Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation (CF Foundation) To Provide Funding and Support for ELX-02 U.S. Pha...
September 23 2019 - 7:30AM
Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) a clinical-stage
biopharmaceutical company dedicated to the discovery and
development of novel therapeutics to treat cystic fibrosis,
cystinosis, inherited retinal disorders, and other diseases caused
by nonsense mutations limiting production of functional proteins,
today announced that the Cystic Fibrosis Therapeutics Development
Network has sanctioned its Phase 2 clinical trial protocol in the
US for ELX-02 in cystic fibrosis, and the CF Foundation will
provide funding and additional resources for the program.
“We are very gratified by the CF Foundation’s
support as we advance our development program for ELX-02 in cystic
fibrosis. The CF Foundation’s funding will accelerate the program
in the US,” said Robert E. Ward, Chairman and Chief Executive
Officer of Eloxx Pharmaceuticals. “We are also pleased that Dr.
Ahmet Uluer, Director of the Adult Cystic Fibrosis Program, Boston
Children’s Hospital has agreed to be the lead investigator in the
U.S, and we look forward to reporting top line data from this trial
later this year.”
Eloxx’s Phase 2 clinical trial program will
consist of two open label, dose escalation studies to evaluate the
safety, tolerability, PK and PD of multiple doses of ELX-02 in
patients with CF with at least one G542X allele. The U.S.
study, EL-012, will enroll up to eight patients, and the CF
Foundation has provided a financial award of up to $1.6 million to
support this study. The European study, EL-004, will enroll up to
16 patients. Eloxx anticipates reporting top line data in the
fourth quarter of 2019.
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 and cystinosis. 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 recently
announced a new program focused on rare ocular genetic disorders.
Eloxx is headquartered in Waltham, MA, with operations in Rehovot,
Israel. 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; 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.
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