{"title":"Muscarinic M1 and M4 agents as treatments for schizophrenia: what do they do and who do they do it for?","authors":"Philip D Harvey","doi":"10.1080/14728214.2025.2458053","DOIUrl":"10.1080/14728214.2025.2458053","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-5"},"PeriodicalIF":2.7,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002924","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kevin Mok, Olivia Chen, Johnny Yau, Landon L Chan, Stephen L Chan
{"title":"Systemic therapy for child-pugh B patients with hepatocellular carcinoma.","authors":"Kevin Mok, Olivia Chen, Johnny Yau, Landon L Chan, Stephen L Chan","doi":"10.1080/14728214.2025.2456774","DOIUrl":"https://doi.org/10.1080/14728214.2025.2456774","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002928","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kyle Valentino, Kayla M Teopiz, Sabrina Wong, Melanie C Zhang, Gia Han Le, Hayun Choi, Hana Ballum, Christine Dri, William Cheung, Roger S McIntyre
{"title":"Seltorexant for major depressive disorder.","authors":"Kyle Valentino, Kayla M Teopiz, Sabrina Wong, Melanie C Zhang, Gia Han Le, Hayun Choi, Hana Ballum, Christine Dri, William Cheung, Roger S McIntyre","doi":"10.1080/14728214.2025.2452514","DOIUrl":"10.1080/14728214.2025.2452514","url":null,"abstract":"<p><strong>Introduction: </strong>Preclinical and clinical pharmacologic evidence indicates that orexin systems are relevant to sleep-wake cycle regulation and dimensions of reward and cognition, providing the basis for hypothesizing that they may be effective as therapeutics in mental disorders. Due to the limited efficacy and tolerability profiles of existing treatments for Major Depressive Disorder (MDD), investigational compounds in novel treatment classes are needed; seltorexant, an orexin receptor antagonist, is a potential new treatment currently under investigation.</p><p><strong>Areas covered: </strong>Mechanisms implicated in MDD, including reward and sleep, are first overviewed. Then, the safety, tolerability, and efficacy profiles of seltorexant and the wider context of orexin receptor antagonism for depression are discussed in focus. Preclinical and clinical data are also discussed. PubMed, Medline, Cochrane Library, Embase, Scopus, and Web of Science were systematically searched from inception to 10 October 2024, in accordance with PRISMA guidelines.</p><p><strong>Expert opinion: </strong>Early clinical evidence suggests that seltorexant is effective in treating MDD, both in individuals diagnosed with insomnia and those not, although greater antidepressant effects are observed in individuals with severe sleep disturbance. Results from large phase III clinical trials are needed to confirm efficacy and safety.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-11"},"PeriodicalIF":2.7,"publicationDate":"2025-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142947157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Davide Politano, Davide Tonduti, Roberta Battini, Elisa Fazzi, Simona Orcesi
{"title":"Exploring emerging JAK inhibitors in the treatment of Aicardi-Goutières syndrome.","authors":"Davide Politano, Davide Tonduti, Roberta Battini, Elisa Fazzi, Simona Orcesi","doi":"10.1080/14728214.2024.2445508","DOIUrl":"10.1080/14728214.2024.2445508","url":null,"abstract":"<p><strong>Introduction: </strong>Aicardi-Goutières syndrome (AGS) is a genetically heterogeneous monogenic autoinflammatory disorder classified as an 'interferonopathy'. Nine genes have been implicated in AGS, encoding proteins involved in nucleic acid clearance, repair, sensing, or histone pre-mRNA processing. Dysregulation in these pathways leads to excessive type I interferon production, the primary driver of the disease. AGS typically presents with early-life neurological regression, followed by stabilization with varying degrees of neurological impairment and common extra-neurological features, such as chilblains. Advances in understanding AGS pathogenesis have enabled the development of new therapies, with JAK inhibitors emerging as the most studied option for reducing interferon-mediated effects.</p><p><strong>Areas covered: </strong>This review discusses the clinical features, genetic basis, and molecular pathways of AGS while tracing the evolution of its therapeutic strategies. Particular emphasis is placed on JAK inhibitors, which target proteins activated by type I interferons, providing a novel direction in treatment.</p><p><strong>Expert opinion: </strong>Inhibitors effectively reduce extra-neurological symptoms in AGS, though their impact on neurological outcomes remains unclear. The unknown natural history of AGS limits treatment evaluation. Despite growing insights, key aspects of pathogenesis and treatment optimization - including timing, administration, and long-term effects - remain unresolved, highlighting the need for further research.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-19"},"PeriodicalIF":2.7,"publicationDate":"2024-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142863686","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman
