Eloxx Pharmaceuticals Announces Initiation of Phase 2 Clinical Trial for ELX-02 in Cystinosis
August 05 2019 - 8:00AM
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 that it has initiated a Phase 2 clinical trial
(Study 003) for ELX-02 in cystinosis in Canada.
Study 003 is a single arm, open label
study designed to assess the safety, tolerability,
pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous
(SC) ELX-02 in patients with nephropathic cystinosis with at least
1 nonsense mutation in the cystinosin gene. Six patients will
be enrolled in the trial, and will be treated with ELX-02 for four
weeks with three escalating dose levels.
“I am excited to have the
opportunity to offer ELX-02 to my cystinosis patients, a new
potential treatment option which has exhibited positive results in
cellular and animal models with dose-dependent reductions in white
blood cell cystine levels, a biomarker used to monitor cysteamine
therapy and to predict clinical benefit,” said Dr. Paul Goodyer,
Professor of Pediatrics at McGill University. “There remains a high
unmet medical need in cystinosis, which is diagnosed in infancy,
and new therapeutic options with the potential to alter the course
of this progress disease could dramatically improve the lives of
patients and their families.”
“We are very pleased to have initiated a Phase 2
clinical trial for ELX-02 in cystinosis patients with nonsense
mutations in Canada and we are grateful for the support and
non-dilutive funding we have received for this trial from Genome
Quebec and Genome Canada,” said Dr. Greg Williams, COO of Eloxx
Pharmaceuticals. “We look forward to reporting top line data from
this trial early in the fourth quarter of this year. We believe
that the results to date, as well as the preliminary results from
our renal impairment study provide support for both continuing our
clinical development programs and evaluating the suitability of our
ERSG library for development in additional renal disorders,
including autosomal dominant polycystic kidney disease and
cystinuria.”
Dr. Goodyer previously reported data at the
WORLDSymposium which showed that ELX-02 decreases the cystine
content in cellular and animal models. Dr. Goodyer extended his
research to show ELX-02 cystine decreases when used in combination
with cystamine. These data were presented in March at the
Cystinosis Research Foundation “Day of Hope” meeting in Irvine,
California.
In support of the cystinosis program, where
preserving renal function is a major concern, Eloxx has completed a
renal study in mild, moderate, and severe renal impairment cohorts.
Previously, the U.S. Food and Drug Administration granted orphan
drug designation for ELX-02 for the treatment of cystinosis and an
IND is currently open. ELX-02 is an investigational drug that has
not been approved by any global regulatory body.
Conference Call
Information:
Date: Wednesday, August 7,
2019
Time: 8:30 a.m. ET
Domestic Dial-in
Number: (866) 913-8546
International Dial-in
Number: (210) 874-7715
Conference ID: 1754316
Live Webcast: accessible from the
Company's website at www.eloxxpharma.com under Events and
Presentations or with this link:
https://edge.media-server.com/mmc/p/9axiqnvt
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
203-274-2825barbarar@eloxxpharma.com
SOURCE: Eloxx Pharmaceuticals, Inc.
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