Expert opinion on investigational drugs最新文献

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Mirdametinib, an FDA-Approved MEK1/2 inhibitor for adult and pediatric NF1-associated plexiform neurofibromas. Mirdametinib, fda批准用于成人和儿童nf1相关丛状神经纤维瘤的MEK1/2抑制剂。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-09-11 DOI: 10.1080/13543784.2025.2558656
Gorkem Oztosun, Amy Armstrong, Angela C Hirbe
{"title":"Mirdametinib, an FDA-Approved MEK1/2 inhibitor for adult and pediatric NF1-associated plexiform neurofibromas.","authors":"Gorkem Oztosun, Amy Armstrong, Angela C Hirbe","doi":"10.1080/13543784.2025.2558656","DOIUrl":"https://doi.org/10.1080/13543784.2025.2558656","url":null,"abstract":"<p><strong>Introduction: </strong>Neurofibromatosis type 1 (NF1) is an autosomal-dominant cancer predisposition syndrome that leads to the development of plexiform neurofibromas (PNs), which can cause significant morbidity and are at risk to transform into malignant peripheral nerve sheath tumors (MPNSTs). Targeted therapies such as mirdametinib, a selective oral MEK1/2 inhibitor, have offered new treatment options for patients with symptomatic, inoperable PNs.</p><p><strong>Areas covered: </strong>This review summarizes the pathophysiology of NF1, the role of MEK inhibition, and the development of mirdametinib as a treatment for NF1-associated plexiform neurofibromas. Relevant literature was identified through PubMed searches using keywords related to NF1, plexiform neurofibromas, MEK inhibition, and mirdametinib. Key aspects discussed include mirdametinib's pharmacologic properties, clinical efficacy, safety profile, and comparison with other MEK inhibitors.</p><p><strong>Expert opinion: </strong>Mirdametinib offers an effective, targeted, and orally available treatment for NF1-associated plexiform neurofibromas, with the added advantage of CNS penetration and durable clinical benefit. However, resistance and toxicity remain challenges, and future research should focus on optimizing patient selection and developing combination strategies to further expand its role in NF1-PN management.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032943","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Investigational agents for ischaemic cardiomyopathy treatment: preclinical and early phase insights. 缺血性心肌病治疗的研究药物:临床前和早期的见解。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-09-10 DOI: 10.1080/13543784.2025.2558655
Paolo Ossola, Claudio Mario Ciampi, Francesco Politi, Andrea Sultana, Andrea Farina, Gabriele Fragasso, Roberto Spoladore
{"title":"Investigational agents for ischaemic cardiomyopathy treatment: preclinical and early phase insights.","authors":"Paolo Ossola, Claudio Mario Ciampi, Francesco Politi, Andrea Sultana, Andrea Farina, Gabriele Fragasso, Roberto Spoladore","doi":"10.1080/13543784.2025.2558655","DOIUrl":"10.1080/13543784.2025.2558655","url":null,"abstract":"<p><strong>Introduction: </strong>Ischemic heart disease (IHD) constitutes the most prevalent form of cardiac disease in the general population. Although current therapeutic interventions have significantly improved both quality of life and survival rates, no available treatment can reverse the loss of cardiomyocytes resulting from ischemic injury. Existing therapies are limited to attenuating myocardial damage, reducing its extent, and mitigating its clinical consequences.</p><p><strong>Area covered: </strong>Advances in pharmacological and biomedical research have paved the way for novel therapeutic modalities. Tissue engineering, gene therapy, microRNAs (miRNAs), small interfering RNAs (siRNAs), and stem cell-based approaches represent promising avenues for promoting myocardial regeneration. In the present review, we aim to provide a succinct yet comprehensive review of the principal areas of current scientific investigation in this evolving field.</p><p><strong>Expert opinion: </strong>Although current scientific evidence remains limited, primarily due to the lack of large-scale clinical trials in vivo, the prospects of these therapeutic strategies are highly promising, particularly for patients with limited conventional treatment options. It remains to be determined when and how these approaches may be effectively implemented in clinical practice.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-9"},"PeriodicalIF":4.1,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145014398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Nonamyloid-beta active immunization for the treatment of Alzheimer's disease. 非淀粉样蛋白- β主动免疫治疗阿尔茨海默病。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-09-07 DOI: 10.1080/13543784.2025.2551352
