August 6, 2020 – 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 reported its financial
results for the three and six months ended June 30, 2020 and
provided a business update.
“We are pleased that enrollment in our Phase 2 cystic fibrosis
clinical trial has been resumed in Europe and Israel after having
been temporarily paused in response to the COVID-19 pandemic in
support of global healthcare providers and our shared commitment to
ensure patient safety. In the U.S., we are gratified to have
received orphan drug designation for ELX-02 for cystic fibrosis
from the FDA which confers substantial benefits to the program
including seven years of exclusivity upon marketing approval and a
potential waiver of the PDUFA application fee,” said Dr. Gregory
Williams, Chief Executive Officer of Eloxx Pharmaceuticals. “We are
working very closely with our clinical investigators and study
sites and evaluating additional sites where patient enrollment may
be feasible. Our highest priority is to complete our Phase 2 proof
of concept clinical trial program for ELX-02 in cystic fibrosis as
soon as possible, as we believe the data from these trials will
represent a substantial value inflection point for the
Company.”
Company Updates
- On August 4, 2020, we announced that the U.S. FDA had granted
orphan drug designation for ELX-02 for the treatment of cystic
fibrosis. The orphan drug designation confers several important
benefits to support development for 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 eligibility for marketing
exclusivity for seven years post approval, tax credits on qualified
U.S. clinical trial expenses, potential grant funding opportunities
that can be used for clinical trials and a potential waiver of the
PDUFA application fee, which is currently set at just under $3
million dollars. ELX-02 had previously been granted orphan
medicinal product designation by the European Medicines Agency for
the treatment of cystic fibrosis.
- Our scientific manuscript titled: “ELX-02 generates
protein via premature stop codon read-through without inducing
native stop codon read-through protein” has been published
in the August 2020 edition of by the Journal of
Pharmacology and Experimental Therapeutics. This
manuscript demonstrates that while ELX-02 mediates read-through of
premature stop codons, the fidelity of stop codons found at the end
of healthy transcripts is maintained. This indicates that
translation integrity is preserved with target-therapeutic exposure
of ELX-02, consistent with the favorable tolerability profile
across our preclinical and clinical datasets.
- On June 17, 2020, we announced that enrollment in our cystic
fibrosis Phase 2 clinical trial in Europe and Israel had been
resumed, while the trial in the U.S. remains paused due to the
COVID-19 pandemic.
- On May 6, 2020, we presented new preclinical data in a
scientific presentation at the virtual Association for
Research in Vision 2020 (ARVO 2020) Annual
Meeting in a presentation entitled “Intravitreal
administration of small molecule read-through agents demonstrate
functional activity in a nonsense mutation mouse model”.
The studies demonstrated restoration of melanin production in a
nonsense mouse model and helped validate the potential to promote
read-through activity in ocular tissues via intravitreal injection.
- In April 2020, we applied for and received a loan of
approximately $800,000 through the U.S. SBA’s “Paycheck Protection
Program”, which was a component of the CARES Act, signed into law
in late March. PPP loans are eligible for partial forgiveness,
which we will apply for, based on using the proceeds for payroll,
maintaining headcount, and other specified costs. The remaining
balance of the loan bears interest at the rate of 1% and is to be
repaid commencing at the end of 2020.
Cystic Fibrosis Phase 2 Program
- Our Phase 2 program consists of two trials, one currently
enrolling patients at sites in Europe and Israel and the second in
the U.S., where enrollment remains temporarily paused due to the
COVID-19 pandemic.
- In the U.S., partial funding is being provided by the Cystic
Fibrosis Foundation (CFF) for a portion of the trial and our
protocol has been sanctioned by the Cystic Fibrosis Therapeutics
Development Network (TDN).
- In Europe, the European Cystic Fibrosis Society Clinical Trial
Network (ECFS-CTN) has given our trial a “high priority”
ranking.
- Professor Eitan Kerem, M.D., Head of the Division of
Pediatrics, Children’s Hospital, Hadassah Medical Center, is the
Global Lead Investigator and 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.
- We are participating in the European HIT-CF consortium to
support the collection of cystic fibrosis patient-derived organoids
and the initiative to conduct a prospective clinical trial to
confirm the translational potential of the organoid model. The
intent of the program is to use these positive results to enroll
patients with responsive organoids in a prospective trial with
ELX-02. We believe this program will continue to expand the
application of organoid technology from drug discovery through drug
approval, and also offers possible label expansion
opportunities.
ADPKD Kidney Program Update
- ADPKD is a relatively common inherited genetic kidney disease,
which in the U.S. affects between 300,000 and 600,000 individuals
and is the leading cause of end stage renal disease.
- In our preclinical studies in ADPKD, we have observed
dose-dependent read-through with our ERSG compounds across the most
common PKD1 alleles and have expanded our studies to include PKD2.
