Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, today announced
that the first patients have now been enrolled in its Phase 2 study
of ELX-02 for the treatment of Alport syndrome in patients with
nonsense mutations.
“With enrollment of the first patients in our Phase 2 study of
ELX-02 for the treatment of Alport syndrome in patients with
nonsense mutations, we remain on track to deliver topline clinical
results from this trial in the first half of 2023,” said Sumit
Aggarwal, President and Chief Executive Officer of Eloxx. “ELX-02
treatment has demonstrated restoration of full-length protein in
multiple preclinical models, including collagen IV and collagen VII
in cells, and also clinical activity in our Phase 2 cystic fibrosis
trial. We believe our results, previously conducted in vivo studies
showing improvement of kidney function with collagen IV restoration
and the ability to achieve high kidney drug levels at tolerable
doses support the clinical development of ELX-02 in Alport
syndrome.”
This Phase 2 trial is targeting dosing of up to eight Alport
syndrome patients with nonsense mutations in the COL4 gene.
Patients will be dosed for two months with a three month follow-up.
In addition to the primary endpoint of safety, the key secondary
efficacy endpoint of proteinuria will be measured every two weeks.
For eligible patients, induction of COL IV will also be measured at
the end of two months. Topline results are expected in the first
half of 2023.
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-ZM™ 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, statements regarding our expected cash burn and future
financial results, the expected timing of trials and results from
clinical studies 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 September 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/financial-information/sec-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, Inc.
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