Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) a clinical-stage
biopharmaceutical company dedicated to the discovery and
development of novel therapeutics to treat cystic fibrosis,
cystinosis, inherited retinal disorders, 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, 2019 and provided a business update.
“We are pleased to be rapidly advancing our Phase 2
programs for ELX-02. Our IND for a Phase 2 clinical trial in cystic
fibrosis is open in the U.S. and the protocol has been endorsed by
the Cystic Fibrosis Foundation (CFF). We believe that the
positive data we have generated for ELX-02 in cystic fibrosis
patient-derived organoids substantially de-risk our Phase 2
program,” said Robert E. Ward, Chairman and CEO of Eloxx
Pharmaceuticals. “We have initiated a Phase 2 clinical trial in
Canada in cystinosis and we look forward to reporting top line data
from both of these programs this year.”
“We are gratified to have two leading CF
experts leading our Phase 2 clinical trial of ELX-02 in cystic
fibrosis patients: Dr. Ahmet Uluer, Director of the Adult Cystic
Fibrosis Program at Boston Children’s Hospital/Brigham and Women’s
Hospital CF Center, has agreed to be the lead study investigator in
the U.S., and Dr. Eitan Kerem, Head of the Division of Pediatrics,
Children’s Hospital, Hadassah Medical Center, will serve as the
Global Lead Investigator, said Dr. Greg Williams, Chief Operating
Officer of Eloxx. “ELX-02 is the only read-through agent to have
demonstrated positive results in organoids derived from cystic
fibrosis patients across the majority of nonsense mutations and
studies have shown the organoid model to be highly predictive of
clinical benefit. We believe these data de-risk our Phase 2
clinical trial program in cystic fibrosis.”
Cystic Fibrosis Program Updates
- Our IND in the U.S. is open and the CFF has endorsed the
protocol for our Phase 2 clinical in cystic fibrosis. We are on
track to report top line data from a Phase 2 clinical trial in
cystic fibrosis patients with the G542X CFTR mutation in the U.S.
and Europe this year.
- Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program
at the Boston Children’s Hospital/Brigham and Women’s Hospital CF
Center, will be the lead study investigator in the U.S., and Dr.
Eitan Kerem, Head of the Division of Pediatrics, Children’s
Hospital, Hadassah Medical Center, will serve as the Global Lead
Investigator.
- Our Phase 2 program will include up to 24 patients in the U.S.,
Europe and Israel. The protocol calls for 4 increasing doses of
ELX-02 ranging from 0.3 up to 3.0 mg/kg/day in order to identify an
optimal dose to carry into further development. While patient
safety is the primary endpoint in Phase 2, we will be evaluating
changes in sweat chloride at multiple ascending doses of ELX-02 as
the primary biomarker, which is consistent with other successful
Phase 2 programs for approved drugs to measure CFTR activity. We
will also be evaluating changes in FEV1.
- We are pleased with our participation in the European HIT-CF
program and the progress being made. The program is now in the
screening phase and has already identified around 40 cystic
fibrosis patients with nonsense mutations who are participating in
organoid development and further testing.
- Three abstracts have been accepted for presentation at the
North American Cystic Fibrosis Conference on October 31st through
November 2nd, 2019 in Nashville, Tennessee:
- “Pharmacokinetics, Safety, and Tolerability of Single
Ascending Doses of ELX-02 in Healthy Volunteers, a Potential
Treatment for Cystic Fibrosis Caused by Nonsense Mutations”
Thursday, October 31, 2019 11:15 a.m. – 1:45 p.m. CDT, Poster
Session 1
- “Pharmacokinetics, Safety, and Tolerability of Multiple
Ascending Doses of ELX-02 in Healthy Volunteers, a Potential
Treatment for Cystic Fibrosis Caused by Nonsense
Mutations” Thursday, October 31, 2019 11:15 a.m. –
1:45 p.m. CDT, Poster Session 1
- “Investigational Drug ELX-02 Mediates
CFTR Nonsense Mutation Read-through to Increase CFTR MRNA, CFTR
Protein Translation and CFTR Function” Friday,
November 1, 2019 2:15 p.m. – 3:50 p.m. CDT, Oral Workshop “New
& Emerging Therapies to Correct the Basic Defect”
Workshop
Cystinosis Program Updates
- We have initiated a Phase 2 clinical trial in cystinosis in
Canada. Dr. Paul Goodyer, a Professor of Pediatrics at McGill
University and recognized leader in hereditary renal disease, is
the principal investigator. The Phase 2 clinical trial is a
single arm, open label study designed to assess the safety,
tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of
subcutaneous (SC) ELX-02 in 6 patients with nephropathic cystinosis
with at least 1 nonsense mutation in the cystinosis gene. The
study will measure the dose-dependent effect of ELX-02 on cysteine
levels in white blood cells, the biomarker used in the development
of the most recently approved drugs for cystinosis. The study will
include three nominal doses ranging from 0.5 to 2.0 mg/kg/day in
order to identify an optimal dose to carry into further
development.
