCQB: MBCOF ), and its wholly-owned subsidiary, Marvel Biotechnology Inc. (collectively the “Company” or “Marvel”), a drug discovery company developing novel therapeutics for autism spectrum disorder (“ASD”) and related neurodevelopmental disorders, is pleased to announce preliminary results from its preclinical study of MB-204 in the Shank3Δex13-16 mouse model , a model associated with Phelan-McDermid syndrome. The study was conducted in collaboration with Dr. Julie Le Merrer and Dr. Jérôme Becker at the iBraiN Institute in Tours, France , and evaluated MB-204 at oral doses of 1 mg/kg and 2.5 mg/kg compared with Istradefylline at 1 mg/kg, the highest dose tested that did not affect locomotion. Animals were studied for one to three hours post dosing. Istradefylline is a clinically approved drug for the treatment of Parkinson’s Disease. Preliminary Results Demonstrate Broad Activity of MB-204 The preliminary results indicate that MB-204 produced improvements across multiple behavioural endpoints associated with repetitive and abnormal behaviours in the Shank3 model. MB-204 continues to demonstrate activity across increasingly diverse genetic models, supported by the emerging Shank3 data, potentially advancing a broader therapeutic opportunity as the company approaches human clinical development. Key Data Highlights Low-dose MB-204 (1 mg/kg) matched or exceeded Istradefylline across the majority of measured endpoints : performance was matched on the number and time of nose contacts, number and time of paw contacts, the number of grooming episodes and number of same arm returns (SARs) in the Y-maze test of repetitive behaviour. Low dose of MB-204 demonstrated superior performance to Istradefylline on the mean duration of paw contacts, number of following episodes and the number of spontaneous and alternate arm returns (AARs) in the Y-maze. Only the number of rearing episodes was Istradefylline more effective than low dose MB-204. High-dose MB-204 (2.5 mg/kg) was superior to Istradefylline across the majority of measured endpoints: performance was matched only in the number of grooming episodes, rearing episodes and SARs. On all other endpoints as per above, MB-204 was superior to Istradefylline. All treatments showed demonstrable improvement in multiple behavioural endpoints over untreated Shank3 animals except for the number of following episodes and AARs in the Istradefylline treated animals. The study also provides a direct head-to-head comparison between MB-204 and its parent compound, Istradefylline , supporting the Company's objective of demonstrating differentiated activity for its proprietary fluorinated derivative. Final statistical analyses and additional endpoints remain pending. * The Company cautions that these are preliminary preclinical findings and that additional analyses may modify the interpretation of individual endpoints. “This is the third model where MB-204 has had a profound effect on socialization and repetitive behaviours,” said Drs. Le Merrer and Becker, “which are the key hallmarks of autism. MB-204 nearly reversed all the social deficits and repetitive behaviours in the Shank3 model as we previously saw in the Oprm1 mouse model and Rett syndrome models. This study also confirms the superiority of MB-204 over the parental drug Istradefylline in a head-to-head study. Together with the recent Fragile X results, the data suggests MB-204 may be a pan-spectrum treatment for autism. We look forward to presenting the data in upcoming conferences and publishing the data on MB-204 for the wider scientific community to review.” Expanding Evidence Across Genetically Distinct Neurodevelopmental Models The Shank3 findings add to a growing body of preclinical evidence generated for MB-204 across genetically distinct models of neurodevelopmental disorders. Marvel has previously reported positive preclinical results in: Oprm1: an autism model in which MB-204 demonstrated restoration of social interaction and improvements in repetitive behaviours following oral administration. Mecp2/Rett syndrome: MB-204 demonstrated near-reversal of social and behavioural deficits and outperformed Trofinetide across multiple endpoints in the Company's disclosed preclinical study. Fragile X (Fmr1): MB-204 produced statistically significant improvements in behavioural and cognitive function, including a carry-over effect after treatment cessation. Shank3: representing another genetically distinct model associated with autism and Phelan-McDermid syndrome. The Company believes that the emergence of consistent behavioural activity across models associated with different underlying genetic causes may be important in evaluating the potential breadth of MB-204's mechanism and therapeutic application. “The Shank3 results are particularly encouraging because they add another genetically distinct model to the growing body of evidence supporting MB-204,” said Dr. Mark Williams, President and Chief Science Officer of Marvel Biosciences. “Together with our previously reported Oprm1, Mecp2 and Fragile X results, these findings continue to support our hypothesis that targeting the adenosine A2A receptor may address behavioural and social deficits across multiple neurodevelopmental disorders. We look forward to completing the statistical analysis and presenting the full dataset to the scientific community.” About Marvel Biosciences Corp. Marvel Biosciences Corp. (TSXV:MRVL | OTCQB:MBCOF), and its wholly-owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company. The Company is developing MB-204, a novel fluorinated derivative of the approved anti-Parkinson’s drug Istradefylline, the only clinically approved adenosine A2a antagonist. A significant and growing body of scientific evidence suggests drugs that block the adenosine A2a receptor, such as MB-204, could be useful in treating other neurological diseases such as autism, depression and Alzheimer’s Disease. The Company is actively investigating its potential in addressing other neurodevelopmental disorders, such as Rett Syndrome and Fragile X Syndrome, to expand its therapeutic reach. Contact Information: Marvel Biosciences Corp. J. Roderick (Rod) Matheson, Chief Executive Officer Email: rod@marvelbiosciences.com Dr. Mark Williams, President and Chief Science Officer Email: mark@marvelbiosciences.com Tel: 403 770 2469 Website: | Twitter/X | LinkedIn Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is deﬁned in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this press release. All information contained in this news release with respect to the Company and its subsidiary,(collectively, the "Parties") were supplied by Marvel, respectively, for inclusion herein and each parties' directors and oﬃcers have relied on each other for any information concerning such Party. This news release may contain forward-looking statements and other statements that are not historical facts. Forward-looking statements are often identiﬁed by terms such as "will", "may", "should", "anticipate", "expects" and similar expressions. All statements other than statements of historical fact, included in this release, including, without limitation, statements regarding the future plans and objectives of the Company are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could diﬀer materially from those anticipated in such statements. Important factors that could cause actual results to diﬀer materially from the expectations of the Company and include other risks detailed from time to time in the ﬁlings made by the Company under securities regulations. The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to diﬀer materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. As a result, the Company cannot guarantee that the above events on the terms will occur and within the time disclosed herein or at all. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may diﬀer materially from those anticipated. Forward-looking statements contained in this news release are expressly qualiﬁed by this cautionary statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company will update or revise publicly any of the included forward-looking statements as expressly required by Canadian securities law.

