lumacaftor (VX-809) News

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|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
Journal: Trikafta restores thermodynamic coupling between two nucleotide binding domains for potentiating CFTR activity. (Pubmed Central) - Jan 11, 2026
The results demonstrated that comparable thermostability between dimerized NBD1 and NBD2 was required to stabilize an activated intermediate for the channel activity potentiation by VX-770. Thus, tight coupling between dimerized NBD1 and NBD2 upon a global induced fit across the interdomain interfaces is still required for Trikafta modulators to rescue the gating defect of the F508del mutant.

|||||||||| Journal: Barriers to the Pharmacologic Rescue of W1282X CFTR. (Pubmed Central) - Jan 6, 2026
Additionally, acute in vitro treatments with approved modulators VX-809 or VX-661 result in immediate potentiation of W1282X-dependent ion transport, showing that F508del CFTR correctors also augment W1282X CFTR channel activity...Clinically approved CFTR correctors VX-445, VX-121, and VX-809 elicited potentiation of G551D CFTR...Moreover, unlike other CFTR mutations such as F508del, proteasome blockade using ALLN partially rescues W1282X at the plasma membrane. These results highlight ways in which detailed mechanistic analysis and modulator profiling are needed to characterize CFTR mutations such as W1282X and that modulator function in rare variants can be quite distinct from classical findings based strictly upon F508del CFTR.

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex, tezacaftor (VX-661) / Vertex
Journal: Inflammatory response in CF airway epithelial cells: a comparative study of modulators and wild-type CFTR rescue. (Pubmed Central) - Jan 5, 2026
These findings suggest that beyond ion transport, the proper folding and structural integrity of CFTR are important for regulating inflammation, potentially through interactions with other cellular proteins involved in inflammatory pathways. This work highlights the need to develop therapeutic strategies that not only restore chloride channel function but also fully correct CFTR misfolding to better control inflammation in CF.

|||||||||| Review, Journal, IO biomarker: Exploration of the protein and pharmacological landscape of monkeypox virus treatment: from entry point to end point. (Pubmed Central) - Dec 22, 2025
This integrative framework advocates for combination therapies, personalized regimens based on clade profiling, and global collaboration to mitigate MPXV's adaptive potential. By bridging virology and pharmacology, the review charts pathways for innovative drug development to combat this zoonotic threat effectively.

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex
Journal: Developing Type II F508del-CFTR correctors with a protective effect against respiratory viruses. (Pubmed Central) - Oct 11, 2025
In addition to its biological activity, compound 3b exhibited a favorable preclinical pharmacokinetic profile in vitro. These findings collectively highlight compound 3b as a promising multitarget candidate for cystic fibrosis, providing a solid foundation for the development of a simplified CF therapy to mitigate PE.

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex
Journal: Thermodynamic Coupling between Folding Correctors and the First of Dimerized Nucleotide Binding Domains in CFTR. (Pubmed Central) - Aug 27, 2025
However, the binding of folding correctors allosterically protected the ?-subdomain from misfolding until subsequent core formation. This thermodynamic protective mechanism, unlike the chaperone-based one in cotranslational NBD1 folding, may restore posttranslational NBD1 folding for tight Mg/ATP-mediated NBD dimerization in the F508del mutation and also potentially apply to treating other cystic fibrosis patients with rare mutations.

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex, tezacaftor (VX-661) / Vertex
Journal: Structure-guided combination of novel CFTR correctors to improve the function of F508del-CFTR in airway epithelial cells. (Pubmed Central) - Aug 23, 2025
Through rational selection based on molecular docking studies and mechanisms of action, we showed that combination of compounds (7a+1b and 2a+2b) targeting distinct domains of CFTR, can additively/synergistically rescue F508del-CFTR function in both CFBE cell line and primary nasal cells. Our study demonstrated that in silico and in vitro approaches to develop and investigate the mechanism of action of novel CFTR correctors could be a tool to optimize the combination correctors therapy to synergistically rescue mutated CFTR.

