Expert Opinion on Emerging Drugs最新文献_第3页

Injectable, oral, gene-based, and vaccine-driven PCSK9-targeted therapies: emerging clinical insights shaping the future of hypercholesterolemia management. 注射、口服、基于基因和疫苗驱动的pcsk9靶向治疗：塑造高胆固醇血症管理未来的新兴临床见解

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-04-29 DOI: 10.1080/14728214.2026.2662915

Thusyanthy Parameswaran, Amanda J Hooper, John R Burnett

引用次数: 0

Potassium channel agonists emerging as treatment options for focal epilepsy: are we breaking new ground? 钾通道激动剂成为局灶性癫痫的治疗选择：我们是否有新的突破？

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-05-19 DOI: 10.1080/14728214.2026.2675274

Cristiana Pelorosso, Simona Balestrini, Renzo Guerrini

{"title":"Potassium channel agonists emerging as treatment options for focal epilepsy: are we breaking new ground?","authors":"Cristiana Pelorosso, Simona Balestrini, Renzo Guerrini","doi":"10.1080/14728214.2026.2675274","DOIUrl":"10.1080/14728214.2026.2675274","url":null,"abstract":"Introduction: Focal epilepsy is a leading cause of neurological disability, with about one-third of patients failing to achieve seizure freedom despite numerous antiseizure medications (ASMs) are available. Most current therapies broadly modulate synaptic transmission, leading to dose-limiting cognitive, psychiatric, and systemic adverse effects. Kv7 (KCNQ) potassium channels, responsible for the neuronal M-current, represent a high-precision therapeutic target that regulates intrinsic excitability and provides a fundamental 'molecular brake' against pathological firing.Areas covered: This mini-review summarizes the clinical evolution of Kv7 modulation, from the first-generation prototype ezogabine to more selective second-generation candidates. We outline the scientific rationale for targeting the M-current, review emerging clinical data for agents such as azetukalner (XEN1101) and opakalim (BHV-7000), and highlight preclinical strategies including dual-mechanism modulators and drug repurposing.Expert opinion: Kv7 agonists offer a mechanistically elegant approach to restoring seizure resistance. Second-generation agents provide encouraging mechanistic and clinical proof-of-concept, but long-term success will depend on clear advantages in patient-centered outcomes over established ASMs. Future value is likely to lie in precision-medicine strategies for KCNQ2/3-related encephalopathies and a carefully defined role in managing neuropsychiatric comorbidities.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"29-36"},"PeriodicalIF":2.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147971385","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}

引用次数: 0

Emerging small molecules for ulcerative colitis: clinical advancements and trial insights. 溃疡性结肠炎新出现的小分子：临床进展和试验见解。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-06-07 DOI: 10.1080/14728214.2026.2680722

Cristina Lanzotti, Fabrizio Fanizzi, Ferdinando D'Amico, Alessandra Zilli, Federica Furfaro, Mariangela Allocca, Virginia Solitano, Clelia Cicerone, Sara Massironi, Tommaso Lorenzo Parigi, Laurent Peyrin-Biroulet, Silvio Danese

{"title":"Emerging small molecules for ulcerative colitis: clinical advancements and trial insights.","authors":"Cristina Lanzotti, Fabrizio Fanizzi, Ferdinando D'Amico, Alessandra Zilli, Federica Furfaro, Mariangela Allocca, Virginia Solitano, Clelia Cicerone, Sara Massironi, Tommaso Lorenzo Parigi, Laurent Peyrin-Biroulet, Silvio Danese","doi":"10.1080/14728214.2026.2680722","DOIUrl":"10.1080/14728214.2026.2680722","url":null,"abstract":"Introduction: Ulcerative colitis (UC) is an inflammatory bowel disease with a relapsing- remitting course, whose etiology is still unknown. Mild forms are generally managed with mesalazine, whereas moderate-to-severe disease may be addressed through a stepwise approach, often starting with corticosteroids and immunomodulators, and, when needed, progressing to advanced therapies targeting specific molecular pathways. Even though these therapies have led to substantial improvements in patients' quality of life and prognosis over recent decades, consistent and lasting therapeutic responses remain challenging to attain.Areas covered: This review aims to outline emerging small molecules for UC, emphasizing efficacy and safety data from recent phase II-III clinical trials, including both published studies and ongoing research. We performed a thorough literature search using PubMed, Scopus, and ClinicalTrials.gov, excluding earlier-phase studies, and identified additional relevant articles through reference screening.Expert opinion: It is essential to investigate innovative, safe, and easy-to-administer therapies that target alternative immunological pathways to enhance UC treatment. Some molecules, such as miRNA-124 upregulators, JAKi, S1P modulators, and LANCL2 agonists, may enrich our therapeutic armamentarium in UC management. To further optimize therapeutic strategies, combining small molecules with biologic agents could enhance treatment effectiveness, reduce immunogenicity, and improve long-term durability by targeting complementary inflammatory pathways.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"37-54"},"PeriodicalIF":2.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148194728","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}