- Genome Quebec and Genome Canada are providing non-dilutive
funding for the Phase 2 clinical trial of ELX-02 in cystinosis. The
Cystinosis Research Foundation provided non-dilutive funding for
the preclinical phase of the program.
- We expect to report top line data in cystinosis early in the
fourth quarter of 2019.
- In support of the cystinosis program where many patients have
impaired renal function, we have successfully completed a renal
impairment study with ELX-02 in subjects with mild, moderate, and
severe renal impairment. To date, the preliminary results support
continuing our existing clinical programs and expanding our
research in other kidney disorders such as autosomal dominant
polycystic kidney disease. We expect to present the results from
the completed renal impairment study at a scientific meeting later
this year.
ELX-02 is an investigational agent not approved
by any regulatory agency for therapeutic use.
Second Quarter 2019 Financial Results
As of June 30, 2019, we had cash, cash
equivalents and marketable securities of $76.3 million, which we
expect will be sufficient to fund our operations through top line
data from our Phase 2 clinical trials in cystic fibrosis and
cystinosis in 2019 and our current and planned operations into the
first quarter of 2021.
For the three months ended June 30, 2019, we
incurred a loss of $14.4 million or $0.40 per share, which includes
$3.0 million non-cash expense related to stock-based compensation.
For the same period in the prior year, we incurred a net loss of
$13.4 million, or $0.42 per share.
Our research and development expenses were $7.3 million for the
three months ended June 30, 2019 which includes $2.3 million
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $4.2 million.
Quarter over quarter increases in R&D expenditures were
driven by our Phase 1 clinical studies, the renal impairment study,
and preparations for our multiple Phase 2 clinical trials, along
with pre-clinical and CMC operations.
Our general and administrative expenses were $7.0 million for
the three months ended June 30, 2019 which includes $0.7 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, G&A expenses were $9.6 million. The
year over year decrease in our G&A expenses was primarily
related to lower non-cash expense related to stock-based
compensation in the 2019 period, partially offset by an increase in
salary related costs reflective of our year over year headcount
growth and increases in professional service fees.
First Half 2019 Financial Results
For the six months ended June 30, 2019, we
incurred a loss of $26.4 million or $0.73 per share, which includes
$5.7 million in non-cash expense related to stock-based
compensation. For the same period in the prior year, we incurred a
net loss of $22.0 million, or $0.74 per share.
Our research and development expenses were $13.3 million for the
six months ended June 30, 2019 which includes $4.4 million in
non-cash expense related to stock-based compensation. For the same
period in the prior year, R&D expenses were $8.5 million. The
year over year increase in R&D expenditures was driven
primarily by growth in our clinical and preclinical operations.
Our general and administrative expenses were $12.9 million for
the six months ended June 30, 2019 which includes $1.3 million in
non-cash stock-based compensation. For the same period in the prior
year, G&A expenses were $13.0 million. Lower non-cash expense
related to stock-based compensation million in the 2019 period, was
partially offset by an increase in salary related costs reflective
of our year over year headcount growth and increases in
professional service fees.
Conference Call
Information:Date:
Wednesday, August 7, 2019Time: 8:30 a.m.