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
Journal: Thermodynamic basis for CFTR activity potentiation. (Pubmed Central) - Aug 20, 2025
The results demonstrated that comparable thermostability between dimerized NBD1 and NBD2 was required to stabilize an activated intermediate for the channel activity potentiation by VX-770. Thus, a global induced fit across the interdomain interfaces upon ligand binding may optimize cooperative ligand-mediated NBD dimerization and improve the treatment of cystic fibrosis.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Targeting programmed death ligand 1 for anticancer therapy using computational drug repurposing and molecular simulations. (Pubmed Central) - Aug 7, 2025
Two of them, Lumacaftor and Vedaprofen, showed appropriate drug profiles and biological activities and stood out as highly potent binding partners of the PD-L1...While the findings are promising, they remain computational and require experimental validation to confirm biological efficacy and specificity. This study also emphasizes the role of bioinformatics approaches in drug repurposing that can help in the identification of novel anticancer agents.

|||||||||| lumacaftor (VX-809) / Vertex, Tasigna (nilotinib) / Novartis, Inhibikase
FDA event, Journal: Repurposing FDA-Approved Drugs to Target MTH1 for Anticancer Therapeutics. (Pubmed Central) - Jun 26, 2025
The findings suggest that Nilotinib could be repurposed to enhance cancer therapy, particularly in combating drug resistance through the novel mechanism of MTH1 inhibition. This approach provides new avenues for tackling chemoresistance and improving therapeutic outcomes in cancer patients.

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
PK/PD data, Review, Journal: Pharmacokinetics of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor in Special Cystic Fibrosis Populations: A Systematic Review. (Pubmed Central) - Jun 24, 2025
Knowledge gaps persist for adults with severe hepatic impairment (Child-Pugh Class C), children with CF-induced hepatic impairment, and pregnant or lactating pwCF. Future research addressing these gaps, through incorporating routine clinical data, is crucial for improving clinical guidelines and optimizing dosing regimens, thereby advancing towards evidence-based utilization of CFTR modulators.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Lumacaftor inhibits channel activity of rescued F508del cystic fibrosis transmembrane conductance regulator. (Pubmed Central) - May 27, 2025
Future research addressing these gaps, through incorporating routine clinical data, is crucial for improving clinical guidelines and optimizing dosing regimens, thereby advancing towards evidence-based utilization of CFTR modulators. Lumacaftor, the corrector of Orkambi, enhances the processing of F508del cystic fibrosis transmembrane conductance regulator (CFTR), but its impact on the channel activity of rescued F508del CFTR (rF508del) is unclear...At 37

|||||||||| Journal: Quantification of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor and their active metabolites in plasma using UHPLC-MS/MS: Doors open to the application of therapeutic drug monitoring in cystic fibrosis treatment. (Pubmed Central) - May 3, 2025
All seven components were stable in EDTA plasma for ten days in the autosampler after sample preparation and through four freeze-thaw cycles. The developed assay was applied in routine TDM analysis to investigate exposure to elexacaftor, tezacaftor, ivacaftor and their metabolites in people with CF undergoing treatment with Kaftrio

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
Review, Journal: Unraveling the Mechanism of Action, Binding Sites, and Therapeutic Advances of CFTR Modulators: A Narrative Review. (Pubmed Central) - Feb 25, 2025
Currently, four CFTR modulators are clinically approved: the potentiator ivacaftor (VX-770), either as monotherapy or in combination with the correctors lumacaftor (VX-809), tezacaftor (VX-661), and elexacaftor (VX-445). Among these, the triple combination VX-445/VX-661/VX-770 (marketed as Trikafta

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Defective Cystic Fibrosis Transmembrane Conductance Regulator Accelerates Skeletal Muscle Aging by Impairing Autophagy/Myogenesis. (Pubmed Central) - Jan 31, 2025
Age-related reduction in skeletal muscle expression of CFTR impairs autophagy and myogenesis, exacerbating skeletal muscle aging. Enhancing CFTR might be a potential treatment strategy for age-related skeletal muscle disorders.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: E-pharmacophore based virtual screening of potent lead molecules against Cystic Fibrosis: An in silico study. (Pubmed Central) - Dec 13, 2024
The compound Anguibactin (NPC41982) has been identified as a top lead that exhibits higher binding affinity and stability than the reference compound Lumacaftor, suggesting their potential to bind to the active site of the CFTR protein. These compounds could serve as starting points for the development of drug-like molecules for treating cystic fibrosis.

