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
Podocyte foot process effacement is a hallmark of Alport syndrome
Together with previously announced rapid and sustained proteinuria remission in one patient in Phase 2 clinical study, these biopsy results strongly support decision to advance ELX-02 into a pivotal trial in Alport syndrome
Alport syndrome, a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, is characterized by podocyte injury and impaired kidney filter function leading to proteinuria. 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. This is the first potential therapy for Alport syndrome that has shown an improvement of podocyte foot process effacement.
“These are profound results. The improvement in podocyte foot process effacement in all three patients treated confirms the disease modifying potential of ELX-02. Combined with the reduction of proteinuria achieved in one patient, these data strengthen our confidence in ELX-02’s potential to treat Alport syndrome patients with nonsense mutations,” said
Electron microscopy assessment of the kidney biopsies in the Phase 2 study showed a reduction in podocyte foot process effacement, indicative of the disease modifying impact of ELX-02 all three patients. 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. Representative TEM images from Patient 4401-02 can be accessed here
| Patient | Pre-treatment | Post -treatment |
| 4401-01 | Widespread foot process effacement present | Segmental foot process effacement present |
| 4401-02* | Widespread foot process effacement present | Segmental foot process effacement present |
| 4402-01 | Moderate to severe foot process effacement present | Moderate segmental foot process effacement present |
*Patient that achieved remission.
“As a physician scientist with a longtime focus on podocytes, I find these electron microscopy results compelling since the demonstrated improvement in podocyte foot process effacement shows that ELX-02 has substantial potential to treat Alport syndrome,” said Dr.
Eloxx previously announced achievement of a rapid and sustained remission in one patient in the Phase 2 clinical trial. Based on the results from the Phase 2 trial, Eloxx intends to advance ELX-02 into pivotal trial for the treatment of Alport syndrome with nonsense mutations.
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.
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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 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 meet 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
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SOURCE:
Source: Eloxx Pharmaceuticals
