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.

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

[+1]

NCT02934139

/ Completed临床1期

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of N91115 in Healthy Subjects

The present study is designed to assess the safety and tolerability of escalating, multiple ascending doses of Cavosonstat (N91115) in healthy subjects.

开始日期2016-10-01

申办/合作机构

Nivalis Therapeutics, Inc.

100 项与 Cavosonstat 相关的临床结果

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100 项与 Cavosonstat 相关的转化医学

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100 项与 Cavosonstat 相关的专利（医药）

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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 ; Singh, Inderjit ; Touhidul Islam, S M ; Fei, Qiao ; Singh, Avtar K ; 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 ; Southern, Kevin W ; Nevitt, Sarah J ; Murphy, Jared

BACKGROUND:

Cystic 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.

OBJECTIVES:

To 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 METHODS:

We 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 CRITERIA:

Randomised controlled trials (RCTs) (parallel design) comparing CFTR correctors to control in pwCF with class II mutations.

DATA COLLECTION AND ANALYSIS:

Two authors independently extracted data, assessed risk of bias and evidence quality (GRADE); we contacted investigators for additional data.

MAIN RESULTS:

We 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' CONCLUSIONS:

There 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 fibrosis : official journal of the European Cystic Fibrosis Society2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: Donaldson, Scott H ; Mandagere, Arun ; Shoemaker, Steven A ; Troha, Janice M ; Chmiel, James F ; Casey, Alicia ; McCoy, Karen ; Zemanick, Edith T ; Flume, Patrick A ; Zeitlin, Pamela L ; Taylor-Cousar, Jennifer L ; Solomon, George M

BACKGROUND:

Cavosonstat (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.

METHODS:

A 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.

RESULTS:

Cavosonstat 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.

CONCLUSIONS:

The 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）

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

2017-04-19

·BioSpace

Ex-Dendreon CEO Returns to Biotech, Throws a Lifeline to Struggling Nivalis

April 19, 2017 By Mark Terry , BioSpace.com Breaking News Staff Nivalis Therapeutics , located in Boulder, Colo., and Alpine Immune System , a private company located in Seattle, have agreed to merge . Alpine will merge with a wholly-owned subsidiary of Nivalis in an all-stock deal. The resulting public company will focus on inflammation and immuno-oncology utilizing its novel protein-based discovery platform. Alpine utilizes a variant immunoglobulin domain (vigD) technology platform. The company has plans to begin a Phase I trial with its first compound, a dual ICOS/CD28 antagonist that has been engineered for autoimmune and inflammatory diseases. It expects to initiate the trial in the second half of 2018. Nivalis had traditionally focused on developing drugs for cystic fibrosis (CF). Its developmental compounds have selectively targeted the enzyme S-nitrosoglutathione reductase (GSNOR), which regulates S-nitrosoglutathione (GSNO). Low levels of GSNO have been associated with CF, asthma, inflammatory bowel diseases and some cardiovascular diseases. In November 2016, Nivalis’ Phase II trial evaluating cavosonstat in adult CF patients who had two copies of the F508del-CTFR mutation and who were being treated with Orkambi, failed to meet its primary endpoint. John Carroll , writing for Endpoints News , says , “After Nivalis’ drug failed in a disastrous attempt to come up with an add-on to Vertex’s cystic fibrosis combo, about the only thing left after the inevitable slash and burn on staff and costs was a virtually empty public shell. Nivalis had managed to catch one of the last waves in the old IPO boom in cashing in on investors’ interest in biotech. But by the time the market closed today the market cap had shrunk down to a mere $41.3 million. And now [Mitch] Gold’s Alpine Immune System is taking that over in a reverse merger that will scoot it out into the public market.” As part of the merger, Frazier Healthcare Partners , Mitch Gold’s Alpine BioVentures , and OrbiMed Advisors plan to invest a combined $17 million into Alpine Immune Sciences. With the cash Alpine and Nivalis have, with the additional funds, the combined company will have about $90 million in cash. “This merger provides a unique opportunity to accelerate the development of our novel immunotherapy platform focused on both inflammation and immuno-oncology, Gold , Alpine’s executive chairman and chief executive officer, said in a statement. “We look forward to building on our early success by taking multiple novel programs into the clinic to help patients with significant medical needs.” The new company, named Alpine, will be led by Gold as chairman and chief executive officer. The board of directors will expand to include two Nivalis representatives. Ben Fidler , writing for Xconomy , says , “Nivalis’s failure has created an opportunity for Alpine to sidestep an IPO, a strategy a number of privately held biotechs have used over the years to go public more quickly and cheaply.” Gold is best known as the chief executive officer of Dendreon . During his tenure, he led the development of sipuleucel-T (Provenge), an immunotherapy for prostate cancer. He raised about $2 billion to bring Provenge to market and came away with about $27 million personally in two days after the U.S. Food and Drug Administration (FDA) approved the compound. Fidler writes, “But the treatment was a commercial bust for a variety of reasons, so Dendreon faltered, was buried in more than $600 million in debt, and filed for bankruptcy in 2014. Sipuleucel-T was ultimately sold to Valeant Pharmaceuticals .” Dendreon and Gold were ahead of the curve in terms of immuno-oncology, and it has since gotten to be a more stable and growing field. It still struggles with problems related to the personalized nature of the treatments, which were part of Dendreon’s downfall, although company’s seem to be working their way around that obstacle. After leaving Dendreon in 2012, Gold founded Alpine Biosciences , which sold to Oncothyreon (now Cascadian Therapeutics ) for $27 million in stock. Gold then launched Alpine BioVentures in 2013 and seeded Alpine in 2015.