|||||||||| MK-3207 / Merck (MSD), lumacaftor (VX-809) / Vertex
Journal: Structure-based virtual screening and drug repurposing studies indicate potential inhibitors of bovine papillomavirus E6 oncoprotein. (Pubmed Central) - Oct 28, 2024
Our results reveal that Lumacaftor and MK-3207 are promising candidates for controlling BPV1 infection. The findings of this study may contribute to the development of E6 oncoprotein blockers in an accelerated and cost-effective manner.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Pd-Catalyzed C(sp2)-C(sp3) Suzuki Coupling and Synthesis of Lumacaftor Using Designed Monophosphine Ligands. (Pubmed Central) - Oct 9, 2024
The aryl thianthrene salt was further applied for late-stage methylation in 97% yield. The active pharmaceutical ingredient lumacaftor was synthesized by aryl-alkyl and aryl-aryl Suzuki coupling reactions using L1 and L2.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: The ER Stress Induced in Human Neuroblastoma Cells Can Be Reverted by Lumacaftor, a CFTR Corrector. (Pubmed Central) - Sep 27, 2024
Here, we evaluated the activity of Vx-809 (Lumacaftor), a drug used in cystic fibrosis, in SH-SY5Y neuronal cells, in which an ER stress condition was induced by Thapsigargin, to verify whether the drug could improve protein folding, suggesting its possible therapeutic use in proteinopathies, such as neurodegenerative diseases (NDs). Our data show that Vx-809 is involved in the significant reduction in protein produced under ER stress, particularly in the levels of Bip, ATF4, and ATF6 by Western blotting analysis, the reduction in ROS in the cytosol and mitochondria, and the reduction in the activation of the apoptotic pathway, measured by flow cytofluorimetry analysis and in restoring calcium homeostasis.

|||||||||| lumacaftor (VX-809) / Vertex
Adeno-Associated Virus 1 (AAV1) CFTR Gene Therapy Successfully Reduces Cysts in a Mouse Model of Autosomal Dominant Polycystic Kidney Disease (Exhibit Hall, Convention Center) - Sep 23, 2024 - Abstract #KIDNEYWEEK2024KIDNEY_WEEK_1506;
These experiments demonstrate convincingly that kidneys of RC/RC animals can be transduced by AAV1 and that phenotypic correction of defective function can be achieved by over expression of CFTR. Also the data suggests that CFTR plays a critical role in ADPKD.

|||||||||| Partial rescue of p.Phe08del-CFTR trafficking and stability defects by dual and triple corrector combinations () - Sep 4, 2024 - Abstract #NACFC2024NACFC_1013;
The lack of additivity of PTI-801 with VX-445 suggests that these compounds may act by a similar MoA to rescue p.Phe508del-CFTR. Nevertheless, despite the higher level of functional rescue by dual- and triple-corrector combinations, p.Phe508del-CFTR still presents instability and accelerated degradation.

|||||||||| Development of a drug testing platform for CFTR premature termination codon variants based on rectal organoids () - Sep 4, 2024 - Abstract #NACFC2024NACFC_1011;
Incubation with the aminoglycoside G418, as expected from the literature, caused a significantly greater in FIS rate than control (vehicle) and ELX-02 or PTC124, with further improvement in combination with CFTR modulators (elexacaftor/tezacaftor/ivacaftor [ETI]). Our preliminary data indicate that the levels of swelling induced by G418+ETI reached reference AUC values of F508del/F508del organoids treated with VX809/ VX770 (mean 2,487.9

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex
Off-target identification of cystic fibrosis pharmacological correctors using functionalized photocrosslinker analogs () - Sep 4, 2024 - Abstract #NACFC2024NACFC_991;
Background: Cystic fibrosis (CF) pharmacological correctors such as VX-809 and VX-445 are used widely in combinations as highly effective modulator therapy (HEMT) at CF clinics because of their efficacy in improving CFTR protein trafficking and function, yet several side-effects from these correctors and adverse drug interactions have been reported, emphasizing the need to understand the etiology of side-effects that may arise from off-target use. The off targets identified may provide insights into indirect correction mechanisms and provide explanations for the side-effects observed in patients at the cellular level, allowing further optimization of corrector combinations to enhance efficacy and decrease adverse effects.

|||||||||| lumacaftor (VX-809) / Vertex, tezacaftor (VX-661) / Vertex
Journal: Synthesis and Biological Evaluation of Pyrazole-Pyrimidones as a New Class of Correctors of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). (Pubmed Central) - Aug 13, 2024
Molecular docking and three-dimensional quantitative structure-activity relationship (3D-QSAR) studies were conducted to propose a plausible binding site and design more potent and effective analogs. We identified three optimized compounds, which, in combination with VX-809 and the investigational corrector 3151, increased the plasma membrane density and function of F508del-CFTR and other rare CFTR mutants resistant to the currently approved therapies.