{"title":"Monoclonal antibodies in phase II and III trials for moderate to severe atopic dermatitis.","authors":"Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman","doi":"10.1080/14728214.2024.2416114","DOIUrl":"10.1080/14728214.2024.2416114","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a prevalent chronic inflammatory skin disease that significantly affects quality of life and mental health, especially in children. Traditional treatments include chemotherapeutics, topical corticosteroids, and immunomodulatory agents, but recent advances have introduced novel monoclonal antibody therapies. Through this comprehensive review paper, we aim to discuss these therapeutic options and their role in treating atopic dermatitis.</p><p><strong>Areas covered: </strong>A comprehensive search of the NIH Clinical Trials database was conducted from September 2023 to January 2024, focusing on phase 2 and 3 trials for AD treatments. Trials were filtered using keywords such as 'atopic dermatitis,' 'monoclonal antibody,' and 'phase 2/3.' Out of 25 trials analyzed, 11 were in phase 2 and 14 in phase 3. Only U.S.-based trials comparing novel therapies to placebo were included. In addition to the clinical trial database, we utilized the companies' websites and relevant abstracts to gather the latest results.</p><p><strong>Expert opinion: </strong>Currently investigated monoclonal antibodies have the ability to transform management by targeting specific mediators implicated in the inflammatory pathway of AD. The results of Phase II and III trials for monoclonal antibodies demonstrated strong therapeutic potential with significant reductions in EASI scores and represent a promising new targeted treatment option.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"361-368"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399909","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour
{"title":"Promising PD-1 antagonists for liver cancer: an evaluation of phase II and III results.","authors":"Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour","doi":"10.1080/14728214.2024.2430493","DOIUrl":"10.1080/14728214.2024.2430493","url":null,"abstract":"<p><strong>Introduction: </strong>Hepatocellular carcinoma (HCC), the most common primary liver cancer, is a major cause of cancer-related morbidity and mortality. Limited treatment options for advanced stages highlight the need for effective therapies.</p><p><strong>Areas covered: </strong>This review explores immune checkpoint inhibitors (ICIs), specifically PD-1, PD-L1, and CTLA-4 inhibitors, as emerging treatments for advanced HCC. It discusses data from phase II and III trials evaluating ICI combinations with tyrosine kinase inhibitors (TKIs), anti-angiogenic agents, and locoregional treatments like Transarterial Chemoembolization (TACE). Clinical outcomes, including progression-free survival and response rates, were analyzed alongside the incidence and management of immune-related adverse events (irAEs). A systematic review approach ensured comprehensive, high-quality study inclusion.</p><p><strong>Expert opinion: </strong>ICI-based therapies and their combinations are transforming advanced HCC treatment, offering improved outcomes and potential survival benefits. However, these therapies need optimization in sequencing and selection, particularly considering variations in liver function and disease stage. Effective management of adverse effects is critical to maximize clinical benefits. Further research is required to develop personalized strategies, tailoring treatments to patient-specific factors and enhancing safety and effectiveness in HCC management.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"369-382"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643385","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Current and emerging drug treatment strategies to tackle sickle cell anemia.","authors":"Yogindra Persaud, Alexis Leonard, Parul Rai","doi":"10.1080/14728214.2024.2379260","DOIUrl":"10.1080/14728214.2024.2379260","url":null,"abstract":"<p><strong>Introduction: </strong>Since its discovery in the early 1900s, sickle cell disease (SCD) has contributed significantly to the scientific understanding of hemoglobin and hemoglobinopathies. Despite this, now almost a century later, optimal medical management and even curative options remain limited. Encouragingly, in the last decade, there has been a push toward advancing the care for individuals with SCD and a diversifying interest in options to manage this disorder.</p><p><strong>Areas covered: </strong>Here, we review the current state of disease modifying therapies for SCD including fetal hemoglobin inducers, monoclonal antibodies, anti-inflammatory modulators, and enzyme activators. We also discuss current curative strategies with specific interest in transformative gene therapies.