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Eloxx Pharmaceuticals, Inc. ("Eloxx"), (NASDAQ: ELOX), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced positive data that ELX-02 showed significant increases in CFTR functional assay
"We are extremely pleased with the emerging profile of ELX-02, as the first read-through agent to be clinically-based in mRNA in organoids derived from cystic fibrosis patients with nonsense mutations," said Neal Sharpe, Ph.D. Vice President of Translational Sciences at Eloxx. "There is a high unmet medical burden among 13% of cystic fibrosis patients with a nonsense mutation, as they have a high burden of disease and few, if any, treatment options."
Recent work with cystic fibrosis patient-derived organoids have extended the potential of the FIS assay to include the use of stratified patient disease severity (1) and the potential predictor of CF patient response to drug therapy (2).
Eloxx plans to initiate a Phase 2 study in cystic fibrosis patients carrying at least one G542X mutation. The European Cystic Fibrosis Society- Clinical Trial Network has reviewed the program and assigned a "high priority" rating. Eloxx expects to report top-line data from the Phase 2 study in 2019.
In a Poster presentation titled "Measuring mRNA levels in cystic fibrosis organoids with nonsense mutations following treatment with ELX-02," presented by Neal Sharpe, PhD, VP Translational Science, Eloxx reported that:
- ELX-02 in a responsive dose increase in CFTR function and mRNA expression when tested in a correlative assay using organoids from cystic fibrosis patients with homozygous and heterozygous nonsense mutations. The FIS is based on a range of concentrations of the swelling inducing agent, forskolin, and did not saturate the timeframe of the assay. Furthermore, the swelling response has been demonstrated to be dependent on CFTR activity and the presence of a nonsense mutation. The response is shown to be potentially predictive of clinical efficacy.
- Using nanoString ™ technology, the ELX-02 mediated organoid swelling was found to correlate with increased CFTR mRNA, with elevations above wild-type. ELX-02 appears to increase the steady state concentrations of CFTR mRNA suggesting that ELX-02 can be modulating nonsense mediated decay.
- The increased CFTR function demonstrated with ELX-02 was further enhanced with the addition of a potentiator and corrector in some organoids derived from patients with heterozygous nonsense mutations.
- These data demonstrate that ELX-02 promotes the translation of functional CFTRs and supports the further development of ELX-02 in patients with cystic fibrosis.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use.
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