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, tezacaftor (VX-661) / Vertex
Journal: Identification of 6,9-dihydro-5H-pyrrolo[3,2-h]quinazolines as a new class of F508del-CFTR correctors for the treatment of cystic fibrosis. (Pubmed Central) - Aug 10, 2024
Synergy was also observed with corr-4a (class 2 corrector) but not with VX-445 and PP028 (class 3 correctors) indicating that the new compounds behave as class 3 correctors. These results suggest that tricyclic pyrrolo-quinazolines interact with CFTR at a site different from that of VX-809 and represent a novel class of CFTR correctors suitable for combinatorial pharmacological treatments for the basic defect in CF.

|||||||||| lumacaftor (VX-809) / Vertex, exaluren (ELX-02) / Eloxx Pharma, eragidomide (CC-90009) / BMS
Rescue of CFTR nonsense mutations is enhanced under inflammatory stimuli (157 A-C) - Jul 4, 2024 - Abstract #NACFC2024NACFC_549;
These results suggest that tricyclic pyrrolo-quinazolines interact with CFTR at a site different from that of VX-809 and represent a novel class of CFTR correctors suitable for combinatorial pharmacological treatments for the basic defect in CF. Cells were also treated in the last 24 hours with ELX-02 (200

|||||||||| lumacaftor (VX-809) / Vertex
Journal, Cancer stem: Lumacaftor as a potential repurposed drug in targeting breast cancer stem cells: insights from in silico study. (Pubmed Central) - Jun 24, 2024
This study opens avenues for further investigation and may pave the way for developing therapeutic potential in breast cancer treatment. Further confirmation is warranted through in vitro and clinical studies to validate the findings of this study.

|||||||||| lumacaftor (VX-809) / Vertex
Preclinical, Journal: Female mice display sex-specific differences in cerebrovascular function and subarachnoid haemorrhage-induced injury. (Pubmed Central) - Apr 15, 2024
Female mice possess a distinct cerebrovascular phenotype compared to males, putatively due to functional differences in CFTR regulation. This sex difference eliminates the CFTR-dependent cerebrovascular effects of SAH and may alter the therapeutic efficacy of lumacaftor compared to males.

|||||||||| Partial Rescue of p.Phe08del-CFTR Trafficking and Stability Defects by Dual and Triple Corrector Combinations (Exhibition and Poster Area) - Mar 22, 2024 - Abstract #ECFS2024ECFS_130;
The lack of additivity of PTI-801 with VX-445 suggests that these compounds may act by a similar MoA to rescue p.Phe508del-CFTR. Nevertheless, despite the higher level of functional rescue by dual and triple corrector combinations, p.Phe508del-CFTR still presents instability and accelerated degradation.

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
Review, Journal: Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor. (Pubmed Central) - Feb 28, 2024
In this review, we exploit the organic synthesis of ivacaftor, tezacaftor, and elexacaftor by providing a retrosynthetic drug analysis for these CFTR modulators. Furthermore, we describe the current understanding of the mechanisms of action (MoA's) of these compounds by discussing several studies that report the key findings on the molecular mechanisms underlying their action on the CFTR protein.

|||||||||| elexacaftor (VX-445) / Vertex, posenacaftor (PTI-801) / Kineta
Journal: PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR. (Pubmed Central) - Feb 26, 2024
Despite the high efficacy of PTI-801 in combination with ABBV-2222, FDL-169, VX-661, or VX-809, these dual corrector combinations only partially restored p.Phe508del-CFTR conformational stability, as shown by the lower half-life of the mutant protein compared to that of WT-CFTR. In summary, PTI-801 likely shares a common MoA with VX-445 in rescuing p.Phe508del-CFTR, thus being a feasible alternative for the development of novel corrector combinations with greater capacity to rescue mutant CFTR folding and stability.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Cyclic diacyl thioureas enhance activity of corrector Lumacaftor on F508del-CFTR. (Pubmed Central) - Feb 19, 2024
All compounds proved to be non-cytotoxic against different cancer cell lines. Good pharmacokinetic properties were predicted for derivatives 5 and 6, thus supporting the value of these compounds for the development of novel modulators potentially useful for cystic fibrosis.

