Full Press Release Details
Eloxx Pharmaceuticals
Reports Third Quarter 2019 Financial and Operating Results and Provides Business Update
-Presented Positive Phase 1 Data from SAD and MAD Studies
at North American Cystic Fibrosis Conference
-On track to report top line Phase 2 data in nephropathic
cystinosis this quarter, preliminary results show cystine reduction with 1mg/kg dose
-Actively enrolling Phase 2 cystic
fibrosis trial in IL with additional sites in EU and US to open this quarter. Expect fully enrolled Phase 2 cystic fibrosis
trials in the first quarter of 2020
-Company to present Phase 1 Renal Impairment
Study results and additional preclinical data at Kidney Week
-Maintained Strong Balance Sheet with $64.9
Million in Cash and Equivalents as of September 30, 2019
-Company to host webcast and conference call
today, Tuesday, November 5, 2019, at 4:30 pm ET
MA. - November 5, 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 and nine months ended September 30, 2019 and provided
"We are very pleased to have presented
the results of our MAD study at NACFC which reinforced the overall favorable tolerability profile and pharmacokinetics of ELX-02,
with no serious adverse events or nephrotoxity. To date, ELX-02 safety and tolerability has been evaluated in 105 healthy volunteers,"
said Dr. Gregory Williams, Chief Operating Officer of Eloxx Pharmaceuticals. "We are encouraged by the preliminary results
of our phase 2 clinical trial in nephropathic cystinosis, as ELX-02 has demonstrated a statistically significant reduction in white
blood cell cystine levels at the second dose level of 1.0 mg/kg. We are on track to complete this cohort and report top line data
this quarter. We believe that achievement of proof of concept for ELX-02 in cystinosis will provide a basis for expansion to studies
of additional kidney diseases, as well as increasing our probability of success in other clinical uses across this dose range."
"This week the Eloxx team will be presenting the results
from our completed Phase 1 Renal Impairment Study at Kidney Week. These data enable dose adjustment based on individual patient's
kidney function," said Robert Ward, Chairman and CEO of Eloxx Pharmaceuticals. "We are pleased to be actively enrolling
patients in our Cystic Fibrosis program, and we expect additional clinical trial sites to open in the US and EU this quarter and
full enrollment to be achieved during the first quarter of 2020. With this change, we expect to report top line data from these
trials in the first half of 2020."
During the quarter ended September 30, 2019, Eloxx appointed
Dr. Thomas Haverty as our Chief Medical Officer. Dr. Haverty has over 30 years of pharmaceutical and biotechnology experience leading
clinical research and operations teams with responsibility for large development portfolios in virtually all classes of molecules
and indications. Prior to joining us in his current capacity, Dr. Haverty, a Board-certified Nephrologist, served as a consultant
to us following a long tenured career at Johnson & Johnson, Schering Plough and Merck Research Labs. Dr. Haverty has successfully
led the development and approval of over 20 leading drugs.
Cystic Fibrosis Program Updates
Cystinosis Program Updates
Additional Development Programs
ELX-02 is an investigational
agent not approved by any regulatory agency for therapeutic use which is currently in Phase 2 clinical trials in cystic fibrosis
Third Quarter 2019 Financial Results
As of September 30, 2019, we had cash,
cash equivalents and marketable securities of $64.9 million, which we expect will be sufficient to fund our operations through
top line data from our Phase 2 clinical trials in cystic fibrosis and cystinosis and our current and planned operations into the
first quarter of 2021.
For the three months ended September 30,
2019, we incurred a loss of $12.9 million or $0.32 per share, which includes $3.0 million non-cash expense related to stock-based
compensation. For the same period in the prior year, we incurred a net loss of $11.2 million, or $0.32 per share.
Our research and development expenses were $6.8 million for
the three months ended September 30, 2019 which includes $0.7 million non-cash expense related to stock-based compensation. For
the same period in the prior year, R&D expenses were $5.4 million. Quarter to quarter increases in R&D expenditures
were largely driven by preparations for our multiple Phase 2 clinical trials, along with pre-clinical and CMC operations.
Our general and administrative expenses were $6.0 million for
the three months ended September 30, 2019 which includes $2.3 million in non-cash expense related to stock-based compensation.
For the same period in the prior year, G&A expenses were $5.9 million. Our G&A expenses were relatively flat year over
year with higher professional service fees offset by a decrease in salary related costs.
First Nine Months 2019 Financial Results
For the nine months ended September 30,
2019, we incurred a loss of $39.2 million or $1.05 per share, which includes $8.6 million in non-cash expense related to stock-based
compensation. For the same period in the prior year, we incurred a net loss of $33.2 million, or $1.05 per share.
Our research and development expenses were $20.2 million for
the nine months ended September 30, 2019 which includes $2.0 million in non-cash expense related to stock-based compensation. For
the same period in the prior year, R&D expenses were $14.0 million. The year over year increase in R&D expenditures was
driven primarily by growth in our clinical and preclinical operations.
Our general and administrative expenses were $18.9 million for
the nine months ended September 30, 2019 which includes $6.6 million in non-cash stock-based compensation. For the same period
in the prior year, G&A expenses were $18.9 million. Our G&A expenses were relatively flat year over year despite increases
in salary related costs reflective of our year over year headcount growth and increases in professional service fees, due to lower
non-cash expense related to stock-based compensation in the 2019 period.
Conference Call and Webcast Information:
Date: Tuesday, November 5, 2019
Domestic Dial-in Number: (866) 754-6374
International Dial-in Number: (210) 874-7715
Conference ID: 8339658
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/ni34pajh
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
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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