Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, reported its
financial results for the three months ended June 30, 2022 and
provided a business update.
“We continue to make significant progress advancing our pipeline
of novel products for patients with substantial unmet medical
needs. With multiple potential value-creating events expected over
the next twelve months, including topline data readouts for ELX-02
in both CF and Alport syndrome, we are approaching an exciting and
eventful period for the company,” said Sumit Aggarwal, President
and Chief Executive Officer of Eloxx. “As further evidence of the
potential of our technology platform, today we are pleased to
announce results of a repeat study of ZKN-013 in a validated model,
which provides further confirmation for the potential of ZKN-013 to
treat FAP,” said Sumit Aggarwal, President and CEO of Eloxx
Pharmaceuticals. “Based on the continuing positive preclinical
results, we now expect to submit an IND to the U.S. Food and Drug
Administration in the fourth quarter of 2023 for ZKN-013 for
FAP.”
Second Quarter 2022 and Subsequent
Highlights
Class 1 Cystic Fibrosis
- Phase 2 clinical trials in CF
patients, with expansion arms designed to evaluate the safety of
ELX-02 and assess short-term biological activity in patients,
remains ongoing. Topline results are expected before the end of the
third quarter of 2022 once complete patient data sets are
received.
- A head-to-head preclinical study of
inhaled (nebulizer-based) delivery of the current subcutaneous
formulation of ELX-02 compared to subcutaneous dosing in a CF
rodent model has been completed with data analysis ongoing. Human
exposure, modeled based on previous animal pharmacokinetic studies,
suggests that the lung drug exposure with inhaled delivery of
ELX-02 is at least 50-fold greater than with subcutaneous delivery.
Higher drug exposure, achievable with inhaled delivery, has been
associated with 3 to 6 fold higher activity in CF models. We expect
to report topline results before the end of the third quarter of
2022 and remain on track to submit an IND application in the fourth
quarter of 2022.
Alport Syndrome
- Eloxx previously announced it has
expanded its clinical development pipeline to include the potential
treatment with ELX-02 of a subset of Alport Syndrome patients with
nonsense mutations in the Collagen Type 4 genes, (COL4A3, COL4A4,
and COL4A5). Alport syndrome is a rare genetic disorder
characterized by kidney disease with high levels of proteinuria,
hearing loss and eye abnormalities.
- Eloxx remains on track to initiate
a proof-of-concept clinical trial in up to eight Alport syndrome
patients with nonsense mutations in the second half of 2022.
Patients will be dosed for two months with a three month follow-up.
Trial primary endpoints include safety while secondary endpoints
are reduction in proteinuria and induction of COL4A5 protein
expression in the kidney. Initial topline results are expected in
the first half of 2023.
Recessive Dystrophic Epidermolysis Bullosa and Junctional
Epidermolysis Bullosa (JEB)
- Eloxx continues to expect to file
an IND application in the fourth quarter of 2022 and be ready to
start a First in Human (FIH) Phase 1 study with ZKN-013 after
recently completing 28-day non Good Laboratory Practice (GLP)
animal studies.
Familial Adenomatous Polyposis
- Eloxx today announced positive
results from a repeat study in the APCMin (multiple intestinal
neoplasia) model evaluating the potential of ZKN-013 to treat FAP.
These results provide further confirmation of ZKN-013’s potential
to treat FAP, following the previously reported single-dose data in
the APCMin model. The APCMin mouse is a translationally validated
model for drug development for FAP, a rare inherited disease, with
no approved drug therapies, characterized by proliferation of colon
polyps. Eloxx is targeting a subset of patients that have nonsense
mutations in the Adenomatous Polyposis Coli (APC) gene that is
truncated in these patients.
- In the repeat study, APCMin mice
received daily oral doses of ZKN-013 at 50, 25, 12.5 mg/kg body
weight for 8 weeks.
- 100% survival was achieved at the
lowest dose of ZKN-013 tested.
- ZKN-013 significantly lessened
anemic conditions, generally considered to be the major cause of
animal death associated with APCMin model.
- Eloxx intends to file an IND
application to the U.S. Food and Drug Administration for ZKN-013
for the treatment of FAP in the second half of 2023.
Second Quarter 2022 Financial Results
For the three months ended June 30, 2022, we incurred a net loss
of $10.6 million, or $0.12 per share, which included $0.7 million
in stock-based compensation. For the same period in the prior year,
we incurred a net loss of $36.1 million, or $0.54 per share. Since
the closing date of Eloxx’s acquisition of Zikani Therapeutics,
Inc. in April 2021, the results of Zikani’s operations have been
included in the Eloxx’ condensed consolidated financial statements.
The second quarter of 2021 included a $22.7 million expense for in
process research and development (R&D) related to the Zikani
acquisition.
