A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of Cavosonstat Administered Twice Daily Compared With Placebo for 24 Weeks in Adult Subjects With Chronic Obstructive Pulmonary Disease (COPD)

Brief Summary: The purpose of this study is to to evaluate the efficacy and safety of Cavosonstat administered twice daily compared with Placebo for 24 Weeks in adult subjects with Chronic Obstructive Pulmonary Disease (COPD)
Detailed Description:To investigate the effect of cavosonstat compared with placebo, on the annualized rate of moderate-to-severe acute exacerbations of COPD (AECOPD) over 24 weeks of treatment.
To investigate:
The effect of cavosonstat compared with placebo, on the duration from baseline to first moderate AECOPD event
The effect of cavosonstat compared with placebo, on respiratory function, as assessed by post-bronchodilator percent-predicted forced expiratory volume in one second (ppFEV1)
The effect of cavosonstat compared with placebo on annualized rate of moderate AECOPD over 24 weeks of treatment
The effect of cavosonstat compared with placebo on annualized rate of severe AECOPD over 24 weeks of treatment
The safety and tolerability of cavosonstat compared with placebo
The pharmacokinetics of cavosonstat
Assessment of quality of life

开始日期2021-02-03

申办/合作机构

GSNOR Therapeutics, Inc.

CTR20201787

/ Completed临床1期

评价N91115在健康受试者中的药代动力学特征以及安全性、耐受性的随机双盲、安慰剂对照I期临床研究

主要目的：评价N91115在健康受试者中的药代动力学特征；次要目的：评价N91115在健康受试者中的安全性和耐受性；探索性研究目的：评价N91115在健康受试者中的药效动力学特征。

开始日期2020-11-06

申办/合作机构

朗煜医药科技（杭州）有限公司

NCT04129931

/ Active, not recruiting临床2期IIT

PrecISE (Precision Interventions for Severe and/or Exacerbation-Prone Asthma) Network Study

The primary objective of this study is to evaluate several interventions given to participants with severe asthma. Interventions are administered in a crossover manner with 16-week treatment periods followed by 8 to 16 week washout.

开始日期2019-12-19

申办/合作机构

The University of North Carolina at Chapel Hill

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项与 Cavosonstat 相关的文献（医药）

2025-01-01·Neuroscience

The role of S-nitrosoglutathione reductase (GSNOR) in T cell-mediated immunopathology of experimental autoimmune encephalomyelitis (EAE)

Article

作者: Kim, Judong ; Touhidul Islam, S.M. ; Singh, Avtar K ; Singh, Inderjit ; Fei, Qiao ; Touhidul Islam, S M ; Won, Jeseong

Previously, we reported that both S-nitrosoglutathione (GSNO), a carrier of cellular nitric oxide, and N6022, an injectable form of GSNO reductase (GSNOR) inhibitor that increases endogenous GSNO levels, alleviate experimental autoimmune encephalomyelitis (EAE) in mice by suppressing Th1 and Th17 immune responses. Building on these findings, we explored the role of GSNOR in EAE pathogenesis and evaluated the efficacy of an orally active GSNOR inhibitor (N91115) in treating the EAE disease. EAE mice exhibited heightened expression/activity of GSNOR in the spinal cord, and the knockout of the GSNOR gene resulted in much milder clinical manifestations of EAE, with lower degrees of demyelination and axonal loss, reduced microglial and astrocyte activations, as well as suppressed Th1 and Th17 cell responses, alongside bolstered Treg immune responses. Next, we evaluated the efficacy of N91115 against EAE immunopathology. Consistent with our findings in GSNOR deficient EAE mice, daily N91115 administration reduced clinical EAE severity, with less spinal cord demyelination and axonal loss compared to untreated EAE mice. Furthermore, N91115 treated EAE mice showed diminished Th1 and Th17 immune responses and enhanced Treg responses. This observation underscores the potential of increased GSNOR expression and activity as a risk factor exacerbating EAE immunopathology, while simultaneously highlighting its potential as a target for modifying the disease. Furthermore, the balanced immune regulation provided by orally active N91115 (IL-6/IL-17a vs. IL-10) presents a promising alternative to immunosuppressive drugs, reducing the risk of opportunistic infections.