||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
Trial completion, Trial completion date, Trial primary completion date:  Bioequivalence and Food Effect Bioavailability Study of Lumacaftor Film-Coated Tablets (clinicaltrials.gov) -  Dec 25, 2023
P1, N=39, Completed,  Sponsor: Qanatpharma Canada LTD
Good pharmacokinetic properties were predicted for derivatives 5 and 6, thus supporting the value of these compounds for the development of novel modulators potentially useful for cystic fibrosis. Recruiting --> Completed

|||||||||| Kalydeco (ivacaftor) / Vertex
Journal: Synthesis and Evaluation of Ivacaftor Derivatives with Reduced Lipophilicity. (Pubmed Central) - Dec 11, 2023
Molecular modeling predicts that the increased polarity of compound 3 allows engagement with polar amino acids present in the binding pocket with hydrogen bonding and ionic interactions, which are collectively higher in strength as compared to hydrophobic interactions that stabilize ivacaftor. Overall, the data suggests that reduced lipophilicity may improve the efficacy of this class of CFTR potentiators when used for folding-rescued ?F508-CFTR.

|||||||||| lumacaftor (VX-809) / Vertex
CFTR mRNA co?delivered with the modulator VX809 in lipid?nanoparticles in Cystic Fibrosis cells in air?liquid interface culture induced CFTR protein expression at the same level as normal cells () - Oct 15, 2023 - Abstract #ESGCT2023ESGCT_1075;
The flexibility of the RTN formulation allows co?delivery of CFTR mRNA with VX?809, which significantly improved CFTR expression. It can induce the same level of CFTR expression as NHBE in CF cells.

||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
Enrollment open:  Bioequivalence and Food Effect Bioavailability Study of Lumacaftor Film-Coated Tablets (clinicaltrials.gov) -  Oct 10, 2023
P1, N=39, Recruiting,  Sponsor: Qanatpharma Canada LTD
It can induce the same level of CFTR expression as NHBE in CF cells. Not yet recruiting --> Recruiting

|||||||||| lumacaftor (VX-809) / Vertex
Journal: M Protein from Dengue virus oligomerizes to pentameric channel protein: in silico analysis study. (Pubmed Central) - Oct 9, 2023
The virtual screening with 165 different ion channel inhibitors from the ion channel library shows monovalent ion channel blockers, namely lumacaftor, glipizide, gliquidone, glisoxepide, and azelnidipine to be the inhibitors with high docking scores. Understanding the three-dimensional structure of M protein will help design therapeutics and vaccines for Dengue infection.

|||||||||| elexacaftor (VX-445) / Vertex
Journal: Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics. (Pubmed Central) - Sep 10, 2023
Partial inhibition of protein translation also mildly sensitizes P67L CFTR to VX-445 correction, supporting a role for translational dynamics in the rescue mechanism of VX-445. Our results provide a better understanding of VX-445 biological mechanism of action and reveal cellular targets that may sensitize unresponsive CFTR variants to known and available correctors.

|||||||||| lumacaftor (VX-809) / Vertex, Lynparza (olaparib) / Merck (MSD), AstraZeneca
Journal: Targeting Staphylococcal Cell-Wall Biosynthesis Protein FemX Through Steered Molecular Dynamics and Drug-Repurposing Approach. (Pubmed Central) - Aug 21, 2023
This is the first experimental report where staphylococcal FemX has been targeted for the discovery of new drugs. It is suggested that Lumacaftor may be a potential lead molecule against S. aureus.

||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
New P1 trial:  Bioequivalence and Food Effect Bioavailability Study of Lumacaftor Film-Coated Tablets (clinicaltrials.gov) -  Aug 1, 2023
P1, N=36, Not yet recruiting,  Sponsor: Qanatpharma Canada LTD

|||||||||| lumacaftor (VX-809) / Vertex
Journal: CFTR and PC2, partners in the primary cilia in ADPKD. (Pubmed Central) - Jul 31, 2023
Our data suggest that CFTR is present in the cilia and plays a role there, perhaps through its conductance of Cl. We also postulate that septin 2 is important for localizing CFTR to the apical membrane in cystic epithelia.

