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 December 31, 2022
and provided a business update.
“With topline data expected for ELX-02 in Alport syndrome in the
first half of 2023, we believe we are approaching a significant
milestone for the company, to advance into our first Phase 3 study,
with the potential to create significant value for both patients
and shareholders,” said Sumit Aggarwal, President and Chief
Executive Officer of Eloxx. “Positive data from the trial would
also be a validation of our potential to treat other rare kidney
diseases and develop small molecule genetic therapy products.”
Fourth Quarter 2022 and Subsequent
Highlights
Alport Syndrome
- Eloxx has now dosed three patients
with ELX-02 in the ongoing proof-of-concept Phase 2 open-label
clinical trial (NCT05448755) in up to eight Alport syndrome
patients with nonsense mutations in the Collagen Type 4 genes,
(COL4A3, COL4A4, and COL4A5). Encouraging initial reduction in
proteinuria has been observed in one patient to date. Alport
syndrome is a rare genetic disorder characterized by kidney disease
with high levels of proteinuria, hearing loss and eye
abnormalities. We will be evaluating expression of COL IV protein
in these three patients at the end of dosing and measuring
proteinuria every two weeks. Topline results are expected in the
first half of 2023.
- Eloxx presented a poster
highlighting the activity of ELX-02 across a range of COL4A5
mutations in preclinical models at the American Society of
Nephrology (ASN) Kidney Week 2022 Conference in early November
2022.
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional
Epidermolysis Bullosa (JEB)
- In March 2023, Eloxx announced the
submission of an Investigational New Drug (“IND”) application with
the U.S. Food and Drug Administration for ZKN-013 for the treatment
of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense
mutations. RDEB is a rare skin disease characterized by mutations
in Collagen 7 gene.
- Recent preclinical results
demonstrated read-through activity of ZKN-013 in multiple COL7
genotypes across multiple RDEB patient derived fibroblasts and
keratinocytes. Read-through activity resulted in up to an 18-fold
increase in full-length COL VII protein levels. Prolonged treatment
with ZKN-013 further increased COL VII protein levels.
Functionality of the restored full-length COL VII protein was
confirmed. These results have been accepted for presentation at an
upcoming medical conference.
Familial Adenomatous Polyposis (FAP)
- Eloxx also plans to develop ZKN-013
to treat FAP, targeting a subset of patients that have nonsense
mutations in the Adenomatous Polyposis Coli (APC) gene that is
truncated in these patients. An additional IND filing for ZKN-013
for treatment of FAP is planned in the first half of 2023.
- In January 2023, Eloxx published positive results from a study
in the APCMin (multiple intestinal neoplasia) model evaluating the
potential of ZKN-013 to treat FAP. The APCMin mouse is a
translationally validated model for drug development for FAP. In
the APCMin model, treatment with ZKN-013 demonstrated a decrease in
intestinal polyps and adenomas, resulting in increased survival.
The publication also included in vitro and in vivo results
demonstrating that ZKN-013 inhibited the growth of human colon
carcinoma cells with APC nonsense mutations, and promoted read
through of premature stop codons in the APC gene, leading to
functional restoration of full-length APC protein.
Fourth Quarter 2022 Financial Results
For the three months ended December 31, 2022, we incurred a net
loss of $6.3 million, or $2.92 per share, which included $0.6
million in stock-based compensation. For the same period in the
prior year, we incurred a net loss of $12.1 million, or $5.60 per
share, which included $1.6 million in stock-based compensation.
R&D expenses were $3.3 million for the three months ended
December 31, 2022, which includes $0.3 million in stock-based
compensation. For the same period in the prior year, R&D
expenses were $7.9 million, which included $0.6 million of
stock-based compensation. The decrease was related to a decrease in
clinical trial expenses related primarily to Cystic Fibrosis
Foundation funded activities, and a decrease in salaries and other
personnel costs.
General and administrative (G&A) expenses were $2.7 million
for the three months ended December 31, 2022, which includes $0.3
million in stock-based compensation. For the same period in the
prior year, G&A expenses were $3.7 million, which included $1.0
million of stock-based compensation. The decrease was primarily
related to a decrease in salaries and other personal related costs
due to reduced headcount and outsourcing certain functions, a
decrease in expenses attributable to professional and consulting
fees, and a decrease in stock-based compensation expense.
