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 a business update.
“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
- Our Phase 2 program consists of two trials, one for clinical
investigators enrolling patients at sites in Europe and Israel. We
are actively enrolling patients in Israel and expect to open
European sites this quarter. Our second Phase 2 trial is in the
U.S., where today, Boston Children’s Hospital is open for
enrollment and we are expecting additional U.S. clinical trial
sites to open this quarter. The expansion of our cystic fibrosis
program to the U.S. has been made possible in part by the support
provided by the Cystic Fibrosis Foundation and by the sanctioning
of our protocol by the Cystic Fibrosis Therapeutics Development
Network (TDN).
- We are aware that there are available patients who have
expressed interest in the trials and the extended period for
opening clinical sites will mean that our cystic fibrosis trials
will be fully enrolled during the first quarter of 2020. As a
result, we are changing our guidance for top line data in cystic
fibrosis to the first half of 2020.
- During October 2019, we completed an interim CMC review meeting
with the U.S. Food and Drug Administration, and we have gained
alignment with the agency on our manufacturing formulation process,
which we believe will be suitable for our expected drug supply
needs through completion of our pivotal trials.
- Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program
at the Boston Children’s Hospital/Brigham and Women’s Hospital CF
Center, is the lead study investigator in the U.S., and Dr. Eitan
Kerem, Head of the Division of Pediatrics, Children’s Hospital,
Hadassah Medical Center, will serve as the Global Lead
Investigator.
- We are pleased with our participation in the European HIT-CF
program and the progress being made. The program will conduct
experiments with ELX-02 in organoids from cystic fibrosis patients
with nonsense mutations who are participating in the program.
- We presented positive data in three scientific presentations at
the North American Cystic Fibrosis Conference on October
31-November 2, 2019 in Nashville, Tennessee, from our completed
Phase 1 clinical trial program for ELX-02, including results from
the SAD study, the MAD study, and additional preclinical data for
ELX-02. These data demonstrated that ELX-02 increases functional
CFTR protein in patient-derived organoids and human bronchial
epithelial cells, Ussing chamber systems, FRT and transgenic mice,
and that it restores CFTR mRNA to healthy control levels. ELX-02
results in a pronounced increase in both CFTR protein expression
and mRNA stability and the preclinical efficacy-associated
exposures from the completed MAD study translate to the selected
Phase 2 clinical trial ascending dose ranges and exposures. ELX-02
shows linear and dose proportional PK following subcutaneous
administration twice weekly. To date, ELX-02 has been generally
well tolerated in clinical studies, with 105 volunteers exposed, no
reported SAEs or renal findings.
Cystinosis Program Updates
- Our Phase 2 cystinosis trial involves two sequential cohorts of
three patients with three escalating doses. A cohort is complete
when each patient has escalated through each of the three dosing
levels or if the Safety Review Committee (SRC) recommends halting
escalation. Following the completion of each dose, the SRC meets to
review the patient safety data prior to escalation to the next dose
level. The first two doses in the first cohort are complete and the
SRC has authorized us to start the final dose in this first cohort
which is currently ongoing. To date, based upon preliminary
results, ELX-02 has been well tolerated through the first two dose
levels, and at the second 1 mg/kg dose level, ELX-02 demonstrated a
statistically significant reduction in white blood cell cystine
levels. Upon completion of the first cohort, we will review with
the Principal Investigator and conduct a separate review with a
panel of cystinosis scientific and clinical experts before
reporting top line data later this year.
- We believe that the emerging profile of ELX-02 is suitable for
continued development and we intend to seek regulatory advice,
following top line results, on initiating an extension study for
patients in the first cohort and to expand the trial to include
sites and patients in the United States. We believe that proof of
concept for ELX-02 in cystinosis will provide a basis for expansion
to studies of additional kidney diseases caused by nonsense
mutations, as well as increase our probability of success in other
clinical uses of this dosage range.
- Genome Quebec and Genome Canada have provided non-dilutive
funding for the Phase 2 clinical trial of ELX-02 in cystinosis. The
Cystinosis Research Foundation provided non-dilutive funding for
the preclinical phase of the program.
