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, 2019 and provided a
business update.
“I am highly confident that we have the right
team and resources in place to achieve our clinical and portfolio
objectives. Our highest priority is to complete enrollment of our
Phase 2 clinical trials for Cystic Fibrosis and report topline data
in the first half of this year,” said Dr. Gregory Williams, Chief
Executive Officer of Eloxx Pharmaceuticals. “We believe that proof
of concept data in cystic fibrosis will be a substantial value
inflection point for the Company. Our recent organizational
realignment extends our cash runway through the end of 2021 and
strengthens our strategic flexibility.”
Organizational Realignment
- On February 26, 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.
- The realignment strengthens our commitment to cystic fibrosis
by ensuring that we have the appropriate resources and the
strategic flexibility to accomplish our key objectives, which
includes delivering topline Phase 2 proof of concept data for
ELX-02 in cystic fibrosis in the first half of 2020.
- These actions underscore our commitment to ensuring that we
deliver value to investors and fulfill our mission to provide
treatment options to patients with unmet medical needs in the most
safe and expeditious manner possible.
- The realignment included the succession of Dr. Gregory Williams
to the position of CEO and Neil Belloff, Esq. to COO in addition to
his role as General Counsel. Dr. Williams has a long track record
of success in developing and gaining the approval of new drugs at
companies including the Medicines Company, and most recently Tymlos
at Radius Health, which is now the number one prescribed anabolic
for postmenopausal osteoporosis.
Cystic Fibrosis Program Updates
- We expect to complete enrollment and report topline data from
our open-label Phase 2 clinical trial program for ELX-02 in cystic
fibrosis in the first half of 2020.
- Our Phase 2 program consists of two trials, one enrolling
patients at sites in Europe and Israel and the second in the U.S.
The expansion of our cystic fibrosis program to the U.S. has been
made possible in part by the funding 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.
- In a recent discussion with the members of the Cystic Fibrosis
Program Advisory Group, a joint advisory group with CFF, we had the
opportunity to share the positive results of the completed first
cohort of the Phase 2 clinical trial for ELX-02 in nephropathic
cystinosis. The Advisory Group viewed the safety and efficacy data
as exciting for cystinosis patients and was pleased that the trial
met the primary endpoint of safety. Together with the supportive
pharmacokinetics from this trial, these data further de-risk the
Phase 2 cystic fibrosis clinical trials.
- A scientific abstract for our Phase 2 clinical trial for ELX-02
in cystic fibrosis has been accepted for an Oral Presentation in a
Workshop titled: “Beyond modulators: approaches involving
gene editing or alternative channels” at the 43rd European
Cystic Fibrosis Conference June 3rd through the 6th, 2020 in Lyon
France.
- As previously reported, we completed an interim CMC review
meeting with the U.S. Food and Drug Administration and gained
alignment with the agency on manufacturing formulation process,
which we believe will be suitable for our expected drug supply
needs through completion of pivotal trials.
- 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. HIT-CF recently announced completion of the
first phase of the program with the collection of organoids from
patients at 47 of the biggest cystic fibrosis centers in 16
countries throughout Europe. Organoids from over 100 individuals
bearing rare nonsense mutations in the CFTR have been collected and
are being tested for responsiveness to ELX-02 in the laboratory.
The first dataset evaluated ELX-02 in 31 patient-derived organoids
and demonstrated activity in the majority of them. 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.
Kidney Program Updates
- In January 2020, we announced positive data from the first
cohort of the Phase 2 study of ELX-02 in the treatment of patients
with nonsense mutation-mediated nephropathic cystinosis. The
results of the first cohort met the primary safety endpoint and the
reductions in white blood cell cystine provided a clear indication
of biologic activity in these patients at nominal doses > 0.5
mg/kg/day. Following review of the safety and pharmacokinetic data
by an independent Safety Review Committee (SRC), the SRC approved
progressing to the second cohort that would enable enrolling
patients ages 12 and older. Due to study design limitations,
patients across all dose groups in cohort 1 had elevated and
uncontrolled pretreatment white blood cell cystine levels which
made it difficult to fully evaluate ELX-02-mediated white blood
cell cystine reductions. Therefore, we have discontinued this study
and will not proceed with the second cohort as contemplated in the
original protocol. We will continue to review these data with
a panel of scientific and clinical experts to determine appropriate
modifications for a possible new study design.