As of December 31, 2022, we had unrestricted cash and cash
equivalents of $19.2 million. In March 2023, we amended the terms
of the Hercules Term Loan Agreement and repaid $7.5 million of the
outstanding principal. The minimum qualified cash balance
requirement was reduced to $2.25 million and Eloxx will be required
to make principal payments, plus interest, beginning in September
2023. Assuming that we initiate Phase 3 clinical trial activities
in the third quarter of 2023, our expectation is that our current
cash position and assuming maintaining compliance with our debt
covenants, will be sufficient to fund our operations into the third
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 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 cash runway and our ability to
comply with the covenants in our debt agreement, the expected
timing of and results from trials 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; our ability to meet
the continued listing requirements of the Nasdaq Capital Market;
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
Annual Report on Form 10-K for the fiscal year ended December 31,
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/financials-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) |
|
|
|
|
|
|
|
December 31,2022 |
|
December 31,2021 |
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
19,207 |
|
|
$ |
42,268 |
Restricted cash |
|
|
261 |
|
|
|
299 |
Prepaid expenses and other current assets |
|
|
661 |
|
|
|
913 |
Total current assets |
|
|
20,129 |
|
|
|
43,480 |
Property and equipment, net |
|
|
169 |
|
|
|
216 |
Operating lease right-of-use assets |
|
|
825 |
|
|
|
1,443 |
Total assets |
|
$ |
21,123 |
|
|
$ |
45,139 |
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’ (DEFICIT)
EQUITY |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
3,020 |
|
|
$ |
1,379 |
Accrued expenses |
|
|
2,799 |
|
|
|
4,196 |
Current portion of long-term debt |
|
|
3,980 |
|
|
|
- |
Advances from collaboration partners |
|
|
12,535 |
|
|
|
3,723 |
Current portion of operating lease liabilities |
|
|
712 |
|
|
|
657 |
Derivative liabilities |
|
|
45 |
|
|
|
- |
Total current liabilities |
|
|
23,091 |
|
|
|
9,955 |
Long-term debt |
|
|
8,557 |
|
|
|
11,996 |
Operating lease liabilities |
|
|
135 |
|
|
|
804 |
Total liabilities |
|
|
31,783 |
|
|
|
22,755 |
Total stockholders’ (deficit) equity |
|
|
(10,660 |
) |
|
|
22,384 |
Total liabilities and stockholders’ (deficit)
equity |
|
$ |
21,123 |
|
|
$ |
45,139 |
|
|
|
|
|
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES |
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS |
(Amounts in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
Three Months EndedDecember 31, |
|
Year EndedDecember 31, |
|
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
3,297 |
|
|
$ |
7,912 |
|
|
$ |
23,727 |
|
|
$ |
22,899 |
|
General and administrative |
|
|
2,731 |
|
|
|
3,718 |
|
|
|
10,692 |
|
|
|
20,449 |
|
In process research and development |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
22,670 |
|
Total operating expenses |
|
|
6,028 |
|
|
|
11,630 |
|
|
|
34,419 |
|
|
|
66,018 |
|
Loss from operations |
|
|
(6,028 |
) |
|
|
(11,630 |
) |
|
|
(34,419 |
) |
|
|
(66,018 |
) |
Other expense, net |
|
|
291 |
|
|
|
460 |
|
|
|
1,646 |
|
|
|
709 |
|
Net loss |
|
$ |
(6,319 |
) |
|
$ |
(12,090 |
) |
|
$ |
(36,065 |
) |
|
$ |
(66,727 |
) |
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per share |
|
$ |
(2.92 |
) |
|
$ |
(5.60 |
) |
|
$ |
(16.65 |
) |
|
$ |
(38.15 |
) |
Weighted average number of common shares used in computing net loss
per share, basic and diluted |
|
|
2,166,356 |
|
|
|
2,159,658 |
|
|
|
2,166,311 |
|
|
|
1,749,071 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
SOURCE: Eloxx Pharmaceuticals, Inc.
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