A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Efficacy and Safety of Brensocatib in Adults With Moderate to Severe Hidradenitis Suppurativa - The CEDAR Study

The primary purpose of the study is evaluate the effect of brensocatib compared with placebo.

开始日期2024-12-16

申办/合作机构

Insmed, Inc.

NCT06344728

/ Completed临床1期

A Phase 1, Open-Label, Randomized, Two-Period Crossover Study to Assess the Relative Bioavailability of Brensocatib Pediatric Oral Solution to Oral Tablets Following Single Oral Doses in Healthy Subjects

The primary purpose of this study is to determine the relative bioavailability of brensocatib between the pediatric oral solution and oral tablets in healthy participants.

开始日期2024-04-30

申办/合作机构

Insmed, Inc.

NCT06178783

/ Completed临床1期

A Phase 1, Double-blind, Randomized, Single-center Study to Assess the Palatability and Acceptability of Different Brensocatib Oral Liquid Formulations in Healthy Subjects

The primary purpose of this study is to assess the palatability and compare acceptability of brensocatib oral solutions.

开始日期2023-12-27

申办/合作机构

Insmed, Inc.

100 项与 Brensocatib 相关的临床结果

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100 项与 Brensocatib 相关的转化医学

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100 项与 Brensocatib 相关的专利（医药）

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项与 Brensocatib 相关的文献（医药）

2025-01-01·BRAIN BEHAVIOR AND IMMUNITY

Cathepsin C exacerbates EAE by promoting the expansion of Tfh cells and the formation of TLSs in the CNS

Article

作者: Zhao, Xinnan ; Li, Xia ; Zhao, Henan ; Liu, Shuang ; Ma, Jianmei ; Du, Cong ; Fan, Kai ; Liu, Gang ; Yang, Xiaohan ; Liu, Jing

Multiple sclerosis (MS) is a chronic autoimmune disease of the central nervous system (CNS) mediated by CD4⁺ T helper (Th) cells, and characterized by immune cell infiltration, demyelination and neurodegeneration, with no definitive cure available. Thus, it is pivotal and imperative to acquire more profound comprehension of the underlying mechanisms implicated in MS. Dysregulated immune responses are widely believed to play a primary role in the pathogenesis of MS. Recently, a plethora of studies have demonstrated the involvement of T follicular helper (Tfh) cells and tertiary lymphoid-like structures (TLSs) in the pathogenesis and progression of MS. Cathepsin C (CatC) is a cysteine exopeptidase which is crucial for the activation of immune-cell-associated serine proteinases in many inflammatory diseases in peripheral system, such as rheumatoid arthritis and septicemia. We have previously demonstrated that CatC is involved in neuroinflammation and exacerbates demyelination in both cuprizone-induced and experimental autoimmune encephalomyelitis (EAE) mouse models. However, the underlying immunopathological mechanism remains elusive. In the present study, we established a recombinant myelin oligodendrocyte glycoprotein 35-55 peptide-induced EAE model using conditional CatC overexpression mice to investigate the effects of CatC on the alteration of CD4⁺ Th subsets, including Th1, Th2, Th17, Tfh and T regulatory cells. Our findings demonstrated that CatC particularly enhanced the population of Tfh cell in the brain, resulting in the earlier onset and more severe chronic syndrome of EAE. Furthermore, CatC promoted the formation of TLSs in the brain, leading to persistent neuroinflammation and exacerbating the severity of EAE in the chronic phase. Conversely, treatment with AZD7986, a specific inhibitor of CatC, effectively attenuated the syndrome of EAE and its effects caused by CatC both in vivo and in vitro. These findings provide a novel insight into the critical role of CatC in innate and adaptive immunity in EAE, and specific inhibitor of CatC, AZD7986, may contribute to potential therapeutic strategies for MS.

2025-01-01·ERJ Open Research

Brensocatib in patients with bronchiectasis: subgroup analyses from the WILLOW trial

Article

作者: Metersky, Mark L ; Lauterio, Melanie ; Chalmers, James D ; McShane, Pamela J ; Loebinger, Michael R ; Teper, Ariel ; Fernandez, Carlos ; van der Laan, Roald ; Haworth, Charles S ; Fucile, Sebastian ; Shih, Vivian H

Introduction:

Bronchiectasis is a chronic inflammatory airway disease. Brensocatib, an oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), reduces pulmonary inflammation by preventing the activation of neutrophil serine proteases. In the phase II WILLOW trial, brensocatib prolonged time to first exacerbation in patients with bronchiectasis. In thispost hocanalysis we compare clinical outcomes in patients from WILLOW according to baseline disease characteristics.

