Expert Opinion on Emerging Drugs最新文献_第9页

Assessing the developing pharmacotherapeutic landscape in hepatitis B treatment: a spotlight on drugs in phase II clinical trials 评估乙型肝炎治疗药物治疗的发展前景：II期临床试验中药物的关注

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-04-03 DOI: 10.1080/14728214.2022.2074977

R. Hui, L. Mak, W. Seto, M. Yuen

{"title":"Assessing the developing pharmacotherapeutic landscape in hepatitis B treatment: a spotlight on drugs in phase II clinical trials","authors":"R. Hui, L. Mak, W. Seto, M. Yuen","doi":"10.1080/14728214.2022.2074977","DOIUrl":"https://doi.org/10.1080/14728214.2022.2074977","url":null,"abstract":"ABSTRACT Introduction Functional cure, defined as sustained HBsAg seroclearance, is associated with favorable outcomes in chronic hepatitis B (CHB). While nucleos(t)ide analogues (NAs) are effective in suppressing HBV replication, NAs are unable to induce functional cure at high rates. A range of novel HBV antivirals, aiming to induce functional cure, are currently under development. Areas covered This article covered novel hepatitis B virus (HBV) antivirals that have entered phase II trials. Virus-directing agents covered include entry inhibitors, transcription inhibitors, RNA silencers, core protein allosteric modulators, noncompetitive polymerase inhibitors, and viral protein export inhibitors. Immunomodulators covered include innate immune stimulators, T-cell modulators, therapeutic vaccines, and monoclonal antibodies. Upcoming developmental directions would also be discussed. Expert opinion Among novel HBV antivirals, RNA silencers, viral protein export inhibitors (with pegylated interferon), and entry inhibitors (with pegylated interferon) appear to be effective in suppressing HBsAg and may even induce functional cure. The other virus-targeting agents have variable effects on HBV DNA, HBsAg, HBeAg, and HBcrAg. Immunomodulators have modest effects on HBsAg but may have important roles in combination therapy. Upcoming trials will answer important questions on ideal dosing, long-term drug effects, and efficacy of combination regimens.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"127 - 140"},"PeriodicalIF":3.4,"publicationDate":"2022-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41594487","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: 5

Emerging drugs for the treatment of hepatocellular carcinoma 治疗肝细胞癌的新兴药物

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-04-03 DOI: 10.1080/14728214.2022.2083107

W. Ayoub, Patricia D. Jones, J. D. Yang, Paul M. Martin

{"title":"Emerging drugs for the treatment of hepatocellular carcinoma","authors":"W. Ayoub, Patricia D. Jones, J. D. Yang, Paul M. Martin","doi":"10.1080/14728214.2022.2083107","DOIUrl":"https://doi.org/10.1080/14728214.2022.2083107","url":null,"abstract":"ABSTRACT Introduction Hepatocellular carcinoma (HCC) remains a leading cause of liver-related mortality. Cirrhosis of any etiology is the major risk factor although HCC can develop in its absence in patients with hepatitis B and increasingly in those with nonalcoholic fatty liver disease. When detected at an early stage, curative options include surgical resection, liver transplantation, and/or ablative therapies. Unfortunately, most cases of HCC are recognized at an advanced stage when options are limited and noncurative. However, new systemic therapies with tyrosine kinase inhibitors and immunotherapy have expanded therapeutic options in advanced HCC. Advances in systemic therapy have given patients with advanced HCC hope and prolonged their survival. Areas covered We discuss recent data and ongoing research efforts to improve the treatment of hepatocellular carcinoma with discussion of current and upcoming systemic therapies combining agents of different classes. Expert opinion Systemic therapy for HCC is in evolution. The inclusion of immunotherapy to systemic therapy has revolutionized the field of HCC treatment. Identification of the appropriate combination and sequence of systemic therapy coupled with discovery of reliable HCC biomarkers will lead to improved survival and individualized HCC therapy.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"141 - 149"},"PeriodicalIF":3.4,"publicationDate":"2022-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45627778","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: 4

Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials. 癫痫:专家对2/3期临床试验新兴药物的意见

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-03-01 Epub Date: 2022-04-01 DOI: 10.1080/14728214.2022.2059464

Amanda W Pong, Jonathan Ross, Ivana Tyrlikova, Alexander J Giermek, Maya P Kohli, Yousef A Khan, Roger D Salgado, Pavel Klein

