Expert opinion on investigational drugs最新文献_第2页

Investigational new drugs for the treatment of leishmaniasis. 研究治疗利什曼病的新药。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-09-10 DOI: 10.1080/13543784.2024.2400139

Shyam Sundar, Vishal Kumar Singh, Neha Agrawal, Om Prakash Singh, Rajiv Kumar

{"title":"Investigational new drugs for the treatment of leishmaniasis.","authors":"Shyam Sundar, Vishal Kumar Singh, Neha Agrawal, Om Prakash Singh, Rajiv Kumar","doi":"10.1080/13543784.2024.2400139","DOIUrl":"10.1080/13543784.2024.2400139","url":null,"abstract":"Introduction: Over the past 20 years, significant progress has been made in anti-leishmanial therapy. Three new drugs/formulations are available for the treatment of various forms of leishmaniasis, namely oral miltefosine, paromomycin and liposomal amphotericin B. However, these advances in drug development have added considerable complexity for clinicians including toxicity, emergence of resistance and decreased sensitivity of available drugs. The development of newer drugs with less toxicity and more efficacy is urgently needed.Areas covered: This review comprehensively examines the latest developments and current status of antileishmanial drugs for the treatment of leishmaniasis across the world. Several new investigational drugs that showed anti-leishmanial activity under in vitro or in vivo conditions and either underwent the phase-I/II clinical trials or are on the verge of entering the trials were reviewed. We also delve into the challenges of drug resistance and discuss the emergence of new and effective antileishmanial compounds.Expert opinion: The available treatments for leishmaniasis are limited in number, toxic, expensive, and demand extensive healthcare resources. Every available antileishmanial drug is associated with several disadvantages, such as drug resistance and toxicity or high cost. Miltefosine is potentially teratogenic. New antileishmanial drugs/treatment modalities are sorely needed for expanding future treatment options.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1029-1046"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142119279","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

Application of engineered antibodies (scFvs and nanobodies) targeting pathological protein aggregates in Alzheimer's disease. 针对阿尔茨海默病病理蛋白聚集体的工程抗体（scFvs 和纳米抗体）的应用。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-08-26 DOI: 10.1080/13543784.2024.2396911

Yuan Zhang, Wanpeng Yu, Lei Zhang, Peifeng Li

{"title":"Application of engineered antibodies (scFvs and nanobodies) targeting pathological protein aggregates in Alzheimer's disease.","authors":"Yuan Zhang, Wanpeng Yu, Lei Zhang, Peifeng Li","doi":"10.1080/13543784.2024.2396911","DOIUrl":"10.1080/13543784.2024.2396911","url":null,"abstract":"Introduction: The misfolding and aggregation of proteins are associated with various neurodegenerative diseases, such as Alzheimer's disease (AD). The small-molecule engineered antibodies, such as single-chain fragment variable (scFv) antibodies and nanobodies (Nbs), have gained attention in recent years due to their strong conformational specificity, ability to cross the blood-brain barrier (BBB), low immunogenicity, and enhanced proximity to active sites within aggregates.Areas covered: We have reviewed recent advances in therapies involving scFvs and Nbs that efficiently and specifically target pathological protein aggregates. Relevant publications were searched for in MEDLINE, GOOGLE SCHOLAR, Elsevier ScienceDirect and Wiley Online Library.Expert opinion: We reviewed the recent and specific targeting of pathological protein aggregates by scFvs and Nbs. These engineered antibodies can inhibit the aggregation or promote the disassembly of misfolded proteins by recognizing antigenic epitopes or through conformational specificity. Additionally, we discuss strategies for improving the effective application of engineered antibodies in treating AD. These technological strategies will lay the foundation for the clinical application of small-molecule antibody drugs in developing effective treatments for neurological diseases. Through rational application strategies, small-molecule engineered antibodies are expected to have significant potential in targeted therapy for neurological disorders.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1047-1062"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142035612","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

Progress in the clinical development of investigational systemic agents for recurrent and metastatic nasopharyngeal carcinoma. 治疗复发性和转移性鼻咽癌的研究性系统药物的临床开发进展。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-09-19 DOI: 10.1080/13543784.2024.2401910