引用次数: 0

Early investigational agents for the treatment of rosacea: drugs in phase II and III clinical development. 治疗酒渣鼻的早期研究药物：处于II期和III期临床开发的药物。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-05-14 DOI: 10.1080/14728214.2026.2655714

Kejia Tian, Ji Li, Ben Wang

{"title":"Early investigational agents for the treatment of rosacea: drugs in phase II and III clinical development.","authors":"Kejia Tian, Ji Li, Ben Wang","doi":"10.1080/14728214.2026.2655714","DOIUrl":"10.1080/14728214.2026.2655714","url":null,"abstract":"Introduction: Rosacea is a chronic inflammatory facial skin disease with an incompletely understood pathogenesis and limited efficacy of current treatments. A number of clinical trials are underway for the development of new drugs to improve patient care.Areas covered: Based on a literature search of PubMed, Embase, Cochrane Library, and Clinicaltrials.gov using the keywords 'rosacea clinical trials', 'rosacea treatment ' we discuss treatments undergoing phase II and III clinical trials for rosacea.Expert opinion: As the pathogenesis of rosacea is better understood and researched, many clinical trials are being conducted to develop new drugs for the treatment of rosacea based on new mechanisms and targets. Currently under investigation are biologics, antidepressants, PDE4 inhibitors, and neuropeptide modulation. Current treatments often focus on monotherapy targeting specific phenotypes, such as inflammatory papules or persistent erythema. However, single-agent therapy is often insufficient to address the multiple clinical manifestations of rosacea simultaneously. Therefore, we need to explore how combination or sequential treatments can be tailored to a patient's specific symptoms to minimize the patient's psychological burden.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"55-65"},"PeriodicalIF":2.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147927986","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}

引用次数: 0

Gene therapy for wet AMD: a paradigm shift in the standard of care. 湿性AMD的基因治疗：护理标准的范式转变。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2026-04-06 DOI: 10.1080/14728214.2026.2651330

Samuel Asanad, David S Boyer

{"title":"Gene therapy for wet AMD: a paradigm shift in the standard of care.","authors":"Samuel Asanad, David S Boyer","doi":"10.1080/14728214.2026.2651330","DOIUrl":"10.1080/14728214.2026.2651330","url":null,"abstract":"Introduction: Neovascular age-related macular degeneration (nAMD), or wet AMD, remains a leading cause of vision loss in older adults. Current standard of care relies on repeated intravitreal anti-vascular endothelial growth factor (anti-VEGF) injections, which create significant treatment burdens for patients and healthcare systems. Emerging gene therapies aim to address these challenges by delivering sustained therapeutic effects via single administration.Areas covered: Next-generation gene therapies are transforming AMD treatment by enabling sustained, autonomous production of therapeutic proteins within ocular tissues, enabling the eye to synthesize its own anti-VEGF agents. A comprehensive literature search was performed using both PubMed and ClinicalTrials.gov to identify pertinent manuscripts and clinical trials for this narrative review. Keywords utilized included: Gene therapy, wet AMD, neovascular AMD, Viral Vectors, Adeno-associated viral vectors, Subretinal, Suprachoroidal, and Retinal Pigment Epithelium.Expert opinion: Advances in gene therapy for wet AMD may revolutionize treatment by enabling sustained intraocular anti-VEGF protein, reducing frequent injections, improving patient adherence, and potentially lowering long-term healthcare costs. Although promising, challenges including long-term safety, complex delivery procedures, immune responses, regulatory hurdles, and the need for optimized vectors and clinical protocols must be addressed before widespread adoption and integration into standard care.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"241-249"},"PeriodicalIF":3.8,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147618667","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}