Methods:

Adults with bronchiectasis treated with brensocatib (10 or 25 mg) or placebo once daily were analysed by baseline Bronchiectasis Severity Index (BSI) score (≤4 (mild), 5–8 (moderate), or ≥9 (severe)), exacerbation history (2 or ≥3 in the previous year), blood eosinophil count (<300 cells per µL or ≥300 cells per µL), long-term macrolide use (≥6 months; no or yes) andPseudomonas aeruginosaculture at screening (negative or positive). End-points were time to first exacerbation, annualised exacerbation rate, change in lung function from baseline, and safety. All patients who received brensocatib were pooled and compared with placebo.

Results:

Treatment with brensocatibversusplacebo was associated with a longer time to first exacerbation (hazard ratio (95% confidence interval), BSI: ≤4, 0.28 (0.08–0.96); 5–8, 0.75 (0.35–1.60); ≥9, 0.61 (0.35–1.04); prior exacerbations: 2, 0.56 (0.34–0.90); ≥3, 0.71 (0.32–1.59); blood eosinophils per µL: <300, 0.66 (0.42–1.06); ≥300, 0.49 (0.20–1.20); long-term macrolide use: no, 0.60 (0.38–0.94); yes, 0.60 (0.25–1.45);P. aeruginosaculture: negative, 0.54 (0.32–0.92); positive, 0.68 (0.37–1.27)). Safety results were similar across subgroups.

Discussion:

Patients treated with brensocatib had a numerically longer time to first exacerbation and reduced annualised rate of exacerbationversusplacebo across all key baseline disease characteristics.

2024-11-22·JCI Insight

Increased expression of cathepsin C in airway epithelia exacerbates airway remodeling in asthma

Article

作者: Yao, Ye ; Qin, Qingwu ; Liu, Huijun ; Liu, Chi ; Wu, Xinyu ; Qu, Xiangping ; Ji, Ming ; Yang, Ming ; Chen, Long ; Xiang, Yang ; Wang, Weijie ; Qin, Xiaoqun ; Yuan, Lin ; Qing, Bei ; Xia, Zhenkun ; Du, Xizi ; Qin, Qiuyan

Airway remodeling is a critical factor determining the pathogenesis and treatment sensitivity of severe asthma (SA) or uncontrolled asthma (UA). The activation of epithelial-mesenchymal trophic units (EMTUs) regulated by airway epithelial cells (AECs) has been proven to induce airway remodeling directly. However, the triggers for EMTU activation and the underlying mechanism of airway remodeling are not fully elucidated. Here, we screened the differentially expressed gene cathepsin C (CTSC; also known as dipeptidyl peptidase 1 [DPP-1]) in epithelia of patients with SA and UA using RNA-sequencing data and further verified the increased expression of CTSC in induced sputum of patients with asthma, which was positively correlated with severity and airway remodeling. Moreover, direct instillation of exogenous CTSC induced airway remodeling. Genetic inhibition of CTSC suppressed EMTU activation and airway remodeling in two asthma models with airway remodeling. Mechanistically, increased secretion of CTSC from AECs induced EMTU activation through the p38-mediated pathway, further inducing airway remodeling. Meanwhile, inhibition of CTSC also reduced the infiltration of inflammatory cells and the production of inflammatory factors in the lungs of asthmatic mice. Consequently, targeting CTSC with compound AZD7986 protected against airway inflammation, EMTU activation, and remodeling in the asthma model. Based on the dual effects of CTSC on airway inflammation and remodeling, CTSC is a potential biomarker and therapeutic target for SA or UA.

