Eloxx Pharmaceuticals Announces Fast Track Designation for ELX-02 for the Treatment of Cystic Fibrosis Patients with Nonsense Mutations
September 09 2021 - 8:00AM
Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), today announced that
the U.S. Food and Drug Administration (FDA) has granted Fast Track
designation for ELX-02, a drug candidate intended to treat cystic
fibrosis patients with nonsense mutations. ELX-02 is currently in
Phase 2 clinical trials in CF patients affected by nonsense
mutations in the CFTR (CF transmembrane conductance regulator) gene
for whom there are no effective disease modifying therapies. The
Phase 2 trials are designed to evaluate the safety of ELX-02 and
assess its biological activity, and Eloxx expects to present data
from the first four treatment arms in the fourth quarter of 2021.
“We are delighted to receive Fast Track
Designation from the FDA for ELX-02 as the need for patients
remains significant. The ability to have greater access to the FDA
and their guidance on the regulatory pathway for ELX-02 can help
provide the ability to work with the urgency needed on behalf of CF
patients with nonsense mutations,” said Sumit Aggarwal, President
and CEO of Eloxx.
Fast Track Designation is granted to drugs being
developed for the treatment of serious or life-threatening diseases
or conditions where there is an unmet medical need. The purpose of
the provision is to help facilitate development and expedite the
review of drugs to treat serious or life-threatening conditions so
that an approved product can reach the market expeditiously.
Sponsors of drugs that receive Fast Track Designation have the
opportunity for more frequent interactions with the FDA review team
throughout the development program.
ELX-02 has previously been granted orphan drug
designation by the FDA and orphan medicinal product designation by
the European Medicines Agency.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the
science of ribosome modulation, leveraging both 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 clinical development focusing on cystic
fibrosis. ELX-02 is an investigational drug that has not been
approved by any global regulatory body. 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 the expected timing of
trials and results from clinical studies of our product candidate
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 quarterly period ended June 30, 2021, 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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