Full Press Release Details
Eloxx Pharmaceuticals Reports Third Quarter
2022 Financial and Operating Results and Provides Business Update
Started proof-of-concept Phase 2 clinical trial
with ELX-02 in up to eight Alport syndrome patients with nonsense mutations; topline results expected in the first half of 2023
On track to submit IND application for ZKN-013
for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) patients with nonsense mutations cells by the end of 2022 or early
November 10, 2022 - 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 September 30, 2022
and provided a business update.
"With the recent start of our Phase 2 proof-of-concept trial
for ELX-02 for the treatment of Alport Syndrome, we are on track to deliver topline results for the program in the first half of 2023.
Alport patients with nonsense mutations have significantly worse clinical outcomes than other Alport patients and have no disease modifying
treatment options," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. "We have also made significant advancements
in our Ribosome Modulating Agents programs, approaching clinical development for ZKN-013 for the treatment of RDEB and generating encouraging
preclinical data in multiple indications."
Third Quarter 2022 and Subsequent Highlights
| Eloxx started a 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). Alport syndrome is a rare genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities. 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. | ||
| 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)
Familial Adenomatous Polyposis (FAP)
Class 1 Cystic Fibrosis (CF)
| o | Two posters were presented at the 34 th EORTC-NCI-AACR Symposium in late October 2022 demonstrating the potential of RMAs to treat MYC-driven cancers. Preclinical data demonstrated activity against subtypes of colorectal cancer (ZKN-157) and small cell lung cancer (ZKN-217). | |
| o | A poster was presented at the International Symposium on Medicinal Chemistry in early September. A phenotypic screen of our RMA library identified showed selective hits for cancer cell lines and demonstrated responsive structure-activity relationship. | |
| o | In Eloxx's program to identify RMAs for further development in the treatment of Class 1 CF, which is supported by the CF Foundation, screening has identified approximately 50 oral RMAs with promising functional CFTR activity. |
available on Eloxx's website at link.
Third Quarter 2022 Financial Results
For the three months ended September 30, 2022, we incurred a net loss
of $7.5 million, or $0.09 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 $9.9 million, or $0.11 per share, which included $2.2 million in stock-based compensation.
Our R&D expenses were $4.9 million for the three months ended September
30, 2022, which includes $0.3 million in stock-based compensation. For the same period in the prior year, R&D expenses were $5.2 million,
which included $0.4 million of stock based compensation. The decrease was related to a decrease in clinical trial expenses related primarily
to CF Foundation funded activities, and a decrease in salaries and other personnel costs.
Our general and administrative (G&A) expenses were $2.3 million
for the three months ended September 30, 2022, which includes $0.4 million in stock-based compensation. For the same period in the prior
year, G&A expenses were $5.0 million, which included $1.9 million of stock-based compensation. The decrease was primarily related
to a decrease in salaries and other personal related costs due to reduced headcount, a decrease in expenses attributable to professional
and consulting fees, and a decrease in stock-based compensation expense due primarily to option modifications of former executives in
As of September 30, 2022, we had unrestricted cash and cash equivalents
of $24.6 million. The Hercules Term Loan Agreement contains customary affirmative and negative covenants, which among others require us
to maintain a minimum qualified cash balance. As of September 30, 2022, we were in compliance with all debt covenants. Our expectation
is that our current cash position will be sufficient to fund our operations into the fourth quarter of 2023 assuming that we can comply
with our lender's minimum cash covenant by either raising additional capital or working with our lender to restructure the covenants.
About Eloxx Pharmaceuticals
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 and ability to comply with the covenants in our debt agreement, 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.
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, 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.
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(Amounts in thousands, except share and per share data)
| September 30, 2022 | December 31, 2021 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 24,554 | $ | 42,268 | ||||
| Restricted cash | 263 | 299 | ||||||
| Prepaid expenses and other current assets | 854 | 913 | ||||||
| Total current assets | 25,671 | 43,480 | ||||||
| Property and equipment, net | 215 | 216 | ||||||
| Operating lease right-of-use assets | 993 | 1,443 | ||||||
| Total assets | $ | 26,879 | $ | 45,139 | ||||
| LIABILITIES AND STOCKHOLDERS' (DEFICIT) EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,595 | $ | 1,379 | ||||
| Accrued expenses | 4,518 | 4,196 | ||||||
| Current portion of long-term debt | 2,530 | - | ||||||
| Advances from collaboration partners | 12,223 | 3,723 | ||||||
| Derivative liabilities | 116 | - | ||||||
| Current portion of operating lease liabilities | 698 | 657 | ||||||
| Total current liabilities | 21,680 | 9,955 | ||||||
| Long-term debt, net of current portion | 9,863 | 11,996 | ||||||
| Operating lease liabilities | 318 | 804 | ||||||
| Total liabilities | 31,861 | 22,755 | ||||||
| Total stockholders' (deficit) equity | (4,982 | ) | 22,384 | |||||
| Total liabilities and stockholders' (deficit) equity | $ | 26,879 | $ | 45,139 |
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
(Amounts in thousands, except share and per share data)
| Three Months Ended | Nine Months Ended | |||||||||||||||
| September 30, | September 30, | |||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 4,880 | $ | 5,210 | $ | 20,430 | $ | 14,987 | ||||||||
| General and administrative | 2,262 | 5,035 | 7,961 | 16,731 | ||||||||||||
| In process research and development | - | - | - | 22,670 | ||||||||||||
| Total operating expenses | 7,142 | 10,245 | 28,391 | 54,388 | ||||||||||||
| Loss from operations | (7,142 | ) | (10,245 | ) | (28,391 | ) | (54,388 | ) | ||||||||
| Other expense (income), net | 366 | (360 | ) | 1,355 | 249 | |||||||||||
| Net loss | $ | (7,508 | ) | $ | (9,885 | ) | $ | (29,746 | ) | $ | (54,637 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.09 | ) | $ | (0.11 | ) | $ | (0.34 | ) | $ | (0.85 | ) | ||||
| Weighted average number of shares of common stock used in computing net loss per share, basic and diluted | 86,656,221 | 86,208,754 | 86,653,811 | 64,428,187 |