项与 Brensocatib 相关的新闻（医药）

2025-02-26

·CPHI制药在线

备受期待的2025年上市新药

关注并星标CPHI制药在线 2025年预计有多款里程碑式的疗法将获得FDA的批准，并有可能获得良好的商业回报。例如Vertex的囊性纤维化药物Alyftrek，已于去年年底获得批准，预计将在2030年实现80亿美元以上的年销售额。阿斯利康和第一三共的乳腺癌ADC药物Datroway于今年1月获得FDA批准，有望在2030年达到50亿美元的年销售额高度。一、Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) ●开发商：Vertex　　 ●模态：小分子　　 ●适应症：囊性纤维化　　 ●监管状态：已批准（2024年12月20日）　　 ●预测2030年销售额：83.4亿美元　　 ●背景与意义： Alyftrek代表着一种崭新的三重组合 CFTR 调节剂疗法：vanzacaftor、tezacaftor 和 deutivacaftor，适用于6岁及以上携带至少一个对该疗法有反应突变的囊性纤维化（CF）患者。囊性纤维化是由CFTR基因突变引起的，这个基因负责CFTR蛋白质的生成，基因突变导致这种蛋白质功能障碍或缺失，从而导致体内产生异常粘稠的粘液，积聚在器官中，引发大多数CF症状。CFTR调节剂可以改善那些由CFTR基因中特定突变引起的囊性纤维化患者的CFTR蛋白功能。Alyftrek为CF患者提供了崭新的选择，特别是对于无法耐受Trikafta（同样为Vertex产品）的患者。除此之外，Alyftrek只需每日服用一次，相较于其他调节剂每日两次的服用方案，可能使患者的用药更加便利。二、Datroway (datopotamab deruxtecan) ●开发商：阿斯利康/第一三共　　 ●模态：ADC　　 ●适应症：既往接受过治疗的转移性 HR 阳性、HER2 阴性乳腺癌　　 ●监管状态：已批准（2025年1月17日）　　 ●预测2030年销售额：49.8亿美元　　 ●背景与意义：美国每年新检测出的乳腺癌患者超过30万人。虽然早期乳腺癌的生存率较高，但转移性患者存活时间超过5年的比例只有30%。大约70%的乳腺癌确诊病例属于HR阳性、HER2阴性乳腺癌。HR阳性转移性乳腺癌的早期治疗中，内分泌疗法被广泛使用。然而，初步治疗后，内分泌治疗的疗效通常有限。目前在内分泌治疗后的标准治疗是化疗，但化疗的反应率和治疗效果较差。在这样的情况下，Datroway的获批显得意义非常重大。Datroway用于治疗无法手术切除或转移性HR阳性、HER2阴性乳腺癌的成人患者，这些患者已接受过内分泌疗法和化疗的治疗。这种新型的TROP2靶向ADC药物的批准标志着一个重要的治疗里程碑，为转移性乳腺癌患者提供了常规化疗之外的新选择。三、Nivolumab Enhanze ●开发商：百时美施贵宝　　 ●模态：单抗　　 ●适应症：多发性硬化症　　 ●监管状态：已递交申请（PDUFA日期2025年2月28日）　　 ●预测2030年销售额：39.1亿美元　　 ●背景与意义： Halozyme与Bristol Myers Squibb合作，已提交Opdivo® (nivolumab) 与 Enhanze® 联合制剂的BLA申请，用于治疗多发性硬化症 (MS)。Opdivo 是BMS的王牌PD-1 免疫检查点抑制剂。该药物利用 Halozyme 的 Enhanze® 技术来改善药物输送。Enhanze®通过提高药物的皮下给药效率，改善患者的治疗体验。该技术的核心是利用重组人透明质酸酶PH20（rHuPH20）来分解皮下组织中的透明质酸，透明质酸是皮肤和结缔组织中的一种天然屏障物质。通过暂时降低透明质酸的粘性，Enhanze® 技术能够加速皮下药物的吸收，使大分子药物更快、更高效地进入血液循环。这种方式不仅可以减少药物给药时间，改善患者的舒适度，还能在某些情况下减少静脉注射的需求，使得许多原本需要通过静脉注射的药物可以通过皮下注射给药。四、Journavx (suzetrigine) ●开发商：Vertex　　 ●模态：小分子　　 ●适应症：中度至重度急性疼痛　　 ●监管状态：已批准（2025年1月30日）　　 ●预测2030年销售额：29亿美元　　 ●背景与意义： Vertex的Journavx（suzetrigine）获得FDA批准，用于治疗成人中度至重度急性疼痛，成为近20年来首个非阿片类镇痛药。Journavx通过阻断NaV1.8钠离子通道发挥作用，这一新机制标志着疼痛治疗领域的重要突破。与传统阿片类药物不同，它不具有成瘾性，并且在临床试验中显示出与阿片类药物相当的止痛效果，但副作用较少。 Journavx的成功推动了NaV1.8抑制剂的进一步开发，目前有多个候选药物正在进行临床试验。