Olivia Triplett, Nicole Varda, Boris Decourt, Marwan N Sabbagh
{"title":"Nonamyloid-beta active immunization for the treatment of Alzheimer's disease.","authors":"Olivia Triplett, Nicole Varda, Boris Decourt, Marwan N Sabbagh","doi":"10.1080/13543784.2025.2551352","DOIUrl":"10.1080/13543784.2025.2551352","url":null,"abstract":"<p><strong>Introduction: </strong>Alzheimer's disease (AD) is characterized by the formation of senile plaques composed of amyloid-beta (Aβ) peptides and the intraneuronal accumulation of neurofibrillary tangles composed of abnormal, hyperphosphorylated tau proteins. These act in concert to drive cognitive decline, but it is widely held that tau spread correlates better with cognitive decline than does amyloid burden. Control of AD neuropathologies with active immunizations is studied as a potential therapeutic avenue because it could build innate immunity. Although most active immunization studies have focused on Aβ targets, tau and other targets continue to be explored.</p><p><strong>Areas covered: </strong>This study aimed to identify and describe non-Aβ active immunization trials for AD treatment. A narrative review was conducted to analyze the current status of non-Aβ active immunization for AD using PubMed as the research database.</p><p><strong>Expert opinion: </strong>The potential of active immunization beyond Aβ was explored as a therapeutic strategy for AD with a focus that targets tau and provides insights into its effectiveness, associated challenges, and limitations. Results from preclinical and clinical studies were examined, highlighting the progress and the hurdles that exist. Active immunization against non-Aβ targets, such as tau, for the treatment of AD remains a promising and expanding field.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-9"},"PeriodicalIF":4.1,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Acute chest syndrome (ACS) in sickle cell disease (SCD): pathogenesis and pharmacotherapies in early clinical development. 镰状细胞病(SCD)的急性胸综合征(ACS):发病机制和早期临床发展中的药物治疗
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-08-31 DOI: 10.1080/13543784.2025.2551353
Subarna Chakravorty, Anne Greenough
{"title":"Acute chest syndrome (ACS) in sickle cell disease (SCD): pathogenesis and pharmacotherapies in early clinical development.","authors":"Subarna Chakravorty, Anne Greenough","doi":"10.1080/13543784.2025.2551353","DOIUrl":"https://doi.org/10.1080/13543784.2025.2551353","url":null,"abstract":"<p><strong>Introduction: </strong>Sickle cell disease (SCD) is a monogenic disorder caused by a point mutation in the <i>HBB</i> gene, leading to the production of sickle hemoglobin (HbS). Under hypoxic or acidic conditions, HbS polymerizes within erythrocytes, leading to a series of downstream events resulting in tissue ischemia. Acute chest syndrome (ACS) is a severe and often life-threatening complication of SCD and the leading cause of intensive care unit admission and mortality in children.</p><p><strong>Areas covered: </strong>This review covers the latest understanding of ACS pathology involving infectious triggers, pulmonary fat embolism, endothelial activation, hypercoagulability, and sterile inflammation. We discuss the role of neutrophil extracellular traps, inflammasomes, and platelet - leukocyte aggregates in pulmonary microvasculature causing tissue infarction, ventilation perfusion mismatch, and respiratory failure. We explore the emergence of targeted therapies in preventing and treating ACS.</p><p><strong>Expert opinion: </strong>Although understanding of ACS pathogenesis has improved, current treatments are mainly supportive, with hydroxyurea as the primary preventive therapy. Newer treatments focus on specific mechanisms such as heme scavenging, P-selectin inhibition, toll-like receptor blockade, nitric oxide pathway modulation, and anti-thrombotic strategies. Given ACS's complex nature, a multi-drug targeted approach is likely needed. Scaling up hydroxyurea use remains essential to improving outcomes for millions affected globally by this life-threatening condition.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-9"},"PeriodicalIF":4.1,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948455","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Investigational agents for pancreatic neuroendocrine tumors: what clinical progress have we seen in the last 5 years? 胰腺神经内分泌肿瘤的研究药物:在过去的5年中我们看到了哪些临床进展?
