Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Pharmacology and Experimental Therapeutics
July 15 2020 - 1:08PM
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 a scientific manuscript
titled: “ELX-02 Generates Protein via Premature Stop Codon
Read-through Without Inducing Native Stop Codon Read-through
Protein”, has been published in the
Journal of
Pharmacology and Experimental Therapeutics.
“We are very pleased that our scientific manuscript on ELX-02
which demonstrates that ELX-02 mediates read-through of premature
stop codons while maintaining the fidelity of stop codons found at
the end of healthy transcripts has been published in the
Journal of Pharmacology and Experimental
Therapeutics,” said Dr. Matthew Goddeeris, PhD, Vice
President of Research, Eloxx Pharmaceuticals. “This is an important
finding indicating that translation integrity is preserved with
target-therapeutic exposure of ELX-02, consistent with the
favorable tolerability profile demonstrated across our preclinical
and clinical datasets. ELX-02 is currently in Phase 2 clinical
trials in cystic fibrosis patients with nonsense mutations, for
whom there are few, if any, treatment options available.”
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 rare ocular genetic disorders and polycystic
kidney disease . 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.
Eloxx Pharmaceuticals (NASDAQ:ELOX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Eloxx Pharmaceuticals (NASDAQ:ELOX)
Historical Stock Chart
From Apr 2023 to Apr 2024