- The clear indications of biologic activity in the first cohort
of the Phase 2 study of ELX-02 in the treatment of patients with
nonsense mutation-mediated nephropathic cystinosis 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 also provide a basis for expansion to studies
of additional kidney diseases caused by nonsense mutations such as
autosomal dominant polycystic kidney disease (ADPKD).
- 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 intend
to evaluate additional cellular and/or animal models of ADPKD and,
with positive results, continue to advance this program toward an
IND submission.
- The results of the completed renal impairment study and
additional preclinical data in cystinosis were presented on
November 7, 2019, at the American Society of Nephrology (ASN)
Kidney Week Conference in Washington, DC 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”
- “Cystinosis nonsense mutation read-through mediated by
ELX-02 restores protein function using in vitro and in vivo
models”
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 by addressing new therapeutic indications.
- In our inherited retinal disease program, 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
disease-relevant tissue layers beyond the photorecepters.
- We will present preclinical data in a scientific presentation
at the Association for Research in Vision 2020 (ARVO
2020) Meeting on May 3 – 7, 2020 in Baltimore, MD.
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.
Fourth Quarter 2019 Financial Results
As of December 31, 2019, we had cash, cash
equivalents and marketable securities of $56.3 million, which we
expect will be sufficient to fund our operations through the end of
2021, well beyond topline data from our Phase 2 clinical trials in
cystic fibrosis.
For the three months ended December 31, 2019, we
incurred a loss of $11.6 million or $0.29 per share, which includes
$2.7 million non-cash expense related to stock-based compensation.
For the same period in the prior year, we incurred a net loss of
$14.0 million, or $0.40 per share.
Our research and development expenses were $5.7 million for the
three months ended December 31, 2019 which includes $0.5 million
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $6.5 million.
The quarter to quarter decrease in R&D expenditures was driven
by the timing of significant activities in our Phase 2 clinical
trials, along with pre-clinical and CMC costs, and lower non-cash
stock compensation.
Our general and administrative expenses were $5.8 million for
the three months ended December 31, 2019 which includes $2.2
million in non-cash expense related to stock-based compensation.
For the same period in the prior year, G&A expenses were $7.6
million. The change was primarily driven by decreases in
professional service fees and non-cash stock compensation
expense.
Full Year 2019 Financial Results
For the twelve months ended December 31, 2019,
we incurred a loss of $50.9 million or $1.34 per share, which
includes $11.3 million in non-cash expense related to stock-based
compensation. For the prior year, we incurred a net loss of $47.2
million, or $1.45 per share.
Our research and development expenses were $25.8 million for the
twelve months ended December 31, 2019 which includes $2.5 million
in non-cash expense related to stock-based compensation. For the
same period in the prior year, R&D expenses were $20.5 million.
The year over year increase in R&D expenditures was driven
primarily by growth in salaries and other personnel costs and
increases in clinical operations costs related to our Phase 2
clinical trials.
Our general and administrative expenses were $24.7 million for
the twelve months ended December 31, 2019 which includes $8.9
million in non-cash stock-based compensation. For the prior year,
G&A expenses were $26.5 million. The year over year decline in
our G&A expenses was primarily related to lower non-cash
expense related to stock-based compensation and a decrease in
infrastructure-related costs offset by increases in salaries and
other personnel costs.
Conference Call and Webcast
Information:Date:
Thursday, March 5, 2020Time: 4:30 p.m.
