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 is to learn about a single dose of fenretinide in healthy volunteers, in both a fasted and fed state. The main questions it aims to answer are: •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. Researchers 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.

NCT04417257 / Completed临床2/3期

RESOLUTION: A Double-blind, Randomized, Placebo-controlled, Phase II/III Study of the Efficacy and Safety of LAU-7b in the Treatment of Adult Hospitalized Patients With COVID-19 Disease

A randomized, double-blind, placebo-controlled Phase 2/3 Study of LAU-7b against confirmed COVID-19 Disease in hospitalized patients at a higher risk of complications.

开始日期2020-06-29

申办/合作机构

Laurent Pharmaceuticals, Inc.

100 项与芬维A胺相关的临床结果

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100 项与芬维A胺相关的转化医学

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100 项与芬维A胺相关的专利（医药）

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项与芬维A胺相关的文献（医药）

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 ; Li, Ran ; Ke Li

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.

2024-11-01·ANTICANCER RESEARCH

Immunoliposomes for Neuroblastoma: Review of the Past Experience and Design of a Novel Nanoparticle

Review

作者: Panosyan, William S ; Panosyan, Daniel E

BACKGROUND/AIM:

High-risk/refractory neuro-blastoma (NBL) treatments include anti-GD2-monoclonal antibodies (mAbs). Several immunoliposomes (ILs) covered with anti-GD2-mAbs (GD2-ILs) have been tested pre-clinically. We aimed to review literature on GD2-IL for characteristics of nanoparticles/payloads, conjugation of mAb/fragments and preclinical data, as well as to explore the feasibility of a recently proposed GD2-IL loaded with the antimetabolite oxamate.

MATERIALS AND METHODS:

Initial PubMed search was generalized for immunoliposomes in cancer. Further search was focused on papers for GD2-IL [keywords: "Immunoliposomes and cancer (or neuroblastoma)"].

RESULTS:

There were 811 results on "immunoliposomes"; >50% were on "immunoliposomes, cancer" (n=439, June 2024). Seventeen items resulted from "immunoliposomes, neuroblastoma" (one was "publishers' correction"). Sixteen GD2-IL references were reviewed (1993-current). The mean±SD GD2-ILs size was 124.8±31 nm (range=86-171). Six papers described GD2-ILs with DNA-damaging agents [doxorubicin (n=4), etoposide (n=1), irinotecan+HDAC inhibitor (n=1)]. Other payloads included: fenretinide (n=4 papers), C-myb antisense (n=2), survivin inhibitor (n=1), tyrosine kinase inhibitor (n=1), IL15 (n=1), and oxamate (n=1). These 9 drug-loads included both hydrophilic and hydrophobic molecules. Except for IL15 and C-myb antisense with high molecular weights (MWs), and oxamate with low MW, the remaining compounds had comparable MWs (496±100 g/mol, range=349-588.6). The overall encapsulation efficiency was 66.2±25.6%. There were 17-30 mAb molecules attached to an IL with PEGylation. Experiments with GD2-positive/GD2-negative cells demonstrated selective efficacy/tropism of GD2-ILs. Mouse models confirmed efficacy, GD2-specific tumor accumulation, decreased toxicity, and improved pharmacokinetic-pharmacodynamics.

CONCLUSION:

PEGylated anti-GD2-IL may allow NBL tropism. A size of approximately 100 nm could allow vascular permeability and packaging of oxamate in amounts needed for profound/selective lactate dehydrogenase-A inhibition. Thus, oxamate-loaded GD2-ILs may allow exploring the great translational potential of Warburg effect inhibition in GD2-positive cancers.

2024-10-02·Journal of chemotherapy (Florence, Italy)

Prognostic and immunological values of SKA3 for overall survival in lung adenocarcinoma and its RNA binding protein involved mechanisms

