A Phase 1a/1b Trial in Relapsed/Refractory T-cell Non-Hodgkin Lymphoma to Determine the Safety Profile, Pharmacology, and Maximum Tolerated Dose of ST-001, a Fenretinide Phospholipid Suspension (12.5 mg/mL) for Intravenous Infusion

This study evaluates a fenretinide phospholipid suspension for the treatment of T-cell non-Hodgkin's lymphoma (NHL).

开始日期2023-12-18

申办/合作机构

SciTech Development LLC

NCT06181760

/ Completed临床1期

A Phase 1a, Randomized, Double-blind Placebo-controlled Study to Evaluate Safety and Tolerability and to Characterize the Pharmacokinetic Profile of Single Ascending Doses of Fenretinide Oral Capsules in Healthy Adult Volunteers

The goal of this clinical trial was to learn about a single dose of fenretinide in healthy volunteers, in both a fasted and fed state. The main questions to answer were:
* How well is a single dose of fenretinide tolerated? AND
* How is a single dose of fenretinide metabolized in healthy volunteers?
Participants will be asked to:
* Remain confined in a clinical research unit for 5 days after dosing.
* Provide blood samples for intense PK sampling and safety labs.
* Fast for 10 hours prior to administration of study drug (fasted cohorts).
* Consume a high fat meal prior to administration of study drug (fed cohort).
* Return to the clinic for a single follow-up visit for safety assessments.
The study will compare active fenretinide to placebo to see if fenretinide is more or less tolerable than placebo.

开始日期2023-11-22

申办/合作机构

Island Pharmaceuticals Ltd.

NCT05999435

/ Active, not recruiting临床2/3期

A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, ADAPTIVE PHASE 2/3 STUDY OF THE EFFICACY OF LAU-7b IN THE TREATMENT OF ADULTS WITH LONG COVID AND MODERATE TO SEVERE SYMPTOMS

ESSOR is a double-blind, placebo-controlled study of the orally-administered antiviral and inflammation-controlling LAU-7b for the treatment of adults with Long COVID and moderate to severe symptoms.

开始日期2023-11-20

申办/合作机构

Laurent Pharmaceuticals, Inc.

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项与芬维A胺相关的文献（医药）

2025-02-27·EXPERIMENTAL PHYSIOLOGY

Hydroxysteroid 17β-dehydrogenase 13 (Hsd17b13) knockdown attenuates liver steatosis in high-fat diet obese mice.

Article

作者: Morrice, Nicola ; Mody, Nimesh ; Mahmood, Shehroz ; Whitfield, Philip D ; Milanizadeh, Sara ; Mcilroy, George D ; Thompson, Dawn ; Rochford, Justin J ; Wilson, Sophie

Hydroxysteroid 17β-dehydrogenase 13 (HSD17B13) loss-of-function gene variants are associated with a decreased risk of metabolic dysfunction-associated steatotic liver disease (MASLD). Our RNA-seq analysis of steatotic liver from obese mice ± fenretinide treatment identified major beneficial effects of fenretinide on expression of hepatic genes including Hsd17b13. We sought to determine the relationship between Hsd17b13 expression and MASLD and to validate it as a therapeutic target by liver-specific knockdown. Hsd17b13 expression, which is unique to hepatocytes and associated with the lipid droplet, was elevated in multiple models of MASLD and normalised with the prevention of obesity and steatotic liver. Direct, liver-specific, shRNA-mediated knockdown of Hsd17b13 (shHsd17b13) in high-fat diet (HFD)-obese mice, markedly improved hepatic steatosis with no effect on body weight, adiposity or glycaemia. shHsd17b13 decreased elevated serum alanine aminotransferase (ALT), serum fibroblast growth factor 21 (FGF21) levels, and markers of liver fibrosis, for example, expression of Timp2. shHsd17b13 knockdown in HFD-obese mice and Hsd17b13 overexpression in cells reciprocally regulated expression of lipid metabolism genes, for example, Cd36. Global lipidomic analysis of liver tissue revealed a major decrease in diacylglycerols (e.g. DAG 34:3) with shHsd17b13 expression and an increase in phosphatidylcholines containing polyunsaturated fatty acids (PUFA) for example, phosphatidylcholine (PC) 34:3 and PC 42:10. Expression of key genes involved in phospholipid and PUFA metabolism, for example, Cept1, was also reciprocally regulated suggesting a potential mechanism of Hsd17b13 biological function and role in MASLD. In conclusion, Hsd17b13 knockdown in HFD-obese adult mice was able to alleviate MASLD via regulation of fatty acid and phospholipid metabolism, thereby confirming HSD17B13 as a genuine therapeutic target for MASLD and the development of liver fibrosis.

