Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, today reported
its financial results for the three months ended June 30, 2021, and
provided a business update.
“Since the acquisition of Zikani Therapeutics in early April, we
have made tremendous progress across our portfolio of novel
therapeutic programs,” said Sumit Aggarwal, President and Chief
Executive Officer of Eloxx. “In CF, we remain on track to report
data from the ongoing Phase 2 clinical trials for ELX-02, while we
are also preparing for combination studies of ELX-02 and ivacaftor
and commencing efforts to evaluate inhaled delivery of ELX-02.
Beyond CF, we continue to advance our preclinical programs to
demonstrate the potential of our oral RMAs. We intend to provide
additional insight into these programs in the coming quarters.”
Second 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.
- As previously announced, based on
enrollment as of the end of June 2021, we remain on track to report
data from the monotherapy arms of the ELX-02 clinical trials in the
fourth quarter of 2021. We are continuing to enroll additional
patients to support Phase 3 clinical trial planning.
- Biological activity is being
assessed by changes in sweat chloride, a surrogate marker for
restoring CFTR activity. For comparison, Orkambi, an FDA-approved
CF agent, demonstrated mid-single digit reductions in sweat
chloride over one- to two-week study durations in a similar trial
setting.
- 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. Previously disclosed preclinical organoid experiments have
demonstrated that the addition of Kalydeco to ELX-02 can enhance
activity up to three-fold.
- Safety Review Committee has
approved advancement to highest dose level to be studied.
- Began evaluation of inhaled
(nebulizer-based) delivery of the current subcutaneous formulation
of ELX-02. Preclinical rodent studies of ELX-02 and other
aminoglycosides have demonstrated 30- to 100-fold higher drug
concentration in the lung versus plasma. This has the potential to
further improve the activity of ELX-02 as both a single agent and
in combination with other drugs.
- Received an award of up to $2.6M
from the Cystic Fibrosis Foundation to identify optimized oral
Ribosome Modulating Agents (RMAs) with our TURBO-ZM™ Platform for
further development for the treatment of CF patients with nonsense
mutations.
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional
Epidermolysis Bullosa (JEB)
- Nominated ZKN-013 as the drug
candidate to move 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 submission in 2022.
- In preclinical experiments,
treating RDEB patient-derived fibroblasts and keratinocytes with
ZKN-013 showed clinically relevant restoration of full-length
Collagen 7A (COL7A) across multiple genotypes in a dose dependent
manner.
- In a non-GLP animal toxicity
study, ZKN-013 demonstrated a substantial safety margin with
therapeutically sufficient drug levels in skin with chronic
dosing.
Rare Inherited and Targeted Oncology
- Continued to advance our
preclincial 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).
- We have initiated 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.
- Prior studies of erythromycin in
APCMin mice showed reductions in colon polyps that successfully
translated to demonstrating clinical efficacy.1
- 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.
- Initiated cancer cell line and
xenograft mouse studies to evaluate the response to treatment
with RMAs to advance first-in-class onco-ribosome targeted
inhibitors for oncology therapy.
Corporate
- An underwritten public offering
of 38,333,334 shares of common stock, including the full exercise
of the underwriters’ option to purchase additional shares, at a
public offering price of $1.35 per share was closed in May 2021.
Aggregate gross proceeds from the offering were approximately
$51.75 million, before deducting underwriting discounts and
commissions and offering expenses.
- Pedro Huertas, M.D., Ph.D.,
joined Eloxx as a Senior Adviser. Dr. Huertas previously served as
CMO of Eloxx. He has also served as CMO of Inozyme Pharma and
Sentien Biotechnologies and in various roles at Pfizer, Shire,
Massachusetts General Hospital, and Genzyme.
Second Quarter 2021 Financial Results
For the three months ended June 30, 2021, we incurred a net loss
of $36.1 million or $0.54 per share, which includes $4.0 million in
stock-based compensation. For the same period in the prior year, we
incurred a net loss of $7.9 million, or $0.20 per share. Results
for the second quarter of 2021 included a $22.7 million acquired
in-process research and development expense related to the
acquisition of Zikani.