{"title":"Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials.","authors":"Amanda W Pong, Jonathan Ross, Ivana Tyrlikova, Alexander J Giermek, Maya P Kohli, Yousef A Khan, Roger D Salgado, Pavel Klein","doi":"10.1080/14728214.2022.2059464","DOIUrl":"10.1080/14728214.2022.2059464","url":null,"abstract":"Introduction: Despite the existence of over 30 anti-seizure medications (ASM), including 20 over the last 30 years, a third of patients with epilepsy remain refractory to treatment, with no disease-modifying or preventive therapies until very recently. The development of new ASMs with new mechanisms of action is therefore critical. Recent clinical trials of new treatments have shifted focus from traditional common epilepsies to rare, genetic epilepsies with known mechanistic targets for treatment and disease-specific animal models.Areas covered: ASMs in phase 2a/b-3 clinical trials target cholesterol, serotonin, sigma-1 receptors, potassium channels and metabotropic glutamate receptors. Neuroinflammation, protein misfolding, abnormal thalamocortical firing, and molecular deficiencies are among the targeted pathways. Clinically, the current phase 2a/b-3 agents hold promise for variety of epilepsy conditions, from developmental epileptic encephalopathies (Dravet Syndrome, Lennox-Gastaut syndrome, CDKL5 and PCDH19, Rett's Syndrome), infantile spasms, tuberous sclerosis as well as focal and idiopathic generalized epilepsies and acute rescue therapy for cluster seizures.Expert opinion: New delivery mechanisms increase potency and site-specificity of existing drugs. Novel mechanisms of action involve cholesterol degradation, mitochondrial pathways, anti-inflammation, and neuro-regeneration. Earlier identification of genetic conditions through genetic testing will allow for earlier use of disease specific and disease-modifying therapies.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"75-90"},"PeriodicalIF":2.7,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48611252","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 drugs for the treatment of cutaneous T-cell lymphoma. 治疗皮肤t细胞淋巴瘤的新药物。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-03-01 DOI: 10.1080/14728214.2022.2049233

Melissa Cheng, Jasmine Zain, Steven T Rosen, Christiane Querfeld

{"title":"Emerging drugs for the treatment of cutaneous T-cell lymphoma.","authors":"Melissa Cheng, Jasmine Zain, Steven T Rosen, Christiane Querfeld","doi":"10.1080/14728214.2022.2049233","DOIUrl":"https://doi.org/10.1080/14728214.2022.2049233","url":null,"abstract":"Introduction: Cutaneous T cell lymphoma (CTCL) is a rare and incurable group of non-Hodgkin lymphomas that manifest as patches, plaques, tumors, and/or erythroderma in the skin. Standard skin-directed therapies for CTCL are effective in patients with indolent early-stage disease, but more advanced/refractory stage patients require systemic therapies. However, none of the treatments are considered curative and most patients suffer from relapses. Biologic therapies and immunotherapy provide novel treatment options for patients with advanced or refractory disease.Areas covered: This review provides a discussion of recently approved biological and novel therapeutics that are actively developed for the management of the heterogeneous group of CTCL.Expert opinion: Mogamulizumab and brentuximab vedotin have reached the market and are approved for the treatment of CTCL, providing valuable options. Additionally, therapies utilizing immune checkpoint inhibitors, miRNA inhibitors, and peptide inhibitors show promising results in clinical trials. Durvalumab, pembrolizumab, TTI-621, BNZ-1, and MRG-106 are several of the emerging treatments still in trials. Further combinatorial studies are needed as none of the treatments have demonstrated long-term remissions.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"45-54"},"PeriodicalIF":3.4,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320301/pdf/nihms-1905596.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9754829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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: 2

Emerging therapies in the management of Irritable Bowel Syndrome (IBS) 肠易激综合征（IBS）治疗的新兴疗法

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-01-02 DOI: 10.1080/14728214.2022.2052043