2020-12-17·The Cochrane database of systematic reviews

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).

Review

作者: Sinha, Ian P ; Murphy, Jared ; Southern, Kevin W ; Nevitt, Sarah J

BACKGROUNDCystic fibrosis (CF) is a common life-shortening genetic condition caused by a variant in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A class II CFTR variant F508del (found in up to 90% of people with CF (pwCF)) is the commonest CF-causing variant. The faulty protein is degraded before reaching the cell membrane, where it needs to be to effect transepithelial salt transport. The F508del variant lacks meaningful CFTR function and corrective therapy could benefit many pwCF. Therapies in this review include single correctors and any combination of correctors and potentiators.OBJECTIVESTo evaluate the effects of CFTR correctors (with or without potentiators) on clinically important benefits and harms in pwCF of any age with class II CFTR mutations (most commonly F508del).SEARCH METHODSWe searched the Cochrane Cystic Fibrosis and Genetic Disorders Cystic Fibrosis Trials Register, reference lists of relevant articles and online trials registries. Most recent search: 14 October 2020.SELECTION CRITERIARandomised controlled trials (RCTs) (parallel design) comparing CFTR correctors to control in pwCF with class II mutations.DATA COLLECTION AND ANALYSISTwo authors independently extracted data, assessed risk of bias and evidence quality (GRADE); we contacted investigators for additional data.MAIN RESULTSWe included 19 RCTs (2959 participants), lasting between 1 day and 24 weeks; an extension of two lumacaftor-ivacaftor studies provided additional 96-week safety data (1029 participants). We assessed eight monotherapy RCTs (344 participants) (4PBA, CPX, lumacaftor, cavosonstat and FDL169), six dual-therapy RCTs (1840 participants) (lumacaftor-ivacaftor or tezacaftor-ivacaftor) and five triple-therapy RCTs (775 participants) (elexacaftor-tezacaftor-ivacaftor or VX-659-tezacaftor-ivacaftor); below we report only the data from elexacaftor-tezacaftor-ivacaftor combination which proceeded to Phase 3 trials. In 14 RCTs participants had F508del/F508del genotypes, in three RCTs F508del/minimal function (MF) genotypes and in two RCTs both genotypes. Risk of bias judgements varied across different comparisons. Results from 11 RCTs may not be applicable to all pwCF due to age limits (e.g. adults only) or non-standard design (converting from monotherapy to combination therapy). Monotherapy Investigators reported no deaths or clinically-relevant improvements in quality of life (QoL). There was insufficient evidence to determine any important effects on lung function. No placebo-controlled monotherapy RCT demonstrated differences in mild, moderate or severe adverse effects (AEs); the clinical relevance of these events is difficult to assess with their variety and small number of participants (all F508del/F508del). Dual therapy Investigators reported no deaths (moderate- to high-quality evidence). QoL scores (respiratory domain) favoured both lumacaftor-ivacaftor and tezacaftor-ivacaftor therapy compared to placebo at all time points. At six months lumacaftor 600 mg or 400 mg (both once daily) plus ivacaftor improved Cystic Fibrosis Questionnaire (CFQ) scores slightly compared with placebo (mean difference (MD) 2.62 points (95% confidence interval (CI) 0.64 to 4.59); 1061 participants; high-quality evidence). A similar effect was observed for twice-daily lumacaftor (200 mg) plus ivacaftor (250 mg), but with low-quality evidence (MD 2.50 points (95% CI 0.10 to 5.10)). The mean increase in CFQ scores with twice-daily tezacaftor (100 mg) and ivacaftor (150 mg) was approximately five points (95% CI 3.20 to 7.00; 504 participants; moderate-quality evidence). At six months, the relative change in forced expiratory volume in one second (FEV₁) % predicted improved with combination therapies compared to placebo by: 5.21% with once-daily lumacaftor-ivacaftor (95% CI 3.61% to 6.80%; 504 participants; high-quality evidence); 2.40% with twice-daily lumacaftor-ivacaftor (95% CI 0.40% to 4.40%; 204 participants; low-quality evidence); and 6.80% with tezacaftor-ivacaftor (95% CI 5.30 to 8.30%; 520 participants; moderate-quality evidence). More pwCF reported early transient breathlessness with lumacaftor-ivacaftor, odds ratio 2.05 (99% CI 1.10 to 3.83; 739 participants; high-quality evidence). Over 120 weeks (initial study period and follow-up) systolic blood pressure rose by 5.1 mmHg and diastolic blood pressure by 4.1 mmHg with twice-daily 400 mg lumacaftor-ivacaftor (80 participants; high-quality evidence). The tezacaftor-ivacaftor RCTs did not report these adverse effects. Pulmonary exacerbation rates decreased in pwCF receiving additional therapies to ivacaftor compared to placebo: lumacaftor 600 mg hazard ratio (HR) 0.70 (95% CI 0.57 to 0.87; 739 participants); lumacaftor 400 mg, HR 0.61 (95% CI 0.49 to 0.76; 740 participants); and tezacaftor, HR 0.64 (95% CI, 0.46 to 0.89; 506 participants) (moderate-quality evidence). Triple therapy Three RCTs of elexacaftor to tezacaftor-ivacaftor in pwCF (aged 12 years and older with either one or two F508del variants) reported no deaths (high-quality evidence). All other evidence was graded as moderate quality. In 403 participants with F508del/minimal function (MF) elexacaftor-tezacaftor-ivacaftor improved QoL respiratory scores (MD 20.2 points (95% CI 16.2 to 24.2)) and absolute change in FEV₁ (MD 14.3% predicted (95% CI 12.7 to 15.8)) compared to placebo at 24 weeks. At four weeks in 107 F508del/F508del participants, elexacaftor-tezacaftor-ivacaftor improved QoL respiratory scores (17.4 points (95% CI 11.9 to 22.9)) and absolute change in FEV₁ (MD 10.0% predicted (95% CI 7.5 to 12.5)) compared to tezacaftor-ivacaftor. There was probably little or no difference in the number or severity of AEs between elexacaftor-tezacaftor-ivacaftor and placebo or control (moderate-quality evidence). In 403 F508del/F508del participants, there was a longer time to protocol-defined pulmonary exacerbation with elexacaftor-tezacaftor-ivacaftor over 24 weeks (moderate-quality evidence).AUTHORS' CONCLUSIONSThere is insufficient evidence that corrector monotherapy has clinically important effects in pwCF with F508del/F508del. Both dual therapies (lumacaftor-ivacaftor, tezacaftor-ivacaftor) result in similar improvements in QoL and respiratory function with lower pulmonary exacerbation rates. Lumacaftor-ivacaftor was associated with an increase in early transient shortness of breath and longer-term increases in blood pressure (not observed for tezacaftor-ivacaftor). Tezacaftor-ivacaftor has a better safety profile, although data are lacking in children under 12 years. In this population, lumacaftor-ivacaftor had an important impact on respiratory function with no apparent immediate safety concerns; but this should be balanced against the blood pressure increase and shortness of breath seen in longer-term adult data when considering lumacaftor-ivacaftor. There is high-quality evidence of clinical efficacy with probably little or no difference in AEs for triple (elexacaftor-tezacaftor-ivacaftor) therapy in pwCF with one or two F508del variants aged 12 years or older. Further RCTs are required in children (under 12 years) and those with more severe respiratory function.