Article

作者: Li, Jinjin ; Gu, Yinfeng ; Guan, Hongjun ; Sun, Changpeng

This article aimed to investigate the correlations among SKA3 expression and prognosis, clinical relevance, tumor immunity, and RNA-binding protein (RBP)-involved mechanisms for overall survival (OS) in lung adenocarcinoma (LUAD). To explore the SKA3 expression level in LUAD by analyzing the genomic data as well as related clinical characteristics from the database of TCGA. Nomogram and gene set enrichment analysis (GSEA) were applied, respectively, to evaluate the performance of SKA3 in LUAD. Correlations between SKA3 and immunity and RBP-involved mechanisms were also performed. SKA3 had a higher expression level in LUAD samples than in adjacent normal lung samples, with shorter survival times in the high-SKA3-expressed LUAD subgroup (P < 0.05). qRT-PCR results remained consistent (P < 0.05). Uni-/multivariate Cox analyses revealed that SKA3 could have independent prognostic ability for LUAD (both P < 0.05). The nomogram model constructed with clinical pathological parameters and SKA3 expression levels predicted OS rates for LUAD and GSEA revealed SKA3-related pathways. In aspects of tumor immunity, SKA3 was significantly involved with tumor neoantigen burden, tumor mutational burden, immune cell pathways, and immune checkpoint inhibitor (ICI) molecules (all P < 0.05). The CellMiner database also found significant correlations between SKA3 and the antitumor drug sensitivity of chemotherapy, fenretinide, and PX-316. Besides, a total of nine LncRNA/RBP/SKA3 networks were revealed in LUAD for their RBP-involved mechanisms. SKA3 could serve as a potential biomarker for OS prognosis and immunotherapy in LUAD. LncRNA/RBP/SKA3 networks were identified in LUAD for their RBP-involved mechanisms, paving the way for further experimental verifications.

项与芬维A胺相关的新闻（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

2022-11-03

·BioSpace

Laurent Pharmaceuticals Presents Results from Phase 2 Clinical Study of LAU-7b in Cystic Fibrosis at the NACFC 2022

LAU-7b first inflammation-controlling drug to demonstrate clinical benefit in adults with CF, complementary to administration of CFTR modulators Study achieves clinically meaningful reduction in lung function loss at 6 months, measured as absolute change in ppFEV1, reaching statistical significance in the Per Protocol population MONTREAL--(BUSINESS WIRE)-- Laurent Pharmaceuticals Inc. (the “Company”), a clinical-stage biopharmaceutical company developing LAU-7b (oral fenretinide) as a first-in-class inflammation-controlling drug candidate for Cystic Fibrosis (CF), announces the presentation of results from its placebo-controlled Phase 2 APPLAUD 24-week clinical study in adults with CF at the North American Cystic Fibrosis Conference (NACFC) currently held in Philadelphia, United States. Study results achieved a statistically significant treatment difference in favour of LAU-7b in the absolute change in the percent predicted forced expiratory volume in one second (ppFEV1), the primary variable, through 24 weeks (1.23 percentage points; p=0.0486) in people that completed at least five months of treatment (Per Protocol population, N=122), and a clinically meaningful reduction of 49% in the loss of lung function at 24 weeks, relative to placebo. Statistically significant clinical benefit was also observed in the relative change in ppFEV1 through 24 weeks (2.34 percentage points; p=0.0351), as well as in the plasma levels of C-reactive protein (p=0.029), an important marker of systemic inflammation. Pre-specified subgroup analysis suggests a higher benefit in subjects with ppFEV1 greater than 70% at baseline, and the effect appears to be maintained in the subgroup of subjects already receiving CFTR modulators, including TRIKAFTA®. The results also passed robustness analyses, despite heterogeneous study population and pandemic interference. The study confirmed that LAU-7b has a predictable and acceptable safety profile, with no unexpected serious adverse events reported. “The potential clinical benefits of reducing the loss of lung function demonstrated with LAU-7b in the APPLAUD study, especially when administrated on top of CFTR modulators, are very promising and represent a first with a drug candidate addressing inflammation in adults with CF,” said Michael Konstan, MD, Vice Dean for Translational Research