Clinical ResultDrug ApprovalClinical Study

28 Sep 2026

·www.biospace.com

Acadia Pharmaceuticals Announces Allowance of U.S. Patent Covering Weight-Banded Dosing of Trofinetide, extending U.S. patent protections to March 2041

-- The allowance strengthens the intellectual property portfolio for trofinetide and extends protections in the U.S. from July 2036 to March 2041 SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. Patent Application No. 17/590,496, titled “Methods and Compositions for Treatment of Rett Syndrome.” The Notice of Allowance indicates that the USPTO has determined that the application meets the requirements for patentability, and the patent is expected to issue following completion of standard administrative steps. The patent application covers weight-banded dosing of trofinetide for the treatment of Rett syndrome. Following issuance and including six months of pediatric exclusivity, the patent is expected to provide protection for trofinetide in the United States through March 28, 2041, extending patent protections by almost five years. Trofinetide is also protected by a method of use patent for the treatment of Rett syndrome, which expires in July 2036. “This Notice of Allowance is an important addition to the intellectual property protections for trofinetide and reflects our commitment to supporting both current formulations of DAYBUE and the Rett syndrome community for the long term,” said Catherine Owen Adams, Chief Executive Officer of Acadia. “We are pleased that this patent is expected to extend protections for trofinetide in the United States from 2036 into 2041.” The Notice of Allowance relates to the weight-banded dosing regimen used for trofinetide, which is marketed in the United States as DAYBUE ® and DAYBUE ® STIX for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. About Rett Syndrome Rett syndrome is a rare, complex, neurodevelopmental disorder and occurs in approximately one of every 10,000 to 15,000 female births worldwide. 1-3 A child with Rett syndrome generally exhibits an early period of apparently normal development until six to 18 months, when many of their skills seem to slow down or stagnate. This is typically followed by a regression phase when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they could show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those individuals living with Rett may continue to experience a stage of motor deterioration, which can last the rest of the patient’s life. 2 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene. 4 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication and brain plasticity, and the deficits in synaptic function may be associated with Rett manifestations. 4-6 Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. 7 Most individuals living with Rett syndrome typically live into adulthood and require intense round-the-clock care. 1,8 Rett syndrome is associated with a substantial physical, psychological, social, and economic burden, significantly affecting the quality of life of both patients and their caregivers. 9-11 About DAYBUE ® (trofinetide) and DAYBUE ® STIX (trofinetide) Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals. 10 About Acadia Pharmaceuticals Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. 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 “may,” “will,” “should,” “expects,” “expected”, “anticipates,” 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: (i) the future issuance of U.S. Patent Application No. 17/590,496, which, if issued, will expire in March 2041, and the benefits Acadia expects to realize from its issuance; (ii) the expected expiry of the trofinetide method of use patent for the treatment of Rett syndrome in July 2036; and (iii) the efficacy and safety pro DAYBUE and DAYBUE STIX. 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: the inherent uncertainty of the patent issuance process and the intellectual property protections provided by Acadia’s patents, our ability to continue to successfully commercialize DAYBUE and DAYBUE STIX 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 period ended June 30, 2026 filed with the Securities and Exchange Commission on August 5, 2026, 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. References 1 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:e000717. 2 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216. 3 May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J Med Econ. 2023;26(1):1570-1580. 4 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188. 5 Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6):537-544. 6 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1):217-227. 7 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6):944-950. 8 Tarquinio DO, Hou W, Neul JL, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5):402-411. 9 Prange EO, Beisang A, Pehlivan D, et al. Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method. Annals of the Child Neurology Society. 2026; 38–51. 10 Kaufmann WE, Percy AK, Neul JL, et al. Burden of illness in Rett syndrome: initial evaluation of a disorder-specific caregiver survey. Orphanet J Rare Dis. 2024;19(1):296. 11 Gold WA, Percy AK, Neul JL, et al. Rett syndrome. Nat Rev Dis Primers. 2024;10(1):84. Contacts Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858) 261-2872 ir@acadia-pharm.com Acadia Pharmaceuticals Inc. Jessica Tieszen (858) 261-2950 ir@acadia-pharm.com Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043 media@acadia-pharm.com

