Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis
August 04 2020 - 7:30AM
Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a clinical-stage
biopharmaceutical company dedicated to the discovery and
development of novel therapeutics to treat cystic fibrosis and
other diseases caused by nonsense mutations limiting production of
functional proteins, today announced that the U.S. Food and Drug
Administration (FDA) has granted the Company orphan drug
designation for ELX-02 for the treatment of Cystic Fibrosis.
The FDA’s Office of Orphan Drug Products grants orphan status to
support the development of medicines for underserved patient
populations, or rare disorders, that affect fewer than 200,000
people in the U.S. Orphan-drug designation qualifies Eloxx for
certain benefits, including seven years of market exclusivity upon
regulatory approval if received, exemption of FDA application fees,
tax credits on qualified U.S. clinical trials and eligibility for
grant funding opportunities that can be used for clinical trial
costs.
“We are pleased to have received orphan drug designation from
the FDA for ELX-02 in the U.S. Combined with our previous EMA
orphan medicinal product designation in Europe, and upon approvals
in the U.S. and the E.U., we will be able to obtain significant
periods of marketing exclusivity in these important markets and
provide treatment options to patients with high unmet medical
needs, particularly in patients with nonsense mutations for whom
there are few, if any, treatments available,” said Dr. Gregory
Williams, Chief Executive Officer of Eloxx Pharmaceuticals. “As
previously announced, we have resumed enrollment in our Phase 2
proof of concept clinical trials in Israel and Europe, with our
trial in the U.S. continuing to be temporarily paused due to the
COVID-19 pandemic. We look forward to reporting top line results as
soon as feasible, and believe that this will be a major value
inflection point for our Company.”
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; the impact of global
health concerns, such as the COVID-19 global pandemic, on our
ability to continue our clinical and preclinical programs and
otherwise operate our business effectively; 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 203-274-2825
barbarar@eloxxpharma.com
SOURCE: Eloxx Pharmaceuticals, Inc.
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