at Case Western Reserve University School of Medicine in Cleveland, and Co-Lead Investigator for the study. “It is really exciting to see this significant progress toward bringing medicines targeting loss of lung function in people with CF, an important and rather neglected aspect of this disease,” added Larry Lands, MD, PhD, Director, Pediatric Respiratory Medicine at McGill University Health Centre in Montreal, and Co-Lead Investigator for the study. “These results showed that the proposed LAU-7b treatment approach is complementary to CFTR modulation, confirming earlier preclinical research by McGill researchers demonstrating LAU-7b’s potential to rescue and preserve CFTR modulator’s effect during cellular stress.” “These results underscore the important clinical benefit potential of LAU-7b and Laurent’s commitment to delivering the next scientific breakthrough in this disease,” said Radu Pislariu, MD, President and CEO of Laurent Pharmaceuticals. “We want to thank all study participants and their families who put their trust in us, the staff at the clinical sites, the Cystic Fibrosis Foundation, and all our collaborators for their support and partnership throughout this study, conducted in unprecedented pandemic conditions.” The key outcomes from the study are presented in NACFC Poster #168, and were selected for oral presentation as part of the Session W21 (Evidence Based Medicine - Update on Clinical Trials in CF). About the Phase 2 APPLAUD clinical study APPLAUD was a double-blind, placebo-controlled study in adults with CF, randomized (1:1) to LAU-7b or placebo for 6 months. The goal of the study was to evaluate the safety of LAU-7b and its effect on preservation of lung function by reducing inflammation. Eligible subjects had ppFEV1 between 40% and 100% at screening and had had at least one pulmonary exacerbation (PEx) in the prior year. The study drug was administered orally once daily, in addition to standard of care, including all commercially available CFTR modulators. The primary efficacy endpoint was the absolute change from baseline in ppFEV1 at 24 weeks. Secondary endpoints included parameters related to PEx, systemic inflammatory and lipidomic biomarkers, and quality of life. Study randomized 166 adults with CF at 40 sites in the US, Canada and Australia, of which 122 completed a minimum five months of study medication (Per Protocol population). Both arms were very comparable in terms of age, BMI, gender distribution and lung function at baseline. The safety and efficacy pro this study supports the use of ppFEV1 as the primary efficacy variable for confirmatory studies, with longer treatment duration. About Cystic Fibrosis Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people worldwide. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene and is characterized by viscous secretions in different exocrine tissues, and an aberrant inflammatory response leading to chronic pulmonary infection and loss in lung function over time. Severe pulmonary dysfunction is the primary cause of death in CF. About LAU-7b LAU-7b (oral fenretinide) is a novel drug candidate that acts on cell membrane lipids to modulate inflammation signaling and protein trafficking. Membrane lipid imbalance is believed to play a role in aberrant pulmonary inflammation in individuals with CF, leading to irreversible lung damage over time. Fenretinide was shown to correct the levels of certain membrane phospholipids and sphingolipids in multiple in vitro and in vivo models of CF, improving resolution of inflammation and the stabilization of CFTR in the epithelial apical membrane during inflammatory stress, a self-protective mechanism complementary to CFTR modulators. About Laurent Pharmaceuticals Laurent Pharmaceuticals is a clinical stage biotech company focusing on the modulation of cell membrane lipids for addressing life-threatening diseases. The company’s lead drug candidate, LAU-7b, is a proprietary oral formulation of fenretinide, an atypical retinoid with a well-documented safety pro in more than 3,000 patients. Existing data suggest that low-dose fenretinide has the potential to modulate certain membrane lipids and exerts antiviral, inflammatory-controlling, and antifibrotic properties. LAU-7b is currently being evaluated as a potential therapeutic for COVID-19 and Cystic Fibrosis. For more information, please visit .

