- |||||||||| Daybue (trofinetide) / Acadia Pharma
Genetics, Molecular Neuroscience, and Clinical Trials on the Path to the First FDA-Approved Treatment in Rett Syndrome () - Nov 2, 2025 - Abstract #AACAP2025AACAP_530;
The intersection of genetic discovery, preclinical disease model evaluation, and natural history studies focused on clinical trial readiness enabled the identification and evaluation of novel therapies in RTT. The successful approval of trofinetide for the treatment of RTT provides evidence of the potential to develop new treatments for RTT as well as other neurodevelopmental disorders.GS, ND, RCT
- |||||||||| Daybue (trofinetide) / Acadia Pharma, plerixafor / Generic mfg., memantine / Generic mfg.
Journal: Network-Medicine-Guided Drug Repurposing for Alzheimer's Disease: A Multi-Dimensional Systems Pharmacology Approach. (Pubmed Central) - Oct 29, 2025
Successful identification of the FDA-approved AD therapeutics memantine and donepezil among the top candidates validated the computational performance, while the predominance of mechanistic evidence classifications (86.7%) highlighted that network predictions represent hypothesis-generating tools requiring systematic experimental validation rather than definitive therapeutic recommendations. The framework bridges computational predictions with pharmaceutical development requirements, providing actionable prioritization for systematic preclinical investigation addressing AD intervention.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Journal: Visual Hallucinations in Serotonergic Psychedelics and Lewy Body Diseases. (Pubmed Central) - Oct 6, 2025
Examining cortical and neuromodulatory similarities between LBD- and SP-induced VH may elucidate the link between sensory degradation, excitation, and hallucinogenesis. Future research should employ real-time neuroimaging of discrete hallucinatory episodes to identify shared mechanisms and develop targeted interventions for LBD hallucinations.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Journal: Pimavanserin -induced decreased appetite and weight loss: A case report. (Pubmed Central) - Sep 29, 2025
Future research should employ real-time neuroimaging of discrete hallucinatory episodes to identify shared mechanisms and develop targeted interventions for LBD hallucinations. No abstract available
- |||||||||| ACP-01 / Hemostemix, Aspire Health Science, intranasal carbetocin (ACP-101) / Acadia Pharma
Journal: Signature motif identification and enzymatic characterization of a protein tyrosine phosphatase in Metarhizium anisopliae (Pubmed Central) - Sep 25, 2025
Functional analysis confirmed that the L-protein retained the PTP activity despite the absence of arginine in its signature motif, while AcP101 functioned as an acid phosphatase. This study provides the first functional validation of an arginine-deficient PTP motif, expanding the definition of PTP signature motifs and offering new insights for phosphatase classification.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
7814: From Collapse to Control: Rewriting OSA Treatment With Targeted Pharmacotherapy (McCormick Place - Lakeside Center Exhibit Hall Experience CHEST) - Sep 10, 2025 - Abstract #CHEST2025CHEST_7035;
This session will explore the latest clinical data and mechanistic rationale behind a diverse group of agents, including tirzepatide (weight loss/passive critical closing pressure), atomoxetine-oxybutynin (dilator muscle tone), pimavanserin and buspirone (arousal threshold), and acetazolamide (loop gain). Attendees will learn how to think beyond CPAP and match the right therapy to the right OSA phenotype.
