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 nine months ended September 30, 2020 and provided a
business update.
“Having resumed our Phase 2 cystic fibrosis
clinical trials in Europe, Israel and the U.S., we now expect to
report top line data in the first half of 2021, contingent on no
further disruptions due to the COVID-19 pandemic. It is our highest
priority 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,” said Dr. Gregory Williams,
Chief Executive Officer of Eloxx Pharmaceuticals. “We are pleased
that, following several planned meetings, the independent Safety
Review Committees for this clinical trial program have allowed dose
escalation up to the highest dose level and, to date, no drug
related serious adverse events have been reported.”
Company
Updates
- On October 27, 2020, we announced the publication of two
manuscripts on ELX-02 and our ERSG library in two leading journals.
- A publication entitled: “ELX-02: an investigational
read-through agent for the treatment of nonsense mutation-related
genetic disease” in the Expert Opinion on Investigational Drugs
Journal.
- The review by Professor Eitan Kerem M.D., Senior Attending
Physician at the Hadassah CF Center in Jerusalem, Israel and Senior
Medical Consultant for Eloxx, details the development of ELX-02 for
the restoration of functional protein in nonsense-mediated disease
in support of our ongoing Phase 2 clinical trials in cystic
fibrosis.
- A publication entitled: “Intravitreal administration of small
molecule read-through agents demonstrate functional activity in a
nonsense mutation mouse model” was published in the Journal of
Experimental Eye Research.
- Multiple small molecules in our ERSG library mediate
dose-dependent read-through at the back of the eye after a single
intravitreal injection.
- Collectively, these manuscripts demonstrate the wide-ranging
potential of our small molecule read-through approach to rare
genetic disorders mediated by nonsense mutations, from targeted
delivery for inherited retinal disorders to systemic delivery for
multi-system disorders like cystic fibrosis. IND enabling studies
are also underway for our library of ERSG compounds in ADPKD.
- On October 22, 2020, we announced that we had presented data
from two scientific abstracts at the North American Cystic Fibrosis
Virtual Conference (NACFC). The two abstracts were also showcased
in the NACFC virtual poster gallery and electronically published as
a supplement to Pediatric Pulmonology.The details for the two
ELX-02 poster presentations are:Poster Session Presentation Title:
“ELX-02 Generates Protein Via Premature Stop Codon
Read-through Without Inducing Native Stop Codon Read-through
Proteins” Poster #: 433Presenter: Dr. Dan
Crawford, Eloxx Pharmaceuticals○ ELX-02 produces
significant read-through of premature stop codons leading to full
length proteins, demonstrated using DMS-114 cells with the R213X
nonsense mutation in the TP53 gene.○ Using three
complementary techniques, no evidence of native stop codon
read-through products could be detected. These data suggest that
ELX-02 does not promote native stop codon read-through at
concentrations relevant to premature stop codon read-though.○
The results of studies are consistent with the acceptable
tolerability profile of ELX-02 across preclinical and clinical
studies to date.Poster Session Presentation Title: “CFTR
Restoration By ELX-02 Across CF Nonsense Genotypes: Utilizing
Patient-Derived Organoids to Survey Responsive
Alleles”Poster #: 383Presenter: Dr. Matthew
Goddeeris, Eloxx Pharmaceuticals○ The patient derived
organoid CFTR FIS assay has enabled the screening of a wide
selection of cystic fibrosis nonsense alleles representing >75%
of the cystic fibrosis nonsense population. Using this method, we
continue to identify new responsive genotypes.○ The response
of W1282X patient-derived organoids to ELX-02 mediated through
read-through positively correlates with CFTR mRNA
expression.○ Increasing the available CFTR mRNA pool through
inhibition of nonsense mediated decay has a synergistic effect on
ELX-02 mediated functional CFTR read-through.○ These results
help guide the interpretation of the patient-derived organoid CFTR
FIS assay data by highlighting the importance of considering CFTR
expression differences across patient-derived organoids for the
applicability of ELX-02 as a potential therapeutic option for
cystic fibrosis patients with nonsense alleles.