- In support of the cystinosis program where many patients have
impaired renal function, we have successfully completed a renal
impairment study with ELX-02 in subjects with mild, moderate, and
severe renal impairment. The results of the renal impairment study
and additional preclinical data in cystinosis will be presented on
Thursday, November 7, 2019, at the American Society of Nephrology
(ASN) Kidney Week Conference in Washington, DC November 5-10, 2019
in two posters titled:
- “An open label-single dose, parallel-group study to
evaluate the effects of renal impairment on the pharmacokinetics of
ELX-02: Results from subjects with mild and moderate renal
impairment” - November 7, 2019 10:00 am – 12:00
pm
- “Cystinosis nonsense mutation read-through mediated by
ELX-02 restores protein function using in vitro and in vivo
models” – November 7, 2019 10:00 am – 12:00 pm
Additional Development Programs
- We have continued to develop our library of molecules and
believe that there are multiple opportunities to expand our
pipeline by advancing these novel molecules in new routes of
administration and or by addressing new therapeutic indications.
This quarter, we evaluated the three most prevalent autosomal
dominant polycystic kidney disease (ADPKD) nonsense mutations in an
in vitro read-through assay and have demonstrated significant
levels of read-through for ELX-02 and several library compounds,
which is the first step in our preclinical development toward IND.
We intend to evaluate additional cellular and/or animal models for
ADPKD and with positive results, advance towards IND.
- In our inherited retinal disease program, we are conducting
feasibility and IND enabling studies for several ERSG compounds
from our library. We believe that our intravitreal ERSG approach
could provide restoration of critical proteins to preserve or
restore visual function across the nonsense related inherited
retinal disorder landscape. We have demonstrated dose-dependent
read-through for our compounds using our in vitro assay platform.
During the quarter, we achieved another important milestone by
demonstrating that several of our compounds successfully restored
protein production in an animal model. We evaluated a mouse model
with a naturally occurring nonsense mutation in the OCA2 gene which
results in type 2 oculcutaneous albinism. In this model the R262X
mutation results in a lack of OCA2 channel protein which is needed
to establish the pH of an organelle that produces pigment, the
melanocyte. Loss of the functional OCA2 protein results in a lack
of pigment in the retinal pigment epithelium and the underlying
choroid layers.
- Multiple Eloxx ERSG compounds have demonstrated an increase in
pigment, an indication of functional restoration of OCA2, after a
single intravitreal injection of Eloxx ERSGs. We have used a dose
range of 50 to 200 nanograms per mouse eye which we believe
provides a window for efficacy based on our current tolerability
profile. This outcome demonstrates that ERSG compounds can reach
inherited retinal disease-relevant tissue layers beyond the
photorecepters. These data support that ERSG compounds may be
applicable in a wider range of inherited retinal disease that
impact cells deep to the neurosensory retina, including the retinal
pigment epithelium and choroid such as nonsense mediated Best and
choroideremia. Furthermore, we are encouraged that this is possible
through intravitreal delivery, which is the most common and least
technical administration and provides a global distribution of the
compound to the retina.
- This work has helped us progress our efforts to produce a
sustained release formulation for the program, and we are currently
evaluating sustained release technologies and potential partners.
- We continue to collaborate and engage in our multi-year
partnership with the Foundation Fighting Blindness (FFB) to support
its inherited retinal degenerative disease registry and educational
programs. In October 2019, we presented as part of the FFB New York
Vision Seminar and we believe that the ongoing research and
development consultation and support provided by the FFB will
accelerate our development programs that seek to support patients
with ocular disorders associated with nonsense mutations, an area
of high unmet medical need. At the FFB New York Vision Seminar, we
presented some of our in vivo proof-of-concept data in a nonsense
model of oculocutaneous albinism type 2.
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 and cystinosis.
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, 2019Time: 4:30 p.m.
ETDomestic Dial-in
Number: (866)
754-6374International Dial-in Number: (210)
874-7715Conference ID: 8339658Live
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 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; 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.