2017-05-01·Journal of Cystic Fibrosis2区 · 医学

Pharmacokinetics and safety of cavosonstat (N91115) in healthy and cystic fibrosis adults homozygous for F508DEL-CFTR

2区 · 医学

Article

作者: Zeitlin, Pamela L ; Taylor-Cousar, Jennifer L ; Donaldson, Scott H ; Shoemaker, Steven A ; Troha, Janice M ; Casey, Alicia ; Chmiel, James F ; Mandagere, Arun ; Flume, Patrick A ; Solomon, George M ; Zemanick, Edith T ; McCoy, Karen

BACKGROUNDCavosonstat (N91115), an orally bioavailable inhibitor of S-nitrosoglutathione reductase, promotes cystic fibrosis transmembrane conductance regulator (CFTR) maturation and plasma membrane stability, with a mechanism of action complementary to CFTR correctors and potentiators.METHODSA Phase I program evaluated pharmacokinetics, drug-drug interactions and safety of cavosonstat in healthy and cystic fibrosis (CF) subjects homozygous for F508del-CFTR. Exploratory outcomes included changes in sweat chloride in CF subjects.RESULTSCavosonstat was rapidly absorbed and demonstrated linear and predictable pharmacokinetics. Exposure was unaffected by a high-fat meal or rifampin-mediated effects on drug metabolism and transport. Cavosonstat was well tolerated, with no dose-limiting toxicities or significant safety findings. At the highest dose, significant reductions from baseline in sweat chloride were observed (-4.1mmol/L; P=0.032) at day 28.CONCLUSIONSThe favorable safety and clinical profile warrant further study of cavosonstat in CF. ClinicalTrials.gov Numbers: NCT02275936, NCT02013388, NCT02500667.

项与 Cavosonstat 相关的新闻（医药）

2024-11-08

·健康元

健康元哮喘治疗领域新进展：创新GSNOR抑制剂临床Ⅱ期研究完成首例患者入组

10月16日，健康元全新S-亚硝基谷胱甘肽还原酶（GSNOR）抑制剂顺利完成了其Ⅱ期临床研究的首例受试者入组，标志着该药物在探索中度及重度哮喘治疗新方案上迈出了关键一步。哮喘创新药取得关键进展安全性良好且疗效显著资料显示，这款全新 S-亚硝基谷胱甘肽还原酶（GSNOR）抑制剂为新一代哮喘治疗药物，能够精准调控GSNO-SNO分子通路，对抑制哮喘患者体内的炎症反应及促进支气管舒张具有显著效果。相较于现有治疗手段，该款哮喘创新药将有望为那些对传统疗法反应不佳或病情难以控制的患者提供一种更新更优的治疗选择。哮喘，尤其是重度哮喘，已成为当前我国公共卫生领域不可忽视的问题。据权威数据统计，我国 20岁及以上人群哮喘患病率为4.2%，患者达4570万，其中近一成为重度哮喘。尽管目前针对哮喘有诸多治疗手段，但仍有大量重度哮喘患者未能得到有效控制，生活质量严重受限，且目前吸入疗法是治疗哮喘病的主要给药方式，急需优质的口服哮喘药物辅助治疗。作为国内研发进度领先的哮喘抑制剂，健康元这款创新药物将有望降低哮喘患者的夜间急性发作率，为难以控制的患者群体提供更优效的治疗。未来， GSNOR药物的成功开发将会大大满足临床的迫切需求。前期研究数据显示，该药物显露出了与市场上主流哮喘药物孟鲁斯特相当的疗效，并在健康人群的 I期临床试验中展现出良好的安全性。参考孟鲁斯特制剂2023年在国内药品终端市场近18亿元的销售额，若后续该款哮喘创新药成功获批上市，未来市场潜力将不容小觑。聚焦优势呼吸领域创新药板块加速布局近年来，健康元持续深耕优势呼吸领域，加速研发管线布局，多个创新药或迎来密集爆发期。目前公司已拥有 10个品种、14个品规的吸入制剂，20多个呼吸科领域品种在研。健康元持续加大呼吸创新药的布局，已经推出8款创新药，成功实现现有及在研品种对哮喘和慢阻肺吸入治疗药物的全类别的覆盖。其中，备受市场关注的抗流感创新药已于 8月申报生产，有望明年上市。同时另一款针对COPD的TSLP生物药，目前临床进展也非常顺利。加上公司近期进入II期的两款药物，健康元已经有5款呼吸创新药推进到II期以后。结合近年来健康元在创新药方面的频繁布局，加上公司有意加速聚焦创新药项目，健康元创新药管线正迎来提质扩容的快速上升期。未来随着各个创新药临床研究的稳步推进及商业化落地，健康元的发展也将再迎来新一轮爆发期。