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-08-27 DOI: 10.1080/13543784.2025.2552847
Paola Marino, Antonella Argentiero, Davide Quaresmini, Elena Sapuppo, Dalila Tessitore, Giuliana Ciappina, Massimilano Berretta, Dario Giuffrida, Mariacarmela Santarpia, Nicola Silvestris
{"title":"Investigational agents for pancreatic neuroendocrine tumors: what clinical progress have we seen in the last 5 years?","authors":"Paola Marino, Antonella Argentiero, Davide Quaresmini, Elena Sapuppo, Dalila Tessitore, Giuliana Ciappina, Massimilano Berretta, Dario Giuffrida, Mariacarmela Santarpia, Nicola Silvestris","doi":"10.1080/13543784.2025.2552847","DOIUrl":"https://doi.org/10.1080/13543784.2025.2552847","url":null,"abstract":"<p><strong>Introduction: </strong>Pancreatic neuroendocrine tumors (pNETs), a group of endocrine tumors that arise in the pancreas from the hormonal cells of the islets of Langerhans, are among the most common gastroenteropancreatic neuroendocrine tumors. The incidence of pNETs has increased in recent years to about 1.5 per 100,000 population and prognosis correlates with histologic grade.This review aims to provide an overview of the newly approved or currently developing drugs over the past five years that have shown a significant improvement in prognosis for patients affected by this rare disease.</p><p><strong>Areas covered: </strong>The literature search was conducted using PubMed, focusing on English-language publications from 2020 to 2025. Keywords included 'pancreatic neuroendocrine tumors,' 'new treatments,' 'targeted therapy,' and 'clinical trials.' Studies selected for inclusion comprised clinical trials, systematic reviews, and meta-analyses reporting data on treatment efficacy, safety, and patient quality of life.</p><p><strong>Expert opinion: </strong>Future research holds great potential, especially in improving personalized treatment predictions. Therefore, it is not easy to define a 'definitive endpoint' for research, as discoveries and technological innovations continue to evolve.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-15"},"PeriodicalIF":4.1,"publicationDate":"2025-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948460","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Antibiotics and non-traditional antimicrobial agents for carbapenem-resistant Acinetobacter baumannii in Phase 1, 2, and 3 clinical trials. 抗生素和非传统抗菌剂用于耐碳青霉烯鲍曼不动杆菌的1、2和3期临床试验
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-08-26 DOI: 10.1080/13543784.2025.2552846
Dimitrios S Kontogiannis, Laura T Romanos, Iva D Tzvetanova, Georgios L Voulgaris, Matthew E Falagas
{"title":"Antibiotics and non-traditional antimicrobial agents for carbapenem-resistant <i>Acinetobacter baumannii</i> in Phase 1, 2, and 3 clinical trials.","authors":"Dimitrios S Kontogiannis, Laura T Romanos, Iva D Tzvetanova, Georgios L Voulgaris, Matthew E Falagas","doi":"10.1080/13543784.2025.2552846","DOIUrl":"https://doi.org/10.1080/13543784.2025.2552846","url":null,"abstract":"<p><strong>Introduction: </strong>Carbapenem-resistant <i>Acinetobacter baumannii</i> (CRAB) infections have become common in healthcare settings worldwide, yet current therapeutic options are limited. A pipeline of new antibiotics and non-traditional antimicrobial agents is being developed to address the urgent need for efficacious therapeutic options for patients with CRAB infections.</p><p><strong>Areas covered: </strong>At the time of this writing, 13 traditional antibiotics are in clinical development for CRAB infections, some with a novel mechanism of action. Specifically, 9 antibiotics are in Phase 1 (<i>R</i>-327, xeruborbactam/QPX-7728, upleganan/SPR-206, MRX-8, QPX-9003, zifanocycline/KBP-7072, apramycin/EBL-1003, zosurabalpin/RG-6006, and ANT-3310), two in Phase 2 (BV-100, OMN-6), and two in Phase 3 (zidebactam/WCK-5222, funobactam/XNW-4107) clinical trials. Additionally, there are six non-traditional antimicrobial agents in Phase 1 or 2 clinical trials for treating CRAB infections. In particular, two monoclonal antibodies (TRL-1068, CMTX-101), a phage therapy (Phagebank), an immune-modulating agent (recombinant human plasma gelsolin/Rhu-pGSN), a microbiome-modulating agent (SER-155), and an engineered cationic antibiotic peptide (PLG-0206).