Acquisition of Zikani Therapeutics,
Additional Treatment Arm Added to Ongoing Phase 2 Clinical Studies for Cystic Fibrosis (CF)
ELX-02 Phase 1 Results Published in Multiple Scientific Journals
“The beginning of 2021 has been transformative for Eloxx and was extremely important in laying the groundwork for the future success of ELX-02, our candidate for cystic fibrosis, and in amplifying the potential of Eloxx’s innovative science and be a leader in RNA-targeted genetic therapies. Our enthusiasm has only increased in the short period since the acquisition,” said
First Quarter 2021 and Subsequent Highlights
- We acquired
Zikani Therapeutics, Inc. , a ribosomal modulation company, in an all stock transaction onApril 1, 2021 . We believe the acquisition maximizes the potential for ELX-02 for CF, which is currently in Phase 2 development. The acquisition also adds a preclinical stage pipeline in rare diseases and oncology targeting RNA and ribosomal mutations. Eloxx expects to file an Investigational New Drug application for the first oral drug to treat patients with Recessive Dystrophic and Junctional Epidermolysis Bullosa (RDEB and JEB) in 2022.Sumit Aggarwal , former Zikani President and CEO, was appointed President and CEO of Eloxx. - As previously announced, the new study arm was added to our global Phase 2 clinical program for ELX-02 for the treatment of CF in patients with at least one G542X allele. The Phase 2 clinical program now includes a fifth treatment arm to evaluate safety of ELX-02 in combination with Kalydeco® (ivacaftor), a FDA-approved CF transmembrane conductance regulator (CFTR) potentiator for the treatment of CF in patients who have at least one mutation in their CF gene amenable to ivacaftor. The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity. We remain on track to present data from the first four treatment arms in the second half of this year.
The CF Foundation extended its funding of our clinical trial program to includeEurope andIsrael .- Phase 1 results for ELX-02 were published in multiple medical journals, including:
- “Targeting G542X CFTR Nonsense Alleles with ELX-02 Restores CFTR Function in Human-Derived Intestinal Organoids” in the
Journal of Cystic Fibrosis ; - “A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX-02 in Healthy Subjects” in the journal Clinical Pharmacology in Drug Development; and
- “Phase 1 Renal Impairment Trial Results Enable Targeted Individualized Dosing of ELX-02 in Nephropathic Cystinosis Patients” in the
Journal of Clinical Pharmacology .
- “Targeting G542X CFTR Nonsense Alleles with ELX-02 Restores CFTR Function in Human-Derived Intestinal Organoids” in the
First Quarter 2021 Financial Results
For the three months ended
Our research and development expenses (R&D) 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 the expected timing of trials and results from clinical studies of our product candidate, the expansion of our clinical trial sites 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 Annual Report on Form 10-K for the fiscal year 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.
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UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||
(Amounts in thousands) | ||||||
ASSETS | ||||||
Current assets: | ||||||
Cash and cash equivalents | $ | 18,247 | $ | 24,668 | ||
Restricted cash | 54 | 56 | ||||
Prepaid expenses and other current assets | 1,659 | 1,169 | ||||
Total current assets | 19,960 | 25,893 | ||||
Property and equipment, net | 117 | 133 | ||||
Operating lease right-of-use asset | 289 | 421 | ||||
Other long-term assets | - | 30 | ||||
Total assets | $ | 20,366 | $ | 26,477 | ||
LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||
Current liabilities: | ||||||
Accounts payable | $ | 485 | $ | 481 | ||
Accrued expenses | 2,917 | 2,886 | ||||
Current portion of long-term debt | 5,562 | 5,239 | ||||
Advances from collaboration partners | 3,411 | 805 | ||||
Current portion of operating lease liability | 280 | 389 | ||||
Taxes payable | 38 | 38 | ||||
Total current liabilities | 12,693 | 9,838 | ||||
Long-term debt | 4,913 | 6,376 | ||||
Operating lease liability | 10 | 33 | ||||
Total liabilities | 17,616 | 16,247 | ||||
Total stockholders’ equity | 2,750 | 10,230 | ||||
Total liabilities and stockholders’ equity | $ | 20,366 | $ | 26,477 | ||
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||
(Amounts in thousands, except share and per share data) | |||||||
Three Months Ended |
|||||||
2021 | 2020 | ||||||
Operating expenses: | |||||||
Research and development | $ | 4,073 | $ | 4,767 | |||
General and administrative | 4,341 | 5,006 | |||||
Restructuring charges | — | 3,994 | |||||
Total operating expenses | 8,414 | 13,767 | |||||
Loss from operations | (8,414 | ) | (13,767 | ) | |||
Other expense, net | 280 | 179 | |||||
Net loss | $ | (8,694 | ) | $ | (13,946 | ) | |
Net loss per share, basic and diluted | $ | (0.22 | ) | $ | (0.35 | ) | |
Weighted average number of shares of common stock used in computing net loss per share, basic and diluted | 40,180,131 | 40,074,275 | |||||
Source: Eloxx Pharmaceuticals