Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Cystic Fibrosis
February 09 2021 - 7:00AM
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: “
Targeting G542X CFTR
Nonsense Alleles With ELX-02 Restores CFTR Function in
Human-Derived Intestinal Organoids” was published in the
Journal of Cystic Fibrosis.
“We are very pleased that our scientific manuscript on the
results of our evaluation of ELX-02 mediated read-though, using the
CFTR-dependent Forskolin-induced swelling (FIS) assay across a
selection of G542X homozygous and heterozygous patient-derived
organoids, was published in the Journal of Cystic
Fibrosis,” said Dr. Matthew Goddeeris, PhD, Vice President
of Research, Eloxx Pharmaceuticals. “ELX-02 increased CFTR activity
in a dose-dependent fashion across a variety of forskolin induction
concentrations.The functional increases are similar
to those obtained with tezacaftor/ivacaftor in an F508del
homozygous organoid. Additionally, ELX-02 treatment of these
patient-derived organoids results in a 5-fold increase in CFTR mRNA
when compared with vehicle treated, resulting in normalization of
CFTR mRNA as measured using Nanostring. These data support our
current Phase 2 clinical trial for ELX-02 in cystic fibrosis
patients with G542X nonsense alleles, for whom there are few, if
any, treatment options available. We look forward to reporting top
line data in the first half of 2021.”
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 StatementsThis 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 Ryan203-274-2825
barbarar@eloxxpharma.com
SOURCE: Eloxx Pharmaceuticals, Inc.
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