</p><p><strong>Expert opinion: </strong>SCD is a chronic, progressive disease that despite a century of clinical description, only now is seeing a growth and advance in therapeutic options to improve the lifespan and quality of life for individuals with SCD. We anticipate newly designed and even repurposed therapies that may work as a single agent or combination agents to tackle the progression of SCD. The vast majority of individuals living with SCD are unlikely to receive gene therapy, therefore improved disease management is critical even for those that may ultimately chose to pursue a potentially curative strategy.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"327-346"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141579372","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir
{"title":"A comprehensive review of phase 2/3 trials in osteoarthritis: an expert opinion.","authors":"Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir","doi":"10.1080/14728214.2024.2386174","DOIUrl":"10.1080/14728214.2024.2386174","url":null,"abstract":"<p><strong>Introduction: </strong>Osteoarthritis (OA) is a chronic, degenerative, and debilitating disease associated with significant long-term morbidity and disability. The pathogenesis of OA is not completely understood but involves an interplay between environmental risk factors, joint mechanics, abnormal pain pathways and upregulation of inflammatory signaling pathways. Current therapeutic options for patients are limited to conservative management, minimal pharmacological options or surgical management, with significant caveats to all approaches.</p><p><strong>Areas covered: </strong>In this review, we have set out to investigate current phase II/III clinical trials by undertaking a PubMed search. Examined clinical trials have explored a myriad of potential therapeutics from conventional disease-modifying anti-rheumatic drugs and biologics usually used in the treatment of inflammatory arthritides, to more novel approaches targeting inflammatory pathways implicated in OA, cartilage degeneration or pain pathways.</p><p><strong>Expert opinion: </strong>Unfortunately, most completed phase II/III clinical trials have shown little impact on patient pain scores, with the exception of the traditional DMARD methotrexate and Sprifermin. Methotrexate has been shown to be beneficial when used in the correct patient cohort (MRI proven synovitis). Sprifermin has the longest follow-up data of 5 years and has been shown to reduce loss of MRI-measured cartilage thickness and pain scores.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"347-359"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet
{"title":"ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype?","authors":"Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet","doi":"10.1080/14728214.2024.2426649","DOIUrl":"10.1080/14728214.2024.2426649","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"321-325"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Emerging immunotherapies in the Hodgkin lymphoma armamentarium.","authors":"Michael A Spinner, Ranjana H Advani","doi":"10.1080/14728214.2024.2349083","DOIUrl":"10.1080/14728214.2024.2349083","url":null,"abstract":"<p><strong>Introduction: </strong>Brentuximab vedotin and PD-1 inhibitors have improved outcomes for classic Hodgkin lymphoma (cHL), but better therapies are needed for patients who relapse after these agents. Based on an improved understanding of cHL biology, there is a robust pipeline of novel therapies in development. In this review, we highlight emerging immunotherapeutic agents and combinations for cHL.</p><p><strong>Areas covered: </strong>We review clinical trials of novel PD-1/PD-L1 inhibitors beyond FDA-approved agents, checkpoint inhibitors targeting CTLA-4, LAG-3, TIM-3, TIGIT, and CD47/SIRPα, PD-1 inhibitor combinations with immunomodulatory agents and epigenetic modifying therapies, antibody-drug conjugates, bispecific antibodies, and cellular therapies including anti-CD30 CAR-T and allogeneic NK cell therapy. We review the key safety and efficacy data from published phase 1-2 studies and highlight trials in progress, including the first phase 3 trial for PD-1 inhibitor-refractory cHL.</p><p><strong>Expert opinion: </strong>Many novel immunotherapies hold great promise in cHL. Rational combinations with existing agents and next-generation antibody and CAR-T constructs may improve response rates and durability. Identifying biomarkers of response to these immunotherapies and using more sensitive tools to assess response, such as circulating tumor DNA, may further inform treatment decisions and enable a precision medicine approach in the future.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"263-275"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140862631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}