Kelvin Yan, Darren Wt Lim, Brigette B B Y Ma

{"title":"Progress in the clinical development of investigational systemic agents for recurrent and metastatic nasopharyngeal carcinoma.","authors":"Kelvin Yan, Darren Wt Lim, Brigette B B Y Ma","doi":"10.1080/13543784.2024.2401910","DOIUrl":"10.1080/13543784.2024.2401910","url":null,"abstract":"Introduction: Nasopharyngeal carcinoma (NPC) remains an endemic disease in certain parts of the world, with many patients presenting with advanced disease on diagnosis. Chemotherapy had remained the standard of care with minimal progress made until recent years. This review aims to provide an overview of recent significant breakthroughs and up-and-coming novel strategies in treating this deadly disease.Areas covered: This review focuses on the latest clinical development of promising investigational agents in the treatment of advanced NPC. These include anti-vascular agents, signaling pathways inhibitors and immunotherapy.Expert opinion: The addition of immune-checkpoint inhibitors (CPI) to platinum-based chemotherapy has undoubtedly changed the therapeutic landscape of R/M NPC in the first-line setting. This leaves much room for further research on the optimal treatment strategy in subsequent-line settings, likely including the addition of CPI to anti-vascular agents or novel CPI combinations, with or without chemotherapy as a backbone. Other potential approaches include optimal CPI maintenance therapy after first-line CPI-chemotherapy combination. Potential novel agents on the horizons are antibody-drug conjugates, bi-specific antibodies and signaling inhibitors, with several phase II/III studies currently underway.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1019-1028"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142282771","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

Recent progress in tyrosine kinase 2 inhibitors for atopic dermatitis. 酪氨酸激酶 2 抑制剂治疗特应性皮炎的最新进展。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-08-21 DOI: 10.1080/13543784.2024.2391825

Qi Liu, Yuan Xia, Lin Liu, Yuan Zhou, Yumei Li

{"title":"Recent progress in tyrosine kinase 2 inhibitors for atopic dermatitis.","authors":"Qi Liu, Yuan Xia, Lin Liu, Yuan Zhou, Yumei Li","doi":"10.1080/13543784.2024.2391825","DOIUrl":"10.1080/13543784.2024.2391825","url":null,"abstract":"Introduction: Atopic dermatitis (AD) is a chronic inflammatory skin disease characterized by persistent itching. Conventional treatments for AD include topical corticosteroids and calcineurin inhibitors, but there are emerging therapies targeting the JAK-TYK2 pathway that are promising for the treatment of AD.Areas covered: This review comprehensively explores the pathogenesis, triggers, clinical manifestations, and conventional treatment options for AD. In addition, we discuss novel therapeutic agents targeting alternative signaling pathways, with a focus on clinical trials evaluating tyrosine kinase 2 (TYK2) inhibitors, including systemic and topical agents. We also provide a detailed assessment of ICP-332 efficacy, safety, and potential adverse effects in moderate-to-severe AD.Expert opinion: Janus kinase inhibitors that have been recently approved have shown promise for the treatment of AD, especially for patients with severe phenotypes. Preliminary findings from randomized controlled trials suggest that TYK2 inhibitors exhibit rapid efficacy and acceptable safety in the management of AD; however, additional investigations, including long-term trials, are warranted to fully understand their efficacy and safety profile.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1001-1007"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141982067","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

Pharmacokinetics, pharmacodynamics, safety, and tolerability of a single-dose riliprubart, an anti-C1s humanized monoclonal antibody in East-Asian adults: results from a Phase 1, randomized, open-label trial. 抗 C1s 人源化单克隆抗体利利鲁巴特在东亚成人中的单剂量药代动力学、药效学、安全性和耐受性：1 期随机开放标签试验的结果。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-08-22 DOI: 10.1080/13543784.2024.2394186

Yingxin Li, Joo Young Na, Yunting Zhu, Jaeseong Oh, Amy Zhao, In-Jin Jang, Lei Tang