尽管NaV1.7和NaV1.8钠通道一直被认为是疼痛治疗的潜在靶点，但开发具有高度选择性的药物一直面临挑战，特别是由于这些通道在其他组织中具有高度同源性。Journavx的选择性优势使其在不影响其他身体功能的前提下有效缓解疼痛。未来，Journavx及其同类药物有望取代阿片类药物，解决长期困扰的成瘾性问题，同时在急性和慢性疼痛治疗领域发挥更大作用。五、Brensocatib ●开发商：Insmed　　 ●模态：小分子　　 ●适应症：非囊性纤维化支气管扩张　　 ●监管状态：已递交NDA申请（PDUFA日期2025年8月12日）　　 ●预测2030年销售额：28.4亿美元　　 ●背景与意义：尽管非囊性纤维化支气管扩张（支气管扩张症）的患病率近年来有所增加，但它仍被广泛忽视。患者常表现出慢性咳嗽、痰液产生以及反复的呼吸道感染和加重发作，这些症状严重影响患者的生活质量并增加住院风险。支气管扩张症的病因复杂，病情表现和严重程度因人而异，这种异质性给临床管理带来了巨大挑战。尽管目前患者可以使用一些现有的治疗方法，但FDA和EMA尚未批准任何针对支气管扩张症的专门药物。当前的管理措施主要集中在清理气道、使用黏液活性剂和抗生素治疗急性发作，而长期使用抗生素则面临耐药性问题。目前正在进行的 3 期 ASPEN 试验是支气管扩张症领域规模最大的全球研究，旨在评估 Brensocatib 对支气管扩张症加重发作率的影响。 Brensocatib 是一种口服的选择性 DPP-1 （二肽基肽酶1）抑制剂，可作为中性粒细胞介导疾病（包括支气管扩张和无鼻息肉的慢性鼻窦炎）的潜在治疗方法。如果获得FDA批准，将成为首款支气管扩张症专用药物。六、Aficamten ●开发商：Cytokinetics ●模态：小分子 ●适应症：阻塞性肥厚型心肌病 ●监管状态：已递交NDA申请（PDUFA日期9月26日） ●预测2030年销售额：26亿美元 ●背景与意义：肥厚型心肌病（HCM）是最常见的单基因心脏疾病，估计大约影响1/500的人口。其主要由肌节的染色体显性突变引起，其中β-心脏肌球蛋白（MYH7）和心脏肌球蛋白结合蛋白C（cMYBP-C, MYBPC3）的突变占据了约50%的家族性HCM病例。Ficamten是一种选择性的小分子心脏肌球蛋白抑制剂，通过显著减缓磷酸盐释放，降低ATP酶活性，稳定肌球蛋白与肌动蛋白的弱结合状态。Aficamten具备成为治疗肥厚型心肌病有效药物的潜力。七、Tolebrutinib ●开发商：赛诺菲　　 ●模态：小分子　　 ●适应症：非复发性继发进展型多发性硬化症　　 ●监管状态：III期试验HERCULES于2024年9月实现主要终点　　 ●预测2030年销售额：13.7亿美元　　 ●背景与意义： FDA已于2024年12月13日授予tolebrutinib突破性疗法认定，用于治疗非复发性继发进展型多发性硬化症（nrSPMS）成人患者。与安慰剂相比，Tolebrutinib能将6个月确认的残疾进展（CDP）的发生时间延缓31%（HR 0.69；95% CI 0.55-0.88；p=0.0026）。Tolebrutinib是首个能够穿透大脑的BTK抑制剂，在多发性硬化症领域获得了突破性疗法认定。目前对于非复发性继发进展型多发性硬化症（nrSPMS）尚无获批药物。八、Remibrutinib ●开发商：诺华　　 ●模态：小分子　　 ●适应症：慢性自发性荨麻疹（CSU）　　 ●监管状态：预计2025年初递交NDA申请　　 ●预测2030年销售额：13亿美元　　 ●背景与意义： CSU指持续 6 周或更长时间的慢性荨麻疹，影响全球约 4000 万人。其特征是突然出现瘙痒的荨麻疹或深层组织肿胀（血管性水肿，可发生在面部、喉咙、手和脚上）。CSU 会引起严重的情绪困扰，大多数患者睡眠不足，焦虑或抑郁等精神障碍发生率很高，并且会影响他们的工作效率。抗组胺药通常用于治疗 CSU，因为它们可以阻断组胺受体并阻止组胺的促炎作用，从而导致瘙痒和肿胀。然而，超过 50% 的 CSU 患者无法单靠 H1 抗组胺药来控制。诺华的BTK抑制剂remibrutinib在关键的 III 期研究 REMIX-1 和 REMIX-2 中显示出显著的症状改善能力。Remibrutinib是一款高选择性共价口服 BTK 抑制剂，可阻断 BTK 级联并防止引起瘙痒性荨麻疹和肿胀的组胺释放。除了 CSU，remibrutinib还在其他几种免疫介导疾病中进行研究，例如化脓性汗腺炎，它在 II 期研究中达到了主要终点。九、Depemokimab ●开发商：GSK　　 ●模态：单抗　　 ●适应症：伴有 2 型炎症的哮喘以及慢性鼻窦炎伴鼻息肉 (CRSwNP)　　 ●监管状态：临床III期，向EMA递交申请　　 ●预计2030年销售额：11.3亿美元　　 ●背景与意义：在欧洲估计有超过 4200 万人患有哮喘，给患者的生活和医疗资源带来了沉重的负担，估计每年的社会成本为 460 亿欧元。