ETDomestic Dial-in
Number: (866)
913-8546International Dial-in Number: (210)
874-7715Conference ID: 3149964Live
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/8d96keug
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
CONSOLIDATED BALANCE SHEETS |
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
December 31, |
|
2019 |
|
2018 |
ASSETS |
|
|
|
Current
assets: |
|
|
|
Cash and cash equivalents |
$ |
22,493 |
|
|
$ |
48,606 |
|
Marketable securities |
|
33,783 |
|
|
|
— |
|
Restricted bank deposit |
|
43 |
|
|
|
45 |
|
Prepaid expenses and other current assets |
|
1,390 |
|
|
|
1,690 |
|
Total current assets |
|
57,709 |
|
|
|
50,341 |
|
Property and
equipment, net |
|
201 |
|
|
|
248 |
|
Operating
lease right-of-use asset |
|
924 |
|
|
|
— |
|
Other
long-term assets |
|
113 |
|
|
|
129 |
|
Total
assets |
$ |
58,947 |
|
|
$ |
50,718 |
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
Current
liabilities: |
|
|
|
Accounts payable |
$ |
1,871 |
|
|
$ |
747 |
|
Accrued expenses |
|
4,655 |
|
|
|
6,938 |
|
Current portion of long-term debt |
|
4,336 |
|
|
|
— |
|
Advances from collaboration partners |
|
403 |
|
|
|
— |
|
Current portion of operating lease liability |
|
499 |
|
|
|
— |
|
Taxes payable |
|
43 |
|
|
|
122 |
|
Total current liabilities |
|
11,807 |
|
|
|
7,807 |
|
Long-term
debt |
|
10,502 |
|
|
|
— |
|
Operating
lease liability |
|
425 |
|
|
|
— |
|
Total
liabilities |
|
22,734 |
|
|
|
7,807 |
|
Stockholders’ equity: |
|
|
|
Preferred stock, $0.01 par value per share, 5,000,000 shares
authorized, no shares issued or outstanding at December 31, 2019
and 2018 |
|
— |
|
|
|
— |
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,186,469 and 35,951,537 shares issued and 40,030,763
and 35,860,114 shares outstanding as of December 31, 2019 and 2018,
respectively |
|
402 |
|
|
|
360 |
|
Common stock in treasury, at cost, 155,706 and 91,423 shares as of
December 31, 2019 and 2018, respectively |
|
(1,703 |
) |
|
|
(1,129 |
) |
Additional paid-in capital |
|
174,515 |
|
|
|
129,825 |
|
Accumulated other comprehensive income |
|
18 |
|
|
|
— |
|
Accumulated deficit |
|
(137,019 |
) |
|
|
(86,145 |
) |
Total stockholders’ equity |
|
36,213 |
|
|
|
42,911 |
|
Total
liabilities and stockholders' equity |
$ |
58,947 |
|
|
$ |
50,718 |
|
|
|
|
|
ELOXX
PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS |
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
2019 |
|
2018 |
|
2019 |
|
2018 |
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
$ |
5,682 |
|
|
$ |
6,530 |
|
|
$ |
25,842 |
|
|
$ |
20,489 |
|
General and administrative |
|
5,806 |
|
|
|
7,584 |
|
|
|
24,713 |
|
|
|
26,482 |
|
Reverse merger related expenses |
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
594 |
|
Total operating expenses |
|
11,488 |
|
|
|
14,114 |
|
|
|
50,555 |
|
|
|
47,565 |
|
Loss from
operations |
|
(11,488 |
) |
|
|
(14,114 |
) |
|
|
(50,555 |
) |
|
|
(47,565 |
) |
Other
expense (income), net |
|
145 |
|
|
|
(209 |
) |
|
|
319 |
|
|
|
(502 |
) |
Loss before
income taxes |
|
(11,633 |
) |
|
|
(13,905 |
) |
|
|
(50,874 |
) |
|
|
(47,063 |
) |
Provision
for income taxes |
|
— |
|
|
|
122 |
|
|
|
— |
|
|
|
122 |
|
Net
loss |
$ |
(11,633 |
) |
|
$ |
(14,027 |
) |
|
$ |
(50,874 |
) |
|
$ |
(47,185 |
) |
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
$ |
(0.29 |
) |
|
$ |
(0.40 |
) |
|
$ |
(1.34 |
) |
|
$ |
(1.45 |
) |
Weighted-average number of shares of common stock used in computing
net loss per share, basic and diluted |
|
39,981,335 |
|
|
|
35,259,810 |
|
|
|
38,063,173 |
|
|
|
32,436,506 |
|
Eloxx Pharmaceuticals (NASDAQ:ELOX)
Historical Stock Chart
From Feb 2024 to Mar 2024
Eloxx Pharmaceuticals (NASDAQ:ELOX)
Historical Stock Chart
From Mar 2023 to Mar 2024