- |||||||||| Review, Journal: Posttraumatic Stress Disorder Psychopharmacology Algorithm Update-2024-2025. (Pubmed Central) - Aug 29, 2025
After treating PTSD insomnia, if significant daytime symptoms remain, a selective serotonin reuptake inhibitor ( SSRI, sertraline or paroxetine are suggested), and this may be augmented by an antipsychotic (consider aripiprazole first) if psychotic symptoms, if any, do not respond to the SSRI...This update also evaluates emerging treatments, including transcranial magnetic stimulation, direct current stimulation, stellate ganglion block, and pimavanserin, though concluding that current evidence does not yet warrant their inclusion in the core algorithm. This update provides clinicians with an evidenceinformed approach to pharmacological PTSD management based on the latest available data.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Trial completion date, Trial primary completion date: C-SAPP: Pimavanserin vs. Quetiapine for Treatment of Parkinson's Psychosis (clinicaltrials.gov) - Aug 28, 2025
P4, N=358, Recruiting,
This update provides clinicians with an evidenceinformed approach to pharmacological PTSD management based on the latest available data. Trial completion date: Aug 2026 --> Aug 2027 | Trial primary completion date: Oct 2025 --> Oct 2026
- |||||||||| Daybue (trofinetide) / Acadia Pharma
A new human neuronal model for evaluating therapeutic AAV-MECP2 regulation in Rett syndrome (Fibes 2) - Aug 21, 2025 - Abstract #ESGCT2025ESGCT_691;
Existing treatments primarily manage symptoms without addressing the underlying genetic cause, including trofinetide, the first and only FDA-approved drug to treat RTT...In summary, our innovative MeCP2 KO SK-N-SH neuronal model, coupled with a versatile high-throughput analytical platform, provides a critical advancement for preclinical evaluation and refinement of AAV-based gene therapies. This model promises not only to significantly impact RTT therapeutic strategies, but also to offer critical insights into general mechanisms underlying gene therapy regulation, advancing treatments for RTT and potentially other neurodevelopmental disorders.
- |||||||||| risperidone / Generic mfg., Nuplazid (pimavanserin) / Acadia Pharma, quetiapine / Generic mfg.
Retrospective data, Review, Journal: Subjectively reported sleep improvement with antipsychotic medications in clinical practice: A systematic review, meta-analysis and meta-regression of moderating factors. (Pubmed Central) - Aug 6, 2025
APs, mostly SGAs, improved sleep quality more than placebo, and this effect was more pronounced in anxiety disorders, for anti-histaminergic APs, and in longer trials. These results could add another important tile to the mosaic of precision medicine for prescribing APs.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Journal: Pimavanserin safety in adult and elderly patients with neuropsychiatric symptoms related to neurodegenerative disease: An open-label extension study. (Pubmed Central) - Jun 16, 2025
The mean (standard error) change from DB baseline to OLE Week 52 in MMSE, ESRS-A, CGI-S, EQ-5D-5L, and SDI scores was +0.9 (0.21), -0.3 (0.22), -1.0 (0.05),?+?10.7 (0.87), and -0.9 (0.07), respectively. No patients reported suicidal behavior.ConclusionsPimavanserin was generally well tolerated in frail older adults and elderly patients with neuropsychiatric symptoms related to NDD for up to 52 weeks of treatment.
- |||||||||| Daybue (trofinetide) / Acadia Pharma
Trial completion date, Trial primary completion date: Cognitive Function in Rett Syndrome During Trofinetide Treatment (clinicaltrials.gov) - Jun 12, 2025
P=N/A, N=20, Recruiting,
Recruiting --> Active, not recruiting Trial completion date: May 2025 --> Jun 2026 | Trial primary completion date: May 2025 --> Jun 2026
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma, quetiapine / Generic mfg., clozapine / Generic mfg.
Review, Journal: Sedation and sleep safety of pimavanserin for Parkinsons's disease psychosis: Review and exploratory analysis of clinical study data. (Pubmed Central) - Jun 9, 2025
The results from our exploratory analyses suggest that pimavanserin may be associated with negligible levels of sedation and other sleep?related adverse events and may provide improvements in nighttime sleep compared with placebo. Future head-to-head clinical trials will be needed for direct comparisons between pimavanserin and other atypical antipsychotics, such as quetiapine or clozapine.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Persistent 5-HT2AR Signaling Triggered by Prolonged Exposure to a Serotonergic Psychedelic In Vitro (Napoleon Ballroom A1-3, Third Floor) - Jun 8, 2025 - Abstract #CPDD2025CPDD_262;
Our data indicate that 5-HT2AR signaling induced by prolonged (-)-DOI exposure in vitro is paradoxically sustained despite removal of the agonist from the extracellular space. Enduring 5-HT2AR signaling produced by (-)-DOI may contribute to its ameliorative effects and continuing studies will evaluate if persistent 5-HT2AR signaling supports psychoplastogenesis in vivo.
- |||||||||| morphine sulphate / Generic mfg.