Our R&D expenses were $7.7 million for the three months
ended June 30, 2022, which includes $0.3 million in stock-based
compensation. For the same period in the prior year, R&D
expenses were $5.7 million, which included $0.1 million of
stock-based compensation. The increase was primarily related to
increases in expenses related to preclinical and development
activities, the continued development of ELX-02 related primarily
to Cystic Fibrosis Foundation funded activities, facility and
overhead expenses and salaries and stock-based compensation.
Our general and administrative (G&A) expenses were $2.6
million for the three months ended June 30, 2022, which includes
$0.4 million in stock-based compensation. For the same period in
the prior year, G&A expenses were $7.4 million, which included
$3.9 million of stock-based compensation. The decrease was
primarily related to decreases in salaries and other personnel
related costs, stock-based compensation and facility and overhead
expenses.
As of June 30, 2022, we had unrestricted cash and cash
equivalents of $30.0 million. The Hercules Term Loan Agreement
contains customary affirmative and negative covenants, which among
others require the Company to maintain at all times a minimum
qualified cash balance. As of June 30, 2022, the Company was in
compliance with all debt covenants. If our lender does not
accelerate our debt upon our failure to comply with the minimum
cash covenant, we expect our current cash position will be
sufficient to fund our operations into the fourth quarter of
2023.
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 Ribosome
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. The U.S.
Food and Drug Administration (FDA) has granted Fast Track
designation for ELX-02 for the treatment of CF patients with
nonsense mutations. In addition, ELX-02 has also been granted
Orphan Drug Designation for the treatment of CF patients with
nonsense mutations by the FDA and orphan medicinal product
designation by the European Commission. ELX-02 is in clinical
development, focusing on cystic fibrosis (US Trial NCT04135495,
EU/IL Trial NCT04126473). 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 future financial results, our
cash runway, 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, 2022, 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 30,2022 |
|
December 31,2021 |
|
ASSETS |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
30,048 |
|
$ |
42,268 |
|
Restricted cash |
|
|
263 |
|
|
299 |
|
Prepaid expenses and other current assets |
|
|
1,281 |
|
|
913 |
|
Total current assets |
|
|
31,592 |
|
|
43,480 |
|
Property and
equipment, net |
|
|
190 |
|
|
216 |
|
Operating
lease right-of-use assets |
|
|
1,159 |
|
|
1,443 |
|
Total assets |
|
$ |
32,941 |
|
$ |
45,139 |
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
1,934 |
|
$ |
1,379 |
|
Accrued expenses |
|
|
4,870 |
|
|
4,196 |
|
Current portion of long-term debt |
|
|
1,125 |
|
|
- |
|
Advances from collaboration partners |
|
|
10,723 |
|
|
3,723 |
|
Derivative liabilities |
|
|
166 |
|
|
- |
|
Current portion of operating lease liabilities |
|
|
686 |
|
|
657 |
|
Total current liabilities |
|
|
19,504 |
|
|
9,955 |
|
Long-term
debt, net of current portion |
|
|
11,129 |
|
|
11,996 |
|
Operating
lease liabilities |
|
|
496 |
|
|
804 |
|
Total
liabilities |
|
|
31,129 |
|
|
22,755 |
|
Total
stockholders’ equity |
|
|
1,812 |
|
|
22,384 |
|
Total liabilities and stockholders' equity |
|
$ |
32,941 |
|
$ |
45,139 |
|
|
|
|
|
|
|
ELOXX
PHARMACEUTICALS, INC. AND SUBSIDIARIES |
|
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS |
|
(Amounts in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended |
|
|
Six Months
Ended |
|
June 30, |
June 30, |
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
7,651 |
|
|
$ |
5,704 |
|
|
$ |
15,550 |
|
|
$ |
9,777 |
|
General and administrative |
|
|
2,645 |
|
|
|
7,355 |
|
|
|
5,699 |
|
|
|
11,696 |
|
In process research and development |
|
|
— |
|
|
|
22,670 |
|
|
|
— |
|
|
|
22,670 |
|
Total operating expenses |
|
|
10,296 |
|
|
|
35,729 |
|
|
|
21,249 |
|
|
|
44,143 |
|
Loss from
operations |
|
|
(10,296 |
) |
|
|
(35,729 |
) |
|
|
(21,249 |
) |
|
|
(44,143 |
) |
Other
expense, net |
|
|
322 |
|
|
|
329 |
|
|
|
989 |
|
|
|
609 |
|
Net
loss |
|
$ |
(10,618 |
) |
|
$ |
(36,058 |
) |
|
$ |
(22,238 |
) |
|
$ |
(44,752 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
|
$ |
(0.12 |
) |
|
$ |
(0.54 |
) |
|
$ |
(0.26 |
) |
|
$ |
(0.84 |
) |
Weighted
average number of shares of common stock used in computing net
loss per share, basic and diluted |
|
|
86,654,120 |
|
|
|
66,389,865 |
|
|
|
86,652,587 |
|
|
|
53,357,401 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Source: Eloxx Pharmaceuticals
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