CRSwNP（慢性鼻窦炎伴鼻息肉）是一种慢性疾病，影响多达 4% 的普通人群。 Depemokimab 是一种针对白细胞介素 5 (IL-5) 的单抗，是首个针对2型炎症哮喘和CRSwNP进入III 期试验的超长效生物制剂。Depemokimab 作为 2 型炎症哮喘患者的附加疗法，降低了病情恶化和住院率；与安慰剂相比，depemokimab 可减小鼻息肉大小和减少鼻塞。研究结果支持六个月 (26 周) 的给药方案。在哮喘患者和 CRSwNP 患者中，临床试验表明 depemokimab 可以持续抑制疾病的关键驱动因素，每年仅注射两次。十、Penmenvy (MenABCWY) ●开发商：GSK ●模态：疫苗 ●适应症：五合一脑膜炎疫苗 ●监管状态：已获批（2025年2月14日） ●预计2030年销售额：12.4亿美元 ●背景与意义： 2025年2月18日， FDA批准了新的5合1疫苗Penmenvy，用于预防10至25岁人群中的脑膜炎球菌感染或侵袭性脑膜炎球菌病（IMD）。Penmenvy将两种已获批准的疫苗的活性成分结合在一起，提供更好的保护，同时减少接种次数，简化了接种流程。 IMD是一种罕见但致命的感染，由脑膜炎奈瑟菌引起，可能导致脑膜炎（脑部肿胀）、败血症（血液感染），甚至在数小时内死亡。幸存者可能面临脑损伤、截肢、听力丧失和神经问题。16至23岁的青少年和年轻成年人风险更高。然而，只有不到13%的人完成了两剂疫苗接种，只有32%的人至少接种了一剂。 Penmenvy结合了Bexsero和Menveo疫苗的部分成分，通过一次注射保护免受五种最常见的脑膜炎奈瑟菌（A、B、C、W和Y）菌株的感染。此疫苗的简化接种方式可能会鼓励更多人进行免疫接种。参考文献： 1.Wexler, M. FDA approves Alyftrek, once-daily triple-combination therapy for CF. Cystic Fibrosis. 02. 01. 2025. 2.Datroway (datopotamab deruxtecan) approved in the US for patients with previously treated metastatic HR-positive, HER2-negative breast cancer. AZ Press Release. 17. 01. 2025. 3.Harrison, C. Vertex’s opioid-free drug for acute pain wins FDA approval. Nature Biotechnology. 2025. https://doi.org/10.1038/s41587-025-02590-3 4.Hartman, J. J., et al. Aficamten is a small-molecule cardiac myosin inhibitor designed to treat hypertrophic cardiomyopathy. Nat Cardiovasc Res 3, 1003–1016 (2024). https://doi.org/10.1038/s44161-024-00505-0 5.Press Release: Tolebrutinib designated Breakthrough Therapy by the FDA for non-relapsing secondary progressive multiple sclerosis. Sanofi Press Release. 13. 12. 2024. 6.Novartis Phase III data confirm sustained efficacy and long-term safety of oral remibrutinib in chronic spontaneous urticaria. Novartis Press Release. 31. 05. 2024. 7.Depemokimab accepted for review by the European Medicines Agency for use in asthma with type 2 inflammation and CRSwNP. GSK Press Release. 28. 01. 2025. 8.Das, J. FDA Approves 5-in-1 Vaccine Against Meningococcal Disease. WebMD. 9.McConaghie, A. et al. 2025’s Biggest New Launches. Scrip. 19. 02. 2025. 智药研习社近期直播预告扫码领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