J. Elwing, Hadi Atassi, Benjamin D. Rogers, G. Sayuk

{"title":"Emerging therapies in the management of Irritable Bowel Syndrome (IBS)","authors":"J. Elwing, Hadi Atassi, Benjamin D. Rogers, G. Sayuk","doi":"10.1080/14728214.2022.2052043","DOIUrl":"https://doi.org/10.1080/14728214.2022.2052043","url":null,"abstract":"ABSTRACT Introduction Irritable bowel syndrome (IBS) is a symptom-based disorder of chronic abdominal pain and altered bowel habits. The pathogenesis of IBS is multifactorial, leading to the potential for the development of diverse treatment strategies. This mechanistic heterogeneity suggests that available therapies will only prove effective in a subset of IBS sufferers. Current US Food and Drug Administration (FDA) approved therapies for IBS with diarrhea (IBS-D) and IBS with constipation (IBS-C) are reviewed. Limited symptom responses and side effect experiences lead to considerable patient dissatisfaction with currently available IBS treatments. Only a small percentage of IBS patients are on prescription therapies underscoring the potential market and need for additional therapeutic options. Areas covered Expanding on currently available therapies, the serotonergic and endogenous opioid receptor systems continue to be a focus of future IBS treatment development. Additional novel emerging therapies include the endogenous cannabinoid system, bile acid secretion and sequestration, and exploit our enhanced understanding of visceral sensory signaling and intestinal secretomotor function. Expert opinion While challenges remain for the future development of IBS therapies, the diverse etiologies underlying the disorder present an opportunity for novel therapies. Hence, great potential is anticipated for future IBS treatment options.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"55 - 73"},"PeriodicalIF":3.4,"publicationDate":"2022-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45584212","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 drugs for the acute treatment of relapses in adult neuromyelitis optica spectrum disorder patients 新兴药物急性治疗复发的成人视神经脊髓炎谱系障碍患者

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2022-01-02 DOI: 10.1080/14728214.2022.2059463

E. Carnero Contentti, P. López, J. Rojas

{"title":"Emerging drugs for the acute treatment of relapses in adult neuromyelitis optica spectrum disorder patients","authors":"E. Carnero Contentti, P. López, J. Rojas","doi":"10.1080/14728214.2022.2059463","DOIUrl":"https://doi.org/10.1080/14728214.2022.2059463","url":null,"abstract":"ABSTRACT Introduction Neuromyelitis optica spectrum disorders (NMOSD) are rare but often devastating neuroinflammatory autoimmune diseases of the central nervous system. Acute treatment is critically important and it should be initiated early and aggressively, as relapses result in severe residual disability. Acute treatments are still based on clinical experience and observational studies. The most commonly used treatments are steroids and plasmapheresis. Several new treatments to improve management and recovery after relapses in NMOSD are currently under investigation. Areas covered This review discusses current and the most recent advances in active development of phase II/III clinical trials for acute treatment options and therapeutic strategies that can help management improvement of NMOSD during a relapse. These treatments include bevacizumab, ublituximab and HBM9161. Expert opinion NMOSD relapses require prompt evaluation and timely treatment to restore function and mitigate disability. Timing is critical. Plasmapheresis showed better outcomes in terms of recovery when compared to high-dose intravenous methylprednisolone alone. Some groups suggest that plasmapheresis could be considered as an initial treatment approach in different clinical scenarios due to its higher effectiveness. Future research and/or real-world data will establish the advantages and disadvantages of these new treatments and define the appropriate patient profile.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"27 1","pages":"91 - 98"},"PeriodicalIF":3.4,"publicationDate":"2022-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42615376","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: 6

Emerging drugs for the treatment of myelofibrosis: phase II & III clinical trials. 治疗骨髓纤维化的新兴药物：II期和III期临床试验。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2021-12-01 Epub Date: 2021-12-12 DOI: 10.1080/14728214.2021.2015320

Douglas Tremblay, Ronald Hoffman

{"title":"Emerging drugs for the treatment of myelofibrosis: phase II & III clinical trials.","authors":"Douglas Tremblay, Ronald Hoffman","doi":"10.1080/14728214.2021.2015320","DOIUrl":"https://doi.org/10.1080/14728214.2021.2015320","url":null,"abstract":"Introduction: Myelofibrosis is a clonal hematologic malignancy with clinical manifestations that include cytopenias, debilitating constitutional symptoms, splenomegaly, bone marrow fibrosis and a propensity toward leukemic progression. While allogeneic hematopoietic stem cell transplantation can be curative, this therapy is not available for the majority of patients. Ruxolitinib and fedratinib are approved JAK2 inhibitors that have produced meaningful benefits in terms of spleen reduction and symptom improvement, but there remain several unmet needs.Areas covered: We discuss novel therapies based upon published data from phase II or III clinical trials. Specifically, we cover novel JAK inhibitors (momelotinib and pacritinib), and agents that target bromodomain and extra-terminal domain (pelabresib), the antiapoptotic proteins BCL-2/BCL-xL (navitoclax), MDM2 (navtemadlin), phosphatidylinositol 3-kinase (parsaclisib), or telomerase (imetelstat).Expert opinion: Patients with disease related cytopenias are ineligible for currently approved JAK2 inhibitors. However, momelotinib and pacritinib may be able to fill this void. Novel therapies are being evaluated in the upfront setting to improve the depth and duration of responses with ruxolitinib. Future evaluation of agents must be judged on their potential to modify disease progression, which current JAK2 inhibitors lack. Combination therapy, possibly with an immunotherapeutic agent might serve as key components of future myelofibrosis treatment options.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"351-362"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39789107","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: 3