引用次数: 0

What can we expect from a combination of datopotamab deruxtecan, carboplatin, and pembrolizumab for brain metastases from non-small cell lung cancer? datopotamab deruxtecan，卡铂和派姆单抗联合治疗非小细胞肺癌脑转移，我们能期待什么？

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2026-03-30 DOI: 10.1080/14728214.2026.2643169

Ana C Z Gelatti, William N William

引用次数: 0

Emerging therapeutic strategies in immune thrombocytopenia: an expert evaluation of the evolving treatment landscape. 新出现的治疗策略在免疫性血小板减少症：不断发展的治疗景观的专家评价。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2025-11-15 DOI: 10.1080/14728214.2025.2590155

Nicole Charbel, Joe Rizkallah, Hassan Fawaz, Sacha El Khoury, Mohammad Hassan Hodroj, Ali Taher

{"title":"Emerging therapeutic strategies in immune thrombocytopenia: an expert evaluation of the evolving treatment landscape.","authors":"Nicole Charbel, Joe Rizkallah, Hassan Fawaz, Sacha El Khoury, Mohammad Hassan Hodroj, Ali Taher","doi":"10.1080/14728214.2025.2590155","DOIUrl":"10.1080/14728214.2025.2590155","url":null,"abstract":"Introduction: Immune thrombocytopenia (ITP) is an autoimmune disorder leading to low platelet counts and increased bleeding risk. The management of ITP, particularly in chronic or refractory cases, presents ongoing challenges, necessitating the development of novel therapeutic approaches that target its complex immunopathophysiology.Areas covered: This review evaluates the current ITP treatment landscape, including established first-line and second-line therapies, and provides an in-depth analysis of emerging drug classes. Key areas include neonatal Fc receptor inhibitors, Bruton's tyrosine kinase inhibitors, spleen tyrosine kinase inhibitors, complement inhibitors, and novel immunotherapies. The scientific rationale, clinical trial data, efficacy, safety profiles, and potential positioning of these agents in future ITP management algorithms are discussed. The literature search encompassed PubMed, Embase, and clinical trial registries for articles and data published up to early 2025.Expert opinion: Therapeutic options for ITP are rapidly expanding beyond conventional immunosuppressants and splenectomy. Emerging targeted therapies offer the promise of improved efficacy, better safety profiles, and the potential for durable, treatment-free remission. Future research should focus on personalized medicine approaches, biomarker identification for predicting treatment response, optimizing treatment sequencing, and understanding long-term outcomes to transform ITP care.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"251-262"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145502803","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}

引用次数: 0

Targeting the gut microbiota as a treatment for obesity and cancer cachexia. 靶向肠道微生物群治疗肥胖和癌症恶病质。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2026-03-29 DOI: 10.1080/14728214.2026.2650180

Anna Giannakogeorgou, Tom van den Ende, Barbara J H Verhaar, Nicolien de Clercq, Hanneke W M van Laarhoven, Max Nieuwdorp

{"title":"Targeting the gut microbiota as a treatment for obesity and cancer cachexia.","authors":"Anna Giannakogeorgou, Tom van den Ende, Barbara J H Verhaar, Nicolien de Clercq, Hanneke W M van Laarhoven, Max Nieuwdorp","doi":"10.1080/14728214.2026.2650180","DOIUrl":"10.1080/14728214.2026.2650180","url":null,"abstract":"Introduction: Obesity and cancer cachexia represent two seemingly contrasting yet interrelated ends of the metabolic disorder spectrum, both characterized by disrupted energy homeostasis, inflammation and neuroendocrine dysfunction, and associated with increased morbidity and mortality. Existing treatments often fail to address the complex underlying pathophysiological mechanisms. Emerging research highlights the role of the gut microbiome in the pathophysiology of both conditions and how it can serve as a novel therapeutic target.Areas covered: This review explores shared and distinct pathways linking obesity and cancer cachexia. Key systems discussed include the gut-brain axis as well as skeletal muscle and adipose tissue metabolism. We discuss how the gut microbiota influences these processes through (diet-derived) gut microbial metabolites that affect specific signaling pathways. The review evaluates the efficacy and limitations of current anti-obesity and cachexia therapies and summarizes clinical and preclinical interventions targeting the gut microbiome, including pre-, pro-, postbiotics and fecal microbiota transplantation.Expert opinion: The gut microbiota holds potential as a therapeutic target in metabolic diseases, offering opportunities for precision medicine based on microbial and metabolic profiles. While early microbiota-based therapies show promise, further investigation into mechanistic pathways and novel engineered microbiota is essential to develop effective treatments for obesity and cachexia.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"225-240"},"PeriodicalIF":3.8,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147493849","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}

