Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, today announced
the opening of clinical trial sites for its Phase 2 study of ELX-02
for the treatment of Alport syndrome in patients with nonsense
mutations.
“Rare diseases with nonsense mutations, such as Alport syndrome,
are particularly challenging to treat. We are incredibly pleased to
open the first clinical trial sites for our Phase 2 study of ELX-02
for the treatment of Alport syndrome in patients with nonsense
mutations, reflecting our commitment to advance our science in the
interest of patients affected by rare disease. Additional sites are
expected to be opened and actively recruiting Alport patients in
the coming weeks,” said Sumit Aggarwal, President and Chief
Executive Officer of Eloxx. “Alport patients with nonsense
mutations have significantly worse clinical outcomes than other
Alport patients and have no disease modifying treatment
options.”
This proof-of-concept clinical trial will include up to eight
Alport syndrome patients with nonsense mutations in the second half
of 2022. Patients will be dosed for two months with a three month
follow-up. Trial primary endpoints will include safety, while
secondary endpoints will include reduction in proteinuria and
induction of COL4A5 protein expression in the kidney. Topline
results are expected in the first half of 2023.
Eloxx believes there is a strong rationale to pursue clinical
development of ELX-02 in Alport syndrome, based on encouraging
preclinical results demonstrating potentially therapeutic levels of
readthrough, ability to deliver high drug concentrations in the
kidney relative to plasma at clinically tolerable dose levels, and
the clinical readthrough results seen in our Phase 2 cystic
fibrosis trial.
About Alport syndrome
Alport syndrome is a genetic disorder characterized by kidney
disease with high levels of proteinuria, hearing loss and eye
abnormalities caused by mutations in the genes (COL4A3, COL4A4, and
COL4A5) needed for production of type 4 collagen. Approximately 6%
to 7% of Alport syndrome patients, or approximately 9,400 to 12,750
individuals, are estimated to have nonsense mutations. These
patients have significantly worse clinical outcomes than other
Alport patients and have no disease modifying treatment
options.
About Nonsense Mutations
Nonsense mutations cause a premature stop codon in the mRNA
resulting in less than full length or loss of function proteins.
These remain highly underserved with no approved disease modifying
therapies. An estimated 10-12% patients across over 8,000 inherited
genetic rare diseases harbor nonsense mutations in one or both
alleles harboring nonsense mutations.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of
ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry
technology platform in an effort to develop novel Ribosome
Modulating Agents (RMAs) and its library of Eukaryotic Ribosome
Selective Glycosides (ERSGs). Eloxx’s lead investigational product
candidate, ELX-02, is a small molecule drug candidate designed to
restore production of full-length functional proteins. The U.S.
Food and Drug Administration (FDA) has granted Fast Track
designation for ELX-02 for the treatment of CF patients with
nonsense mutations. In addition, ELX-02 has also been granted
Orphan Drug Designation for the treatment of CF patients with
nonsense mutations by the FDA and orphan medicinal product
designation by the European Commission. ELX-02 is in clinical
development, focusing on cystic fibrosis (US Trial NCT04135495,
EU/IL Trial NCT04126473). Eloxx also has preclinical programs
focused on select rare diseases, including inherited diseases,
cancer caused by nonsense mutations, kidney diseases, including
autosomal dominant polycystic kidney disease, as well as rare
ocular genetic disorders.
For more information, please visit www.eloxxpharma.com.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements other than statements of present and
historical facts contained in this press release, including without
limitation, the expected timing of trials of our product candidates
and the potential of our product candidate to treat nonsense
mutations are forward-looking statements. Forward-looking
statements can be identified by the words “aim,” “may,” “will,”
“would,” “should,” “expect,” “explore,” “plan,” “anticipate,”
“could,” “intend,” “target,” “project,” “contemplate,” “believe,”
“estimate,” “predict,” “potential,” “seeks,” or “continue” or the
negative of these terms similar expressions, although not all
forward-looking statements contain these words. Forward-looking
statements are based on management's current plans, estimates,
assumptions and projections based on information currently
available to us. Forward-looking statements are subject to known
and unknown risks, uncertainties and assumptions, and actual
results or outcomes may differ materially from those expressed or
implied in the forward-looking statements due to various important
factors, including, but not limited to: our ability to progress any
product candidates in preclinical or clinical trials; the
uncertainty of clinical trial results and the fact that positive
results from preclinical studies are not always indicative of
positive clinical results; the scope, rate and progress of our
preclinical studies and clinical trials and other research and
development activities; the competition for patient enrollment from
drug candidates in development; the impact of the global COVID-19
pandemic on our clinical trials, operations, vendors, suppliers,
and employees; our ability to obtain the capital necessary to fund
our operations; the cost of filing, prosecuting, defending and
enforcing any patent claims and other intellectual property rights;
our ability to obtain financial in the future through product
licensing, public or private equity or debt financing or otherwise;
general business conditions, regulatory environment, competition
and market for our products; and business ability and judgment of
personnel, and the availability of qualified personnel and other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarter ended June 30, 2022,
as any such factors may be updated from time to time in our other
filings with the SEC, accessible on the SEC’s website at
www.sec.gov and the “Financials & Filings” page of our website
at https://investors.eloxxpharma.com/financials-filings.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
Contact
InvestorsJohn
Woolfordjohn.woolford@westwicke.com443.213.0506
MediaLaureen Cassidylaureen@outcomescg.com
Source: Eloxx Pharmaceuticals
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