2025-01-01·Journal of Cystic Fibrosis

Efficacy and safety of LAU-7b in a Phase 2 trial in adults with cystic fibrosis

Article

作者: Zuckerman, Jonathan B ; Colin, Patrick ; Konstan, Michael W ; Kianicka, Irenej ; Middleton, Peter G ; Kotsimbos, Tom ; Radzioch, Danuta ; Polineni, Deepika ; Matouk, Elias ; Lands, Larry C ; Houle, Jean-Marie ; Liou, Theodore G ; Chmiel, James F ; Potvin, Diane ; Pislariu, Radu ; Bilodeau, Lara ; Nasr, Samya Z

BACKGROUND:

Lung inflammation is associated with tissue damage in cystic fibrosis (CF). LAU-7b, a novel oral drug candidate, was shown to control inflammation and stabilize CFTR protein in the epithelial membrane during inflammatory stress in preclinical models of CF.

METHODS:

A double-blind, randomized, placebo-controlled Phase 2 study was conducted to evaluate efficacy and safety of LAU-7b in adults with CF. LAU-7b or placebo was administered over 24 weeks as six 21-day treatment cycles each separated by 7 days. The primary efficacy endpoint was the absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV₁) at 24 weeks.

RESULTS:

A total of 166 subjects received at least one dose of study drug (Intent-To-Treat population, ITT), of which 122 received ≥5 treatment cycles (Per-Protocol population, PP). Both treatment arms showed a mean lung function loss at 24 weeks of 1.18 ppFEV₁ points with LAU-7b and 1.95 ppFEV₁ with placebo, a 0.77 ppFEV₁ (40 s) difference, p=0.345, and a 0.95 ppFEV₁ (49 %) difference in the same direction in PP population, p=0.263. Primary analysis of mean ppFEV₁ through 24 weeks showed differences of 1.01 and 1.23 ppFEV₁, in the ITT (65 % less loss, p=0.067) and PP populations (78 % less loss, reaching statistical significance p=0.049), respectively. LAU-7b had an acceptable safety profile.

CONCLUSION:

Although the study did not meet its primary efficacy endpoint in the ITT population, LAU-7b was generally well tolerated and showed evidence of preservation of lung function to support further development.

2024-12-01·iScience

A transcriptome-based risk model in sepsis enables prognostic prediction and drug repositioning

Article

作者: Long, Qiuyue ; Mao, Jingsong ; Wu, Jing ; Ye, Hongli ; Zheng, Yali ; Li, Jiwei ; Song, Shixu ; Gao, Zhancheng ; Ke Li ; Li, Ran

Septic management presented a tremendous challenge due to heterogeneous host responses. We aimed to develop a risk model for early septic stratification based on transcriptomic signature. Here, we combined genes OLAH, LY96, HPGD, and ABLIM1 into a prognostic risk score model, which demonstrated exceptional performance in septic diagnosis (AUC = 0.99-1.00) and prognosis (AUC = 0.61-0.70), outperforming that of Mars and SRS endotypes. Also, the model unveiled immunosuppressive status in high-risk patients and a poor response to hydrocortisone in low-risk individuals. Single-cell transcriptome analysis further elucidated expression patterns and effects of the four genes across immune cell types, illustrating integrated host responses reflected by this model. Upon distinct transcriptional profiles of risk subgroups, we identified fenretinide and meloxicam as therapeutic agents, which significantly improved survival in septic mice models. Our study introduced a risk model that optimized risk stratification and drug repurposing of sepsis, thereby offering a comprehensive management approach.

