Drugs最新文献

{"title":"Zenocutuzumab: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40265-025-02155-3","DOIUrl":"10.1007/s40265-025-02155-3","url":null,"abstract":"<p><p>Zenocutuzumab (zenocutuzumab-zbco; BIZENGRI^®), an IgG1 bispecific human epidermal growth factor receptor (HER)2- and HER3-directed antibody, is being developed by Merus for the treatment of solid tumours with neuregulin 1 (NRG1) gene fusions. On 4 December 2024, zenocutuzumab received its first approval in the USA for the treatment of adults with advanced, unresectable or metastatic pancreatic adenocarcinoma or non-small cell lung cancer (NSCLC) harbouring an NRG1 gene fusion with disease progression on or after prior systemic therapy. This article summarizes the milestones in the development of zenocutuzumab leading to this first approval for the second-line treatment of NRG1+ pancreatic adenocarcinoma or NSCLC.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"591-597"},"PeriodicalIF":13.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143491253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Revolutionary Changes in the Management of Lupus Nephritis: Towards De-Glucocorticoid or No-Glucocorticoid.","authors":"Yoshiya Tanaka","doi":"10.1007/s40265-025-02156-2","DOIUrl":"10.1007/s40265-025-02156-2","url":null,"abstract":"<p><p>Glucocorticoids (GCs) possess potent anti-inflammatory and immunosuppressive properties and are used to treat various diseases, including systemic autoimmune rheumatic diseases, rheumatoid arthritis, and systemic lupus erythematosus (SLE). However, GCs are associated with several adverse events and are considered risk factors for infections and cardiovascular disorders; furthermore, their application as therapeutics has changed with recent progress in molecular-targeted therapies. Although GCs have been the mainstay of SLE treatment for more than 50 years, the latest European Alliance of Association for Rheumatology recommendations for the management of SLE in 2023 has significantly relegated the use of GCs and recommended that these be used as \"bridging therapy\" during periods of SLE disease activity. They also recommended the use of GC pulse therapy followed by relatively low doses of GCs even in patients with high disease-activity lupus nephritis, with a focus on the appropriate use of hydroxychloroquine, immunosuppressive drugs, and biological agents. This combination is essential for improving renal survival, minimizing flares, and reducing the side effects of GC. The GC dose was tapered to < 5 mg/day of prednisolone within half a year, maintained for 3 years, and then discontinued with the concomitant use of combination therapies. In contrast to non-renal SLE, the development of more potent molecular targeted therapies for lupus nephritis is required.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"447-455"},"PeriodicalIF":13.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tasurgratinib Succinate: First Approval.","authors":"Yvette N Lamb","doi":"10.1007/s40265-025-02153-5","DOIUrl":"10.1007/s40265-025-02153-5","url":null,"abstract":"<p><p>Tasurgratinib succinate (TASFYGO^®; Eisai Co., Ltd.) is an orally active, small molecule inhibitor of fibroblast growth factor receptors (FGFRs) 1, 2, and 3 being developed for the treatment of solid tumors, including cholangiocarcinoma (i.e., bile duct cancer) and breast cancer. Tasurgratinib succinate received its first approval on 24 September 2024 in Japan, for the treatment of FGFR2 fusion-positive unresectable biliary tract cancer that has progressed after chemotherapy. The approval was based on the positive results of an open-label, single-arm, multicenter, phase II study conducted in Japan and China. This article summarizes the milestones in the development of tasurgratinib succinate leading to this first approval for biliary tract cancer.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"585-590"},"PeriodicalIF":13.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143540580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emerging Immunotherapies for Disease Modification of Type 1 Diabetes.","authors":"Timothy P Foster, Brittany S Bruggeman, Michael J Haller","doi":"10.1007/s40265-025-02150-8","DOIUrl":"10.1007/s40265-025-02150-8","url":null,"abstract":"<p><p>Type 1 diabetes mellitus (T1DM) is characterized by the progressive, autoimmune-mediated destruction of β cells. As such, restoring immunoregulation early in the disease course is sought to retain endogenous insulin production. Nevertheless, in the more than 100 years since the discovery of insulin, treatment of T1DM has focused primarily on hormone replacement and glucose monitoring. That said, immunotherapies are widely used to interdict autoimmune and autoinflammatory diseases and are emerging as potential therapeutics seeking the preservation of β-cell function among those with T1DM. In the past 4 decades of diabetes research, several immunomodulatory therapies have been explored, culminating with the US Food and Drug Administration approval of teplizumab to delay stage 3 (clinical) onset of T1DM. Clinical trials seeking to prevent or reverse T1DM by repurposing immunotherapies approved for other autoimmune conditions and by exploring new therapeutics are ongoing. Collectively, these efforts have the potential to transform the future of diabetes care. We encapsulate the past 40 years of immunotherapy trials, take stock of our successes and failures, and chart paths forward in this new age of clinically available immune therapies for T1DM.