Preclinical, Journal: 5-HT2A receptor inverse agonist attenuates morphine withdrawal syndrome and its aversiveness in rats. (Pubmed Central) - May 20, 2025
The pimavanserin injection also significantly attenuated the aversiveness of morphine withdrawal, as indicated by reduced conditioned avoidance of the chamber where precipitated withdrawal had occurred. These results indicate a major role for the 5-HT2A receptor in opiate physical dependence and withdrawal syndrome, suggesting this receptor as a potential therapeutic target.
- |||||||||| NNZ-2591 / Neuren, Acadia Pharma
Enrollment change: An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) (clinicaltrials.gov) - May 19, 2025
P2, N=28, Completed,
These results indicate a major role for the 5-HT2A receptor in opiate physical dependence and withdrawal syndrome, suggesting this receptor as a potential therapeutic target. N=16 --> 28
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Review, Journal: Treating psychosis in people with Parkinson's disease. (Pubmed Central) - May 2, 2025
Pimavanserin is the first-line treatment for mild psychotic symptoms that do not require a rapid response and quetiapine and clozapine for psychosis that requires improvement in a short period of time. Antipsychotics used to treat PDP should demonstrate safety, motor tolerability and efficacy over time.
- |||||||||| Daybue (trofinetide) / Acadia Pharma
Biomarker, Review, Journal: A Blueprint for Translational Precision Medicine in Autism Spectrum Disorder and Related Neurogenetic Syndromes. (Pubmed Central) - May 1, 2025
Factors that contributed to the approval of trofinetide for RTT included replicated findings in mouse models, a well-studied natural history of the syndrome, development of RTT-specific outcome measures, and strong engagement of the RTT family community. The translation of our growing understanding of the neurobiology of ASD to human drug discovery will require a precision medicine approach, including the use of multiple model systems for molecular target selection, evaluation of target engagement, and clinical trial design strategies that address heterogeneity, power, and the placebo response.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Clinical, P3 data, Journal: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Pimavanserin as an Adjunctive Treatment for the Negative Symptoms of Schizophrenia (ADVANCE-2) in Patients With Predominant Negative Symptoms. (Pubmed Central) - Apr 4, 2025
While switching strategies are suggested, further study is needed to assess combination antipsychotic therapy. In this study, pimavanserin was well tolerated, and although it demonstrated a similar treatment effect as in the prior phase 2 study favoring pimavanserin, treatment with pimavanserin vs placebo did not result in significant differences for primary or other endpoints.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Trial completion date, Trial termination, Trial primary completion date: Extension Study of Pimavanserin in Irritability Associated With Autism Spectrum Disorder (clinicaltrials.gov) - Apr 4, 2025
P2/3, N=209, Terminated,
In this study, pimavanserin was well tolerated, and although it demonstrated a similar treatment effect as in the prior phase 2 study favoring pimavanserin, treatment with pimavanserin vs placebo did not result in significant differences for primary or other endpoints. Trial completion date: Jul 2025 --> Feb 2025 | Active, not recruiting --> Terminated | Trial primary completion date: Jul 2025 --> Feb 2025; The study was terminated for business reasons and not due to safety concerns.
- |||||||||| Nuplazid (pimavanserin) / Acadia Pharma
Trial completion date, Trial primary completion date: Pimavanserin and Aggression and Social Cognition. (clinicaltrials.gov) - Mar 27, 2025
P2, N=35, Recruiting,
Active, not recruiting --> Completed Trial completion date: Jan 2026 --> Jan 2028 | Trial primary completion date: Jan 2026 --> Jan 2027
- |||||||||| Review, Journal: Third-Generation Antipsychotics: The Quest for the Key to Neurotrophism. (Pubmed Central) - Mar 27, 2025
Although data remain limited and focused primarily on earlier SGAs, emerging findings suggest that some TGAs may exert positive effects on neuroplastic processes, including the modulation of brain-derived neurotrophic factors (BDNFs) and synaptic architecture. However, robust clinical data on their long-term effects and comparative efficacy are lacking; therefore, further research is necessary to validate their role in preventing neurodegenerative changes and improving cognitive outcomes in patients with psychiatric conditions.
|