</p><p><strong>Expert opinion: </strong>Several agents with promising characteristics against CRAB infections are in clinical development (Phases 1, 2, and 3). The urgent need for therapeutic options against CRAB infections necessitates optimizing efforts and time for introducing successfully studied agents into clinical practice.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-12"},"PeriodicalIF":4.1,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144948384","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Tirzepatide for metabolic dysfunction-associated steatohepatitis: results from phase II clinical trials and perspectives. 替西肽治疗代谢功能障碍相关脂肪性肝炎:来自II期临床试验的结果和观点。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-08-09 DOI: 10.1080/13543784.2025.2546812
Stefano Fiorucci, Ginevra Urbani
{"title":"Tirzepatide for metabolic dysfunction-associated steatohepatitis: results from phase II clinical trials and perspectives.","authors":"Stefano Fiorucci, Ginevra Urbani","doi":"10.1080/13543784.2025.2546812","DOIUrl":"https://doi.org/10.1080/13543784.2025.2546812","url":null,"abstract":"<p><strong>Introduction: </strong>Tirzepatide is a once-weekly injectable dual glucose-dependent insulinotropic polypeptide (GIP) receptor and glucagon-like peptide-1 (GLP-1) receptor agonist. GIP and GLP-1 are incretins promoting insulin release from pancreatic β-cells. Results from clinical trials have confirmed that tirzepatide exerts favorable effects on glucose metabolism and insulin resistance, reduces food intake and has been approved for the treatment of adults with type 2 diabetes, and who are overweight/obese or who have weight-related comorbidities.</p><p><strong>Areas covered: </strong>Results from SYNERGY-NASH, a phase 2 study involving patients with biopsy-proven MASH and stage 2 or 3 fibrosis, have shown that tirzepatide achieved MASH resolution with no worsening of fibrosis in a significantly higher percentage of patients than placebo. Additionally, more patients in the tirzepatide group in comparison to placebo achieved a 1-stage or greater fibrosis improvement without worsening of MASH, but the study was not powered to detect this change, and noninvasive biomarkers of fibrosis were not significantly improved.</p><p><strong>Expert opinion: </strong>While these results suggest a potential role for tirzepatide in MASH treatment given its ability to improve insulin sensitivity and reduce food intake, factors already shown to be beneficial in reducing livers steatosis and fibrosis, larger clinical trials are needed.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2025-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144803901","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Development of gene expression inhibitors for the treatment of cutaneous carcinomas. 基因表达抑制剂治疗皮肤癌的研究进展。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-07-01 Epub Date: 2025-07-15 DOI: 10.1080/13543784.2025.2533442
Dhruv Sharma, Aniruddha Roy, Gautam Singhvi
{"title":"Development of gene expression inhibitors for the treatment of cutaneous carcinomas.","authors":"Dhruv Sharma, Aniruddha Roy, Gautam Singhvi","doi":"10.1080/13543784.2025.2533442","DOIUrl":"10.1080/13543784.2025.2533442","url":null,"abstract":"","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"551-555"},"PeriodicalIF":4.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144599873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Promising experimental drugs for obsessive-compulsive personality disorder. 治疗强迫性人格障碍的试验性药物。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-07-01 Epub Date: 2025-07-24 DOI: 10.1080/13543784.2025.2536846
Donatella Marazziti, Riccardo Gurrieri, Matteo Gambini, Gerardo Russomanno, Umberto Albert