First in Class

2021-10-01

·BioSpace

Merck COVID-19 Antiviral Cuts Death, Hospitalizations in Half

Kena Betancur/Getty Images Interim results from the Phase III trial of a potential treatment for mild-to-moderate COVID-19 symptoms by Merck and Ridgeback Biotherapeutics showed promise in preventing hospitalizations and death. Initial analysis from the Phase III MOVe-OUT study on investigational oral antiviral drug molnupiravir (MK-4482, EIDD-2801) shows a significantly reduced risk for hospitalization and death in adult patients by about 50%. Through Day 29, only 7.3% of the participants were either hospitalized or died, compared to the 14.1% who received a placebo. No deaths were reported in the molnupiravir group, as opposed to the eight in the placebo group. The results were based on data from 775 patients enrolled in the trial. To qualify for the study, the patients had to have laboratory-confirmed mild-to-moderate COVID-19, with symptom onset within five days of the study randomization. All patients were required to have at least one risk factor linked with poor disease outcomes. Aside from reduced hospitalization and death risks across all subgroups, the preliminary results also demonstrated consistent efficacy against viral variants Delta, Gamma, and Mu. With these results, an independent Data Monitoring Committee sought the approval of the U.S. Food and Drug Administration (FDA) to halt further recruitment into the trial. The next step now would be for Merck to submit an Emergency Use Authorization application to the FDA and marketing applications to other regulators worldwide. "More tools and treatments are urgently needed to fight the COVID-19 pandemic, which has become a leading cause of death and continues to profoundly affect patients, families, and societies and strain health care systems all around the world. With these compelling results, we are optimistic that molnupiravir can become an important medicine as part of the global effort to fight the pandemic and will add to Merck's unique legacy of bringing forward breakthroughs in infectious diseases when they are needed most," said Robert M. Davis, chief executive officer and president of Merck, in a statement. In fact, because of its high confidence in molnupiravir's potential, Merck has been producing the drug at risk. The company expects to manufacture 10 million courses of treatment by the end of this year, with more doses to be produced in 2022. Merck also signed a procurement agreement with the U.S. Government early this year, which allows it to supply some 1.7 million courses of the drug to the government upon EUA or the FDA's approval. It has also entered into supply and purchase deals with other governments worldwide. Another notable move to demonstrate Merck's commitment to making molnupiravir accessible to everyone is a non-exclusive voluntary licensing agreement with generic manufacturers to distribute the drug in over 100 low- and middle-income countries (LMICs). All these are contingent on local regulatory EUAs and approvals. Laurent’s Oral COVID-19 Drug Could Help Moderate-to-Severe Patients Topline results from the Phase II RESOLUTION trial of Laurent Pharmaceuticals’ once-daily oral COVID-19 drug, LAU-7b, showed effectiveness against death and disease progression in moderate-to-critical patients, though it failed to achieve its overall primary endpoint where the total involved those who were critically ill. The trial tested the efficacy and safety of LAU-7b for 14 days in a total of 232 hospitalized patients,148 of whom had moderate-to-severe disease that did not require oxygen and 84 who were critically ill, requiring high-flow oxygen but not mechanical ventilation. While the drug failed to reach its primary endpoint of improving the total percentage of patients kept alive and free of respiratory failure by Day 29 – the numbers were 69.2% in the treatment group versus 72.2% in the placebo – LAU-7b (plus standard of care) demonstrated a 100% reduction in risk of all causes of death and progression to mechanical ventilation in moderate-to-severe patients by Day 60. None of those who were treated with LAU-7b plus SoC died or required a machine, compared to the placebo group where four died and five progressed to mechanical ventilation. Although the overall outcome is a setback for Laurent, the company said it would focus its future development on patients with moderate-to-severe disease instead, as well as the key endpoints that delivered significant benefits. “The results from the moderate-to-severe subgroup are very promising, suggestive of a potential clinical benefit of LAU-7b in COVID-19 patients that are not yet in respiratory failure, which also represents the patient population with the fewest treatment options. An oral therapeutic that prevents serious COVID-19 illness and death, also allowing patients to continue the treatment at home after discharge from hospital, would be a powerful tool in the therapeutic arsenal against COVID-19," commented Laurent President and CEO Dr. Radu Pislariu, M.D. LAU-7b is an oral form of fenretinide that was initially developed for its potential to trigger the resolution phase of inflammation, a natural mechanism that controls inflammatory response, without inducing immune suppression. Fenretinide was recently shown to have antiviral properties against SARS-CoV-2 in vitro, especially when combined with remdesivir. In addition to its COVID-19 links, LAU-7b is also being evaluated as a potential treatment for cystic fibrosis.