临床2期

2024-04-18

·健康元

健康元TG-1000胶囊获突破性进展抗流感病毒药物赛道将迎重磅产品

4月17日晚间，健康元发布公告称，其创新药产品TG-1000胶囊多中心Ⅲ期临床试验达到主要研究终点，公司将于近期就该药物治疗流感的上市申请向国家药品监督管理局药品审评中心（CDE）进行沟通，推进药物上市进程。 TG-1000为创新抗流感1类新药，是一种新型帽依赖性核酸内切酶抑制剂，适用于12周岁及以上单纯性甲型和乙型流感急性感染无并发症的患者。公开资料显示，健康元是一家创新科研型的综合医药集团，聚焦创新药、高壁垒复杂制剂的开发工作。近年来，公司深耕呼吸系统疾病、消化系统疾病等重点治疗领域，通过自主研发、外部引进、合作开发相结合的研发模式，不断扩充企业优势领域产品线和研发管线。 TG-1000胶囊是健康元于去年3月引进的一款重磅创新药。早期研究数据表明，该药物具有起效快、抑制病毒时间长、耐受性好、口服不受食物影响的特点，能够同时有效抑制甲型、乙型流感病毒。去年 9月，健康元启动了TG-1000胶囊III期临床试验。该III期临床研究是一项多中心、随机、双盲研究的临床试验，主要研究终点是治疗期15天内所有流感症状缓解的时间。初步统计分析结果表明，该药物达到主要疗效指标并具有统计学差异（P<0.0001）且未发生与药物相关的严重不良反应，临床试验表现良好且安全性更高。流感作为世界上常见的病毒性传染病，每年流感季节性流行在全球可导致 300万-500万例重症病例，29万-65万例呼吸道疾病相关死亡病例。抗流感病毒药物是治疗及控制流感的重要手段之一，如 TG-1000胶囊后续成功获批上市，将进一步强化健康元在流感领域的产品布局，助力公司抢占国内抗流感病毒药物市场高地。近两年来，创新药的布局成为药企争相发力的主赛道之一，尤其是今年 “创新药”被首次写入政府工作报告，创新药迎来了极佳的发展机遇。健康元也前瞻性进行了多领域的创新药布局工作。除了抗流感新药 TG-1000胶囊临床进展顺利以外，健康元在优势呼吸领域还达成了多项重要战略合作，实现从注射剂、吸入制剂到口服小分子创新药的多维度覆盖。其中，治疗哮喘双靶点新药 DBM-1152A、治疗哮喘口服新药N91115、针对慢性阻塞性肺疾病的口服药物-拜耳小分子抑制剂均进入临床I期；适用于治疗哮喘、慢性阻塞性肺疾病等呼吸系统疾病注射液QX008N和BA2101分别进入临床Ib期、临床Ⅱ期。此外，健康元还成功引进镇痛新药 FZ008-145，拓展了公司在镇痛领域的研发布局。 FZ008-145 是全球第二、国内首个高选择性第二代 Nav1.8抑制剂，拟适用于疼痛的治疗，预期具有强效、非成瘾性镇痛的优势，目前已获批开展临床试验。

临床3期临床2期申请上市临床1期上市批准

2023-01-03

·PRNewswire

DelveInsight Evaluates a Robust Cystic Fibrosis Pipeline as 75+ Influential Pharma Players to Set Foot in the Domain