{"title":"Promising experimental drugs for obsessive-compulsive personality disorder.","authors":"Donatella Marazziti, Riccardo Gurrieri, Matteo Gambini, Gerardo Russomanno, Umberto Albert","doi":"10.1080/13543784.2025.2536846","DOIUrl":"10.1080/13543784.2025.2536846","url":null,"abstract":"<p><strong>Introduction: </strong>Obsessive-compulsive personality disorder (OCPD) is a common psychopathological condition that generally causes a significant maladjustment in affected individuals. Despite its prevalence, there are no univocal and defined guidelines for the treatment of this condition, and the literature evidence on this topic is scant and controversial.</p><p><strong>Areas covered: </strong>This narrative review synthesizes clinical trials, case reports, and experimental proposals for OCPD treatment in the light of the most recent neurobiological models. We aimed to review current therapeutic evidence for the disorder and evaluate their effectiveness, proposing potential rationales to guide future strategies.</p><p><strong>Expert opinion: </strong>Although no guidelines are available for OCPD, given the paucity of controlled studies, a possibility to overcome this gap might rely on identifying among the currently available and emerging therapies those that may appear most promising for improving the outcome of the disorder and suggesting novel therapeutic targets. In this way, this paper might represent a sort of 'call to action' to the scientific deepening of the mode of action of those treatments that could be of help to improve the detrimental quality of life of OCPD patients.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"623-637"},"PeriodicalIF":4.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144674269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Antibiotics and non-traditional antimicrobial agents for Pseudomonas aeruginosa in clinical phases 1, 2, and 3 trials. 铜绿假单胞菌的抗生素和非传统抗菌药物临床1、2和3期试验。
IF 4.1 2区 医学
Expert opinion on investigational drugs Pub Date : 2025-07-01 Epub Date: 2025-07-14 DOI: 10.1080/13543784.2025.2532443
Laura T Romanos, Dimitrios S Kontogiannis, Chara Tsiampali, Iva D Tzvetanova, Matthew E Falagas
{"title":"Antibiotics and non-traditional antimicrobial agents for <i>Pseudomonas aeruginosa</i> in clinical phases 1, 2, and 3 trials.","authors":"Laura T Romanos, Dimitrios S Kontogiannis, Chara Tsiampali, Iva D Tzvetanova, Matthew E Falagas","doi":"10.1080/13543784.2025.2532443","DOIUrl":"10.1080/13543784.2025.2532443","url":null,"abstract":"<p><strong>Introduction: </strong>Multidrug-resistant <i>Pseudomonas aeruginosa</i> infections have disseminated globally and are associated with high mortality due to the considerable virulence of the pathogen and the limited therapeutic options. This has led to efforts to develop new treatment options for such infections.</p><p><strong>Areas covered: </strong>This review evaluated the most recent literature on the relevant traditional and non-traditional antibiotics currently being developed in Phases 1, 2, and 3 clinical trials. We conducted a PubMed literature search, as well as a backward citation search of relevant studies. Traditional agents in clinical development include β-lactam/β-lactamase inhibitors (funobactam, taniborbactam, QPX2014-xeruborbactam), aminoglycosides (apramycin), polymyxin derivatives (upleganan, MRX-8, and SPR741), fluoroquinolones (MP-376), and lipopolysaccharide transport inhibitors (murepavadin). Non-traditional antibiotics in clinical development include anti-virulence agents (fluorothiazinone), monoclonal antibodies (INFEX-702, TRL-1068, and CMTX-101), bacteriophages (AP-PA02, YPT-01, BX004-A, and WRAIR-PAM-CF1), and miscellaneous agents (AR-501, PLG-0206, SNSP-113, OligoG CF-5/20, and ALX-009).</p><p><strong>Expert opinion: </strong>A considerable number of antimicrobial agents, some with novel mechanisms of action, are in clinical phases of development for treating <i>Pseudomonas aeruginosa</i> infections. The urgent need for more therapeutic options necessitates the rapid optimization of progress to introduce new agents into clinical practice.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"639-653"},"PeriodicalIF":4.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144599872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
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