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 twelve months ended December 31, 2020 and provided a
business update.
“We are on track to report top line data from
our global Phase 2 cystic fibrosis clinical trials in the first
half of this year. In addition to Europe, Israel and the U.S., we
are opening additional clinical sites in Australia and Canada. 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 the CF Foundation has increased its financial
support for the global clinical trial program and that the
independent Safety Review Committees 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 February 9, 2021, we announced that a scientific manuscript
titled: “Targeting G542X CFTR
Nonsense Alleles With ELX-02 Restores CFTR Function in
Human-Derived Intestinal Organoids” was published in the
Journal of Cystic Fibrosis.
- Our evaluation of ELX-02 mediated read-through, using the
CFTR-dependent, Forskolin-induced swelling (FIS) assay across a
selection of G542X homozygous and heterozygous patient-derived
organoids, demonstrated that ELX-02 increased CFTR activity in a
dose-dependent fashion across a variety of forskolin induction
concentrations. These functional increases are similar to those
obtained with tezacaftor/ivacaftor in a F508del homozygous
organoid. Additionally, in some cases, ELX-02 treatment of these
patient-derived organoids resulted in about a 5-fold increase in
CFTR mRNA when compared with vehicle treated as measured using
Nanostring. These data support our Phase 2 clinical trial for
ELX-02 in cystic fibrosis patients with G542X alleles.
- On January 26, 2021, we announced that a scientific manuscript
titled: “A Randomized, Double-Blind, Placebo-Controlled,
Multiple Dose Escalation Study to Evaluate the Safety and
Pharmacokinetics of ELX-02 in Healthy Subjects” was
published in the journal Clinical Pharmacology in Drug
Development.
- In this Phase 1B multiple-ascending dose trial evaluating the
safety and pharmacokinetics of ELX-02 in healthy volunteers there
were no severe or serious side effects, which is consistent with
the favorable tolerability profile demonstrated across our
preclinical and clinical datasets.
- On January 20, 2021, we announced that a scientific manuscript
titled: “Phase 1 Renal Impairment Trial Results Enable
Targeted Individualized Dosing of ELX-02 in Nephropathic Cystinosis
Patients” was published in the Journal of Clinical
Pharmacology.
- This study was designed to define the relationship of eGFR and
drug exposure and urinary clearance in patients with renal
impairment. The results demonstrated that ELX-02 was well tolerated
by patients with renal insufficiency and nephropathic cystinosis
patients. Across increasing degrees of renal insufficiency with
reduced clearance, ELX-02 pharmacokinetics demonstrated increased
exposure and prolonged renal elimination proportional to eGFR. The
data from the renal impairment study enabled the development of an
eGFR-PK model of ELX-02 which was successfully used to implement
individualized daily dosing of ELX-02 in a Phase 2 study in
patients with nephropathic cystinosis to achieve the targeted
exposure.
- Collectively, our published scientific manuscripts support our
current Phase 2 global clinical trial for ELX-02 in cystic fibrosis
patients with nonsense alleles. Additionally, they 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 January 13, 2021, we announced that the CF Foundation
extended its partial funding for our clinical trial program beyond
the U.S. portion of the trial to include Europe and Israel.
Cystic Fibrosis Phase 2 Program
- We are on track to report top line data from our
proof-of-concept Phase 2 program in the first half of this
year.
- Our global Phase 2 program consists of two trials, one
currently enrolling patients at sites in Europe, Israel and opening
in Australia, and the second in the U.S. and opening in Canada.
- The Cystic Fibrosis
Foundation’s (CFF) partial funding of the U.S. trial has been
extended to include Europe and Israel and our protocol has been
sanctioned by CFF’s Therapeutics Development Network.
- Opening additional
clinical trial sites in Australia and Canada.
- 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 fourth and
highest dose level. To date, no drug related serious adverse events
have been reported.
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 building the models to evaluate the ADPKD nonsense patient
population, we enlisted the support of Dr. Benjamin Freedman,
Associate Professor of the Division of Nephrology at the University
of Washington. Dr. Freedman is an expert in differentiating induced
pluripotent stem cells into 3-dimensional kidney organoids, capable
of modeling cyst formation observed in ADPKD. We have modeled the
most prevalent ADPKD nonsense mutations in these cells, and we
anticipate providing updates on ELX-02’s ability to prevent or
reduce cysts in these organoids along with other program progress
over the coming year.
- In our preclinical studies in ADPKD, we have observed
dose-dependent read-through with our ERSG compounds across the most
common PKD2 alleles and have expanded our studies to include PKD1.
Using a patient-derived organoid with the most common PKD2 nonsense
allele, we have reproducibly observed reduced cystogenesis and cyst
size with administration of Eloxx ERSGs.
Ocular Program
- In order to expand our ocular
research footprint and ensure we are evaluating the most relevant
cellular and animal models of nonsense-mediated blindness we have
established research collaborations with ocular disease experts at
the University of Maryland, University of Wisconsin and UCLA, and
look forward to sharing more results as these programs
progress.
- 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 last 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 developing 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.