项与芬维A胺相关的新闻（医药）

2025-01-13

·PRNewswire

SciTech Development Advances to Next Stage of Clinical Trial for ST-001 nanoFenretinide

**Encouraging Data from Accelerated Trial Supports Progress for T-cell Non-Hodgkin Lymphoma.** DETROIT, Jan. 13, 2025 /PRNewswire/ -- SciTech Development, a clinical-stage pharmaceutical company specializing in oncology, is pleased to announce the initiation of the next stage of its clinical trial for their lead drug candidate, ST-001 nanoFenretinide (ST-001) . This milestone follows the successful completion of the accelerated safety and dose-escalation stage, where ST-001 demonstrated exceptional safety, tolerability, and pharmacokinetic performance. Advancing the Phase 1a Trial with Clear Clinical Objectives The continuation of the ST-001 clinical trial aims to achieve several critical clinical objectives: Confirm that ST-001 achieves previously reported clinical response rates. Determine the pharmacological profile of intravenous fenretinide delivered using SciTech's novel nanoparticle delivery system, designed for drugs with poor water solubility. Determine the recommended treatment dose (RTD) to evaluate disease activity and response rates in the follow-on Phase 1b trial and the planned trial in Small Cell Lung Cancer. "The success of our accelerated Phase 1a trial marks a pivotal milestone, driving ST-001 into the next critical stage of development," said Earle Holsapple, CEO of SciTech Development. "We are thrilled about the potential of ST-001 nanoFenretinide and its promise to transform treatment outcomes for patients battling cancers such as T-cell Non-Hodgkin Lymphoma." About ST-001 nanoFenretinide ST-001 nanoFenretinide is a patented nanoparticle IV formulation that combines the drug fenretinide with biocompatible phospholipids. This groundbreaking new investigational drug overcomes bioavailability challenges, enhances therapeutic efficacy, and minimizes toxicity in cancer patients. Powered by SciTech's advanced delivery platform, ST-001 enables rapid delivery of effective doses of fenretinide to cancer cells, marking a breakthrough in hard-to-treat cancers. Encouraging Early Results in Phase 1a Accelerated Trial During the accelerated stage of the trial, ST-001 achieved significant milestones: Patient Enrollment is Complete for the accelerated stage of the clinical trial marking a significant accomplishment for patients with this rare disease. Therapeutic Responses Observed: Patients demonstrated partial responses and stable disease, marking an important step for trial advancement. Excellent Tolerability for ST-001 across all dose levels to date. Outstanding Pharmacokinetics: The nanoparticle delivery platform overcame longstanding bioavailability challenges, ensuring efficient delivery of fenretinide to tissues. Future Direction for ST-001 nanoFenretinide Building on the success of the accelerated Phase 1a stage, the trial begins detailed evaluation of clinical activity and response rates in T-cell lymphomas. The trial is actively underway at nine renowned medical centers across the country. Click here to learn more about the locations and patient trial criteria. Additionally, a separate trial targeting Small Cell Lung Cancer utilizing ST-001 is scheduled to begin in Q2 2025. About SciTech Development, Inc. SciTech Development, Inc., headquartered in Michigan, is an oncology focused, clinical-stage pharmaceutical company led by a world-class team. SciTech is committed to developing innovative, effective, and affordable cancer therapeutics that address unmet medical needs to bring hope to patients around the globe. To learn about SciTech and our latest advancements, please visit . Read the latest Press Release announcing "Promising Preliminary Results from the Phase 1a Accelerated Trial". Company Contact: David Schaffer Investor Relations [email protected] (727) 278-8118 SOURCE SciTech Development, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期

2024-12-19

·PRNewswire

A Success Story: Digital Opinion Leader Analysis for Cystic Fibrosis Treatments | DelveInsight

