Cystic Fibrosis Foundation (
Topline data from cystic fibrosis (CF) Phase 2 expansion treatment arms evaluating combination with ivacaftor expected at the end of the first half of 2022
Expanded development of ELX-02 for the treatment of Alport syndrome, a rare kidney genetic disorder, with Phase 2 expected to start in second half of 2022
Expect to submit an Investigational New Drug (IND) application for the inhaled delivery of ELX-02 in the second half of 2022
On track to start First in Human Phase 1 study in 2022 with ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) patients with nonsense mutations cells
Cash and equivalents expected to be sufficient to fund operations into the second quarter of 2023
“During the first quarter, we made significant progress, highlighted by the funding award from the
First Quarter 2022 and Subsequent Highlights
Class 1 Cystic Fibrosis
March 2022, Eloxx announced additional funding from a Therapeutic DevelopmentAward of up to $15.9 millionfrom the CF Foundationto support the ongoing global Phase 2 ELX-02 clinical development of ELX-02 in Class 1 CF. Following an upfront funding of $7.0 million, the funding will be tranched based on the achievement of certain clinical milestones.
- Phase 2 clinical trials in CF patients, with expansion arms designed to evaluate the safety of ELX-02 and assess short-term biological activity in patients, remains ongoing. Topline results are expected at the end of the first half of 2022.
- Evaluation of inhaled (nebulizer-based) delivery of the current subcutaneous formulation of ELX-02 remains ongoing. We believe that the increased drug exposure in the lung versus plasma with inhaled delivery has the potential to further improve the activity of ELX-02. We remain on track to submit an Investigational New Drug (IND) application in the second half of 2022.
March 2022, Eloxx announced it has expanded its clinical development pipeline to include the potential treatment with ELX-02 of a subset of 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.
- Eloxx believes there is a strong rationale to pursue clinical development of ELX-02 in Alport syndrome based on encouraging preclinical results and clinical results.
- Clinical readthrough results in our Phase 2 cystic fibrosis trial at well tolerated dose levels confirm activity of ELX-02 to restore proteins.
- In recently published preclinical studies, ELX-02 has demonstrated significant readthrough in COL4A5 mutations, which represent 85% of nonsense mutations in this population. Previously published in vivo studies have shown that even low levels of Collagen IV restoration may result in significant reduction in proteinuria.
- ELX-02 is preferentially taken up in the kidney with an expected greater than 50-fold exposure in the kidneys compared to plasma. As a result, we expect that a low dose of 0.75mg/kg/day of ELX-02 could restore therapeutically relevant levels of Collagen IV.
- Eloxx intends to initiate a proof-of-concept clinical trial in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022. 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.
Recessive Dystrophic Epidermolysis Bullosa and Junctional Epidermolysis Bullosa (JEB)
- ZKN-013 continues to demonstrate dose dependent inducement of functional Collagen VIIA protein (truncated in patients with nonsense mutations patients), in RDEB patient cells.
- Eloxx continues to expect to file an IND application to start a First in Human (FIH) Phase 1 study in 2022 with ZKN-013 after recently completing 28-day non Good Laboratory Practice (GLP) animal studies.
Familial Adenomatous Polyposis
- FAP is a rare inherited disease characterized by proliferation of colon polyps with no approved drug therapies. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.
- As previously announced, Eloxx reported observed encouraging results from an 8-week treatment study for ZKN-013 in the APCMin (multiple intestinal neoplasia) model to evaluate the potential of ZKN-013 to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP.
- 10-week old APCMin mice were randomized for treatment with ZKN-013 for 8 weeks (n=10 in each group)
- Treatment with ZKN-013 resulted in a significant 39% reduction in the number of colon polyps and an approximately 50% reduction in polyp burden. There were substantial reductions in both lesion area and area of adenoma with no progression to carcinomas
- This led to an observed 50% survival benefit with no deaths in mice treated with ZKN-013.
First Quarter 2022 Financial Results
For the three months ended
Our research and development expenses (R&D) were
Our general and administrative (G&A) expenses were
About Nonsense Mutations
Nonsense mutations cause a premature stop codon in the mRNA resulting in less than full length or loss of function proteins. These remain highly underserved with no approved disease modifying therapies. An estimated 10-12% patients across over 8,000 inherited genetic rare diseases harbor nonsense mutations in one or both alleles harboring nonsense mutations.
For more information, please visit www.eloxxpharma.com.
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, the sufficiency of our cash and cash equivalents to fund our operations, 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 quarterly period 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.
|UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS|
|(in thousands, except share and per share data)|
|Cash and cash equivalents||$||39,768||$||42,268|
|Prepaid expenses and other current assets||2,082||913|
|Total current assets||42,147||43,480|
|Property and equipment, net||206||216|
|Operating lease right-of-use assets||1,265||1,443|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Advances from collaboration partners||10,723||3,723|
|Current portion of operating lease liabilities||647||657|
|Total current liabilities||19,174||9,955|
|Operating lease liabilities||638||804|
|Total stockholders’ equity||11,686||22,384|
|Total liabilities and stockholders' equity||$||43,618||$||45,139|
|UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|(in thousands, except share and per share data)|
|Three Months Ended
|Research and development||$||7,899||$||4,073|
|General and administrative||3,054||4,341|
|Total operating expenses||10,953||8,414|
|Loss from operations||(10,953||)||(8,414||)|
|Other expense, net||667||280|
|Basic and diluted net loss per share||$||0.13||$||0.22|
|Weighted average number of common shares used in computing net loss per share, basic and diluted||86,651,036||40,180,131|
Source: Eloxx Pharmaceuticals