Eloxx Pharmaceuticals Reports First Quarter 2020 Financial and Operating Results and Provides Business Update
May 07 2020 - 4:01PM
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 months ended March 31, 2020 and provided a business update.
“Our highest priority is to resume and complete
our Phase 2 proof of concept clinical trial program for ELX-02 in
cystic fibrosis, as we believe these data represent a substantial
value inflection point for the Company. As previously announced,
these trials have been temporarily paused in response to the
COVID-19 global pandemic in support of global healthcare providers
and our shared commitment to ensure patient safety,” said Dr.
Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals.
“We are working very closely with our clinical investigators and
study sites to ensure that we can resume and complete our Phase 2
trials as rapidly as possible and report top line results.”
Company Updates
- We are pleased to announce today that our scientific manuscript
titled: “ELX-02 generates protein via premature stop codon
read-through without inducing native stop codon read-through
protein” has been accepted for publication 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. The pre-publication version of the manuscript can be
found within the “Fast Forward” section of the Journal’s
website.
- 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.
- On March 25, 2020, we announced that enrollment in our Phase 2
clinical trials for ELX-02 in cystic fibrosis has been temporarily
paused in response to the COVID-19 global pandemic. Our goals are
to avoid unnecessary exposure in at-risk populations, to maintain
the integrity of our study data and to support global healthcare
providers in their commitment to ensure patient safety. Public
health authorities worldwide have recommended that people at high
risk stay at home as much as possible, cancel non-essential
doctor’s visits and avoid unnecessary exposure to people and public
spaces. Cystic fibrosis patients, especially those with nonsense
mutations, have compromised lung function and may be at increased
risk of severe illness in the event of a COVID-19 infection.
- On February 24, 2020, our Board of Directors approved a
leadership and organizational realignment intended to reduce
operating expenses and extend the Company’s cash runway to the end
of 2021.
Cystic Fibrosis Phase 2 Program
- Our Phase 2 program consists of two trials, one enrolling
patients at sites in Europe and Israel and the second in the U.S.o
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).o 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 pleased with our participation 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
- ELX-02 results from the first cohort of the Phase 2 study in
the treatment of patients with nonsense mutation-mediated
nephropathic cystinosis met the primary safety endpoint and the
reductions in white blood cell cystine provided a clear indication
of biologic activity. These data provide human clinical proof of
concept for ELX-02 and de-risk other clinical applications of our
ERSG library using this dosage range. These encouraging results
provide the basis for expanding our studies to additional kidney
diseases caused by nonsense mutations, such as ADPKD.o 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. 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.
- We are pleased to report that using a reporter assay we have
already observed dose-dependent read-through with our ERSGs across
the most common PKD1 alleles and have now demonstrated
dose-dependent read-through across the most common PKD2 alleles. We
are now applying this information to our functional model efforts
in order to confirm that the read-through we observe has an impact
on cyst formation and growth. Our Cystic Fibrosis platform has
highlighted the utility of organoid technology to assess function
in a translational model. Similarly, for ADPKD, organoids derived
from patient cells or induced pluripotent stem cells can be
differentiated in a manner that recapitulates the cellular
diversity of the kidney and generate the cysts characteristic of
the disease state.
- Using a patient-derived organoid with the most common PKD2
nonsense allele, we have observed encouraging results of reduced
cystogenesis.
- Results of our preclinical research to date demonstrate that a
read-through approach can have a direct impact on a meaningful
metric of ADPKD progression, cyst number. We intend to evaluate
additional models of ADPKD and, with positive results, advance
toward an IND submission.
Ocular Program
Update
- In our ocular program, focusing on inherited retinal disorders,
we have reported that multiple ERSG compounds have demonstrated
dose-dependent read-through using our in vitro assay platform, and
an acceptable intravitreal tolerability in animal models. We have
achieved an important preclinical milestone demonstrating an
increase in pigment, an indication of functional restoration of
OCA2, after a single intravitreal injection of Eloxx ERSGs. This
outcome demonstrates that ERSG compounds can reach inherited
retinal disorder-relevant tissue layers beyond the photoreceptors.
- On May 6, 2020, we presented new preclinical data in a
scientific presentation at the Association for Research in
Vision 2020 (ARVO 2020) Virtual Meeting
in a presentation entitled “Intravitreal administration of
small molecule read-through agents demonstrate functional activity
in a nonsense mutation mouse model”. This presentation
described our studies in a mouse model with a naturally occurring
nonsense mutation in the OCA2 gene which results in a form of
albinism present in human type 2 oculocutaneous albinism. In this
model, the R262X mutation results in a lack of OCA2 channel protein
which is needed to establish the pH of the organelle that produces
pigment, the melanosome. The results showed a significant increase
in melanin production which validates the potential to promote
read-through activity in our target tissue via intravitreal
injection.
- Our intravitreal read-through approach provides the opportunity
to reach the totality of the retina. To extend the duration of the
delivery, our team is actively working to achieve a desired
sustained release formulation. We are exploring several
biodegradable, controlled release, polymer technologies and 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 against nonsense mutations in disease
causative genes such as USH2a, Myo7a, CEP290 and PDE6B, 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 agency for therapeutic use which is currently in Phase 2
clinical trials in cystic fibrosis.
