Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation (CF Foundation) To Provide Increased Funding and Support for ELX-0...
14 January 2021 - 1:00AM
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 an expansion of its agreement
with the CF Foundation beyond the U.S. portion of the trial to
include additional funding for the global clinical trial program
which includes Europe and Israel.
“We are gratified by the CF Foundation’s
expanded support for our Phase 2 proof of concept global clinical
trial program for ELX-02 for the treatment of patients with
nonsense mediated cystic fibrosis, for whom there remains a high
unmet medical need,” said Dr. Gregory Williams, Chief Executive
Officer of Eloxx Pharmaceuticals. “We Look forward to reporting top
line data from this program in the first half of this year and are
pleased that ELX-02 has been granted an orphan drug designation by
the FDA and orphan medicinal product designation by the EMA.”
Cystic Fibrosis Phase 2 Program
- Our Phase 2 program
consists of two trials, one currently enrolling patients at sites
in Europe and Israel and the second in the U.S.
- In the U.S., in
addition to the partial funding being provided by the CF
Foundation, our protocol has been sanctioned by the Cystic Fibrosis
Therapeutics Development Network (TDN).
- In Europe, the
European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN)
has given our trial a “high priority” ranking.
- Dr. Ahmet Uluer,
Director of the Adult Cystic Fibrosis Program at the Boston
Children’s Hospital/Brigham and Women’s Hospital CF Center, is the
lead study investigator in the U.S.
- We expect to report
top line data from our proof of concept Phase 2 program in the
first half of 2021, which is contingent on no further disruptions
due to COVID-19.
- The Safety Review
Committee has held several planned meetings and approved dose
escalation up to the highest dose level. To date, we have had no
reported serious adverse events in the clinical trial.
- We are participating in the European HIT-CF consortium to
support the collection of cystic fibrosis patient-derived organoids
and the initiative to conduct a prospective clinical trial to
confirm the translational potential of the organoid model. The
intent of the program is to use these positive results to enroll
patients with responsive organoids in a prospective trial with
ELX-02. We believe this program will continue to expand the
application of organoid technology from drug discovery through drug
approval, and also offers possible label expansion
opportunities.
ELX-02 is an investigational agent not approved by any
regulatory authority for therapeutic use, which is currently in
Phase 2 clinical trials in cystic fibrosis.
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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