|||||||||| lumacaftor (VX-809) / Vertex, tezacaftor (VX-661) / Vertex, galicaftor (GLPG2222) / AbbVie
Journal: Computational Exploration of Potential CFTR Binding Sites for Type I Corrector Drugs. (Pubmed Central) - Jul 12, 2023
This data may serve to better understand the structural changes induced by mutation of CFTR and how correctors bind to the protein. Additionally, it may aid in the design of new, more effective CFTR corrector drugs.

|||||||||| elexacaftor (VX-445) / Vertex, tezacaftor (VX-661) / Vertex
Journal: Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination. (Pubmed Central) - Jul 2, 2023
Overall, our data show a relevant response to VX-661-VX-445 in rectal organoids and hNEC with CFTR genotype A559T/A559T. This could provide a strong rationale for treating patients carrying this variant with VX-661-VX-445-VX-770 combination.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Drug repurposing for Mpox: Discovery of small molecules as potential inhibitors against DNA-dependent RNA polymerase using molecular modeling approach. (Pubmed Central) - May 22, 2023
After due validation in wet-lab using available in vitro and in vivo experiments, these repurposed drugs can be further utilized for the treatment of contagious Mpox virus. The outcome of this study may establish a solid foundation to screen repurposed and natural compounds as potential antiviral therapeutics against different highly pathogenic viruses.

|||||||||| lumacaftor (VX-809) / Vertex, Tasigna (nilotinib) / Novartis, Inhibikase
A NOVEL PROGNOSTIC MODEL OF DLBCL PATIENTS BASED ON CUPROPTOSIS RELATED GENES (Poster area) - May 12, 2023 - Abstract #EHA2023EHA_1388;
The outcome of this study may establish a solid foundation to screen repurposed and natural compounds as potential antiviral therapeutics against different highly pathogenic viruses. A prognostic model based on 5 cuproptosis-related genes was constructed, and 3 potential targeted inhibitors of CDKN2A were screened out by molecular docking.

|||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
Beneficial effect of cystic fibrosis transmembrane conductance regulator modulators on adults with cystic fibrosis liver disease (Poster Area) - Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_899;
Liver stiffness was also improved in treated patients. However, more patients and longer follow-up are needed for solid conclusions to be drawn.

|||||||||| elexacaftor (VX-445) / Vertex, lumacaftor (VX-809) / Vertex
A Drug Screening System for Cystic Fibrosis Utilizing Stem-cell and Functional Imaging Technologies (Walter E. Washington Convention Center, Area D, Hall C (Lower Level)) - Mar 25, 2023 - Abstract #ATS2023ATS_8477;
Next, we performed phenotypic screening using both MQAE and label-free FIS assay on drug repositioning library, resulting in identification of candidate compounds. CONCLUSION We established a novel phenotypic screening system by combination of iPSC-based disease models and fluorescent/label-free imaging assays, which will be promising for efficient identification of drug candidates for CF.

|||||||||| lumacaftor (VX-809) / Vertex
Journal: Vx-809, a CFTR Corrector, Acts through a General Mechanism of Protein Folding and on the Inflammatory Process. (Pubmed Central) - Mar 2, 2023
Here, we demonstrate that this drug reduces the ER stress and, consequently, the inflammation that is caused by such events. Thus, this molecule is a promising drug to treat several pathologies that present an etiopathogenesis due to the accumulation of protein aggregates that lead to chronic reticular stress.

|||||||||| lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
Analysis of synthetic derivatives with reduced lipophilicity as candidate potentiators towards better treatment alternatives for cystic fibrosis (ePoster Corner B -Room 0.16) - Feb 27, 2023 - Abstract #ECFS2023ECFS_281;
Thereafter, Orkambi, a combination of ivacaftor and the trafficking corrector lumacaftor was approved for patients with homozygous F508del mutation. Our studies suggest that the synthesized derivatives which exert reduced lipophilicity and destabilization may open new avenues in paving the way for future clinical studies with better patient outcomes in CF.

|||||||||| lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
Impact of cholesterol on the functioning of CFTR modulators (Hall G1) - Feb 27, 2023 - Abstract #ECFS2023ECFS_215;
Our experiments reveal that cholesterol plays an important role in the functioning of Orkambi and in the interaction of this drug with the membrane. These findings help to better understand the role of cholesterol in CFTR-modulator interactions, further bridging the gap in knowledge relating to existing CF drugs and paving the way for the design of more effective CF treatments.



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