{"title":"Pharmacokinetics, pharmacodynamics, safety, and tolerability of a single-dose riliprubart, an anti-C1s humanized monoclonal antibody in East-Asian adults: results from a Phase 1, randomized, open-label trial.","authors":"Yingxin Li, Joo Young Na, Yunting Zhu, Jaeseong Oh, Amy Zhao, In-Jin Jang, Lei Tang","doi":"10.1080/13543784.2024.2394186","DOIUrl":"10.1080/13543784.2024.2394186","url":null,"abstract":"Objectives: This Phase 1 trial was planned to investigate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of a single dose of riliprubart in healthy East-Asian adult participants.Methods: A single-center, parallel-group, randomized, open-label, single-dose study was performed to evaluate the PK, PD, safety, and tolerability of riliprubart (50 mg/kg intravenous [IV] or 600 mg subcutaneous [SC]) in 37 healthy East-Asian (Chinese, Japanese, and Korean) participants.Results: Riliprubart was slowly absorbed after SC administration (median tmax: 7.01-10.48 days) and showed a long half-life after IV or SC administration (mean: 9.52-11.0 weeks), with a bioavailability of 74.6% after SC administration. The PD profiles, which are evaluated by classical complement pathway activity or CH50, were similar and largely overlapped across East-Asian participants after a single IV or SC dose. Riliprubart was safe and well tolerated in participants following a single IV or SC dose.Conclusions: Riliprubart was safe and well tolerated and demonstrated favorable PK and PD profiles in healthy East-Asian participants following a single IV or SC dose. These results are comparable to first-in-human study results from non-East-Asian participants and support the same dosing regimen of riliprubart for global simultaneous clinical development.Clinical trial registration: This trial is registered at https://cris.nih.go.kr (identifier: KCT0006571).","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1063-1074"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016875","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

Experimental drugs for erosive esophagitis: what is in the clinical development pipeline? 治疗侵蚀性食管炎的实验性药物：临床开发项目有哪些？

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-08-30 DOI: 10.1080/13543784.2024.2393868

Daniel Martin Simadibrata, Elvira Lesmana, Yeong Yeh Lee

{"title":"Experimental drugs for erosive esophagitis: what is in the clinical development pipeline?","authors":"Daniel Martin Simadibrata, Elvira Lesmana, Yeong Yeh Lee","doi":"10.1080/13543784.2024.2393868","DOIUrl":"10.1080/13543784.2024.2393868","url":null,"abstract":"Introduction: Proton pump inhibitor (PPI) has revolutionized the treatment of erosive esophagitis (EE) in the past few decades. However, roughly 30-40% of the patients, especially those with severe EE (Los Angeles Grade C/D), remain poorly responsive to this medication. Novel drugs have been formulated and/or repurposed to address this problem.Areas covered: This review highlights novel drugs that have been investigated for use in EE, such as mucosal protectants, prokinetics, transient lower esophageal sphincter relaxation (TLESR) reducers, novel PPIs, and the new potassium-competitive acid blocker (PCAB). Studies have demonstrated that PCAB has promising results (efficacy and safety) compared to PPI for the healing of EE, especially in severe diseases.Expert opinion: PCAB has gained interest in recent years, with pharmacokinetics and pharmacodynamics properties surpassing PPI. Although recent data on PCABs, which comprised mainly of Vonoprazan, have shown promising results, more randomized controlled trials for other PCAB drugs are needed to elucidate and confirm the superiority of this drug class to PPI, the current first-line treatment of EE.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1009-1018"},"PeriodicalIF":4.9,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141995570","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

Olaparib monotherapy or in combination with abiraterone for treating mutated metastatic castration-resistant prostate cancer: alone or stronger together? 奥拉帕利单药或与阿比特龙联合治疗突变的转移性去势抵抗性前列腺癌：单独治疗还是强强联合？

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-10-01 Epub Date: 2024-08-20 DOI: 10.1080/13543784.2024.2391828