ETDomestic Dial-in
Number: (866)
913-8546International Dial-in Number: (210)
874-7715Conference ID: 1754316
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/9axiqnvt
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 203-274-2825barbarar@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) |
|
|
|
|
|
June 30, 2019 |
|
December 31, 2018 |
ASSETS |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
49,326 |
|
|
$ |
48,606 |
|
Marketable securities |
|
26,973 |
|
|
|
— |
|
Restricted bank deposit |
|
45 |
|
|
|
45 |
|
Prepaid expenses and other current assets |
|
1,392 |
|
|
|
1,690 |
|
Total current assets |
|
77,736 |
|
|
|
50,341 |
|
Property and equipment, net |
|
214 |
|
|
|
248 |
|
Operating lease right-of-use asset |
|
989 |
|
|
|
— |
|
Other long-term assets |
|
100 |
|
|
|
129 |
|
Total assets |
$ |
79,039 |
|
|
$ |
50,718 |
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
2,473 |
|
|
$ |
747 |
|
Accrued expenses |
|
5,773 |
|
|
|
6,938 |
|
Current portion of long-term debt |
|
2,231 |
|
|
|
— |
|
Current portion of operating lease liability |
|
473 |
|
|
|
— |
|
Taxes payable |
|
43 |
|
|
|
122 |
|
Total current liabilities |
|
10,993 |
|
|
|
7,807 |
|
Long-term debt |
|
12,306 |
|
|
|
— |
|
Operating lease liability |
|
516 |
|
|
|
— |
|
Stockholders’ equity: |
|
|
|
Common stock, $0.01 par value per share, 500,000,000 shares
authorized, 40,043,365 and 35,951,537 shares issued and 39,914,668
and 35,860,114 shares outstanding as of June 30, 2019 and
December 31, 2018, respectively |
|
402 |
|
|
|
360 |
|
Common stock in treasury, at cost, 128,697 and 91,423 shares at
June 30, 2019 and December 31, 2018, respectively |
|
(1,539 |
) |
|
|
(1,129 |
) |
Additional paid in capital |
|
168,848 |
|
|
|
129,825 |
|
Accumulated other comprehensive income |
|
24 |
|
|
|
— |
|
Accumulated deficit |
|
(112,511 |
) |
|
|
(86,145 |
) |
Total stockholders’ equity |
|
55,224 |
|
|
|
42,911 |
|
Total liabilities and stockholders' equity |
$ |
79,039 |
|
|
$ |
50,718 |
|
|
|
|
|
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED CONSOLIDATED INCOME STATEMENTS |
(Amounts in thousands, except share and per share
data) |
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
$ |
7,340 |
|
|
$ |
4,150 |
|
|
$ |
13,359 |
|
|
$ |
8,544 |
|
General and administrative |
|
6,971 |
|
|
|
9,560 |
|
|
|
12,929 |
|
|
|
12,953 |
|
Reverse merger related expenses |
|
— |
|
|
|
(167 |
) |
|
|
— |
|
|
|
594 |
|
Total operating expenses |
|
14,311 |
|
|
|
13,543 |
|
|
|
26,288 |
|
|
|
22,091 |
|
Loss from operations |
|
(14,311 |
) |
|
|
(13,543 |
) |
|
|
(26,288 |
) |
|
|
(22,091 |
) |
Other expense (income), net |
|
138 |
|
|
|
(137 |
) |
|
|
78 |
|
|
|
(94 |
) |
Net loss |
$ |
(14,449 |
) |
|
$ |
(13,406 |
) |
|
$ |
(26,366 |
) |
|
$ |
(21,997 |
) |
|
|
|
|
|
|
|
|
Basic and diluted net loss per share |
$ |
(0.40 |
) |
|
$ |
(0.42 |
) |
|
$ |
(0.73 |
) |
|
$ |
(0.74 |
) |
Weighted average number of Common Shares used in computing basic
and diluted net loss per share |
|
36,278,567 |
|
|
|
31,839,303 |
|
|
|
36,098,171 |
|
|
|
29,695,430 |
|
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