ELOXX PHARMACEUTICALS, INC. Added Additional Treatment Arm and Provided Enrollment Update for Ongoing ELX-02 Phase 2 Clinical Trials for Cystic Fibrosis (CF)
Nominated ZKN-013 as Lead Candidate for Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB) Programs
Advanced Multiple Preclinical Rare Inherited and Targeted Oncology Programs
Received Funding Award from
Raised Approximately
“Since the acquisition of Zikani Therapeutics in early April, we have made tremendous progress across our portfolio of novel therapeutic programs,” said
Second Quarter 2021 and Subsequent Highlights
Class 1 Cystic Fibrosis
- Ongoing ELX-02 Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene are designed to evaluate the safety of ELX-02 and assess short-term biological activity in patients.
- As previously announced, based on enrollment as of the end of
June 2021 , we remain on track to report data from the monotherapy arms of the ELX-02 clinical trials in the fourth quarter of 2021. We are continuing to enroll additional patients to support Phase 3 clinical trial planning. - Biological activity is being assessed by changes in sweat chloride, a surrogate marker for restoring CFTR activity. For comparison, Orkambi, an FDA-approved CF agent, demonstrated mid-single digit reductions in sweat chloride over one- to two-week study durations in a similar trial setting.
- Patient dosing in the expansion arm, which includes a combination of ELX-02 and the CFTR protein potentiator, Kalydeco (ivacaftor), is expected to begin by the end of 2021. Previously disclosed preclinical organoid experiments have demonstrated that the addition of Kalydeco to ELX-02 can enhance activity up to three-fold.
- Safety Review Committee has approved advancement to highest dose level to be studied.
- As previously announced, based on enrollment as of the end of
- Began evaluation of inhaled (nebulizer-based) delivery of the current subcutaneous formulation of ELX-02. Preclinical rodent studies of ELX-02 and other aminoglycosides have demonstrated 30- to 100-fold higher drug concentration in the lung versus plasma. This has the potential to further improve the activity of ELX-02 as both a single agent and in combination with other drugs.
- Received an award of up to
$2.6M from theCystic Fibrosis Foundation to identify optimized oral Ribosome Modulating Agents (RMAs) with our TURBO-ZM™ Platform for further development for the treatment of CF patients with nonsense mutations.
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB)
- Nominated ZKN-013 as the drug candidate to move into IND-enabling studies for the treatment of RDEB and JEB. Good Laboratory Practice (GLP) safety studies are expected to begin by the end of 2021. We remain on track to file an IND submission in 2022.
- In preclinical experiments, treating RDEB patient-derived fibroblasts and keratinocytes with ZKN-013 showed clinically relevant restoration of full-length Collagen 7A (COL7A) across multiple genotypes in a dose dependent manner.
- In a non-GLP animal toxicity study, ZKN-013 demonstrated a substantial safety margin with therapeutically sufficient drug levels in skin with chronic dosing.
Rare Inherited and Targeted Oncology
- Continued to advance our preclincial oncology pipeline, which is focused on rare inherited cancers with driver nonsense mutations and cancers with driver mutations in the ribosome.
- Our lead program is for the treatment of patients with Familial Adenomatous Polyposis (FAP).
- We have initiated an 8-week study in APCMin (multiple intestinal neoplasia) mice to evaluate the potential of RMAs to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP.
- Prior studies of erythromycin in APCMin mice showed reductions in colon polyps that successfully translated to demonstrating clinical efficacy.1
- This study in APCMin will evaluate polyp number and size in ZKN013 treated mice versus control mice. We expect to report results from this study in the fourth quarter of 2021.
- We have initiated an 8-week study in APCMin (multiple intestinal neoplasia) mice to evaluate the potential of RMAs to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP.
- Initiated cancer cell line and xenograft mouse studies to evaluate the response to treatment with RMAs to advance first-in-class onco-ribosome targeted inhibitors for oncology therapy.
Corporate
- An underwritten public offering of 38,333,334 shares of common stock, including the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of
$1.35 per share was closed inMay 2021 . Aggregate gross proceeds from the offering were approximately$51.75 million , before deducting underwriting discounts and commissions and offering expenses. Pedro Huertas , M.D., Ph.D., joined Eloxx as a Senior Adviser.Dr. Huertas previously served as CMO of Eloxx. He has also served as CMO of Inozyme Pharma andSentien Biotechnologies and in various roles at Pfizer, Shire,Massachusetts General Hospital , andGenzyme .
Second 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
1 J Mol Med (Berl) 2016 Apr;94(4):469-82) and Int J Cancer 2020 Feb 15;146(4):1064-1074)
About
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 expected cash burn and future financial results, 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, 2021, as any such factors may be updated from time to time in our other filings with the
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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Media
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| UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
| (Amounts in thousands, except share and per share data) | |||||||
2021 | 2020 | ||||||
| ASSETS | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 56,734 | $ | 24,668 | |||
| Restricted cash | 246 | 56 | |||||
| Prepaid expenses and other current assets | 1,579 | 1,169 | |||||
| Total current assets | 58,559 | 25,893 | |||||
| Property and equipment, net | 185 | 133 | |||||
| Operating lease right-of-use asset | 1,866 | 421 | |||||
| Other long-term assets | - | 30 | |||||
| Total assets | $ | 60,610 | $ | 26,477 | |||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 2,024 | $ | 481 | |||
| Accrued expenses | 3,302 | 2,886 | |||||
| Current portion of long-term debt | 5,686 | 5,239 | |||||
| Advances from collaboration partners | 3,411 | 805 | |||||
| Current portion of operating lease liability | 753 | 389 | |||||
| Taxes payable | 34 | 38 | |||||
| Total current liabilities | 15,210 | 9,838 | |||||
| Long-term debt | 3,637 | 6,376 | |||||
| Operating lease liability | 1,120 | 33 | |||||
| Total liabilities | 19,967 | 16,247 | |||||
| Total stockholders’ equity | 40,643 | 10,230 | |||||
| Total liabilities and stockholders' equity | $ | 60,610 | $ | 26,477 | |||
| UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
| (Amounts in thousands, except share and per share data) | ||||||||||||||||
| Three Months Ended | Six Months Ended | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 5,704 | $ | 3,738 | $ | 9,777 | $ | 8,505 | ||||||||
| General and administrative | 7,355 | 3,848 | 11,696 | 8,854 | ||||||||||||
| In process research and development | 22,670 | — | 22,670 | — | ||||||||||||
| Restructuring charges | — | — | — | 3,994 | ||||||||||||
| Total operating expenses | 35,729 | 7,586 | 44,143 | 21,353 | ||||||||||||
| Loss from operations | (35,729 | ) | (7,586 | ) | (44,143 | ) | (21,353 | ) | ||||||||
| Other expense, net | 329 | 301 | 609 | 480 | ||||||||||||
| Net loss | $ | (36,058 | ) | $ | (7,887 | ) | $ | (44,752 | ) | $ | (21,833 | ) | ||||
| Basic and diluted net loss per share | $ | (0.54 | ) | $ | (0.20 | ) | $ | (0.84 | ) | $ | (0.54 | ) | ||||
| Weighted average number of common shares used in computing net loss per share, basic and diluted | 66,389,865 | 40,129,304 | 53,357,401 | 40,101,789 | ||||||||||||
SOURCE:
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