- On September 14, 2020, we announced that Professor Eitan Kerem,
M.D. had joined the Company as a Senior Consultant. Dr.
Kerem will continue to advise on our cystic
fibrosis program focusing on strategic leadership, cystic fibrosis
medical safety review and medical communications, as well as
liaising with patient advocacy groups and regulatory authorities.
Dr. Kerem joined Eloxx upon his retirement from Hadassah Medical
Center where he most recently served as Head of Pediatrics and
Professor of Pediatrics at Hebrew University Hadassah Medical
School. Dr. Kerem was a board member of the European Cystic
Fibrosis Society where he contributed to the development of the
European Cystic Fibrosis Registry and was the President of CIPP,
the Annual International Congress on Pediatric
Pulmonology. Dr. Kerem was previously a member of the
editorial boards of the leading journals in the field of
pulmonology, “Pediatric Pulmonology”,
"Chest" and the “American Journal of
Respiratory and Critical Care Medicine”.
- 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” was published in
the August 2020 edition of 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.
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.
- 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 CFF’s Therapeutics Development
Network.
- In Europe, the European Cystic Fibrosis Society Clinical Trial
Network has given our trial a “high priority” ranking.
- 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.
- The independent Safety Review Committees have held several
planned meetings and approved dose escalation up to the highest
dose level. To date, no drug related serious adverse events have
been reported.
- We expect to report top line data from our proof of concept
Phase 2 program in the first half of 2021, which is contingent on
no further disruptions due to COVID-19.
ADPKD Kidney
Program
- 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 efforts 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.
Ocular Program
- 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.
- 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.
Third Quarter 2020
Financial Results
As of September 30, 2020, we had cash and cash
equivalents of $30.6 million, which we expect will be sufficient to
fund our operations through the end of 2021.
For the three months ended September 30, 2020,
we incurred a net loss of $6.6 million or $0.16 per share, which
includes $1.4 million in non-cash stock-based compensation. For the
same period in the prior year, we incurred a net loss of $12.9
million, or $0.32 per share.
Our research and development expenses were $3.2 million for the
three months ended September 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
$6.8 million. The quarter to quarter decrease in R&D
expenses of $3.6 million was driven by reduced professional service
fees primarily the result of the temporary pause in our clinical
trials due to COVID-19, and reduced headcount and related salaries
for the 2020 period.
Our general and administrative expenses were $3.1 million for
the three months ended June 30, 2020, which includes $1.1 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 headcount and
professional services costs.
First Nine Months 2020
Financial Results
For the nine months ended September 30, 2020, we
incurred a net loss of $28.5 million, or $0.71 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 $5.3 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 $39.2 million, or $1.05 per
share.
Our research and development expenses were $11.3 million for the
nine months ended September 30, 2020, which includes $0.8 million
in non-cash expense related to stock-based compensation. For the
same period in the prior year, R&D expenses were
$20.2 million. The year over year decrease in R&D expenses
of $8.9 million was driven by reduced professional service fees
primarily the result of the temporary 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 $12.3 million for
the nine months ended September 30, 2020, which includes $4.5
million in non-cash expense related to stock-based compensation.
For the same period in the prior year, G&A expenses were $18.9
million. The decrease of $6.6 million was primarily driven by lower
headcount and professional services costs.
Conference Call and Webcast
Information:Date: Thursday, November 5,
2020Time: 4:30 p.m. ETDomestic Dial-in
Number: (866) 913-8546International Dial-in
Number: (210) 874-7715Conference ID:
9467336Live 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/gucnkjge.