Designing phase II clinical trials in Friedreich ataxia. 设计弗里德里希共济失调的II期临床试验。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2021-12-01 Epub Date: 2021-12-22 DOI: 10.1080/14728214.2021.1998452

Layne N Rodden, David R Lynch

{"title":"Designing phase II clinical trials in Friedreich ataxia.","authors":"Layne N Rodden, David R Lynch","doi":"10.1080/14728214.2021.1998452","DOIUrl":"https://doi.org/10.1080/14728214.2021.1998452","url":null,"abstract":"Introduction: Friedreich ataxia (FRDA) is an autosomal recessive disorder caused by deficiency of frataxin, an essential mitochondrial protein involved in iron sulfur cluster biogenesis, oxidative phosphorylation and other processes. FRDA most notably affects the heart, sensory neurons, spinal cord, cerebellum, and other brain regions, and manifests clinically as ataxia, sensory loss, dysarthria, spasticity, and hypertrophic cardiomyopathy. Therapeutic approaches in FRDA have consisted of two different approaches: (1) augmenting or restoring frataxin production and (2) modulating a variety of downstream processes related to mitochondrial dysfunction, including reactive oxygen species production, ferroptosis, or Nrf2 activation.Areas covered: In this review, we summarize data from major phase II clinical trials in FRDA published between 2015 and 2020, which includes A0001/EPI743, Omaveloxolone, RT001, and Actimmune.Expert opinion: A growing number of drug candidates are being tested in phase II clinical trials for FRDA; however, most have not met their primary endpoints, and none have received FDA approval. In this review, we aim to summarize completed phase II clinical trials in FRDA, outlining critical lessons that have been learned and that should be incorporated into future trial design to ultimately optimize drug development in FRDA.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"415-423"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39556079","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: 6

Emerging biological drugs for the treatment of gastroesophageal adenocarcinoma. 治疗胃食管腺癌的新兴生物药物。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2021-12-01 Epub Date: 2021-11-28 DOI: 10.1080/14728214.2021.2010705

Maria Alsina, Marc Diez, Josep Tabernero

{"title":"Emerging biological drugs for the treatment of gastroesophageal adenocarcinoma.","authors":"Maria Alsina, Marc Diez, Josep Tabernero","doi":"10.1080/14728214.2021.2010705","DOIUrl":"https://doi.org/10.1080/14728214.2021.2010705","url":null,"abstract":"Introduction: Gastric cancer (GC) and gastroesophageal junction cancer (GOJC) patients have a poor prognosis with a 5-year relative survival rate of 6% in the metastatic setting. Despite the well-characterized molecular features, patients have been historically considered for treatment with universal and undistinguishing chemotherapies and targeted agents, except for the HER2-positive population and some immunological approaches.Areas covered: In this review, we discuss the intrinsic characteristics of GC/GOJC from an epidemiological, molecular, and clinical perspective with an exhaustive evaluation of the reported and ongoing phase II/III clinical trials with targeted therapies.Expert opinion: The absence of robust biomarkers, the difficulties in measuring it due to the well-recognized molecular heterogeneity, and in part nonoptimistic clinical trial designs have been a major cause of frequent failure. Current efforts should focus on proper recognition of the distinctive molecular and clinical features of each GC/GOJC patient. Sequencing both tumor tissue DNA and ctDNA could identify targetable alterations, including rare alterations, thus allowing GC/GOJC patients for a precision medicine benefit.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"385-400"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39652428","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: 4

Emerging drugs for hemophilia A: insights into phase II and III clinical trials. 治疗A型血友病的新兴药物:II期和III期临床试验的见解。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2021-12-01 Epub Date: 2021-10-11 DOI: 10.1080/14728214.2021.1988073

Hande Kizilocak, Guy Young

引用次数: 2