Full Press Release Details
Eloxx Pharmaceuticals Reports Second Quarter
2022 Financial and Operating Results and Provides Business Update
Topline data from cystic fibrosis (CF) Phase
2 expansion treatment arms evaluating combination with ivacaftor expected by the end of the third quarter of 2022
On track to initiate a proof-of-concept clinical
trial with ELX-02 in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022
On track to submit an Investigational New Drug
(IND) application for the inhaled delivery of ELX-02 in the fourth quarter of 2022
On track to submit IND application for ZKN-013
for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) patients with nonsense mutations cells in the fourth quarter of
of ZKN-013 Dose Response Study in APCMin Model that confirmed the results from prior study which concluded in March 2022
and expect to submit an IND application for Familial Adenomatous Polyposis (FAP) in the second half of 2023
Cash and equivalents expected to be sufficient
to fund operations into the fourth quarter of 2023
WATERTOWN, MA - August 15,
2022 - 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
Recessive Dystrophic Epidermolysis Bullosa and Junctional Epidermolysis
Familial Adenomatous Polyposis
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.
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 |
PHARMACEUTICALS, INC. AND SUBSIDIARIES
CONSOLIDATED STATEMENTS OF OPERATIONS
(Amounts in thousands, except share and per share data)
| Three Months Ended June 30, | Six Months Ended 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 |