Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, today reported
its financial results for the three and nine months ended September
30, 2021 and provided a business update.
“We continue to make significant progress across our portfolio
of therapeutic programs,” said Sumit Aggarwal, President and Chief
Executive Officer of Eloxx. “We are on track to report data from
the ongoing Phase 2 clinical trials for ELX-02 in the fourth
quarter of 2021. In addition, our preclinical programs continue to
advance demonstrating the potential of our oral RMAs. We intend to
provide additional updates on these programs in the coming
quarters.”
Third Quarter 2021 and Subsequent
Highlights
Class 1 Cystic Fibrosis
- Ongoing ELX-02 Phase 2 clinical
trials in CF patients affected by nonsense mutations in the CFTR
(CF transmembrane conductance regulator) gene are designed to
evaluate the safety of ELX-02 and assess short-term biological
activity in patients.
- We remain on track to report data
from the monotherapy arms of the ELX-02 clinical trials in the
fourth quarter of 2021. In addition, we are continuing to enroll
additional patients to support Phase 3 clinical trial
planning.
- As previously announced, the U.S.
Food and Drug Administration granted Fast Track designation for
ELX-02. Fast Track Designation is granted to drugs being developed
for the treatment of serious or life-threatening diseases or
conditions where there is an unmet medical need.
- Biological activity is being
assessed by changes in sweat chloride, a surrogate marker for
restoring CFTR activity. For comparison, Orkambi, an FDA-approved
combination CF agent (Lumacaftor/Ivacaftor), demonstrated
mid-single digit reductions in sweat chloride over one- to two-week
study durations in a similar trial setting. In addition, Symdeko,
another FDA-approved combination agent, demonstrated low- to
mid-single digit reductions in sweat chloride over 28 days.
- Patient dosing in the expansion
arm, which includes a combination of ELX-02 and the CFTR protein
potentiator, Kalydeco (ivacaftor), is expected to begin by the end
of 2021, with topline results expected in the first half of
2022.
- Began evaluation of inhaled
(nebulizer-based) delivery of the current subcutaneous formulation
of ELX-02. This has the potential to further improve the activity
of ELX-02 as a single agent and in combination with other drugs
given potential for increased drug exposure in the lung versus
plasma. An Investigational New Drug (IND) application is expected
in 2H 2022.
- Presented preclinical data at the
2021 North American Cystic Fibrosis Conference demonstrating that
significantly greater than 75% of patient derived organoids with
ultra rare premature termination codon (PTC) mutations and with no
residual CFTR function show swelling response to treatment with
ELX-02.
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional
Epidermolysis Bullosa (JEB)
- ZKN-013 is advancing into
IND-enabling studies for the treatment of RDEB and JEB. Good
Laboratory Practice (GLP) safety studies are expected to begin by
the end of 2021. We remain on track to file an IND in 2022.
Rare Inherited and Targeted Oncology
- Continued to advance our
preclinical oncology pipeline, which is focused on rare inherited
cancers with driver nonsense mutations and cancers with driver
mutations in the ribosome.
- Our lead program is for the
treatment of patients with Familial Adenomatous Polyposis (FAP).
- An 8-week study in APCMin
(multiple intestinal neoplasia) mice to evaluate the potential of
RMAs to treat FAP. The APCMin mouse is a translationally validated
model for drug development for FAP. This study in APCMin will
evaluate polyp number and size in ZKN013 treated mice versus
control mice. We expect to report results from this study in the
fourth quarter of 2021.
- Cancer cell line and xenograft
mouse studies ongoing to evaluate the response to treatment
with RMAs to advance first-in-class onco-ribosome targeted
inhibitors for oncology therapy.
Corporate
- Eloxx entered into a debt
facility with Hercules Capital, Inc., a specialty financing lender
for life science and technology companies, that provides for
borrowings of up to $30.0 million. Eloxx has drawn the initial
tranche of $12.5 million and used the proceeds to repay in full
amounts outstanding under the Eloxx’ existing debt facility. The
remaining funds under the new facility will be available in
additional tranches upon the achievement of specific clinical
milestones or other terms and conditions.