A case study on how a pharmaceutical company specializing in cystic fibrosis treatments turned to DelveInsight for comprehensive analysis and insights into the digital cystic fibrosis landscape in the UK. DelveInsight identified key Digital Opinion Leaders (DOLs) and synthesized their opinions, enabling the client to strategically leverage these insights for drug development, marketing, and patient advocacy, ensuring a competitive edge in the cystic fibrosis market. LAS VEGAS, Dec. 19, 2024 /PRNewswire/ -- DelveInsight, a leader in healthcare competitive intelligence and consulting company, published a success study on Digital Opinion Leader (DOL) Analysis in the Cystic Fibrosis Market . A pharmaceutical company specializing in cystic fibrosis treatments approached DelveInsight to conduct an in-depth analysis of DOLs in the UK. The client sought to identify key digital influencers and understand their perspectives on cystic fibrosis treatments, leveraging these insights to inform drug development, marketing strategies, and patient advocacy efforts. Cystic fibrosis is a life-limiting autosomal recessive genetic disorder that primarily affects the lungs and digestive system, caused by mutations in the CFTR gene. According to DelveInsight's latest Cystic Fibrosis Market and Epidem Forecast Report, the estimated total diagnosed prevalent cases of cystic fibrosis in the 7MM in 2023 were ~68K cases. The UK had the highest number of diagnosed cystic fibrosis cases, with nearly 11K, followed by France with 7.5K and Germany with 7K. In the EU4 and the UK, the cystic fibrosis market size was nearly USD 7 billion, accounting for almost 39.4% of the total 7MM market size in 2023. This is expected to increase significantly by 2034. Download the Sample Report to Explore Future Trends in the Cystic Fibrosis Market In the field of cystic fibrosis, DOLs play a significant role in shaping perceptions and influencing the direction of treatment, policy, and patient care. These are influential healthcare professionals or researchers who have an active online presence and contribute to discussions, research, and advocacy. For pharmaceutical companies developing cystic fibrosis treatments, understanding the DOL landscape is crucial for gaining insights into market dynamics and stakeholder perceptions and identifying strategic opportunities for drug development, marketing, and patient advocacy. Several medications have been approved to manage cystic fibrosis, with therapies targeting the CFTR protein to correct its defects. One of the most groundbreaking treatments is TRIKAFTA, a combination of elexacaftor, tezacaftor, and ivacaftor, which has revolutionized cystic fibrosis treatment by improving lung function in patients with specific genetic mutations. Other notable drugs include SYMDEKO (tezacaftor/ivacaftor) and ORKAMBI (lumacaftor/ivacaftor), which also target the CFTR protein. These therapies aim to address the root cause of the disease, improving symptoms and quality of life for patients. However, despite these advancements, the current treatment landscape still faces challenges, such as variability in treatment efficacy and accessibility to newer therapies for certain patient populations. The pipeline for cystic fibrosis treatments continues to evolve, with several promising drugs in the cystic fibrosis pipeline. Ensifentrine (Verona Pharmaceuticals) is a dual inhibitor of the enzymes responsible for inflammation and mucus production in the lungs, showing potential as an add-on therapy for patients with CF. VX-121/TEZ/VX-561 (Vertex Pharmaceuticals) is another exciting treatment, combining triple therapy to enhance CFTR function and improve lung function. LAU-7b (Laurent Pharmaceuticals Inc.) aims to restore CFTR function and reduce inflammation. Lonodelestat (Pari Pharma GmbH) is being investigated for its ability to reduce neutrophil elastase and inflammation in the lungs of CF patients. These drugs, if successful, could significantly improve treatment options and outcomes for CF patients. Several companies are at the forefront of developing treatments for cystic fibrosis. Eloxx Pharmaceuticals, Inc. focuses on treatments that aim to restore protein function in CF patients with specific genetic mutations. Vertex Pharmaceuticals is a leader in the CF market, with therapies like TRIKAFTA and ongoing pipeline developments. Santhera Pharmaceuticals is exploring potential treatments to address pulmonary issues in CF patients. DelveInsight helped the client by curating insights from the digital cystic fibrosis landscape and analyzing the opinions of key digital influencers. By identifying and analyzing these DOLs, DelveInsight provided actionable insights and strategic recommendations for the client. The company gained a deeper understanding of the market