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"457-473"},"PeriodicalIF":13.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11949705/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143052018","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and Longevity of Intraocular Pressure Control After Bimatoprost Implant Administration: Interim Analysis of a Phase 3b Clinical Trial (TRITON).","authors":"Steven M Silverstein, Francesco Oddone, Miriam Kolko, Christian K Brinkmann, William C Christie, Amanda K Bicket, Petrus N J Gous, Jan Luebke, Jyotsna Maram, Ashley Nguyen, E Randy Craven, Yongjia Pu, Jenny Jiao, Marina Bejanian, Michael R Robinson","doi":"10.1007/s40265-025-02154-4","DOIUrl":"10.1007/s40265-025-02154-4","url":null,"abstract":"<p><strong>Background: </strong>Bimatoprost implant 10 µg is an intracameral, biodegradable implant that slowly releases bimatoprost to lower intraocular pressure (IOP). This study was designed to evaluate safety and the duration of the IOP-lowering effect after single and as-needed repeat administration of the bimatoprost implant in patients with open-angle glaucoma (OAG) and ocular hypertension (OHT).</p><p><strong>Patients and methods: </strong>This study is an interim analysis of an ongoing, prospective, open-label, multicenter study in patients with OAG or OHT who are inadequately managed with topical IOP-lowering medication for reasons other than efficacy. IOP-lowering rescue treatment is allowed if implant retreatment criteria are not met. The primary endpoint is time to retreatment/rescue after the initial implant administration analyzed with the Kaplan-Meier method. Key safety measures include treatment-emergent adverse events (TEAEs) and reading-center evaluation of central corneal endothelial cell density (CECD). Analysis of data collected through 15 September 2023 focused on outcomes after a single or two implants.</p><p><strong>Results: </strong>In total, 441 patients received the 10-µg bimatoprost implant in the study eye on day 1 (cycle 1), 179 patients received a second administration (cycle 2), and 378 patients had at least 12 months of follow-up data available. The median time (95% confidence interval) from the first administration to a second administration or rescue was 392 (369, 485) days; the probability of not requiring retreatment or rescue by day 360 was 57.5%. A second implant administration similarly provided a long duration of IOP control. The baseline mean (standard error, SE) IOP was 25.6 (0.14) mmHg; the mean (SE) change from baseline IOP in unrescued eyes after a single administration was - 7.5 (0.21) mmHg at week 24 and - 6.4 (0.28) mmHg at month 12. Conjunctival hyperemia, typically associated with the administration procedure, was the most common ocular TEAE (cycle 1, 14.3%; cycle 2, 12.8%). Mean (SE) percentage change in CECD from baseline at 12 months after administration was - 4.3 (0.81)% in cycle 1 and - 8.5 (2.22)% in cycle 2. The cycle 1 implant was no longer visible or ≤ 25% of initial size in 66.3% and 94.3% of study eyes at months 12 and 24, respectively.</p><p><strong>Conclusions: </strong>In this interim analysis based on available data, the IOP-lowering effect of the initial administration of the 10-µg bimatoprost implant was well maintained for > 1 year in most patients. Results after a second administration were comparable. The safety profile of initial and repeat administration was acceptable.</p><p><strong>Trial registry: </strong>ClinicalTrials.gov identifier NCT03850782; registered 20 February 2019.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"557-570"},"PeriodicalIF":13.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11947067/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476367","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Antibacterials with Novel Chemical Scaffolds in Clinical Development.","authors":"Dominik Heimann, Daniel Kohnhäuser, Alexandra Jana Kohnhäuser, Mark Brönstrup","doi":"10.1007/s40265-024-02137-x","DOIUrl":"10.1007/s40265-024-02137-x","url":null,"abstract":"<p><p>The rise of antimicrobial resistance represents a significant global health threat, driven by the diminishing efficacy of existing antibiotics, a lack of novel antibacterials entering the market, and an over- or misuse of existing antibiotics, which accelerates the evolution of resistant bacterial strains. This review focuses on innovative therapies by highlighting 19 novel antibacterials in clinical development as of June 2024. These selected compounds are characterized by new chemical scaffolds, novel molecular targets, and/or unique mechanisms of action, which render their potential to break antimicrobial resistance particularly high. A detailed analysis of the scientific foundations behind each of these compounds is provided, including their pharmacodynamic profiles, current development state, and potential for overcoming existing limitations in antibiotic therapy. By presenting this subset of chemically novel antibacterials, the review highlights the ability to innovate in antibiotic drug development to counteract bacterial resistance and improve treatment outcomes.