抗体药物偶联物临床结果

2025-02-25

·新药前沿

FDA不召开专家会议！支气管扩张将迎首款First-In-Class疗法

2月24日，Insmed Incorporated公司发布公告称：FDA目前未计划召开专家咨询委员会会议讨论其开发的DPP1抑制剂Brensocatib治疗非囊性纤维化支气管扩张症患者的新药申请（NDA）。 FDA重申该申请处于优先审评阶段，PDUFA目标行动日期为2025年8月12日。 FDA不召开专家咨询委员会会议的决定通常被视为一个积极的信号，表明FDA可能没有需要外部咨询的重大问题。然而，最终的批准决定将取决于FDA对数据的全面审查。 Insmed Incorporated于2024年12月向FDA提交Brensocatib了NDA申请。该NDA基于 Aspen研究的数据。Aspen研究是有史以来在支气管扩张患者中进行的最大的III期研究。 Brensocatib有可能成为支气管扩张的第一种也是唯一批准的治疗方法，并且也是用于治疗中性粒细胞介导疾病的二肽基肽酶1（DPP1）抑制剂这种新型药物中的第一种治疗方法。支气管扩张症外，DPP1抑制剂在COPD、α-1抗胰蛋白酶缺乏症（AATD）、囊性纤维化（CF）等多种呼吸系统疾病都具有治疗潜力。值得一提的是，我国也有多家企业参与DPP1抑制剂竞争：海思科开发的HSK31858片已于2024年9月进入到Ⅲ期临床试验阶段。此外，恒瑞等公司也是该靶点积极的竞争者。本文由「新药前沿」微信公众号根据公开资料整理编辑，欢迎个人转发至朋友圈。媒体或机构转载授权请在「新药前沿」微信公众号留言公众号名称，审核通过后开通白名单获取转载授权，转载请标识来源。免责声明：本文仅作信息交流之目的，非投资建议或者治疗方案推荐，「新药前沿」微信公众号不对任何主体因使用本文内容而导致的任何损失承担责任。限于作者水平和专业知识所限，如有谬误，欢迎指正！

优先审批临床3期引进/卖出

2025-02-24

·PRNewswire

Insmed Announces that FDA Does Not Currently Plan to Hold Advisory Committee Meeting to Discuss New Drug Application for Brensocatib in Patients with Bronchiectasis

—FDA Reaffirms Application is Under Priority Review with a PDUFA Target Action Date of August 12, 2025— BRIDGEWATER, N.J., Feb. 24, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has informed the Company that it does not currently plan to hold an advisory committee meeting to discuss the New Drug Application (NDA) for brensocatib for patients with non-cystic fibrosis bronchiectasis. The FDA provided this update in its Day 74 communication to Insmed. "We are very pleased with our ongoing communications with the FDA about the NDA for brensocatib," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "We are committed to working closely with the agency to successfully complete the review and potentially bring forward this much-needed treatment for patients with bronchiectasis." The FDA had previously granted Priority Review to Insmed's NDA for brensocatib and set a target action date of August 12, 2025, under the Prescription Drug User Fee Act (PDUFA). This NDA is based on data from the landmark ASPEN study, the largest Phase 3 study ever conducted in patients with bronchiectasis. Brensocatib has the potential to become the first and only approved treatment for bronchiectasis and the first in a new class of medicines called dipeptidyl peptidase 1 (DPP1) inhibitors for the treatment of neutrophil-mediated diseases. About Bronchiectasis Bronchiectasis is a serious, chronic lung disease in which the bronchi become permanently dilated due to a cycle of infection, inflammation, and lung tissue damage. The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. Symptoms include chronic cough, excessive sputum production, shortness of breath, and repeated respiratory infections, which can worsen the underlying condition. Today, approximately 500,000 patients in the U.S., 600,000 patients in the EU5 (France, Germany, Italy, Spain, and UK), and 150,000 patients in Japan have been diagnosed with bronchiectasis, and there are currently no approved therapies specifically targeting bronchiectasis in these regions. About Brensocatib Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis, chronic rhinosinusitis without nasal polyps, hidradenitis suppurativa, and other neutrophil-mediated diseases. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. Brensocatib is an investigational drug product that has not been approved for any indication in any jurisdiction. About Insmed Insmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inﬂammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's pre-clinical research programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue. Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending four consecutive years as the No. 1 Science Top Employer. Visit to learn more. Forward-looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements. The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: the risk that the full data set from the ASPEN study or data generated in further clinical trials of brensocatib will not be consistent with the topline results of the ASPEN study or any additional results of the ASPEN study; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib in the U.S., Europe or Japan; failure to successfully commercialize brensocatib, if approved by applicable regulatory authorities, in the U.S., Europe or Japan, or to maintain U.S., European or Japanese approval for brensocatib once approved; uncertainties in the degree of market acceptance of brensocatib by physicians, patients, third-party payors and others in the healthcare community; inaccuracies in the Company's estimates of the size of the potential markets for brensocatib or in data the Company has used to identify physicians; expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; inability of the Company, Esteve Química, S.A., Thermo Fisher Scientific, Inc. or the Company's other third-party manufacturers to comply with regulatory requirements related to brensocatib; the Company's inability to obtain adequate reimbursement from government or third-party payors for brensocatib or acceptable prices for brensocatib; development of unexpected safety or efficacy concerns related to brensocatib; failure to obtain regulatory approval for potential future brensocatib indications; restrictions or other obligations imposed on us by agreements related to brensocatib, including our license agreement with AstraZeneca AB, and failure to comply with our obligations under such agreements; failure to successfully conduct future clinical trials for brensocatib, including due to the Company's potential inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval, among other things; risks that the Company's clinical studies will be delayed or that serious side effects will be identified during drug development; failure of third parties on which the Company is dependent to manufacture sufficient quantities of brensocatib for commercial or clinical needs, to conduct the Company's clinical trials, or to comply with the Company's agreements or laws and regulations that impact the Company's business or agreements with the Company; the strength and enforceability of the Company's intellectual property rights or the rights of third parties; the cost and potential reputational damage resulting from litigation to which the Company may become a party, including product liability claims; changes in laws and regulations applicable to the Company's business and failure to comply with such laws and regulations; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; and inability to repay the Company's existing indebtedness and uncertainties with respect to the Company's need and ability to access future capital. The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 and any subsequent Company filings with the Securities and Exchange Commission (SEC). The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Contact: Bryan Dunn Vice President, Investor Relations (646) 812-4030 [email protected] Michael V. Morabito, Ph.D. Director, Investor Relations (917) 936-8430 [email protected] Gianna De Palma Manager, Investor Relations (973) 886-2236 [email protected] Media: Mandy Fahey Vice President, Corporate Communications (732) 718-3621 [email protected] SOURCE Insmed Incorporated WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