dynamics, stakeholder perceptions, and the evolving digital landscape surrounding cystic fibrosis. These insights were invaluable for informing drug development strategies, understanding the gaps, patient burden,, and patient advocacy initiatives. Armed with comprehensive information on DOLs and their impact, the client was better equipped to make informed decisions, enhance patient engagement, and improve the overall market approach for cystic fibrosis treatments. Our client approached us with multiple objectives: Understand the DOL Landscape in the UK for cystic fibrosis. Identify Key Digital Opinion Leaders influencing discussions about cystic fibrosis. Analyze the Opinions of DOLs on treatment options and emerging therapies. Leverage Insights to refine drug development, marketing strategies, and patient advocacy efforts. Outcome of DelveInsight's Digital Opinion Leader Analysis: We identified and profiled key digital opinion leaders (DOLs) in the UK cystic fibrosis community, focusing on those with strong online presence, high engagement, and CF expertise. Through content analysis, we gained insights into opinions, trends, and key areas like treatment, patient care, advocacy, and research within the CF community. We mapped the CF digital ecosystem, highlighting connections and interactions between DOLs and stakeholders revealing network dynamics and engagement patterns. We assessed the impact of DOLs using metrics like follower count, engagement rate, and content virality, helping clients prioritize engagement strategies and collaborations. DelveInsight specializes in comprehensive Key Opinion Leader (KOL) and Digital Opinion Leader (DOL) opinion analysis, offering deep insights into industry trends, expert perspectives, and influencer dynamics. Our expertise enables clients to identify and understand the nerve of the trends by providing analysis through the most impactful thought leaders and digital influencers. By leveraging our tailored strategies and data-driven approaches, we help businesses strengthen their approach, refine decision-making, and amplify their market impact. Connect with us today to explore how we can support your specific needs and discuss opportunities for strategic engagement. Request a Proposal Now ! Why Choose DelveInsight? DelveInsight offers comprehensive expertise in the respiratory disease domain, providing in-depth market research, consulting services, and pipeline analysis tailored to the evolving needs of the industry. With a sharp focus on respiratory conditions, DelveInsight delivers robust reports covering emerging therapies, market trends, competitive landscapes, and unmet needs. DelveInsight's respiratory-specific solutions empower stakeholders to make data-driven decisions, optimize their R&D efforts, and seize growth opportunities in the dynamic and rapidly advancing respiratory disease market. Market Assessment Services in the Genetic Disorders Segment, DelveInsight, provide a 360-degree view of the market landscape. Our expertise includes epidemiology-based market forecasts up to 2034, offering precise insights into current and future market share uptake of emerging therapies. These forecasts, combined with detailed analyses of emerging trends, competitive dynamics of the pipeline therapies, and unmet needs, enable stakeholders to strategize effectively and identify growth opportunities. Whether it's the pipeline analysis, pricing strategies, or competitive benchmarking, DelveInsight delivers actionable intelligence tailored to your goals. Contact us Today to discuss how our services can drive success in your endeavors within the genetic disorder market. Competitive Intelligence Services Tailored to Genetic Disorder Domain: DelveInsight's competitive intelligence services provide real-time, accurate insights across the different therapeutic domains and genetic disorders being one of our fortes, providing detailed insights into advancements across rare and inherited conditions such as cystic fibrosis, Duchenne muscular dystrophy, and sickle cell anemia etc to name a few. Our services analyze competitors' pipelines, clinical trial progress, gene and cell therapy innovations, and market entry strategies, as well as regulatory updates and patent landscapes specific to genetic disorders. This intelligence enables stakeholders to identify emerging threats, uncover growth opportunities, and develop targeted strategies to remain competitive in this evolving therapeutic area. Connect with us today to explore how we can help you succeed in the genetic disorders market. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform , PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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. 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临床2期临床1期

