Eloxx Pharmaceuticals Presents Positive New Data for Lead Investigational Drug, ELX-02, at the European Cystic Fibrosis Society (ECFS) Basic Science Conference
ELX-02 shows significant increases in CFTR protein expression and localization correlating with functional activity in Cystic Fibrosis patient-derived organoids bearing CFTR nonsense mutations
In previous studies, the increased CFTR functional activity associated with ELX-02 was shown to correlate with increases in CFTR mRNA with elevations to wild-type levels
ELX-02 appears to increase CFTR mRNA stability, suggesting that ELX-02 may be modulating nonsense mediated decay
“ELX-02 is the first read-through agent to demonstrate significant increases in CFTR protein expression, localization on the apical surface, and functional activity in a dose responsive manner in organoids derived from cystic fibrosis patients with nonsense mutations” said Dr.
Recent work with cystic fibrosis patient-derived organoids have extended the potential applications of the FIS assay to include use in stratifying patient disease severity (1) and as a potential predictor of CF patient response to drug therapy (2). On
Eloxx is on track to report top line results in 2019 from a Phase 2 study in cystic fibrosis patients carrying at least one G542X mutation in the U.S., and
In a Poster presentation at the
- Consistent with increased CFTR activity observed in the organoid swelling assay, ELX-02 mediates a significant restoration of CFTR protein expression as measured via a capillary-based immunoassay approach in multiple G542X or W1282X nonsense carrying organoids.
- G542X organoids treated with ELX-02 demonstrate proper cell surface CFTR localization on the apical surface, which is consistent with increased CFTR, mRNA, and CFTR function in the swelling assay.
While ELX-02 mediated protein increases have been previously demonstrated, this is the first demonstration reported in cystic fibrosis patient organoids. Within this translational CF organoid model, ELX-02 dose-dependent increases in CFTR mRNA stability and function can now be extended to the demonstration of accompanying increases of CFTR protein.
Previously, Eloxx presented positive data for ELX-02 at the
These data demonstrate that ELX-02 promotes translation of functional CFTR and we believe, de-risks our planned Phase 2 clinical trial of ELX-02 in cystic fibrosis patients with the G542X CFTR mutation on one or both alleles, which is the second most common mutation globally and accounts for about 5% of the cystic fibrosis population.
Eloxx has continued to generate new data for ELX-02 activity from a growing number of patient-derived organoids which represent multiple nonsense mutations across a variety of genotypes representing the top 5 nonsense mutations in the cystic fibrosis population, which cover over 75% of the nonsense bearing cystic fibrosis patients.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use.
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors,including: the development of the Company’s read-through technology; the approval of the Company’s patent applications; the Company’s ability to successfully defend its intellectual property or obtain necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the Company’s ability to obtain applicable regulatory approvals for its current and future product candidates; the acceptance by the market of the Company’s products should they receive regulatory approval; the timing and success of the Company’s preliminary studies, preclinical research, clinical trials, and related regulatory filings; the ability of the Company to consummate additional financings as needed; as well as those discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.
Source: Eloxx Pharmaceuticals