引用次数: 0

Exploring the latest emerging drugs for the treatment of sickle cell disease. 探索治疗镰状细胞病的最新药物。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2026-03-27 DOI: 10.1080/14728214.2026.2650178

Giovanna Cannas

{"title":"Exploring the latest emerging drugs for the treatment of sickle cell disease.","authors":"Giovanna Cannas","doi":"10.1080/14728214.2026.2650178","DOIUrl":"10.1080/14728214.2026.2650178","url":null,"abstract":"Introduction: Sickle cell disease (SCD) is an inherited autosomal recessive hemoglobinopathy that causes significant morbidity and mortality in children and adults. The availability of novel therapeutic agents that are safe, effective, and affordable and new cell therapy techniques remains highly desirable for the treatment of SCD.Areas covered: After a brief reminder of the main SCD complications, this review summarizes emerging disease-modifying agents and promising cell therapy strategies for the treatment of patients with SCD. A comprehensive search to look for the efficacy and safety of new agents was made on PubMed.ncbi.nlm.nih.gov for published studies and ClinicalTrials.gov for registered trials.Expert opinion: The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"263-281"},"PeriodicalIF":3.8,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147497909","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}

引用次数: 0

Future treatments for myelin oligodendrocyte glycoprotein antibody-associated disease: the clinical trial landscape. 髓鞘少突胶质细胞糖蛋白抗体相关疾病的未来治疗：临床试验前景

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2025-12-01 Epub Date: 2025-09-24 DOI: 10.1080/14728214.2025.2565189

Edgar Carnero Contentti, Vinícius de Oliveira Boldrini, Adriana Casallas-Vanegas, Sanja Gluscevic, Emine Rabia Koc, Sara Samadzadeh, Meral Seferoğlu, Natalia Szejko, Michael Levy

{"title":"Future treatments for myelin oligodendrocyte glycoprotein antibody-associated disease: the clinical trial landscape.","authors":"Edgar Carnero Contentti, Vinícius de Oliveira Boldrini, Adriana Casallas-Vanegas, Sanja Gluscevic, Emine Rabia Koc, Sara Samadzadeh, Meral Seferoğlu, Natalia Szejko, Michael Levy","doi":"10.1080/14728214.2025.2565189","DOIUrl":"10.1080/14728214.2025.2565189","url":null,"abstract":"Introduction: Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is an emerging autoimmune demyelinating disorder distinct from multiple sclerosis and AQP4-IgG-positive neuromyelitis optica. Despite increasing recognition, no therapies are currently approved for MOGAD, and treatment remains empirical, with significant variability in clinical response and access to care.Areas covered: This review explores the evolving treatment landscape of adult MOGAD, with a focus on immunotherapies under active clinical investigation: azathioprine, tocilizumab, satralizumab, and rozanolixizumab. For each agent, we discuss mechanisms of action, pharmacokinetics, dosing, safety, and efficacy based on clinical trials and observational data. Literature was identified through PubMed and ClinicalTrials.gov, including ongoing phase 2/3 studies (MOGwAI, TOMATO, METEOROID, and cosMOG).Expert opinion: Targeted immunotherapies have the potential to transform MOGAD management. In the next five years, one or more of these agents may achieve regulatory approval, particularly if biomarker-driven strategies and trial designs are refined. Addressing unmet needs in pediatric populations and low-resource settings will be essential to ensure equitable, personalized treatment.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"283-297"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145124742","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}

引用次数: 0