Eloxx Pharmaceuticals Presents Positive New Data at the Association for Research in Vision and Ophthalmology “ARVO” 2019 Annual Meeting
Eloxx is evaluating several of its eukaryotic ribosomal selective glycoside (ERSG) molecules in IND-enabling studies for use in the treatment of inherited retinal diseases with an initial focus on Usher Syndrome
Preclinical studies have demonstrated:
dose-dependent restoration of missing protein of Usher Syndrome nonsense mutations;
encouraging pharmacokinetics in the retina by intravitreal injection;
a favorable tolerability profile at high doses in sensitive species
Eloxx also made a presentation at the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit on
“We are very pleased with the preclinical results for several of our ERSG compounds demonstrating that they are suitable for intravitreal injection, well-tolerated in a sensitive model species, active against Usher Syndrome mutations and capable of reaching the retina. We look forward to advancing IND-enabling studies in inherited retinal diseases, with an initial focus on Usher Syndrome,” said Dr.
In a Poster presentation at the ARVO Annual Meeting on
- Eloxx has screened multiple compounds from its ERSG library of read-through agents for potential use in the treatment of inherited retinal disorders with an initial focus on Usher Syndrome, beginning with USH2A.
- Multiple Eloxx compounds in preclinical studies have demonstrated:
- Dose-dependent activity against Usher mutations.
- Restoration of missing Usher Syndrome protein.
- Favorable tolerability profile at high doses in sensitive species; preserved electroretinogram (ERG) and no compound-related histopathological changes.
- Encouraging pharmacokinetics demonstrating retina exposure by intravitreal injection.
- IND-enabling studies ongoing.
- Next steps include evaluation of patient-derived cell models and sustained release formulations.
At the request of the Foundation Fighting Blindness, Dr.
Eloxx compounds are investigational agents 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