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"293-323"},"PeriodicalIF":13.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11891108/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rezafungin: A Review in Invasive Candidiasis.","authors":"Simon Fung, Matt Shirley","doi":"10.1007/s40265-024-02134-0","DOIUrl":"10.1007/s40265-024-02134-0","url":null,"abstract":"<p><p>Rezafungin (Rezzayo^®) is a next-generation echinocandin antifungal with improved pharmacokinetic properties over first-generation echinocandins that allows for once-weekly rather than once-daily intravenous administration. It has recently been approved for the treatment of adults with invasive candidiasis in the EU and UK, and is approved for adults who have limited or no alternative options for the treatment of candidaemia and invasive candidiasis in the USA. In the pivotal phase 3 ReSTORE trial, rezafungin was non-inferior to caspofungin (a first-line echinocandin antifungal agent) based both on global cure rates at day 14 and all-cause mortality rates at day 30 in adults with candidaemia or invasive candidiasis. Additionally, the once-weekly administration of rezafungin has the potential advantage of front-loading the dose and increasing drug exposure, with some evidence suggesting that rezafungin may achieve earlier infection clearance relative to caspofungin. Rezafungin was generally well tolerated, with the most common treatment-emergent adverse events being hypokalaemia, pyrexia, diarrhoea and anaemia. Therefore, rezafungin is a useful addition to the treatments currently available for invasive candidiasis in adults, with potential benefits associated with less frequent administration compared with first-generation echinocandins.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"415-423"},"PeriodicalIF":13.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current and Emerging Therapeutic Strategies for the Management of Neurotrophic Keratitis.","authors":"Javier Lacorzana, Yan Ning Neo, Laura Maubon, Daniel Sibley, Sajjad Ahmad","doi":"10.1007/s40265-025-02147-3","DOIUrl":"10.1007/s40265-025-02147-3","url":null,"abstract":"<p><p>Neurotrophic keratitis is a rare eye condition characterised by reduced or absent corneal sensation. This leads to impaired corneal healing through a loss of protective mechanisms such as blinking. The cornea becomes vulnerable to persistent epithelial defects, ulceration, infection and ultimately, vision loss or loss of the eye. Treatment strategies aim to protect the corneal surface and promote re-epithelialisation. Established treatments include specialised eye drops such as blood serum eye drops and topical nerve growth factor. In some cases, surgical interventions or procedures such as amniotic membrane transplantation or corneal neurotisation may be necessary. Emerging therapeutic drug options include insulin drops, BRM424, CSB-001 and varenicline. The aim of this Current Opinion is to introduce and review this field for the general reader, paying particular attention to emerging drug therapies for neurotrophic keratitis.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"283-291"},"PeriodicalIF":13.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022552","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Authors' Reply to Purchase's Comment on \"Trientine Tetrahydrochloride, from Bench to Beside: A Narrative Review\".","authors":"C Omar F Kamlin, Timothy M Jenkins, Jamie L Heise, Naseem S Amin","doi":"10.1007/s40265-024-02142-0","DOIUrl":"10.1007/s40265-024-02142-0","url":null,"abstract":"","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"445-446"},"PeriodicalIF":13.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142968974","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ebronucimab: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40265-025-02146-4","DOIUrl":"10.1007/s40265-025-02146-4","url":null,"abstract":"<p><p>Ebronucimab (^®) is a subcutaneous fully human anti-proprotein convertase subtilisin/kexin type 9 (PCSK9) monoclonal antibody being developed by Akeso Biopharma for the treatment of dyslipidaemia. In September 2024, ebronucimab was approved in China, in combination with statins or statins and other lipid-lowering therapies, for adult patients with primary hypercholesterolaemia [including heterozygous familial hypercholesterolaemia (HeFH)] and mixed hyperlipidaemia who are unable to achieve the low-density lipoprotein cholesterol target after receiving moderate or higher doses of statins. This article summarizes the milestones in the development of ebronucimab leading to this first approval for primary hypercholesterolaemia, mixed hyperlipidaemia and HeFH.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"437-441"},"PeriodicalIF":13.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255051","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}