Cystic fibrosis is quickly becoming the most prevalent genetic illness in every region of the world. According to the Cystic Fibrosis Patient Registry, more than 30,000 persons in the United States are currently afflicted with this ailment, and it is projected that approximately 70,000 people throughout the world are afflicted with this condition. The introduction of potential drugs is on the new horizon, which is bolstering market growth. Several companies are involved in evaluating novel therapies in different stages of development. LAS VEGAS, Jan. 3, 2023 /PRNewswire/ -- DelveInsight's ' Cystic Fibrosis Pipeline Insight – 2022 ' report provides comprehensive global coverage of available, marketed, and pipeline cystic fibrosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the cystic fibrosis pipeline domain. Key Takeaways from the Cystic Fibrosis Pipeline Report DelveInsight's cystic fibrosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for cystic fibrosis treatment. Key cystic fibrosis companies such as Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, AbbVie, Krystal Biotech, BiomX, Inc., Moderna Therapeutics, ReCode Therapeutics, Aeon Respire, Kither Biotech, Carbon Biosciences, SpliSense, Sionna Therapeutics, Lung Therapeutics, Tavanta Therapeutics, Branca Bunús Ltd, and others are evaluating new cystic fibrosis drugs to improve the treatment landscape. Promising cystic fibrosis pipeline therapies in various stages of development include ELX-02, NM002, ARO ENaC, S-1226, P-1037, MRT5005, Cavosonstat, AR-501, QBW276, VX-121, OligoG, PTI-808, JBT-101, GLPG1837, POL6014, CB-280, Galicaftor, SPX-101, RPL554, IONIS-ENaCRx, CHF 6333, Theradux, PRX 110, LAU-7b, LUNAR-CF, ETD001, 4D-710, AP-PA02,Deutivacaftor/tezacaftor/vanzacaftor, Brensocatib, ABBV-576, KB 407, BX004-A, VXc-522, SP-101, mRNA-based therapeutic program, AR-001, KIT-2014, LTI-05, TAVT-135, BrB102, and others. In December 2022, Vertex Pharmaceuticals announced that the US Food and Drug Administration had cleared its Investigational New Drug (IND) application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF) lung disease for the approximately 5,000 people with CF who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF. In December 2022, First Wave BioPharma, Inc. announced that it has selected the initial clinical trial sites for its planned Phase II clinical trial to evaluate an enhanced enteric microgranule delivery formulation of adrulipase as a treatment for exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). Based on anticipated regulatory and clinical trial activities, First Wave BioPharma expects to report topline data from the Phase 2 trial by mid-2023. In December 2022, Sionna Therapeutics, announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for SION-638, a novel small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The first subject has been dosed in a Phase I study to evaluate the safety and pharmacokinetics of SION-638 in healthy volunteers. In December 2022, Armata Pharmaceuticals, Inc. announced that the last subject has completed the company's Phase I/II 'SWARM-P.a.' clinical trial of its lead candidate, AP-PA02, in cystic fibrosis (CF) subjects with chronic pulmonary Pseudomonas aeruginosa infection. In March 2020, Armata announced that it had been awarded up to $5 million in a therapeutic development award from the CF Foundation to advance development of AP-PA02. In October 2021, the Foundation subsequently made an equity investment of $3 million in Armata to further support this program. In November 2022, Aridis Pharmaceuticals, Inc. announced the closing of patient enrollment in the multiple ascending dose (MAD) and dose-ranging cohorts in the Phase IIa clinical trial of AR-501 in cystic fibrosis (CF) patients. The Company is on track to complete database lock, data analyses, and disclose top-line data in the first quarter of 2023. In September 2022, Eloxx Pharmaceuticals, Inc. announced topline results from the Phase II clinical trial of ELX-02 in combination with ivacaftor in Class 1 cystic fibrosis (CF) patients with at least one nonsense mutation. The combination trial of ELX-02 with ivacaftor was well tolerated but did not achieve statistical significance for efficacy endpoints, including changes from baseline in sweat chloride concentration (SCC) and percent forced expiratory volume (FEV1). Request a sample and discover the recent advances in cystic fibrosis drug treatment @ Cystic Fibrosis Pipeline Report The cystic fibrosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage cystic fibrosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the cystic fibrosis clinical trial landscape. Cystic Fibrosis Overview Cystic fibrosis is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. F508del is the most common variation reported globally, but there are over 2000 others, not all of which cause the disease. Cystic fibrosis symptoms typically appear 6-8 months after birth, though this varies greatly from person to person. Cystic fibrosis symptoms vary with age and can affect different body parts. Two tests for cystic fibrosis diagnosis are newborn screening, a genetic test to look for a gene defect, and a blood test to look for pancreatic problems. A sweat test can be performed after initial testing to confirm the cystic fibrosis diagnosis. Currently available cystic fibrosis medications aim at symptom relief, complication prevention, and, more recently, protein rectifiers to correct underlying structural and functional abnormalities. Find out more about drugs for cystic fibrosis @ New Cystic Fibrosis Drugs A snapshot