Fourth Quarter 2020 Financial Results
As of December 31, 2020, we had cash and cash
equivalents of $24.7 million, which we expect will be sufficient to
fund our operations though top line data and into the fourth
quarter.
For the three months ended December 31, 2020, we
incurred a net loss of $6.1 million or $0.15 per share, which
includes $1.3 million in non-cash stock-based compensation. For the
same period in the prior year, we incurred a net loss of $11.6
million, or $0.29 per share.
Our research and development expenses were $2.6 million for the
three months ended December 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
$5.9 million. The quarter-to-quarter decrease in R&D
expenses of $3.3 million was driven by reduced headcount and
related salaries as well as decreases in certain clinical and
pre-clinical research costs for the 2020 period.
Our general and administrative expenses were $3.1 million for
the three months ended December 31, 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 $5.6
million. The decrease was primarily driven by lower headcount and
professional services costs for the 2020 period.
Full Year 2020 Financial Results
For the twelve months ended December 31, 2020,
we incurred a net loss of $34.6 million, or $0.86 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 $6.6 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 $50.9 million, or $1.34 per
share.
Our research and development expenses were $14.6 million for the
twelve months ended December 31, 2020, which includes $1.0 million
in non-cash expense related to stock-based compensation. For the
same period in the prior year, R&D expenses were
$26.3 million. The year over year decrease in R&D expenses
of $11.7 million was driven by reduced headcount and related
salaries for a portion of the 2020 period as well as reduced costs
relating to certain clinical and preclinical activities.
Our general and administrative expenses were $14.8 million for
the twelve months ended December 31, 2020, which includes $5.6
million in non-cash expense related to stock-based compensation.
For the same period in the prior year, G&A expenses were $24.2
million. The decrease of $9.4 million was primarily driven by lower
headcount and professional services costs.
Conference Call
Information:Date: Thursday, March 11,
2021Time: 4:30 p.m. ETDomestic Dial-in
Number: (866) 913-8546International Dial-in
Number: (210) 874-7715Conference ID:
4772135Live 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/m65hdyjn.
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 StatementsThis 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) |
|
|
|
|
|
|
|
December 31, |
|
|
2020 |
|
2019 |
ASSETS |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
24,668 |
|
$ |
22,493 |
Marketable securities |
|
|
— |
|
|
33,783 |
Restricted cash |
|
|
56 |
|
|
43 |
Prepaid expenses and other current assets |
|
|
1,169 |
|
|
1,390 |
Total current assets |
|
|
25,893 |
|
|
57,709 |
Property and
equipment, net |
|
|
133 |
|
|
201 |
Operating
lease right-of-use asset |
|
|
421 |
|
|
924 |
Other
long-term assets |
|
|
30 |
|
|
113 |
Total assets |
|
$ |
26,477 |
|
$ |
58,947 |
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
481 |
|
$ |
1,871 |
Accrued expenses |
|
|
2,886 |
|
|
4,655 |
Current portion of long-term debt |
|
|
5,239 |
|
|
4,336 |
Advances from collaboration partners |
|
|
805 |
|
|
403 |
Current portion of operating lease liability |
|
|
389 |
|
|
499 |
Taxes payable |
|
|
38 |
|
|
43 |
Total current liabilities |
|
|
9,838 |
|
|
11,807 |
Long-term
debt |
|
|
6,376 |
|
|
10,502 |
Operating
lease liability |
|
|
33 |
|
|
425 |
Total
liabilities |
|
|
16,247 |
|
|
22,734 |
Total
stockholders’ equity |
|
|
10,230 |
|
|
36,213 |
Total liabilities and stockholders’ equity |
|
$ |
26,477 |
|
$ |
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 December 31, |
|
Year Ended December 31, |
|
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
2,640 |
|
|
$ |
5,855 |
|
|
$ |
14,590 |
|
|
$ |
26,349 |
|
General and administrative |
|
|
3,142 |
|
|
|
5,633 |
|
|
|
14,847 |
|
|
|
24,206 |
|
Restructuring charges |
|
|
24 |
|
|
|
— |
|
|
|
4,018 |
|
|
|
— |
|
Total operating expenses |
|
|
5,806 |
|
|
|
11,488 |
|
|
|
33,455 |
|
|
|
50,555 |
|
Loss from
operations |
|
|
(5,806 |
) |
|
|
(11,488 |
) |
|
|
(33,455 |
) |
|
|
(50,555 |
) |
Other
expense, net |
|
|
321 |
|
|
|
145 |
|
|
|
1,122 |
|
|
|
319 |
|
Net
loss |
|
$ |
(6,127 |
) |
|
$ |
(11,633 |
) |
|
$ |
(34,577 |
) |
|
$ |
(50,874 |
) |
|
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
|
$ |
(0.15 |
) |
|
$ |
(0.29 |
) |
|
$ |
(0.86 |
) |
|
$ |
(1.34 |
) |
Weighted
average number of shares of common stock used in computing net loss
per share, basic and diluted |
|
|
40,153,552 |
|
|
|
39,981,335 |
|
|
|
40,124,953 |
|
|
|
38,063,173 |
|
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