Eloxx Pharmaceuticals Presents New Positive Data for ELX-02 at the American Society of Nephrology (ASN) Kidney Week 2019
November 07 2019 - 10:00AM
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 new data from two abstracts presented at the
American Society of Nephrology (ASN) Kidney Week
2019 in Washington, D.C. November 5 -10, 2019.
“We are pleased to have presented the results of
our completed renal impairment study at Kidney Week, which have
been important in defining the appropriate doses for patients with
renal insufficiency. These data support our current clinical
programs as well as the expansion of our research in the kidney
beyond nephropathic cystinosis into other areas such as autosomal
dominant polycystic kidney disease where there is a high prevalence
of nonsense mutation patients,” said Dr. Gregory Williams, Chief
Operating Officer of Eloxx Pharmaceuticals. “We are advancing our
Phase 2 clinical trials for ELX-02 in cystic fibrosis and
cystinosis and we look forward to providing an update on our cystic
fibrosis clinical trials and reporting additional top line
cystinosis data later this quarter.”
In a poster session titled “An open
label-single dose, parallel-group study to evaluate the effects of
renal impairment on the pharmacokinetics of ELX-02: Results from
subjects with mild and moderate renal
impairment,” Dr. Gregory Williams, Chief Operating
Officer, Eloxx Pharmaceuticals reported that:
- As degree of renal impairment increased, the exposure to ELX-02
increased and its clearance decreased.
- There were no significant differences in plasma ELX-02
concentrations between the control group and the mildly impaired
renal groups. AUC0-24 was higher in the moderate and severe groups
relative to the control group.
- The observed changes in plasma concentrations enable dose
adjustment based on eGFR/renal function.
- Urinary ELX-02 clearance was similar to plasma clearance, with
decreased rate in subjects with more severe renal impairment.
- To date, ELX-02 has been generally well tolerated in clinical
studies, with 105 volunteers exposed, no reported SAEs or renal
findings.
- Collectively, these data support the future evaluation of
ELX-02 in Phase 2 trials with nonsense mediated diseases.
In a poster session titled “Cystinosis
nonsense mutation read-through mediated by ELX-02 restores protein
function using in vitro and in vivo models,” Dr. Matthew
Goddeeris, Executive Director of Research, Eloxx Pharmaceuticals
reported that:
- ELX-02 read-through is sufficient to produce functional CTNS
protein and increase CTNS mRNA.
- ELX-02 demonstrated that the expressed cystinosin protein
reduced the accumulated lysosomal cystine by one third in the time
frame of the experiment at the given dose of 10 mg/kg.
- Kidney exposure and demonstration of efficacy in vivo support
dose-range selection for a Phase 2 clinical trial of ELX-02 in
Nephropathic Cystinosis.
- Completion of a Phase 1 study in renal insufficient
participants provides modeling necessary for dose adjustments based
on renal function.
- These results support the continued development of ELX-02 for
the potential treatment of nephropathic cystinosis and other
nonsense mutation mediated diseases of the kidney, such as
Autosomal Dominant Polycystic Kidney Disease (ADPDK).
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.
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contains forward-looking statements, which are generally statements
that are not historical facts. Forward-looking statements can be
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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
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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
203-274-2825barbarar@eloxxpharma.com
SOURCE: Eloxx Pharmaceuticals, Inc.
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