- Ali Hariri, M.D., joined Eloxx as
Chief Medical Officer, overseeing clinical development efforts for
the company’s promising pipeline of therapies to treat rare
diseases.
Third Quarter 2021 Financial Results
For the three months ended September 30, 2021, we incurred a net
loss of $9.9 million, or $0.11 per share, which includes $2.2
million in stock-based compensation. For the same period in the
prior year, we incurred a net loss of $6.6 million, or $0.16 per
share. Results for the third quarter of 2021 included costs related
to the acquisition of Zikani. Since the closing date of the
acquisition, the results of Zikani’s operations have been included
in the Eloxx’ condensed consolidated financial statements.
Our research and development expenses (R&D) were $5.2
million for the three months ended September 30, 2021, which
includes $0.4 million in stock-based compensation. For the same
period in the prior year, R&D expenses were $3.4 million. The
increase was primarily related to expenses related to the continued
development of ELX-02 due to the impact of the COVID-19 pandemic in
the prior year period, an increase in salaries and other personnel
related costs, and an increase in operational facilities.
Our general and administrative (G&A) expenses were $5.0
million for the three months ended September 30, 2021, which
includes $1.9 million in stock-based compensation. For the same
period in the prior year, G&A expenses were $2.9 million. The
increase was primarily related to an increase in stock-based
compensation expense, an increase in salaries and other personnel
related costs, as well as an increase in legal, accounting and
other professional fees.
As of September 30, 2021, we had cash and cash equivalents of
$52.4 million, which we expect will be sufficient to fund our
operations into the first quarter of 2023.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of
ribosome modulation, leveraging its innovative
TURBO-ZM™ chemistry technology platform in an effort to
develop novel Ribosome Modulating Agents (RMAs) and its library of
Eukaryotic Ribsome Selective Glycosides (ERSGs). 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 clinical development, focusing on cystic
fibrosis. Eloxx also has preclinical programs focused on select
rare diseases, including inherited diseases, cancer caused by
nonsense mutations, kidney diseases, including autosomal dominant
polycystic kidney disease, as well as rare ocular genetic
disorders.
For more information, please visit www.eloxxpharma.com.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements other than statements of present and
historical facts contained in this press release, including without
limitation, statements regarding our expected cash burn and future
financial results, the expected timing of trials and results from
clinical studies of our product candidates and the potential of our
product candidate to treat nonsense mutations are forward-looking
statements. Forward-looking statements can be identified by the
words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,”
“plan,” “anticipate,” “could,” “intend,” “target,” “project,”
“contemplate,” “believe,” “estimate,” “predict,” “potential,”
“seeks,” or “continue” or the negative of these terms similar
expressions, although not all forward-looking statements contain
these words.
