Full Press Release Details
Eloxx Pharmaceuticals
Reports First Quarter 2019 Financial and Operating Results and Provides Business Update
Positive new preclinical
data for Eloxx ERSG molecules presented at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting
May 2, 2019 demonstrated:
dependent restoration of missing protein of Usher Syndrome nonsense mutations,
pharmacokinetics in the retina by intravitreal injection
tolerability profile
On track to report top
line data from Phase 2 clinical trials for ELX-02 in cystic fibrosis and cystinosis in the U.S. and Europe in 2019
Company to host webcast
and conference call on Thursday, May 9, 2019 at 8:30 am ET
MA. - May 9, 2019 - 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 reported its financial results for the three months ended March 31, 2019 and provided a business update.
"We are very pleased to be completing the final cohort
of our multiple ascending dose study in the U.S. and rapidly advancing ELX-02 into Phase 2 clinical trials in cystic fibrosis and
cystinosis. We look forward to reporting top line data in the U.S. and Europe in 2019 and we believe that the positive data we
have generated for ELX-02 in cystic fibrosis patient-derived organoids substantially de-risk our Phase 2 program," said Robert
E. Ward, Chairman and CEO of Eloxx Pharmaceuticals. "Eloxx had the opportunity to share new positive data for several of
our investigational product candidates in inherited retinal diseases for the first time at ARVO, which was well received given
the high unmet medical need across nonsense mutation-bearing patients."
Cystic Fibrosis Program Updates
ELX-02 mediated protein increases have been previously demonstrated, this is the first demonstration reported in cystic fibrosis
patient organoids. Within this translational CF organoid model, ELX-02 dose-dependent increases in CFTR mRNA stability and
function can now be extended to the demonstration of accompanying increases of CFTR protein.
Previously, Eloxx presented positive data for ELX-02
at the North American Cystic Fibrosis Conference on October 18th, 2018 in Denver, Colorado in a poster presentation
titled: "Measuring mRNA levels in cystic fibrosis organoids with nonsense mutations following treatment with ELX-02,"
which demonstrated ELX-02 mediated dose responsive increases in CFTR function, as measured by FIS
swelling activity, which was found to correlate with increases in CFTR mRNA, as measured
by nanoString technology, with elevations above wild-type. ELX-02 appears to
increase the steady state concentrations of CFTR mRNA suggesting that ELX-02 may be modulating nonsense mediated decay.
These data demonstrate that ELX-02 promotes translation of functional CFTR and we believe, de-risks
our planned Phase 2 clinical trial of ELX-02 in cystic fibrosis patients with the G542X CFTR mutation on one or both alleles, which
is the second most common mutation globally and accounts for about 5% of the cystic fibrosis population.
ELX-02 is an investigational
agent not approved by any regulatory agency for therapeutic use.
Inherited Retinal Disease Program Updates
At the request of the Foundation Fighting
Blindness, Dr. Matthew Goddeeris, also made a presentation at the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit
in Vancouver, BC., on April 26, 2019, to review Eloxx Pharmaceuticals' read-through therapeutic approach to inherited retinal
disorders with its library of ERSG molecules, which was very well received.
First Quarter 2019 Financial Results
As of March 31, 2019, we had cash and cash
equivalents of $53.5 million. We expect our total cash and cash equivalents will be sufficient to fund our operations through top
line data from our Phase 2 clinical trials in cystic fibrosis and cystinosis for our lead investigational product, ELX-02, and
into the second quarter of 2020 based upon our current operating plans.
We incurred a loss for the three months ended March 31, 2019,
of $11.9 million or $0.33 per share, which includes $2.7 million non-cash expense related to stock-based compensation. For the
same period in the prior year, we incurred a net loss of $8.6 million, or $0.31 per share.
Our research and development expenses were $6.0 million for
the three months ended March 31, 2019 which includes $0.5 million non-cash expense related to stock-based compensation. For the
same period in the prior year, R&D expenses were $4.4 million. Quarter to quarter fluctuations were due to normal timing
Our general and administrative expenses were approximately $6.0
million for the three months ended March 31, 2019 compared to approximately $3.4 million for the same period in the prior year,
an increase of approximately $2.6 million. The increase in our general and administrative expenses was primarily related to non-cash
expense related to stock-based compensation of $2.1 million in the 2019 period, and an increase in our headcount and related salaries,
and professional service fees.
Conference Call Information:
Date: Thursday, May 9, 2019
Domestic Dial-in Number:
International Dial-in Number:
Live Webcast: accessible from the Company's website
at www.eloxxpharma.com under Events and Presentations or with this link:
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
Forward-Looking Statements
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.
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