优先审批临床3期申请上市基因疗法

100 项与 Brensocatib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15638

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
非囊性支气管扩张	申请上市	美国	Insmed, Inc.	2024-12-01
新型冠状病毒感染	临床3期	英国	Insmed, Inc.	2020-06-05
化脓性汗腺炎	临床2期	美国	Insmed, Inc.	2024-12-16
慢性鼻窦炎	临床2期	美国	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	阿根廷	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	澳大利亚	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	比利时	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	保加利亚	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	加拿大	Insmed, Inc.	2023-10-30
慢性鼻窦炎	临床2期	捷克	Insmed, Inc.	2023-10-30

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04594369 (ASPEN, PRNewswire) 人工标引	临床3期	支气管扩张	-	Brensocatib 10 mg	蓋積艱鏇構襯淵窪遞餘(鑰蓋衊積憲鏇艱觸夢構) = 鬱願齋鹽範積觸鑰製選鑰獵繭獵衊齋觸餘鹹衊 (淵鹽鑰積製糧衊鹽醖網 ) 更多	积极	2024-10-08
				Brensocatib 25 mg	蓋積艱鏇構襯淵窪遞餘(鑰蓋衊積憲鏇艱觸夢構) = 鑰簾鹹衊積艱廠繭鏇衊鑰獵繭獵衊齋觸餘鹹衊 (淵鹽鑰積製糧衊鹽醖網 ) 更多
NCT04594369 (ASPEN, Company_Website) 人工标引	临床3期	支气管扩张	1,721	Brensocatib 10 mg	壓壓襯鑰糧膚壓襯顧窪(淵夢鏇壓襯網願網繭顧) = 鏇獵醖襯鑰憲艱鹽齋餘壓衊鏇選襯積糧選糧餘 (網夢壓積築繭鬱獵齋襯 ) 更多	积极	2024-05-28
				Brensocatib 25 mg	壓壓襯鑰糧膚壓襯顧窪(淵夢鏇壓襯網願網繭顧) = 遞膚壓廠齋膚鬱蓋蓋窪壓衊鏇選襯積糧選糧餘 (網夢壓積築繭鬱獵齋襯 ) 更多
ATS2024 人工标引	临床1期	非囊性支气管扩张	28	Brensocatib (Subjects with mild renal impairment)	淵鏇築範憲顧憲觸餘齋(糧窪淵簾淵繭鹽鬱憲繭) = There were no treatment-emergent AEs, serious AEs, or death during this study. 選積夢壓鹹顧網衊願艱 (襯淵築鹽襯積構範鹹淵 )	积极	2024-05-22
				Brensocatib (Subjects with moderate renal impairment)
NCT04817332 (STOP-COVID19, CTgov)	临床3期	新型冠状病毒感染	406	Brensocatib (Brensocatib)	製醖積膚餘餘壓鑰築衊 = 構選製齋繭繭窪範鹽夢鬱獵鏇襯構襯衊糧餘範 (衊鹹構繭鑰顧鏇餘夢築, 範選夢鬱糧襯齋選選製 ~ 顧願廠鑰醖遞齋夢醖築) 更多	-	2023-08-22