Drug Approval

24 Sep 2026

·www.biospace.com

Acadia Pharmaceuticals Announces Phase 3 Enabling Topline Results from Phase 2 RADIANT Study of Remlifanserin for the Treatment of Alzheimer’s Disease Psychosis (ADP)

-- Meaningful and consistent efficacy trends across endpoints support continuing with Phase 3 development of 60 mg once daily remlifanserin in ADP, with demonstrated evidence of dose effect -- Narrowly missed primary endpoint of SAPS H+D (p=0.0603) with significance (nominal) on key secondary endpoint of CGI-S-ADP (p=0.0077) -- Favorable safety and tolerability pro both doses -- Detailed results to be presented at upcoming Clinical Trials on Alzheimer's Disease (CTAD) conference in November -- Conference call and webcast to be held today at 8:00 a.m. Eastern Time SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced Phase 3 enabling topline results from the Phase 2 portion of the ongoing RADIANT clinical trial program evaluating remlifanserin for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). For the primary endpoint of change from baseline in the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions subscales (SAPS-H+D), once daily 60 mg remlifanserin demonstrated a change of -12.6 versus a -10.4 change for placebo at week 6, yielding a standardized effect size of 0.26 (p=0.0603). For the key secondary endpoint, Clinical Global Impression – Severity (CGI-S-ADP), the 60 mg dose achieved a change from baseline of -1.3 versus a change of -0.9 for placebo at week 6, yielding a standardized effect size of 0.37 [p=0.0077 (nominal)]. On both efficacy measures, the difference in the 60 mg dose versus placebo increased through the 6-week treatment period. The 30 mg dose showed minimal improvement across endpoints compared to placebo. All analyses were performed in the pre-specified modified full analysis set (mFAS 1 ) population. In this study, across both doses, remlifanserin demonstrated a favorable safety profile, with rates of adverse events, serious adverse events, and discontinuations due to adverse events, similar to placebo. There was no signal of QT prolongation versus placebo. This dataset suggests no negative impact on motor symptoms or cognition. Additionally, there were no deaths in the remlifanserin treatment arms. Given these data, Acadia plans to continue enrolling its two ongoing Phase 3 studies evaluating remlifanserin in ADP while implementing amendments to the Phase 3 program, including removing the 30 mg dosage arm. “We are pleased that the Phase 2 RADIANT data support continuation of the Phase 3 ADP program, with new insights helping us to refine the program for future regulatory success. These results provide valuable information regarding the efficacy, safety and tolerability of once daily remlifanserin in Alzheimer’s disease psychosis,” said Catherine Owen Adams, Chief Executive Officer of Acadia Pharmaceuticals. “We are excited about the potential of remlifanserin in this area of high unmet need, for which there are no currently approved therapies.” “The Phase 2 RADIANT findings provide encouraging evidence that 60 mg once daily remlifanserin may have the potential to reduce hallucinations and delusions in patients with Alzheimer’s disease,” said Jeffrey Cummings, M.D., Sc.D., Director of the Chambers-Grundy Center for Transformative Neuroscience, University of Nevada, Las Vegas. “This patient population has a significant need for an efficacious medication that is well tolerated, convenient to administer, and is compatible with the many concomitant medications commonly used by patients with Alzheimer’s disease. In particular, avoiding negative impacts on motor symptoms and cognition is encouraging, and such a medicine could be an important treatment option for patients.” Acadia plans to present detailed safety and efficacy results from the RADIANT Phase 2 study in an oral presentation at the upcoming Clinical Trials on Alzheimer's Disease (CTAD) conference, taking place November 16-19, 2026 in Boston. Additionally, remlifanserin is currently being investigated in an additional Phase 2 study to evaluate its efficacy, safety and tolerability for the treatment of Lewy body dementia psychosis. Conference Call and Webcast Information Acadia will discuss top-line results from its Phase 2 RADIANT study of remlifanserin for the treatment of Alzheimer’s disease psychosis via conference call and webcast today at 8:00 a.m. Eastern Time. The conference call webcast is available at this registration link and will be available on Acadia’s website, acadia.com under the investors section and will be archived there for at least 90 days following the event. About RADIANT RADIANT is a global, multi-center, randomized, double-blind, placebo-controlled, operationally seamless Phase 2 and 3 clinical trial program evaluating remlifanserin for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). The Phase 2 portion of the program was designed to evaluate the efficacy, safety, and tolerability of remlifanserin at 60 mg and 30 mg once-daily doses compared with placebo. Patients who complete the study will have the option of participating in a long-term open-label extension (OLE) study. About Alzheimer’s Disease Psychosis According to the Alzheimer’s Association, more than 7 million people in the United States are living with Alzheimer’s disease (AD). 