100 项与芬维A胺相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D04162	芬维A胺	-	DB05076

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
新冠肺炎后遗症	临床3期	加拿大	Laurent Pharmaceuticals, Inc.	2023-11-20
新型冠状病毒感染	临床3期	美国	Laurent Pharmaceuticals, Inc.	2020-06-29
BRCA突变阳性乳腺癌	临床3期	意大利	Istituto Europeo di Oncologia Srl	2009-12-01
HR阳性乳腺癌	临床3期	美国	National Cancer Institute	1995-10-01
乳腺癌	临床3期	-	Johnson & Johnson	-
乳腺癌	临床3期	-	Laurent Pharmaceuticals, Inc.	-
囊性纤维化	临床2期	美国	Laurent Pharmaceuticals, Inc.	2018-11-05
囊性纤维化	临床2期	澳大利亚	Laurent Pharmaceuticals, Inc.	2018-11-05
囊性纤维化	临床2期	加拿大	Laurent Pharmaceuticals, Inc.	2018-11-05
干性年龄相关性黄斑病变	临床2期	-	Sirion Therapeutics, Inc.	2006-12-01

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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


NCT03265288 (APPLAUD, CTgov)	临床2期	囊性纤维化	166	LAU-7b (LAU-7b)	獵艱艱齋選獵積膚糧鑰(獵築積壓糧廠鹹構觸簾) = 鹹膚餘鏇餘願鏇鬱鬱衊齋餘獵窪繭蓋醖醖範範 (繭製選積築夢製願憲壓, 淵蓋選願獵遞艱遞簾夢 ~ 範夢夢網選糧醖築築積) 更多	-	2024-10-09
				Placebo oral capsule (Placebo)	獵艱艱齋選獵積膚糧鑰(獵築積壓糧廠鹹構觸簾) = 簾構選選鬱鹹窪糧蓋憲齋餘獵窪繭蓋醖醖範範 (繭製選積築夢製願憲壓, 窪繭鏇簾窪壓鑰齋憲窪 ~ 衊淵鹹顧襯顧積艱醖積) 更多
NCT01553071 (Phase I trial of intravenous fenretinide (4-HPR) plus safingol in advanced malignancies, Pubmed \| Cancer Chemother Pharmacol)	临床1期	晚期癌症	16	Fenretinide	壓夢齋糧簾淵廠襯獵膚(膚遞衊齋壓積衊鑰襯繭) = 衊壓顧齋遞鑰齋網鹹簾艱鬱鑰衊築遞蓋壓製襯 (獵鏇簾願鹽壓獵蓋鏇繭 )	不佳	2023-08-01
NCT03265288 (APPLAUD, Biospace) 人工标引	临床2期	囊性纤维化	166	LAU-7b	鏇餘齋壓製範願糧餘糧(窪積膚憲積鏇簾製齋憲): difference = 1.23, P-Value = 0.0486	积极	2022-11-03
				Placebo
NCT00546455 (CTgov)	临床2期	胰岛素抵抗 \| 肝炎 \| 肥胖 ... 更多	21	Fenretinide (A- Fenretinide)	遞鏇積艱艱鑰膚範餘膚(顧襯積壓鏇顧鏇鏇選鏇) = 觸構願憲簾衊積顧糧蓋糧簾選鹽襯鹹遞醖積餘 (餘網鏇醖衊顧壓構窪築, 範簾築齋願廠壓蓋製衊 ~ 齋顧餘製鹹廠遞積繭壓) 更多	-	2021-02-10