of the Cystic Fibrosis Pipeline Drugs mentioned in the report: Learn more about the emerging cystic fibrosis pipeline therapies @ Cystic Fibrosis Clinical Trials Cystic Fibrosis Therapeutics Assessment The cystic fibrosis pipeline report proffers an integral view of cystic fibrosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Cystic Fibrosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: Cystic fibrosis transmembrane conductance regulator modulators, Dipeptidyl peptidase I inhibitors, Cystic fibrosis transmembrane conductance regulator modulators, Cell membrane permeability enhancers, Bacterial growth inhibitor, Gene transference, Immunomodulators; Peptidyltransferase modulators; Protein 50S ribosomal subunit modulators, Cystic fibrosis transmembrane conductance regulator replacements; Gene transference, Epithelial sodium channel antagonists, Cystic fibrosis transmembrane conductance regulator stimulants, Cystic fibrosis transmembrane conductance regulator expression modulators; RNA interference Key Cystic Fibrosis Companies: Eloxx Pharmaceuticals, Arrowhead Pharmaceuticals, SolAeroMed, Parion Sciences, Translate Bio, Inc., Path BioAnalytics, Aridis Pharmaceuticals, Novartis, Vertex Pharmaceuticals, AlgiPharma, Spyryx Biosciences, Inc., Verona Pharma, Ionis Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Ligand Pharmaceuticals, Boehringer Ingelheim, OrPro Therapeutics, Protalix Biotherapeutics, Laurent Pharmaceuticals, Arcturus Therapeutics, Enterprise Therapeutics, Affinia Therapeutics, Sound Pharmaceuticals, Spirovant, 4D Molecular Therapeutics, Santhera Pharmaceuticals, Armata Pharmaceuticals, AbbVie, Krystal Biotech, BiomX, Inc., Moderna Therapeutics, ReCode Therapeutics, Aeon Respire, Kither Biotech, Carbon Biosciences, SpliSense, Sionna Therapeutics, Lung Therapeutics, Tavanta Therapeutics, Branca Bunús Ltd, and others. Key Cystic Fibrosis Pipeline Therapies: ELX-02, NM002, ARO ENaC, S-1226, P-1037, MRT5005, Cavosonstat, AR-501, QBW276, VX-121, OligoG, PTI-808, JBT-101, GLPG1837, POL6014, CB-280, Galicaftor, SPX-101, RPL554, IONIS-ENaCRx, CHF 6333, Theradux, PRX 110, LAU-7b, LUNAR-CF, ETD001, 4D-710, AP-PA02,Deutivacaftor/tezacaftor/vanzacaftor, Brensocatib, ABBV-576, KB 407, BX004-A, VXc-522, SP-101, mRNA-based therapeutic program, AR-001, KIT-2014, LTI-05, TAVT-135, BrB102, and others. Dive deep into rich insights for new drugs for cystic fibrosis treatment; visit @ Cystic Fibrosis Medications Table of Contents For further information on the cystic fibrosis pipeline therapeutics, reach out @ Cystic Fibrosis Drug Treatment Related Reports Cystic Fibrosis Epidemiology Forecast Cystic Fibrosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and the cystic fibrosis epidemiology trends. Cystic Fibrosis Market Cystic Fibrosis Market Insight, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key cystic fibrosis companies, including Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, among others. Non-Cystic Fibrosis Bronchiectasis Market Non-Cystic Fibrosis Bronchiectasis Market Insight, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key non-cystic fibrosis bronchiectasis companies, including AstraZeneca, Insmed, Novartis, CSL Behring, Arrowhead Pharmaceuticals, among others. Non-Cystic Fibrosis Bronchiectasis Pipeline Non-Cystic Fibrosis Bronchiectasis Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key non-cystic fibrosis bronchiectasis companies, including AstraZeneca, Insmed, Novartis, CSL Behring, Arrowhead Pharmaceuticals, among others. Non-Cystic Fibrosis Bronchiectasis Epidemiology Non-Cystic Fibrosis Bronchiectasis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and the non-cystic fibrosis bronchiectasis epidemiology trends. Airway Clearance Devices for Cystic Fibrosis Market Airway Clearance Devices for Cystic Fibrosis Market Insight, Competitive Landscape, and Market Forecast – 2027 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key airway clearance devices for cystic fibrosis companies, including Dymedso, PARI GmBH, Thayer Medical, Electromed, among others. Other Trending Reports Goitre Market | Thymus Cancer Market | US Healthcare Outlook Report | Venous Stenosis Market | Negative Pressure Wound Therapy Systems Market | Metrorrhagia Market | Dysfunctional Uterine Bleeding Market | Hypereosinophilic Syndrome Market | Age-related Vision Dysfunction Market | Dental Lasers Market | CRISPR Therapies Pipeline Insight | Cell And Gene Therapy For Multiple Myeloma Market | Drug Hypersensitivity Market | Dysthymia Market | Persistent Depressive Disorder Market | Cancer Vaccines Market | Weight Loss/Weight Management (Cystic Fibrosis) Market | Food Allergy Market | Obsessive-Compulsive Disorder Market | Tumor Ablation Market | Physiotherapy Equipment Market | CAR-T Pipeline Insight | Anti-hypertension Market | Radiofrequency Ablation Devices Market | Global Messenger RNA (mRNA)-based Vaccines and Therapeutics Market | Gastro Intestinal Bleeding Market | Trastuzumab Biosimilars Insight | Varicose Veins Market | Germ Cell Tumor Market | Intracardiac Echocardiography Devices Market | India Healthcare Outlook Report | Crows Feet Market Insight | Seborrhoeic Dermatitis Market | Injectable Drug Delivery Devices Market | Structural Heart Devices Market | Substance (Drug) Abuse Market Related Healthcare Blogs Cystic Fibrosis: A Global Concern Weight Loss and Cystic Fibrosis Market Cystic Fibrosis Treatment Market Related Healthcare Services Healthcare Business Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. Connect with us at LinkedIn Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research LLP