2,3 Approximately 30% of patients with AD experience psychosis, commonly consisting of hallucinations and delusions. 4 These symptoms may be frequent and severe and may recur over time. 5 A hallucination is defined as a perception-like experience that occurs without an external stimulus and is sensory (seen, heard, felt, tasted, sensed, smelled) in nature. A delusion is defined as a false, fixed belief despite evidence to the contrary. Serious consequences have been associated with psychosis in patients with dementia, such as increased likelihood of nursing home placement, more severe dementia, and increased risk of morbidity and mortality. 6,7 There is no FDA-approved drug for the treatment of Alzheimer’s disease psychosis. About Remlifanserin Remlifanserin is a novel, highly selective 5HT2A receptor inverse agonist being developed to treat Alzheimer’s disease psychosis and Lewy body dementia psychosis. About Acadia Pharmaceuticals Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. 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: (i) our clinical development plans to continue the Phase 3 portion of the program related to remlifanserin in Alzheimer’s disease psychosis and the Phase 2 study of remlifanserin in Lewy body dementia psychosis, (ii) our plans to implement protocol amendments to the Phase 3 portion of the program, and (iii) the safety pro remlifanserin, and the potential benefits that remlifanserin could provide to patients suffering with psychosis associated with Alzheimer’s disease. 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: risks associated with the discovery, development and regulation of pharmaceutical products in general; risks that our future clinical trials may not support approval of our product candidates; risks that future clinical trial results may not be consistent with interim, initial, preliminary or topline results or results from prior preclinical studies or clinical trials; our dependency on the continued successful commercialization of Nuplazid (Pimavanserin) and Daybue (Trofinetide) and our ability to maintain or increase sales of Nuplazid and Daybue; our ability to generate or obtain the necessary capital to fund our clinical development plans; the timing and results of our clinical trials; 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 period ended June 30, 2026 filed with the Securities and Exchange Commission on August 5, 2026, 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. References: 1 The Modified Full Analysis Set (mFAS) includes all randomized subjects who received at least one dose of study drug and who have a baseline SAPS-H+D total score ≥10 and at least one post-baseline value for SAPS-H+D total score. Subjects were analyzed based on their planned randomized treatment. 2 Alzheimer’s Association. 2025 Alzheimer’s Disease Facts and Figures. Alzheimer’s Dement 2025;21(4). 3 Rajan KB et al. Population estimate of people with clinical Alzheimer’s disease and mild cognitive impairment in the United States (2020-2060). Alzheimer’s and Dementia . 2021: 1-10. 4 Cummings J et al. Criteria for Psychosis in Major and Mild Neurocognitive Disorders: International Psychogeriatric Associations (IPA) Consensus Clinical and Research Definition. Am J of Geriatric Psychiatry . 2020; 28(12): 1256-1269. 5 Ballard C et al. A prospective study of psychotic symptoms in dementia sufferers: psychosis in dementia. Int Psychogeriatr . 1997; 9(1): 57-64. 6 Scarmeas N et al. Delusions and hallucinations are associated with worse outcome in Alzheimer Disease. Arch Neurol . 2005; 62(10): 1601-1608. 7 Peters ME et al. Neuropsychiatric symptoms as predictors of progression to severe Alzheimer’s dementia and death: the Cache County Dementia Progression study. Am J Psychiatry . 2015; 172(5): 460-465. Contacts Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858) 261-2872 ir@acadia-pharm.com Acadia Pharmaceuticals Inc. Jessica Tieszen (858) 261-2950 ir@acadia-pharm.com Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043 media@acadia-pharm.com