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D04162	芬维A胺	-	DB05076

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
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)	膚襯襯鹽簾淵膚衊鏇網(鹹壓獵齋遞願鏇遞築窪) = 獵遞齋糧鹹窪觸獵窪廠膚選壓構觸鹹網簾襯鬱 (窪鬱夢顧衊鑰蓋積窪夢, 鏇鑰鏇醖糧範築醖鏇憲 ~ 願製醖廠願糧廠鹹獵夢) 更多	-	2024-10-09
				Placebo oral capsule (Placebo)	膚襯襯鹽簾淵膚衊鏇網(鹹壓獵齋遞願鏇遞築窪) = 蓋襯築鹽廠獵壓淵糧網膚選壓構觸鹹網簾襯鬱 (窪鬱夢顧衊鑰蓋積窪夢, 齋廠糧齋築襯網簾觸鬱 ~ 鏇鏇選艱窪鹽蓋鑰簾壓) 更多
NCT03265288 (APPLAUD, Biospace) 人工标引	临床2期	囊性纤维化	166	LAU-7b	鬱憲築窪範製艱觸遞鏇(選選願鏇範淵衊鹹積鹽): 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)	壓鹽觸鏇簾廠製製鬱餘(簾觸憲網築製鑰淵遞鹽) = 醖餘鬱夢壓窪積鏇醖願齋願鹹觸衊衊膚餘簾製 (衊蓋選襯觸積醖積艱繭, 鹹蓋餘網襯鏇淵構蓋鑰 ~ 鬱願鹽願襯淵顧鑰糧獵) 更多	-	2021-02-10
				placebo (B-Placebo)	壓鹽觸鏇簾廠製製鬱餘(簾觸憲網築製鑰淵遞鹽) = 襯衊襯簾積獵簾窪壓齋齋願鹹觸衊衊膚餘簾製 (衊蓋選襯觸積醖積艱繭, 壓築夢壓鹹範淵獵選窪 ~ 憲窪築範顧衊餘網鏇構) 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	獵鏇襯餘遞構夢膚齋構(築鏇壓鹽憲衊淵淵鏇鑰) = No dose-limiting toxicities were observed. 醖糧築繭選醖網夢簾壓 (襯鏇憲築鹹顧齋構遞鏇 ) 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])	壓獵鹽齋餘齋齋窪壓醖(簾鹹餘積鑰淵築膚顧積) = 網構選顧鏇廠齋繭衊鹹顧蓋鏇鹹廠網蓋構淵積 (廠齋製鹽獵簾膚積網憲, 廠鑰鏇鹹廠廠壓網願夢 ~ 廠簾鑰遞簾網鑰壓願糧) 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)	窪衊夢夢憲範憲製鹽積(構鏇繭積鏇廠簾築築鹹) = 選構艱積廠壓鹽襯餘艱獵蓋膚顧構顧蓋顧鏇願 (鏇餘憲蓋願範蓋夢艱鏇, 願壓齋餘觸窪鹹鬱繭遞 ~ 鹹鹹廠鬱憲鑰築遞簾顧) 更多	-	2014-10-06
				Rituximab+Fenretinide (Fenretinide and Rituximab (Rituximab Naive))	願積鹹製醖簾醖醖窪齋(願蓋醖繭遞壓積醖構鹹) = 襯簾鑰鹽艱齋築糧蓋鑰構繭觸廠鬱蓋鑰觸選顧 (鑰憲鹽窪願襯簾繭蓋築, 憲夢膚廠願簾廠衊壓願 ~ 遞鏇糧顧鹽範簾製範願)
NCT00295919 (N2004-04, ASCO2014)	临床1期	神经母细胞瘤	80	4-HPR/LXS oral powder	膚膚衊選齋窪窪遞獵廠(蓋觸憲艱繭構築醖積獵) = 觸積繭鬱蓋襯蓋窪構範鏇艱襯範簾積糧襯繭齋 (構獵選簾遞積鑰範鹽窪, 1.35 ~ 36.9+) 更多	-	2014-05-20
				Ketoconazole	遞顧顧窪遞壓夢壓衊積(構選鹹簾顧蓋憲淵積遞) = 製觸襯範廠製壓顧衊繭糧簾壓醖獵膚顧鏇糧壓 (憲鏇範壓蓋襯願夢壓膚, 14.6 ~ 22.1)
PhI-42 (ASCO2012)	临床1期	血液肿瘤	25	Fenretinide (4-HPR) intravenous emulsion	網築簾選膚餘簾糧築繭(範膚觸餘範艱夢壓襯壓) = Reversible hypertriglyceridemia related to the intralipid vehicle accounted for 6/7 DLTs 獵壓壓製網蓋繭獵衊廠 (獵遞獵餘憲夢構鬱願積 )	-	2012-05-20
AACR2012	临床1期	复发性神经母细胞瘤	-	4-HPR/LXS	糧積蓋選積鹽顧顧淵簾(齋憲鏇壓構艱構願糧鑰) = 顧鑰衊鹹夢網鏇膚範憲夢製憲願餘憲顧鬱膚選 (餘鏇簾鏇簾鹽廠構鑰衊 )	-	2012-04-15
ARVO2011	N/A	视网膜变性	-	Brimonidine	構鹹鏇膚襯選醖鹽鏇窪(蓋淵積築蓋鑰憲窪觸遞) = 鏇糧遞製範築襯艱鑰膚獵鑰範糧遞網範淵選繭 (觸膚構夢窪簾窪蓋壓積 ) 更多	-	2011-04-01
				Fenretinide	構鹹鏇膚襯選醖鹽鏇窪(蓋淵積築蓋鑰憲窪觸遞) = 製鏇觸膚廠觸壓鹽構壓獵鑰範糧遞網範淵選繭 (觸膚構夢窪簾窪蓋壓積 ) 更多