Eloxx Pharmaceuticals Presents Positive New Data for Lead Investigational Drug, ELX-02, at the 42nd European Cystic Fibrosis Society Conference (ECFS)
ELX-02 demonstrates dose-responsive pronounced increases in functional CFTR and read-through in organoids, human bronchial epithelial (HBE), and Ussing chamber systems
ELX-02 increases CFTR mRNA to healthy control levels
ELX-02 has shown increased CFTR function in organoids bearing nonsense alleles representing >75% of the cystic fibrosis nonsense genotype population
Eloxx to report top line data from Phase 2 clinical trial in cystic fibrosis in 2019
Phase 2 protocol has been reviewed and approved by the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN) and given a score of “high priority”
“We are extremely pleased with the emerging profile of ELX-02 and believe that previous studies with already approved drugs for cystic fibrosis demonstrating that functional increases in CFTR protein have been highly correlated with improvements in FEV1, and lung function derisk our Phase 2 studies,” said Dr.
In an oral presentation titled: “ELX-02 increases full length CFTR mRNA through nonsense mediated decay interruption” presented by Dr.
- ELX-02 demonstrates dose-responsive pronounced increases in functional CFTR and read-through in plasmid, HBEs, Fisher rat thyroid epithelia, transgenic mice and patient-derived organoids.
- ELX-02 dose-dependently increases CFTR mRNA stability.
- ELX-02 increases CFTR mRNA to healthy control levels.
- ELX-02 increases CFTR function in organoids bearing nonsense alleles representing >75% of the cystic fibrosis nonsense genotype population.
In a second oral presentation titled: “Administration of ELX-02 to Healthy Volunteers Demonstrates Dose Linearity and Proportionality as well as Low Inter-subject Variability” presented by Dr.
- ELX-02 has been generally well tolerated in clinical studies to date, supporting future development in cystic fibrosis patients with nonsense genotypes.
- ELX-02 shows high bioavailability (98%) upon subcutaneous administration and highly reproducible pharmacokinetics.
- ELX-02 administered SC shows linear and dose proportional AUC0-12 and Cmax over the dosage studied (0.3 to 7.5 mg/kg).
- Consistent results were observed across single and multiple dose studies, with no accumulation.
- Elimination is primarily renal as parent compound.
In a poster presentation titled: “ELX-02 Pharmacokinetic profile appropriate for CF patient use” presented by Dr.
- ELX-02 demonstrated pronounced restoration of CFTR activity in organoid, HBE, and Ussing chamber systems.
- Pharmacokinetic nonclinical and clinical results along with physiologically based modeling data support the use of ELX-02 in cystic fibrosis patients with twice weekly or daily dosing.
- Preliminary multiple ascending dose study results are consistent with single ascending dose study results.
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Source: Eloxx Pharmaceuticals