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 2023
“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
Third Quarter 2022 and Subsequent Highlights
Alport Syndrome
- 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 earlyNovember 2022 .
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB)
- Eloxx continues to expect to file an Investigational New Drug (IND) application to start a First in Human (FIH) Phase 1 study of ZKN-013 by the end of 2022 or early 2023.
Familial Adenomatous Polyposis (FAP)
- Eloxx continues to evaluate the potential of 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. Eloxx is currently preparing an IND application.
Class 1 Cystic Fibrosis (CF)
- A Phase 2 combination study data readout of ELX-02 with ivacaftor in CF patients with nonsense mutations did not meet efficacy endpoints but did confirm drug activity for ELX-02 and demonstrated need for higher drug exposure in lung to generate therapeutic benefit. Eloxx aims to complete IND enabling studies for inhaled ELX-02. The path forward in CF is to be determined together with the
CF Foundation .
TURBO-ZM™ Platform
- Recent advancements have demonstrated the potential of Eloxx’s TURBO-ZM chemistry technology platform to develop novel Ribosome Modulating Agents (RMAs).
- Two posters were presented at the 34th 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). - 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.
- 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.
- Two posters were presented at the 34th EORTC-NCI-AACR Symposium in late
Presentations are available on Eloxx’s website at link.
Third Quarter 2022 Financial Results
For the three months ended
Our R&D expenses were
Our general and administrative (G&A) expenses were
As of
About
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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.
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
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
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443.213.0506
Media
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UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
(Amounts in thousands, except share and per share data) | ||||||||
2022 |
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 | ||||
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 | 86,656,221 | 86,208,754 | 86,653,811 | 64,428,187 | ||||||||||||
computing net loss per share, basic and diluted | ||||||||||||||||
Source: Eloxx Pharmaceuticals
Source: Eloxx Pharmaceuticals