Forward-looking statements are based on management's current
plans, estimates, assumptions and projections based on information
currently available to us. Forward-looking statements are subject
to known and unknown risks, uncertainties and assumptions, and
actual results or outcomes may differ materially from those
expressed or implied in the forward-looking statements due to
various important factors, including, but not limited to: our
ability to progress any product candidates in preclinical or
clinical trials; the uncertainty of clinical trial results and the
fact that positive results from preclinical studies are not always
indicative of positive clinical results; the scope, rate and
progress of our preclinical studies and clinical trials and other
research and development activities; the competition for patient
enrollment from drug candidates in development; the impact of the
global COVID-19 pandemic on our clinical trials, operations,
vendors, suppliers, and employees; our ability to obtain the
capital necessary to fund our operations; the cost of filing,
prosecuting, defending and enforcing any patent claims and other
intellectual property rights; our ability to obtain financial in
the future through product licensing, public or private equity or
debt financing or otherwise; general business conditions,
regulatory environment, competition and market for our products;
and business ability and judgment of personnel, and the
availability of qualified personnel and other important factors
discussed under the caption “Risk Factors” in our Quarterly Report
on Form 10-Q for the quarter ended September 30, 2021, as any such
factors may be updated from time to time in our other filings with
the SEC, accessible on the SEC’s website at www.sec.gov and the
“Financials & Filings” page of our website at
https://investors.eloxxpharma.com/financial-information/sec-filings
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
Contact
InvestorsJohn
Woolfordjohn.woolford@westwicke.com443.213.0506
MediaLaureen Cassidylaureen@outcomescg.com
ELOXX PHARMACEUTICALS, INC. AND
SUBSIDIARIES UNAUDITED CONDENSED CONSOLIDATED
BALANCE SHEETS (in thousands, except share and per
share data)
|
|
September 30,2021 |
|
|
December 31,2020 |
|
ASSETS |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
52,432 |
|
|
$ |
24,668 |
|
Restricted cash |
|
|
246 |
|
|
|
56 |
|
Prepaid expenses and other current assets |
|
|
864 |
|
|
|
1,169 |
|
Total current assets |
|
|
53,542 |
|
|
|
25,893 |
|
Property and equipment,
net |
|
|
224 |
|
|
|
133 |
|
Operating lease right-of-use
assets |
|
|
1,617 |
|
|
|
421 |
|
Other long-term assets |
|
|
— |
|
|
|
30 |
|
Total assets |
|
$ |
55,383 |
|
|
$ |
26,477 |
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS’ EQUITY |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
2,509 |
|
|
$ |
481 |
|
Accrued expenses |
|
|
2,611 |
|
|
|
2,924 |
|
Current portion of long-term debt |
|
|
— |
|
|
|
5,239 |
|
Advances from collaboration partners |
|
|
3,723 |
|
|
|
805 |
|
Current portion of operating lease liabilities |
|
|
667 |
|
|
|
389 |
|
Total current liabilities |
|
|
9,510 |
|
|
|
9,838 |
|
Long-term debt |
|
|
11,911 |
|
|
|
6,376 |
|
Operating lease
liabilities |
|
|
964 |
|
|
|
33 |
|
Total liabilities |
|
|
22,385 |
|
|
|
16,247 |
|
Total stockholders’
equity |
|
|
32,998 |
|
|
|
10,230 |
|
Total liabilities and
stockholders’ equity |
|
$ |
55,383 |
|
|
$ |
26,477 |
|
|
|
|
|
|
|
|
|
|
UNAUDITED CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE
LOSS(in thousands, except share and per share
data)
|
|
Three Months EndedSeptember 30, |
|
|
Nine Months EndedSeptember 30, |
|
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
5,210 |
|
|
$ |
3,445 |
|
|
$ |
14,987 |
|
|
$ |
11,950 |
|
General and administrative |
|
|
5,035 |
|
|
|
2,851 |
|
|
|
16,731 |
|
|
|
11,705 |
|
In process research and development |
|
|
— |
|
|
|
— |
|
|
|
22,670 |
|
|
|
— |
|
Restructuring charges |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
3,994 |
|
Total operating expenses |
|
|
10,245 |
|
|
|
6,296 |
|
|
|
54,388 |
|
|
|
27,649 |
|
Loss from operations |
|
|
(10,245 |
) |
|
|
(6,296 |
) |
|
|
(54,388 |
) |
|
|
(27,649 |
) |
Other income (expense),
net |
|
|
360 |
|
|
|
(321 |
) |
|
|
(249 |
) |
|
|
(801 |
) |
Net loss |
|
$ |
(9,885 |
) |
|
$ |
(6,617 |
) |
|
$ |
(54,637 |
) |
|
$ |
(28,450 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(0.11 |
) |
|
$ |
(0.16 |
) |
|
$ |
(0.85 |
) |
|
$ |
(0.71 |
) |
Weighted average number of
shares of common stock used in computing net loss per share, basic
and diluted |
|
|
86,208,754 |
|
|
|
40,142,178 |
|
|
|
64,428,187 |
|
|
|
40,115,351 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Source: Eloxx Pharmaceuticals
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