临床2期临床1期

100 项与 Cavosonstat 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10916	-	-	DB14775

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
中度哮喘	临床2期	中国	健康元药业集团股份有限公司	2024-10-16
重度哮喘	临床2期	中国	健康元药业集团股份有限公司	2024-10-16
脑脊髓炎	临床前	美国	Ralph H. Johnson VA Medical Center	2025-01-01
脑脊髓炎	临床前	美国	The Medical University of South Carolina	2025-01-01
慢性阻塞性肺疾病	药物发现	-	GSNOR Therapeutics, Inc.	2021-02-03
哮喘	药物发现	中国	朗煜医药科技（杭州）有限公司	2020-11-06
囊性纤维化	药物发现	美国	Nivalis Therapeutics, Inc.	2015-11-01

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临床结果

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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


NCT02275936 \| NCT02500667 \| NCT02013388 (Pubmed \| J Cyst Fibros)	临床1期	囊性纤维化 F508DEL-CFTR	-	Cavosonstat	(餘觸獵淵範選築製製餘) = 鏇選網醖鹽襯築顧繭夢餘鑰淵製構膚膚襯鏇醖 (鑰艱鬱遞鹹顧膚衊鹽網 )	-	2017-05-01
NCT02013388 (SNO2, CTgov)	临床1期	-	49	Placebo (Placebo)	膚醖鹽襯鹹鬱願鏇簾構(鬱網壓糧鹹蓋鹽襯鏇製) = 鹹淵獵鏇窪範鏇艱鏇範獵艱積鹹蓋蓋齋壓糧鹽 (網壓淵積窪糧壓鑰襯構, 膚衊鏇襯遞鏇醖壓壓醖 ~ 網繭鏇齋餘衊築齋繭鏇) 更多	-	2015-03-18
NCT02013388 (SNO2, CTgov)	临床1期	-	49	N91115 (10 mg)	膚醖鹽襯鹹鬱願鏇簾構(鬱網壓糧鹹蓋鹽襯鏇製) = 築餘觸網顧壓鑰膚壓構獵艱積鹹蓋蓋齋壓糧鹽 (網壓淵積窪糧壓鑰襯構, 繭醖顧繭餘積衊築觸醖 ~ 構觸鏇窪範範窪製廠醖) 更多	-	2015-03-18