Announced today that all 3 patients (100% response rate) treated with ELX-02 showed an improvement in podocyte foot process effacement post-treatment in kidney biopsies assessed by electron microscopy, demonstrating the disease-modifying effect of ELX-02
Announced achievement of remission in one patient in Phase 2 clinical study of ELX-02 for the treatment of Alport syndrome and decision to advance ELX-02 into a pivotal trial in Alport syndrome
Highlighted significant unmet need in the treatment of Alport syndrome and additional positive data from Phase 2 clinical study evaluating ELX-02 in KOL event
Nasdaq granted Eloxx’s request for an extension to regain compliance with the Market Value of
“This is a transformative time at Eloxx. With today’s confirmation of the disease modifying potential of ELX-02 in all three patient biopsies from the Alport syndrome trial, we look forward to advancing to a pivotal trial of ELX-02 for the treatment of Alport syndrome,” said
Second Quarter 2023 and Subsequent Highlights
- Eloxx intends to advance ELX-02 into pivotal trial for the treatment of Alport syndrome with nonsense mutations, pending obtaining the necessary capital. Alport syndrome is a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, characterized by podocyte injury and impaired kidney filter function leading to proteinuria.
- In a separate press release, Eloxx today announced positive biopsy results from its proof-of-concept Phase 2 open-label clinical trial (NCT05448755) of ELX-02 for the treatment of Alport syndrome after eight weeks of treatment. All three patients (100% response rate) treated with ELX-02 showed an improvement in podocyte foot process effacement post-treatment in kidney biopsies assessed by electron microscopy demonstrating the disease modifying effect of ELX-02 and potential for improvement in proteinuria with longer duration of treatment.
- Podocytes are specialized cells that bind to the glomerular basement membrane and form finger-like extensions called foot processes that enable efficient ultrafiltration. Podocyte injury leads to the effacement (loss) of podocyte foot processes and proteinuria in nearly all cases of Alport syndrome.
- In two patients, widespread foot process effacement was improved to segmental foot process effacement. In the third patient, moderate to severe foot process effacement was improved to moderate only.
- Eloxx previously announced achievement of remission in one patient. One month after the end of treatment, the patient demonstrated a rapid increase in Urine Protein to Creatinine (UPCR), providing additional evidence of drug activity.
- ELX-02 was well-tolerated in the study, with no discontinuations to date.
- An IND application for ELX-02 is expected to be submitted to the FDA in the third quarter of 2023.
- Additional data recently announced regarding the efficacy of ELX-02 in its cystic fibrosis study bolsters the strength of results in Phase 2 Alport syndrome trial, further supporting Eloxx’s decision to advance into a pivotal trial in Alport syndrome.
- Alport syndrome RaDaR natural history data presented at the 60th
European Renal Association Congressindicates that Alport syndrome patients with autosomal recessive COL4A4 mutations have severest disease, with a more rapid progression to kidney failure. The patient that achieved remission in Eloxx Phase 2 trial had autosomal recessive COL4A4 nonsense mutation resulting in a truncated protein.
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB)
May 2023, Eloxx announced that the FDA has cleared the IND application to initiate a SAD clinical trial in healthy volunteers for ZKN-013 for the potential treatment of RDEB with nonsense mutations. RDEB is a rare skin disease characterized by mutations in the Collagen 7 gene. Eloxx plans to initiate the Phase 1 SAD clinical study, assuming sufficient funding, with the first subject expected to be dosed by the end of 2023.
- Further SAD and multiple ascending dose (MAD) testing are expected to be conducted following the completion of the planned dose cohorts in the SAD study and discussion with the FDA. The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by FDA.
- Preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes. In this trial, read-through activity resulted in up to an 18-fold increase in full-length COL VII protein levels. Prolonged treatment with ZKN-013 was shown to further increased COL VII protein levels. Functionality of the restored full-length COL VII protein was observed. These results have been accepted for presentation at an upcoming medical conference.
Familial Adenomatous Polyposis (FAP)
- Eloxx also plans, assuming sufficient funding, to develop 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.
Cancer Research Communicationspublished “A Novel Class of Ribosome Modulating Agents Exploits Cancer Ribosome Heterogeneity to Selectively Target the CMS2 Subtypeof Colorectal Cancer.” The publication demonstrates the of potential the TURBO-ZM chemistry technology platform to develop novel Ribosome Modulating Agents (RMAs) and details preclinical data that demonstrate activity for ZKN-157 against subtypes of colorectal cancer.
- Results suggest that MYC-overexpressing cancers can be targeted by exploiting ribosome heterogeneity in cancer, as preclinical data has demonstrated the activity of ZKN-157 against subtypes of colorectal cancer. This research potentially provides opportunities to selectively target MYC-driven cancers with a novel mechanism and possible synergy with existing cancer therapies.
Second Quarter 2023 Financial Results
For the three months ended
R&D expenses were
General and administrative (G&A) expenses were
Eloxx received notice from the
About Alport syndrome
Alport syndrome is a genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities caused by mutations in the genes (COL4A3, COL4A4, and COL4A5) needed for production of type 4 collagen. Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations. These patients have significantly worse clinical outcomes than other patients with Alport syndrome and have no disease modifying treatment options.
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 cash runway to fund our operating plan, our plans to raise additional capital, and our ability to comply with the covenants in our debt agreement, the expected timing of and results from trials 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; our ability to regain and maintain compliance with the continued listing requirements of the Nasdaq Capital Market; 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|
|(Amounts in thousands, except share and per share data)|
|Cash and cash equivalents||$||4,331||$||19,207|
|Prepaid expenses and other current assets||841||661|
|Total current assets||5,382||20,129|
|Property and equipment, net||130||169|
|Operating lease right-of-use asset||481||825|
|LIABILITIES AND STOCKHOLDERS’ DEFICIT|
|Current portion of long-term debt||2,276||3,980|
|Advances from collaboration partners||12,535||12,535|
|Current portion of operating lease liability||492||712|
|Total current liabilities||21,251||23,091|
|Long-term debt, net of current portion||3,334||8,557|
|Operating lease liability||4||135|
|Total stockholders’ deficit:||(18,596||)||(10,660||)|
|Total liabilities and stockholders’ deficit||$||5,993||$||21,123|
|UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS|
|(Amounts in thousands, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Research and development||$||2,338||$||7,651||$||5,826||$||15,550|
|General and administrative||1,802||2,645||3,797||5,699|
|Total operating expenses||4,140||10,296||9,623||21,249|
|Loss from operations||(4,140||)||(10,296||)||(9,623||)||(21,249||)|
|Other expense, net||201||322||948||989|
|Net loss per share, basic and diluted||$||(1.96||)||$||(4.90||)||$||(4.83||)||$||(10.27||)|
|Weighted average number of shares of common stock used in computing net loss per share, basic and diluted||2,212,364||2,166,352||2,189,487||2,166,314|
Source: Eloxx Pharmaceuticals