First Quarter 2020 Financial Results
As of March 31, 2020, we had cash, cash
equivalents and marketable securities of $44.0 million, which we
expect will be sufficient to fund our operations through the end of
2021. The cash balance as of March 31, 2020 does not include the
loan of $800,000 which we received through the U.S. SBA’s “Paycheck
Protection Program.”
For the three months ended March 31, 2020, we
incurred a loss of $13.9 million or $0.35 per share, which includes
$4.0 million in restructuring charges associated with our
realignment, $2.1 million of which was non-cash stock-based
compensation. Net loss also includes $1.9 million in non-cash
stock-based compensation from ongoing operations. For the same
period in the prior year, we incurred a net loss of $11.9 million,
or $0.33 per share.
Our research and development expenses were $4.5 million for the
three months ended March 31, 2020, which includes $0.2 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $6.0 million.
The quarter to quarter decrease in R&D expenses of $1.5 million
was driven by reduced professional service fees and stock-based
compensation, offset by an increase in headcount and related
salaries for a portion of the 2020 period.
Our general and administrative expenses were $5.2 million for
the three months ended March 31, 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 $6.0 million.
The decrease was primarily driven by lower non-cash stock-based
compensation and other infrastructure-related costs.
Conference Call and Webcast
Information:Date: Thursday,
May 7, 2020Time: 4:30 p.m. ETDomestic
Dial-in Number: (866) 913-8546International
Dial-in Number: (210) 874-7715Conference
ID: 7798542Live 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/tgendj2c
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 body. Eloxx’s preclinical
candidate pool consists of a library of novel ERSG drug candidates
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 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 203-274-2825barbarar@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) |
|
|
|
|
|
|
|
March 31, |
|
December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
25,875 |
|
|
$ |
22,493 |
|
Marketable securities |
|
|
18,082 |
|
|
|
33,783 |
|
Restricted cash |
|
|
43 |
|
|
|
43 |
|
Prepaid expenses and other current assets |
|
|
1,927 |
|
|
|
1,390 |
|
Total current assets |
|
|
45,927 |
|
|
|
57,709 |
|
Property and equipment, net |
|
|
182 |
|
|
|
201 |
|
Operating lease right-of-use asset |
|
|
822 |
|
|
|
924 |
|
Other long-term assets |
|
|
110 |
|
|
|
113 |
|
Total assets |
|
$ |
47,041 |
|
|
$ |
58,947 |
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
1,389 |
|
|
$ |
1,871 |
|
Accrued expenses |
|
|
3,568 |
|
|
|
4,655 |
|
Current portion of long-term debt |
|
|
4,772 |
|
|
|
4,336 |
|
Advances from collaboration partners |
|
|
805 |
|
|
|
403 |
|
Current portion of operating lease liability |
|
|
507 |
|
|
|
499 |
|
Taxes payable |
|
|
43 |
|
|
|
43 |
|
Total current liabilities |
|
|
11,084 |
|
|
|
11,807 |
|
Long-term debt |
|
|
9,385 |
|
|
|
10,502 |
|
Operating lease liability |
|
|
315 |
|
|
|
425 |
|
Total liabilities |
|
|
20,784 |
|
|
|
22,734 |
|
Stockholders’ equity: |
|
|
|
|
Preferred stock, $0.01 par value per share, 5,000,000 shares
authorized, no shares issued or outstanding as of March 31,
2020 or December 31, 2019 |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,316,034 and 40,186,469 shares issued and
40,125,454 and 40,030,763 shares outstanding as of March 31,
2020 and December 31, 2019, respectively |
|
|
403 |
|
|
|
402 |
|
Common stock in treasury, at cost, 190,580 and 155,706 shares as
of March 31, 2020 and December 31, 2019, respectively |
|
|
(1,819 |
) |
|
|
(1,703 |
) |
Additional paid-in capital |
|
|
178,573 |
|
|
|
174,515 |
|
Accumulated other comprehensive income |
|
|
65 |
|
|
|
18 |
|
Accumulated deficit |
|
|
(150,965 |
) |
|
|
(137,019 |
) |
Total stockholders’ equity |
|
|
26,257 |
|
|
|
36,213 |
|
Total liabilities and stockholders' equity |
|
$ |
47,041 |
|
|
$ |
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 March 31, |
|
|
|
2020 |
|
|
|
2019 |
|
Operating expenses: |
|
|
|
|
Research and development |
|
$ |
4,549 |
|
|
$ |
6,019 |
|
General and administrative |
|
|
5,224 |
|
|
|
5,958 |
|
Restructuring charges |
|
|
3,994 |
|
|
|
— |
|
Total operating expenses |
|
|
13,767 |
|
|
|
11,977 |
|
Loss from operations |
|
|
(13,767 |
) |
|
|
(11,977 |
) |
Other expense (income), net |
|
|
179 |
|
|
|
(60 |
) |
Net loss |
|
$ |
(13,946 |
) |
|
$ |
(11,917 |
) |
|
|
|
|
|
Net loss per share, basic and diluted |
|
$ |
(0.35 |
) |
|
$ |
(0.33 |
) |
Weighted average number of shares of common stock used in computing
net loss per share, basic and diluted |
|
|
40,074,275 |
|
|
|
35,910,270 |
|
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