ELOXX
PHARMACEUTICALS, INC. AND SUBSIDIARIES |
|
UNAUDITED
CONDENSED CONSOLIDATED BALANCE SHEETS |
|
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
September 30, 2019 |
|
December 31, 2018 |
|
ASSETS |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
22,155 |
|
|
$ |
48,606 |
|
|
Marketable securities |
|
|
42,781 |
|
|
|
— |
|
|
Restricted bank deposit |
|
|
40 |
|
|
|
45 |
|
|
Prepaid expenses and other current assets |
|
|
1,878 |
|
|
|
1,690 |
|
|
Total current assets |
|
|
66,854 |
|
|
|
50,341 |
|
|
Property and
equipment, net |
|
|
220 |
|
|
|
248 |
|
|
Operating
lease right-of-use asset |
|
|
870 |
|
|
|
— |
|
|
Other
long-term assets |
|
|
113 |
|
|
|
129 |
|
|
Total
assets |
|
$ |
68,057 |
|
|
$ |
50,718 |
|
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
1,687 |
|
|
$ |
747 |
|
|
Accrued expenses |
|
|
5,145 |
|
|
|
6,938 |
|
|
Current portion of long-term debt |
|
|
3,488 |
|
|
|
— |
|
|
Advances from collaboration partners |
|
|
403 |
|
|
|
— |
|
|
Current portion of operating lease liability |
|
|
461 |
|
|
|
— |
|
|
Taxes payable |
|
|
43 |
|
|
|
122 |
|
|
Total current liabilities |
|
|
11,227 |
|
|
|
7,807 |
|
|
Long-term
debt |
|
|
11,193 |
|
|
|
— |
|
|
Operating
lease liability |
|
|
409 |
|
|
|
— |
|
|
Stockholders’ equity: |
|
|
|
|
|
Preferred stock, $0.01 par value per share, 5,000,000 shares
authorized, no shares issued and outstanding at September 30, 2019
and December 31, 2018 |
|
|
— |
|
|
|
— |
|
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,117,186 and 35,951,537 shares issued and 39,977,654
and 35,860,114 shares outstanding as of September 30, 2019 and
December 31, 2018, respectively |
|
|
404 |
|
|
|
360 |
|
|
Common stock in treasury, at cost, 139,532 and 91,423 shares at
September 30, 2019 and December 31, 2018, respectively |
|
|
(1,585 |
) |
|
|
(1,129 |
) |
|
Additional paid-in capital |
|
|
171,783 |
|
|
|
129,825 |
|
|
Accumulated other comprehensive income |
|
|
12 |
|
|
|
— |
|
|
Accumulated deficit |
|
|
(125,386 |
) |
|
|
(86,145 |
) |
|
Total stockholders’ equity |
|
|
45,228 |
|
|
|
42,911 |
|
|
Total
liabilities and stockholders' equity |
|
$ |
68,057 |
|
|
$ |
50,718 |
|
|
|
|
|
|
|
|
ELOXX
PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED
CONSOLIDATED INCOME STATEMENTS |
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
Three Months EndedSeptember 30, |
|
Nine Months EndedSeptember 30, |
|
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
6,801 |
|
|
$ |
5,415 |
|
|
$ |
20,160 |
|
|
$ |
13,959 |
|
General and administrative |
|
|
5,978 |
|
|
|
5,945 |
|
|
|
18,907 |
|
|
|
18,898 |
|
Reverse merger related expenses |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
594 |
|
Total operating expenses |
|
|
12,779 |
|
|
|
11,360 |
|
|
|
39,067 |
|
|
|
33,451 |
|
Loss from
operations |
|
|
(12,779 |
) |
|
|
(11,360 |
) |
|
|
(39,067 |
) |
|
|
(33,451 |
) |
Other
expense (income), net |
|
|
96 |
|
|
|
(199 |
) |
|
|
174 |
|
|
|
(293 |
) |
Net
loss |
|
$ |
(12,875 |
) |
|
$ |
(11,161 |
) |
|
$ |
(39,241 |
) |
|
$ |
(33,158 |
) |
|
|
|
|
|
|
|
|
|
Basic and
diluted net loss per share |
|
$ |
(0.32 |
) |
|
$ |
(0.32 |
) |
|
$ |
(1.05 |
) |
|
$ |
(1.05 |
) |
Weighted
average number of Common Shares used in computing basic and diluted
net loss per share |
|
|
39,944,324 |
|
|
|
35,005,979 |
|
|
|
37,394,310 |
|
|
|
31,485,067 |
|
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