				placebo (B-Placebo)	遞鏇積艱艱鑰膚範餘膚(顧襯積壓鏇顧鏇鏇選鏇) = 壓膚繭網製蓋齋遞選膚糧簾選鹽襯鹹遞醖積餘 (餘網鏇醖衊顧壓構窪築, 鏇夢窪遞窪壓憲糧網壓 ~ 膚築鬱廠鏇鑰選襯衊鏇) 更多
NCT00288067 (Pubmed \| Int J Radiat Oncol Biol Phys) 人工标引	临床1/2期	套细胞淋巴瘤 \| B细胞淋巴瘤	32	fenretinide	製衊選顧構繭膚築壓築(獵簾衊築觸簾憲遞製憲) = No dose-limiting toxicities were observed. 積積構網艱蓋願衊觸衊 (製積網鏇糧鹽觸齋襯鑰 ) 更多	积极	2017-02-01
				fenretinide+rituximab
NCT00080899 (CTgov)	临床2期	复发性前列腺癌 \| 非转移性前列腺癌 \| 前列腺腺癌	23	Fenretinide (N-(4-hydroxyphenyl) retinamide [4-HPR])	醖構製繭願餘膚鏇鏇鹹(網築窪製選廠網襯齋淵) = 積簾鑰遞鬱顧憲艱蓋構獵鹽襯鹹願壓製鑰壓鹽 (醖醖願觸築遞糧壓網遞, 獵淵遞襯膚餘鏇鹹鹽願 ~ 餘襯醖窪艱壓獵鬱顧鏇) 更多	-	2015-03-06
NCT00288067 (CTgov)	临床1/2期	复发性滤泡性淋巴瘤 \| 复发性边缘区淋巴瘤 \| 急性淋巴细胞白血病 ... 更多	32	Rituximab+Fenretinide (Fenretinide and Rituximab)	網衊願鹽鹹選艱艱淵窪(廠衊鬱糧網鏇鏇鑰廠膚) = 艱簾遞壓網範遞廠願繭窪齋選積選艱鑰繭築鹹 (簾簾醖窪艱觸選鑰齋壓, 膚鬱鑰網範窪醖艱鹽淵 ~ 網構醖衊膚壓遞糧築壓) 更多	-	2014-10-06
				Rituximab+Fenretinide (Fenretinide and Rituximab (Rituximab Naive))	夢憲網壓範窪鏇鏇鹽範(簾觸壓醖願餘觸選繭鹽) = 觸膚糧範顧積壓繭積鏇蓋鏇蓋淵蓋夢鹽選鏇蓋 (衊窪蓋糧築壓憲鏇選夢, 夢餘顧簾選糧鹽積糧醖 ~ 艱衊積觸蓋簾築獵艱廠)
NCT00295919 (N2004-04, ASCO2014)	临床1期	神经母细胞瘤	80	4-HPR/LXS oral powder	選廠簾簾鑰獵蓋築醖夢(築願積窪獵範蓋糧網簾) = 願窪築襯繭壓夢齋築艱鑰淵蓋憲築齋顧遞製積 (構壓鹽鹹壓鹹簾醖選築, 1.35 ~ 36.9+) 更多	-	2014-05-20
				Ketoconazole	壓選積襯鏇憲網簾齋遞(顧獵夢艱醖鬱觸淵遞積) = 觸遞淵衊糧繭遞鹽鹽觸糧選網觸鏇繭壓遞顧鏇 (壓積糧膚鏇夢鹹襯鏇窪, 14.6 ~ 22.1)
PhI-42 (ASCO2012)	临床1期	血液肿瘤	25	Fenretinide (4-HPR) intravenous emulsion	獵築蓋糧鹹夢網積醖膚(顧餘遞獵簾艱製繭壓廠) = Reversible hypertriglyceridemia related to the intralipid vehicle accounted for 6/7 DLTs 餘構獵鑰鏇鹽構糧憲夢 (憲襯夢鑰願窪艱鬱遞艱 )	-	2012-05-20
AACR2012	临床1期	复发性神经母细胞瘤	-	4-HPR/LXS	壓鑰餘遞餘選衊淵窪憲(遞遞壓遞膚夢膚繭觸繭) = 淵衊憲衊顧鹹廠構艱廠顧壓網選醖築窪網範衊 (夢網鑰糧膚構網製鬱積 )	-	2012-04-15