100 Deals associated with Trofinetide

External Link

KEGG	Wiki	ATC	Drug Bank
D12400	Trofinetide	-	DB06045

R&D Status

Approved

10 top approved records.

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

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Seizures	Phase 3	Japan	ACADIA Pharmaceuticals, Inc.	10 Jun 2025
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 2	United States	Neuren Pharmaceuticals Ltd.	01 Apr 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


LOTUS (AAN2026)	Not Applicable	Rett Syndrome	77	Trofinetide	qjspgiomfm(bberksjkcj) = vknlefzxwx cfmldrzlqd (rnknlislrc, 24 - 33)	Positive	25 Apr 2026
LILAC (IEC2025)	Not Applicable	Rett Syndrome	154	Trofinetide	lffkdfftlv(crqncohlap) = The most common AEs were diarrhea (74.7%) and vomiting (28.6%) xspmstcdqh (npbybktjfz )	Positive	30 Aug 2025
LILAC (IEC2025)	Not Applicable	Rett Syndrome	154	-		Positive	30 Aug 2025
NCT04988867 (DAFFODIL, Pubmed \| Med)	Phase 2/3	Rett Syndrome	-	Trofinetide	owyrgqxfjk(rvvjfzqvqs) = 80.0% vtuhpculoy (jbksmtvjon ) View more	Positive	01 Jun 2025
NCT04776746 (LILAC-2, CTgov)	Phase 3	Rett Syndrome	77	Trofinetide	szliltlvmn = zvhowijwpe lhzjcpzeda (dsxmgpysme, idznmlzhgt - modcpmqxfz) View more	-	27 Sep 2024
NCT04988867 (DAFFODIL \| DAFFODIL™, CTgov)	Phase 2/3	Rett Syndrome	15	Trofinetide	kxexpgisxi = gpghlzyvup ddwgohkshh (dojvldrdva, fqmpfmzmim - lqlffyzdbj) View more	-	24 Sep 2024
NCT04776746 (LILAC-2, BusinessWire) Manual	Not Applicable	Rett Syndrome	-	DAYBUE (LILAC-1)	fchkkueuhs(gfkjihbtnw) = nrjkpbzxjy ojqjwmdwyd (gvuexetgtw, 1.62) View more	Positive	18 Jul 2024
NCT04776746 (LILAC-2, BusinessWire) Manual	Not Applicable	Rett Syndrome	-	Placebo (LILAC-1)	fchkkueuhs(gfkjihbtnw) = oojpxiavsw ojqjwmdwyd (gvuexetgtw, 1.61) View more	Positive	18 Jul 2024
NCT04776746 (LILAC-2, Pubmed)	Phase 3	Rett Syndrome	77	Trofinetide	qsmwyozzkn(ntdydmlkoy) = 27.3% timowshjbm (eyphoyxwbs ) View more	Positive	16 Jul 2024
AAN2024 Manual	Not Applicable	Rett Syndrome	325	Trofinetide	umucoowctt(bavfphcbcy) = lyitflnozg dckycwwovt (qegkwawcxp )	Positive	13 Apr 2024
NCT04279314 (LILAC™, CTgov)	Phase 3	Rett Syndrome	154	Trofinetide	dwsdtfyzcm = rvuyyezzeb mufvtusecp (uimjtxcggt, gizvkcjita - zsnqjphwdj) View more	-	11 Apr 2024
S37.008 (AAN2024)	Phase 3	Rett Syndrome	33	Trofinetide (LILAC/LILAC-2)	flqfknhxnl(xzccwjmtfn) = qbxqvngnej rvejsqvaue (gnsjdfktwg ) View more	Positive	09 Apr 2024

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