				Placebo (Placebo)	製醖積膚餘餘壓鑰築衊 = 壓艱網簾鹽窪範願簾襯鬱獵鏇襯構襯衊糧餘範 (衊鹹構繭鑰顧鏇餘夢築, 襯鬱願鬱觸鏇襯餘獵築 ~ 鬱觸簾壓廠願選餘衊齋) 更多
ATS2023	N/A	支气管扩张	-	Brensocatib 25mg	鹹範鬱鹹夢蓋膚醖鏇壓(製襯廠鏇衊簾醖醖網襯) = 淵襯鑰蓋鹹壓衊顧憲觸鏇鹽鑰膚獵獵鬱願憲獵 (膚簾糧範遞鬱願鏇醖網 )	-	2023-05-21
				Placebo	鹹範鬱鹹夢蓋膚醖鏇壓(製襯廠鏇衊簾醖醖網襯) = 顧夢鹽構廠觸醖夢願壓鏇鹽鑰膚獵獵鬱願憲獵 (膚簾糧範遞鬱願鏇醖網 )
NCT03218917 (WILLOW, CTgov)	临床2期	非囊性支气管扩张	256	Brensocatib 10 mg (Brensocatib 10 mg)	醖餘淵顧遞築窪糧膚憲(壓餘獵獵觸襯餘鏇顧窪) = 願構獵顧範製廠鬱鹹獵製簾獵鹹選鏇鏇衊壓鏇 (衊鏇願鹽範窪顧窪簾鹽, 製繭糧襯簾鑰積繭鹹遞 ~ 壓顧襯觸願構艱齋鏇衊) 更多	-	2023-02-08
				Brensocatib 25 mg (Brensocatib 25 mg)	醖餘淵顧遞築窪糧膚憲(壓餘獵獵觸襯餘鏇顧窪) = 醖鬱餘鹹糧顧觸簾構鬱製簾獵鹹選鏇鏇衊壓鏇 (衊鏇願鹽範窪顧窪簾鹽, 齋膚膚網選鑰壓鬱淵鹹 ~ 構繭夢網艱醖顧糧鏇襯) 更多
Pubmed \| Clin Pharmacol Drug Dev	临床1期	-	-	Brensocatib 10mg	鏇夢夢壓顧鹽鬱鏇壓網(鏇淵艱淵鏇網觸觸鬱鏇) = The most frequently reported treatment-emergent adverse events were headache 繭襯積遞鬱範夢遞願憲 (鏇醖壓鑰憲膚糧鏇廠糧 ) 更多	-	2022-07-01
				Brensocatib 40mg
NCT04817332 (NEWS) 人工标引	临床3期	新型冠状病毒感染	404	Standard of Care+brensocatib	糧鑰獵鑰顧餘糧鹽獵憲(壓醖鏇製襯簾積衊夢壓) = Brensocatib did not improve the clinical status of patients hospitalized 選獵鏇夢蓋網積衊網鑰 (壓廠齋餘衊鑰窪網醖願 ) 更多	不佳	2022-05-18
				Standard of Care+Placebo
ATS2021	N/A	-	-	Brensocatib 40mg	鹹範夢鏇艱願繭鏇願壓(顧遞選顧獵積齋衊襯顧) = The most frequently reported TEAE was skin exfoliation, reported by 1 participant in each ethnic group, both in the fed state 膚壓窪壓選範衊構鑰顧 (艱齋網獵衊願範憲鑰構 ) 更多	-	2021-05-03
				Placebo
NCT03218917 (WILLOW, Pubmed \| N Engl J Med)	临床2期	支气管扩张 neutrophil elastase	256	Placebo	範膚襯襯醖艱夢願蓋糧(憲願夢鑰選鬱壓選醖鏇) = 獵蓋艱齋顧廠願網願鹹觸廠願積築願觸選鹽繭 (餘淵壓糧積淵窪餘鑰齋 )	积极	2020-11-26
				Brensocatib 10 mg	範膚襯襯醖艱夢願蓋糧(憲願夢鑰選鬱壓選醖鏇) = 獵構鏇積艱餘選膚夢壓觸廠願積築願觸選鹽繭 (餘淵壓糧積淵窪餘鑰齋 )