Eloxx Presents Two Preclinical Posters at the 2020 North American Cystic Fibrosis Virtual Conference
October 22 2020 - 8:57AM
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 it presented data from
two scientific abstracts at the North American Cystic Fibrosis
Virtual Conference (NACFC). The two abstracts were also showcased
in the NACFC virtual poster gallery and electronically published as
a supplement to
Pediatric Pulmonology. The live
sessions and discussions will take place through October 23rd,
2020. These virtual posters are available to registered attendees
on the NACFC online conference platform.
“We were pleased to have the opportunity to present additional
preclinical study results in cystic fibrosis at the 2020 NACFC
virtual conference that 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 believe that
these results de-risk the current Phase 2 proof of concept clinical
trials for ELX-02 in cystic fibrosis. We are continuing to advance
our trials in Europe, Israel and the U.S., and we look forward to
reporting top line data from the Phase 2 clinical trial program 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 #:
433Presenter: Dr. Dan Crawford, Eloxx
Pharmaceuticals
- ELX-02 produces significant read-through of premature stop
codons leading to full length proteins, demonstrated using DMS-114
cells with the R213X nonsense mutation in the TP53 gene.
- Using three complementary techniques, no evidence of native
stop codon read-through products could be detected. These data
suggest that ELX-02 does not promote native stop codon read-through
at concentrations relevant to premature stop codon
read-though.
- The results of studies are consistent with the acceptable
tolerability profile of ELX-02 across preclinical and clinical
studies to date.
Poster Session Presentation Title: “CFTR Restoration By
ELX-02 Across CF Nonsense Genotypes: Utilizing Patient-Derived
Organoids to Survey Responsive Alleles” Poster #:
383Presenter: Dr. Matthew Goddeeris, Eloxx
Pharmaceuticals
- The patient-derived organoid CFTR FIS assay has enabled the
screening of a wide selection of cystic fibrosis nonsense alleles
representing >75% of the cystic fibrosis nonsense population.
Using this method, we continue to identify new responsive
genotypes.
- The response of W1282X patient-derived organoids to ELX-02
mediated through read-through positively correlates with CFTR mRNA
expression.
- Increasing the available CFTR mRNA pool through inhibition of
nonsense mediated decay has a synergistic effect on ELX-02 mediated
functional CFTR read-through.
- These results help guide the interpretation of the
patient-derived organoid CFTR FIS assay data by highlighting the
importance of considering CFTR expression differences across
patient-derived organoids for the applicability of ELX-02 as a
potential therapeutic option for cystic fibrosis patients with
nonsense alleles.
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 Ryan203-274-2825barbarar@eloxxpharma.com
SOURCE: Eloxx Pharmaceuticals, Inc.
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