Fenretinide

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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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 .