Eloxx Pharmaceuticals Announces FDA Clearance to Begin Single Ascending Dose Study of ZKN-013
May 02 2023 - 8:00AM
Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the
“Company”), a leader in ribosomal RNA-targeted genetic therapies
for rare diseases, today announced that the U.S. Food and Drug
Administration (FDA) has cleared the Company’s Investigational New
Drug (IND) application to initiate a single ascending dose (SAD)
clinical trial in healthy volunteers for ZKN-013 for the potential
treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with
nonsense mutations. RDEB is a rare skin disease characterized by
mutations in Collagen7 gene.
“FDA clearance to begin our planned single ascending dose trial
is an important milestone towards providing a potential treatment
option for patients with RDEB and JEB,” said Sumit Aggarwal,
President and Chief Executive Officer of Eloxx. “This update is
also a momentous event for Eloxx, as ZKN-013, our lead TURBO-ZM™
based molecule, is the first program developed from hit to lead and
is now poised to enter clinical development. Beyond ZKN-013, Eloxx
has promising TURBO-ZM based discovery programs in cystic fibrosis
(fully funded by the Cystic Fibrosis Foundation) and in certain
cMYC-overexpressing cancers.”
Further SAD and multiple ascending dose (MAD) testing is
expected to be conducted following the completion of the planned
dose cohorts in the SAD study and discussion with the FDA. The MAD
testing could potentially include RDEB patients given the strong
benefit/risk in patients cited by the FDA.
ZKN-013 Preclinical Activity in RDEB, JEB and
FAP
ZKN-013 has demonstrated robust functional preclinical activity
in RDEB and JEB patient cells and in APCmin (multiple intestinal
neoplasia) mice.
Eloxx also plans to develop ZKN-013 for the treatment of FAP
patients with nonsense mutations characterized by proliferation of
colon polyps and progression to colon cancer. In January 2023,
Eloxx published its preclinical results that showed treatment with
ZKN-013 demonstrated a decrease in intestinal polyps and adenomas,
resulting in increased survival.
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. ELX-02 is in Phase 2 clinical development for the
treatment of Alport syndrome in patients with nonsense mutations.
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 clinical testing of ZKN-013 for
the treatment of RDEB and JEB and the expected timing of trials for
our other product candidates and the potential of our product
candidates 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; our ability to meet the continued
listing requirements of the Nasdaq Capital Market; 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 Annual Report on
Form 10-K for the fiscal year ended December 31, 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, Inc.
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