Using a patient-derived organoid with the most common PKD2 nonsense
allele, we have repeated encouraging results of reduced
cystogenesis and also observe a reduction in cyst size. These
results demonstrate that a read-through approach potentially can
have a direct impact on meaningful metrics of ADPKD progression,
cyst number and size.
- We continue our effort in establishing and evaluating
functional models of ADPKD in order to confirm that the
read-through we observe has an impact on cyst formation and growth.
We are working on this program with Dr. Benjamin Freedman, a
Professor in the Division of Nephrology, Department of Medicine,
University of Washington, and a pioneer in ADPKD organoid
technology.
Ocular Program Update
- In our ocular program focusing on inherited retinal disorders,
our library of compounds has demonstrated dose-dependent
read-through using our in vitro assay platform, acceptable
intravitreal tolerability and restored protein production in an
animal model via ERSG intravitreal injection. Our intravitreal
read-through approach provides the opportunity to reach the
totality of the retina. We achieved an important preclinical
milestone which we reported on at this year’s virtual ARVO Meeting
by showing an increase in pigment, an indication of functional
restoration of OCA2, after a single intravitreal injection of Eloxx
ERSG compounds. This outcome demonstrates that ERSG compounds can
reach inherited retinal disorder-relevant tissue layers beyond the
photoreceptors.
- While we continue in our ocular program efforts, we are
gratified to observe that other researchers are also reporting on
the exciting potential of Eloxx compounds in this area. For
example, the group of Bikash Pattnaik at the University of
Wisconsin-Madison recently published in the American Journal of
Human Genetics that ELX-03 (also known as NB84) was sufficient to
rescue Kir7.1 channel function in a nonsense allele cellular model
derived from an individual with a form of pediatric blindness,
Lebers congenital amaurosis. These promising results represent
another example of how intravitreal delivery of an ERSG may have
broad application across nonsense-related inherited retinal
disorders through restoring production of essential
proteins.
- We are actively working to develop a sustained release
formulation for intravitreal injection and are exploring several
biodegradable, controlled release technologies. We are encouraged
by the in vitro release rates achieved to date which are consistent
with our target release profile of one to three months. When our
tissue exposure data is coupled with our ongoing sustained release
formulation efforts and the read-through potential we observe, we
are encouraged that the intravitreal ERSG approach could provide
restoration of critical proteins to preserve or restore visual
function across nonsense-related inherited retinal disorders.
ELX-02 is an investigational agent not approved by any
regulatory authority for therapeutic use, which is currently in
Phase 2 clinical trials in cystic fibrosis.
Second Quarter 2020 Financial Results
As of June 30, 2020, we had cash, cash
equivalents and marketable securities of $37.1 million, which we
expect will be sufficient to fund our operations through the end of
2021.
For the three months ended June 30, 2020, we
incurred a loss of $7.9 million or $0.20 per share, which includes
$2.0 million in non-cash stock-based compensation. For the same
period in the prior year, we incurred a net loss of $14.4 million,
or $0.40 per share.
Our research and development expenses were $3.5 million for the
three months ended June 30, 2020, which includes $0.3 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $7.3 million.
The quarter to quarter decrease in R&D expenses of $3.8 million
was driven by reduced professional service fees largely due to the
pause in our clinical trials due to COVID-19, and reduced headcount
and related salaries for a portion of the 2020 period.
Our general and administrative expenses were $4.1 million for
the three months ended June 30, 2020, which includes $1.7 million
in non-cash expense related to stock-based compensation. For the
same period in the prior year, G&A expenses were $7.0 million.
The decrease was primarily driven by lower headcount and
professional services costs.
First Half 2020 Financial Results
For the six months ended June 30, 2020, we
incurred a loss of $21.8 million or $0.54 per share, which includes
a one-time restructuring charge of $4.0 million associated with our
realignment in the first quarter (comprised of $2.1 in non-cash
stock based compensation and $1.9 million in cash severance) and
$3.8 million in non-cash stock-based compensation associated with
ongoing operations. For the same period in the prior year, we
incurred a net loss of $26.4 million, or $0.73 per share.
Our research and development expenses were $8.1 million for the
six months ended June 30, 2020, which includes $0.5 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $13.4 million.
The year over year decrease in R&D expenses of $5.3 million was
driven by reduced professional service fees largely due to the
pause in our clinical trials due to COVID-19, and reduced headcount
and related salaries for a portion of the 2020 period.
Our general and administrative expenses were $9.3 million for
the six months ended June 30, 2020, which includes $3.4 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, G&A expenses were $12.9 million. The
decrease of $3.6 million was primarily driven by lower headcount
and professional services costs.
Conference Call and Webcast Information:
Date: Thursday, August 6, 2020
Time: 2:00 p.m. ET
Domestic Dial-in Number: (866)
913-8546
International Dial-in Number: (210)
874-7715
Conference ID: 2191126
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/nxdhnad8.
Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company developing novel RNA-modulating drug
candidates (each designed to be a eukaryotic ribosomal selective
glycoside, or ERSG) 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, currently focusing on
cystic fibrosis. ELX-02 is an investigational drug that has not
been approved by any global regulatory authority. Eloxx’s
preclinical candidate pool consists of a library of novel ERSG drug
candidates identified based on read-through potential. Eloxx also
has preclinical programs focused on kidney diseases including
autosomal dominant polycystic kidney disease, as well as rare
ocular genetic disorders. Eloxx is headquartered in Waltham, MA,
with operations in Rehovot, Israel and Morristown, NJ. 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.
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED CONDENSED CONSOLIDATED BALANCE
SHEETS |
(Amounts in thousands, except share and per share
data) |
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
30,347 |
|
|
$ |
22,493 |
|
Marketable securities |
|
|
6,769 |
|
|
|
33,783 |
|
Restricted cash |
|
|
52 |
|
|
|
43 |
|
Prepaid expenses and other current assets |
|
|
1,800 |
|
|
|
1,390 |
|
Total current assets |
|
|
38,968 |
|
|
|
57,709 |
|
Property and equipment, net |
|
|
165 |
|
|
|
201 |
|
Operating lease right-of-use
asset |
|
|
678 |
|
|
|
924 |
|
Other long-term assets |
|
|
110 |
|
|
|
113 |
|
Total assets |
|
$ |
39,921 |
|
|
$ |
58,947 |
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS’ EQUITY |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
770 |
|
|
$ |
1,871 |
|
Accrued expenses |
|
|
3,485 |
|
|
|
4,655 |
|
Current portion of long-term debt |
|
|
5,149 |
|
|
|
4,336 |
|
Advances from collaboration partners |
|
|
805 |
|
|
|
403 |
|
Current portion of operating lease liability |
|
|
524 |
|
|
|
499 |
|
Taxes payable |
|
|
38 |
|
|
|
43 |
|
Total current liabilities |
|
|
10,771 |
|
|
|
11,807 |
|
Long-term debt |
|
|
8,704 |
|
|
|
10,502 |
|
Operating lease liability |
|
|
155 |
|
|
|
425 |
|
Total liabilities |
|
|
19,630 |
|
|
|
22,734 |
|
Stockholders’ equity: |
|
|
|
|
Preferred stock, $0.01 par value per share, 5,000,000 shares
authorized, no shares issued or outstanding as of June 30,
2020 or December 31, 2019 |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,326,906 and 40,186,469 shares issued, and
40,135,290 and 40,030,763 shares outstanding as of June 30,
2020 and December 31, 2019, respectively |
|
|
403 |
|
|
|
402 |
|
Common stock in treasury, at cost, 191,616 and 155,706 shares as of
June 30, 2020 and December 31, 2019, respectively |
|
|
(1,822 |
) |
|
|
(1,703 |
) |
Additional paid-in capital |
|
|
180,549 |
|
|
|
174,515 |
|
Accumulated other comprehensive income |
|
|
13 |
|
|
|
18 |
|
Accumulated deficit |
|
|
(158,852 |
) |
|
|
(137,019 |
) |
Total stockholders’ equity |
|
|
20,291 |
|
|
|
36,213 |
|
Total liabilities and
stockholders’ equity |
|
$ |
39,921 |
|
|
$ |
58,947 |
|
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS |
(Amounts in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
3,528 |
|
|
$ |
7,340 |
|
|
$ |
8,077 |
|
|
$ |
13,359 |
|
General and administrative |
|
|
4,058 |
|
|
|
6,971 |
|
|
|
9,282 |
|
|
|
12,929 |
|
Restructuring charges |
|
|
— |
|
|
|
— |
|
|
|
3,994 |
|
|
|
— |
|
Total operating expenses |
|
|
7,586 |
|
|
|
14,311 |
|
|
|
21,353 |
|
|
|
26,288 |
|
Loss from operations |
|
|
(7,586 |
) |
|
|
(14,311 |
) |
|
|
(21,353 |
) |
|
|
(26,288 |
) |
Other expense, net |
|
|
301 |
|
|
|
138 |
|
|
|
480 |
|
|
|
78 |
|
Net loss |
|
$ |
(7,887 |
) |
|
$ |
(14,449 |
) |
|
$ |
(21,833 |
) |
|
$ |
(26,366 |
) |
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(0.20 |
) |
|
$ |
(0.40 |
) |
|
$ |
(0.54 |
) |
|
$ |
(0.73 |
) |
Weighted average number of shares
of common stock used in computing net loss per share, basic and
diluted |
|
|
40,129,304 |
|
|
|
36,278,567 |
|
|
|
40,101,789 |
|
|
|
36,098,171 |
|
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