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. 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 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 Ryan203-274-2825
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, |
|
December
31, |
|
|
2020 |
|
2019 |
ASSETS |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
30,592 |
|
|
$ |
22,493 |
|
Marketable securities |
|
|
— |
|
|
|
33,783 |
|
Restricted cash |
|
|
52 |
|
|
|
43 |
|
Prepaid expenses and other current assets |
|
|
1,568 |
|
|
|
1,390 |
|
Total current assets |
|
|
32,212 |
|
|
|
57,709 |
|
Property and
equipment, net |
|
|
149 |
|
|
|
201 |
|
Operating
lease right-of-use asset |
|
|
551 |
|
|
|
924 |
|
Other
long-term assets |
|
|
30 |
|
|
|
113 |
|
Total
assets |
|
$ |
32,942 |
|
|
$ |
58,947 |
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
638 |
|
|
$ |
1,871 |
|
Accrued expenses |
|
|
3,091 |
|
|
|
4,655 |
|
Current portion of long-term debt |
|
|
4,917 |
|
|
|
4,336 |
|
Advances from collaboration partners |
|
|
805 |
|
|
|
403 |
|
Current portion of operating lease liability |
|
|
496 |
|
|
|
499 |
|
Taxes payable |
|
|
38 |
|
|
|
43 |
|
Total current liabilities |
|
|
9,985 |
|
|
|
11,807 |
|
Long-term
debt |
|
|
7,823 |
|
|
|
10,502 |
|
Operating
lease liability |
|
|
56 |
|
|
|
425 |
|
Total
liabilities |
|
|
17,864 |
|
|
|
22,734 |
|
Stockholders’ equity: |
|
|
|
|
Preferred stock, $0.01 par value per share, 5,000,000 shares
authorized, no shares issued or outstanding as of September 30,
2020 or December 31, 2019 |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,343,181 and 40,186,469 shares issued, and 40,150,530
and 40,030,763 shares outstanding as of September 30, 2020 and
December 31, 2019, respectively |
|
|
403 |
|
|
|
402 |
|
Common stock in treasury, at cost, 192,651 and 155,706 shares as of
September 30, 2020 and December 31, 2019, respectively |
|
|
(1,825 |
) |
|
|
(1,703 |
) |
Additional paid-in capital |
|
|
181,969 |
|
|
|
174,515 |
|
Accumulated other comprehensive income |
|
|
— |
|
|
|
18 |
|
Accumulated deficit |
|
|
(165,469 |
) |
|
|
(137,019 |
) |
Total stockholders’ equity |
|
|
15,078 |
|
|
|
36,213 |
|
Total
liabilities and stockholders’ equity |
|
$ |
32,942 |
|
|
$ |
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 September 30, |
|
Nine Months Ended September 30, |
|
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
3,231 |
|
|
$ |
6,801 |
|
|
$ |
11,308 |
|
|
$ |
20,160 |
|
General and administrative |
|
|
3,065 |
|
|
|
5,978 |
|
|
|
12,347 |
|
|
|
18,907 |
|
Restructuring charges |
|
|
— |
|
|
|
— |
|
|
|
3,994 |
|
|
|
— |
|
Total operating expenses |
|
|
6,296 |
|
|
|
12,779 |
|
|
|
27,649 |
|
|
|
39,067 |
|
Loss from
operations |
|
|
(6,296 |
) |
|
|
(12,779 |
) |
|
|
(27,649 |
) |
|
|
(39,067 |
) |
Other
expense, net |
|
|
321 |
|
|
|
96 |
|
|
|
801 |
|
|
|
174 |
|
Net
loss |
|
$ |
(6,617 |
) |
|
$ |
(12,875 |
) |
|
$ |
(28,450 |
) |
|
$ |
(39,241 |
) |
|
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
|
$ |
(0.16 |
) |
|
$ |
(0.32 |
) |
|
$ |
(0.71 |
) |
|
$ |
(1.05 |
) |
Weighted
average number of shares of common stock used in computing net loss
per share, basic and diluted |
|
|
40,142,178 |
|
|
|
39,944,324 |
|
|
|
40,115,351 |
|
|
|
37,394,310 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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