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Eloxx Announces Two Preclinical Abstracts Accepted for Presentation at the 2020 North American Cystic Fibrosis Virtual Conference in October

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Eloxx Pharmaceuticals (NASDAQ: ELOX) has announced the upcoming presentation of two abstracts at the North American Cystic Fibrosis Conference (NACFC) starting October 7, 2020. The abstracts will showcase preclinical study results on the drug ELX-02, which targets cystic fibrosis caused by nonsense mutations. CEO Gregory Williams emphasized ELX-02’s effectiveness in restoring CFTR production in patient-derived organoids. The posters will be available to registered attendees and published in Pediatric Pulmonology.

Positive
  • Presentation of two abstracts at NACFC could enhance visibility and credibility.
  • Data suggests ELX-02 effectively restores functional CFTR production.
  • Phase 2 clinical trials for ELX-02 are advancing in multiple countries.
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  • None.

WALTHAM, Mass., Aug. 26, 2020 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that two abstracts will be presented at the North American Cystic Fibrosis Conference (NACFC) to be held virtually starting on October 7, 2020. The two abstracts will be showcased in the NACFC virtual poster gallery and electronically published as a supplement to Pediatric Pulmonology. These virtual posters will be available to registered attendees on the NACFC online conference platform starting on October 7, 2020.

“We are pleased to be presenting additional preclinical study results in cystic fibrosis at the 2020 NACFC virtual conference which demonstrate ELX-02’s selectivity for read-through of premature stop codons versus native stop codons and its ability to restore production of functional CFTR in patient-derived organoids,” said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. “We are advancing our Phase 2 clinical trials for ELX-02 in cystic fibrosis in Europe, Israel and the U.S., and we believe the data from our preclinical programs substantially de-risk this program. We look forward to reporting top line data from our proof of concept Phase 2 clinical trials as quickly as possible.”

The details for the two ELX-02 poster presentations are:

Poster Session Presentation Title: “ELX-02 Generates Protein Via Premature Stop Codon Read-through Without Inducing Native Stop Codon Read-through Proteins” 
Poster #: 433
Presenter: Dr. Dan Crawford, Eloxx Pharmaceuticals
Time: Available virtually to registered attendees starting on October 7, 2020

Poster Session Presentation Title: “CFTR Restoration By ELX-02 Across CF Nonsense Genotypes: Utilizing Patient-Derived Organoids to Survey Responsive Alleles
Poster #: 383
Presenter: Dr. Matthew Goddeeris, Eloxx Pharmaceuticals
Time: Available virtually to registered attendees starting on October 7, 2020

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx’s preclinical candidate pool consists of a library of novel drug candidates designed to be eukaryotic ribosomal selective glycosides identified based on read-through potential. Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with operations in Rehovot, Israel and Morristown, NJ.  For more information, please visit www.eloxxpharma.com.

Forward-Looking Statements

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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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.

Contact:

Barbara Ryan 
203-274-2825
barbarar@eloxxpharma.com

SOURCE: Eloxx Pharmaceuticals, Inc.


FAQ

What is Eloxx Pharmaceuticals presenting at the NACFC?

Eloxx Pharmaceuticals is presenting two abstracts focused on ELX-02 and its efficacy in treating cystic fibrosis.

When will the NACFC take place?

The NACFC will be held virtually starting October 7, 2020.

What is ELX-02's significance in cystic fibrosis treatment?

ELX-02 aims to restore functional CFTR protein production in patients with cystic fibrosis caused by nonsense mutations.

What are the titles of the posters being presented?

The poster titles are 'ELX-02 Generates Protein Via Premature Stop Codon Read-through' and 'CFTR Restoration By ELX-02 Across CF Nonsense Genotypes.'

Who will present the abstracts?

Dr. Dan Crawford and Dr. Matthew Goddeeris will present the abstracts.

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