Our research and development expenses (R&D) were $5.7
million for the three months ended June 30, 2021, which includes
$0.1 million in stock-based compensation. For the same period in
the prior year, R&D expenses were $3.7 million. The increase in
R&D expenses was primarily related to an increase in expenses
related to the continued development of ELX-02 as a result of the
suspension of our clinical trials due to the impact of the COVID-19
pandemic in the prior year period and an increase in salaries and
other personnel related costs, partially offset by a decrease in
stock-based compensation expense.
Our general and administrative (G&A) expenses were $7.4
million for the three months ended June 30, 2021, which includes
$3.9 million in stock-based compensation. For the same period in
the prior year, G&A expenses were $3.8 million. The increase
was primarily related to an increase in stock-based compensation
expense, an increase in salaries and other personnel-related costs
associated with the merger with Zikani, as well as an increase in
expenses attributable principally to infrastructure related costs
including legal, accounting and other professional fees.
As of June 30, 2021, we had cash and cash equivalents of $56.7
million, which we expect will be sufficient to fund our operations
into the first quarter of 2023.
1 J Mol Med (Berl) 2016 Apr;94(4):469-82) and Int J Cancer 2020
Feb 15;146(4):1064-1074)
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of
ribosome modulation, leveraging its innovative TURBO-ZMTM 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 June 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 |
|
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
June 30, 2021 |
|
December 31, 2020 |
|
ASSETS |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
56,734 |
|
$ |
24,668 |
|
Restricted cash |
|
|
246 |
|
|
56 |
|
Prepaid expenses and other current assets |
|
|
1,579 |
|
|
1,169 |
|
Total current assets |
|
|
58,559 |
|
|
25,893 |
|
Property and
equipment, net |
|
|
185 |
|
|
133 |
|
Operating
lease right-of-use asset |
|
|
1,866 |
|
|
421 |
|
Other
long-term assets |
|
|
- |
|
|
30 |
|
Total assets |
|
$ |
60,610 |
|
$ |
26,477 |
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
2,024 |
|
$ |
481 |
|
Accrued expenses |
|
|
3,302 |
|
|
2,886 |
|
Current portion of long-term debt |
|
|
5,686 |
|
|
5,239 |
|
Advances from collaboration partners |
|
|
3,411 |
|
|
805 |
|
Current portion of operating lease liability |
|
|
753 |
|
|
389 |
|
Taxes payable |
|
|
34 |
|
|
38 |
|
Total current liabilities |
|
|
15,210 |
|
|
9,838 |
|
Long-term
debt |
|
|
3,637 |
|
|
6,376 |
|
Operating
lease liability |
|
|
1,120 |
|
|
33 |
|
Total
liabilities |
|
|
19,967 |
|
|
16,247 |
|
Total
stockholders’ equity |
|
|
40,643 |
|
|
10,230 |
|
Total liabilities and stockholders' equity |
|
$ |
60,610 |
|
$ |
26,477 |
|
|
|
|
|
|
|
ELOXX
PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS |
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
5,704 |
|
|
$ |
3,738 |
|
|
$ |
9,777 |
|
|
$ |
8,505 |
|
General and administrative |
|
|
7,355 |
|
|
|
3,848 |
|
|
|
11,696 |
|
|
|
8,854 |
|
In process research and development |
|
|
22,670 |
|
|
|
— |
|
|
|
22,670 |
|
|
|
— |
|
Restructuring charges |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
3,994 |
|
Total operating expenses |
|
|
35,729 |
|
|
|
7,586 |
|
|
|
44,143 |
|
|
|
21,353 |
|
Loss from
operations |
|
|
(35,729 |
) |
|
|
(7,586 |
) |
|
|
(44,143 |
) |
|
|
(21,353 |
) |
Other
expense, net |
|
|
329 |
|
|
|
301 |
|
|
|
609 |
|
|
|
480 |
|
Net
loss |
|
$ |
(36,058 |
) |
|
$ |
(7,887 |
) |
|
$ |
(44,752 |
) |
|
$ |
(21,833 |
) |
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per share |
|
$ |
(0.54 |
) |
|
$ |
(0.20 |
) |
|
$ |
(0.84 |
) |
|
$ |
(0.54 |
) |
Weighted
average number of common shares used in computing net loss per
share, basic and diluted |
|
|
66,389,865 |
|
|
|
40,129,304 |
|
|
|
53,357,401 |
|
|
|
40,101,789 |
|
SOURCE: Eloxx Pharmaceuticals, Inc.
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