免疫疗法临床2期IPO并购临床1期

2017-01-04

·BioSpace

Nivalis Begins Exploring Strategic Alternatives and Streamlining Ops After Clinical Trial Setback

January 4, 2017 By Mark Terry , BioSpace.com Breaking News Staff Boulder, Colo. – Nivalis Therapeutics announced yesterday that it is evaluating “strategic alternatives,” which may include the possibility of an acquisition, merger, or other strategic transaction. Nivalis is focused on identifying, developing and commercializing drugs for cystic fibrosis (CF). Previously known as N30 Pharmaceuticals , it changed to Nivalis in February 2015. The company went public in July 2015. On November 28, the company announced topline results from its Phase II clinical trial of cavosonstat, 200 mg and 400 mg, in patients with CF who had two copies of the F508del-CFTR mutation that were being treated with Orkambi. Although the drug had minimal side effects, it failed to show benefit in absolute change in percent predicted FEV1, which was the primary endpoint. “While we are disappointed in the outcome of this trial, we plan to continue to investigate the therapeutic potential of cavosonstat and our S-nitrosoglutathione reductase (GSNOR) inhibitor portfolio to determine next steps,” said Jon Congleton , president and chief executive officer of Nivalis, in a statement. In the company’s third-quarter financial report on November 7, 2016, the company published a net loss of $7.4 million, or ($0.48) per share, versus a net loss of $6.1 million ($0.39 per share) in the same quarter in 2015. Most of the loss was attributed to the two Phase II trials of cavosonstat. In the same month, Nivalis announced that Cynthia Smith , then the chief commercial officer of ZS Pharma , a subsidiary of AstraZeneca , had joined the company’s board of directors. She had also previously served as vice president, market access and commercial development at Affymax. In addition to today’s announcement, Nivalis indicated it plans to finish its ongoing SNO-7 clinical trial of cavosonstat in patients with CF currently on Kalydeco (ivacaftor). That trial is expected to be completed this quarter. To assist with its strategic alternatives, Nivalis had hired Ladenburg Thalmann & Co. Inc. , an advisory firm. In addition to a possible merger, sale or partnership, the company indicated it plans to streamline operations. Although that often means layoffs, nothing specific was said in the company announcement regarding job cuts. Greg Avery , writing for the Denver Business Journal, notes that as of September 30, 2016, Nivalis had $16 million in cash and $50 million in marketable securities. “That could make Nivalis a candidate for being acquired by another company looking to go public,” he writes. “Such reverse mergers became more common during the waning months of 2016 as the IPO market struggled and companies sought a cost-effective way to access public markets.” He cites Boulder-based Mirage Therapeutics merging with Carlsbad, California-based Signal Genetics in November as an example of such a deal. Another recent example is when Boston, Mass.-based Tokai Pharmaceuticals and tic Pharma , a privately-held pharma company, merged under the name of OticPharma . Arsalan Arif , writing for Endpoints News, said, “Last August, Tokai became the latest in a long string of public biotechs to hit a brick wall running at full speed. The late-stage failure of its cancer drug galeterone effectively shredded its stock price and its business plan, forcing the company to rapidly ax staffers and start looking for strategic alternatives in the face of an extinction-level event.” Nivalis is currently trading for $2.36.

临床2期临床结果并购高管变更IPO

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
囊性纤维化	临床2期	美国	Nivalis Therapeutics, Inc.	2015-11-01
哮喘	临床1期	中国	朗煜医药科技（杭州）有限公司	2020-11-06
脑脊髓炎	临床前	美国	Medical University of South Carolina	2025-01-01
脑脊髓炎	临床前	美国	Ralph H. Johnson VA Medical Center	2025-01-01

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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	廠壓構獵製願襯衊選壓(夢膚衊淵顧齋網範蓋壓) = 廠壓糧淵選淵製廠淵顧鹽醖觸蓋膚糧鹹築築觸 (製廠鏇願窪觸淵膚窪鹹 )	-	2017-05-01
NCT02013388 (SNO2, CTgov)	临床1期	-	49	Placebo (Placebo)	構製蓋夢醖網蓋鏇淵膚 = 顧衊憲艱壓衊窪壓願網衊蓋襯艱獵製觸衊廠鏇 (選廠願簾淵壓網齋選簾, 鹽襯窪構製鏇餘顧膚餘 ~ 醖獵蓋膚鬱醖壓鏇齋獵) 更多	-	2015-03-18
NCT02013388 (SNO2, CTgov)	临床1期	-	49	N91115 (10 mg)	構製蓋夢醖網蓋鏇淵膚 = 築獵製顧願壓淵鹹壓築衊蓋襯艱獵製觸衊廠鏇 (選廠願簾淵壓網齋選簾, 夢淵醖觸積醖選窪糧糧 ~ 鹽製繭膚廠夢醖製製襯) 更多	-	2015-03-18