Sarah E Fenton, Maha Hussain

引用次数: 0

MASLD and MASH: how a change of nomenclature may impact our approach in treating liver disease. MASLD和MASH：名称的改变会如何影响我们治疗肝病的方法。

IF 6.1 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-10 DOI: 10.1080/13543784.2024.2401907

Kelly Dale,Yasmeen Fallouh,Naim Alkhouri

引用次数: 0

Antiviral agents and therapeutics against respiratory viruses. 针对呼吸道病毒的抗病毒剂和疗法。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-08 DOI: 10.1080/13543784.2024.2401911

Ralph A Tripp, David E Martin

{"title":"Antiviral agents and therapeutics against respiratory viruses.","authors":"Ralph A Tripp, David E Martin","doi":"10.1080/13543784.2024.2401911","DOIUrl":"https://doi.org/10.1080/13543784.2024.2401911","url":null,"abstract":"Introduction: Respiratory viruses are responsible for significant worldwide morbidity and mortality. While vaccines are highly effective at reducing the morbidity and mortality associated with viral infections, this protection is incomplete. It requires a high degree of compliance, which is hindered by vaccine hesitancy. To address these gaps, antiviral agents and therapeutics are crucial in combating diseases caused by respiratory viruses. Antiviral agents are broadly classified into two groups: 1) direct-acting antivirals (DAA) and 2) host-directed antivirals (HDA).Areas covered: This review comprehensively examines Phase II FDA-approved antiviral drugs for influenza virus, SARS-CoV-2, and RSV as published in clinicaltrials.gov. It focuses on DAAs and various monoclonal antibodies (mAbs) that have been approved for the prevention and treatment of viral respiratory tract infections.Expert opinion: Antiviral drugs being developed assess different mechanisms of action to combat viruses and other delivery routes (i.e. oral, inhalation, or parenteral). The associated clinical trials address the impact on disease while determining the appropriate dosage levels for further investigation in Phase III. A robust pipeline of agents is necessary to meet the global need for effective antiviral therapeutics.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"1-5"},"PeriodicalIF":4.9,"publicationDate":"2024-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153501","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

Emerging treatment landscape for Guillain-Barré Syndrome (GBS): what's new? 新出现的吉兰-巴雷综合征（GBS）治疗方法：有何新进展？

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-07-10 DOI: 10.1080/13543784.2024.2377323

Alina Sprenger-Svačina, Martin K R Svačina, Tong Gao, Gang Zhang, Kazim A Sheikh

{"title":"Emerging treatment landscape for Guillain-Barré Syndrome (GBS): what's new?","authors":"Alina Sprenger-Svačina, Martin K R Svačina, Tong Gao, Gang Zhang, Kazim A Sheikh","doi":"10.1080/13543784.2024.2377323","DOIUrl":"10.1080/13543784.2024.2377323","url":null,"abstract":"Introduction: Guillain-Barré syndrome (GBS) is a monophasic immune neuropathic disorder characterized by acute paralysis. A significant portion of patients are left with residual deficits, which presents a considerable global healthcare challenge. The precise mechanisms underlying GBS pathogenesis are not fully elucidated. Recent studies have focused on postinfectious molecular mimicry and identified involvement of IgG autoantibodies and innate immune effectors in GBS. Intravenous immunoglobulins (IVIg) and plasma exchange (PE) are two established evidence-based immunomodulatory treatments for GBS, but a significant proportion of GBS patients fails to respond adequately to either therapy. This emphasizes an urgent need for novel and more potent treatments.Areas covered: We discuss novel immunomodulatory therapies presently at different phases of preclinical and clinical investigation. Some drugs in development target pathophysiologic mechanisms such as IgG autoantibody catabolism and complement activation, which are relevant to GBS.Expert opinion: There is an unmet need for more effective immune therapies for GBS. New immunomodulatory therapies under development may provide more potent options for GBS patients who do not respond to IVIg or PE. Future directions may include incorporating neuroprotective interventions based on evolving understanding of mechanisms underlying nerve injury and axonal degeneration.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"881-886"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11424254/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141558495","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0