Kidney morphology improved in all three patients with protein re-expression consistent with disease regression in its Phase 2 open-label clinical trial of ELX-02 for the treatment of Alport Syndrome
ELX-02 improved podocyte foot process effacement in all three treated patients by an average of 60% based on blinded biopsy analysis by
Clinical data from Phase 2 study of ELX-02 for Alport Syndrome included in presentations at the
Significant progress toward completion of a strategic partnership for ZKN-013
- Completed Phase 2 open label trial of ELX-02 for the treatment of Alport Syndrome patients with nonsense mutations.
- Analysis of biopsy samples provided unequivocal clinical evidence of both morphology and ELX-02’s disease modifying potential.
- Collagen Alpha 5 protein expression observed in the glomerular basement membrane post treatment in all patients.
- Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the
American Society of Nephrology (ASN) Kidney Week 2023. - Significant progress towards completing a strategic partnership for ZKN-013
- Raised additional cash in ongoing efforts to strengthen balance sheet.
“We are excited about the recent progress across our pipeline and remain committed to advancing our programs for the benefit of our patients and stakeholders,” said
Third Quarter 2023 and Subsequent Highlights
ELX-02 in Nonsense Mutation Alport Syndrome
- Confirmed that all three patients showed an improvement in morphology and proteinuria in 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 patients had Autosomal Recessive Alport Syndrome with a nonsense mutation on one allele. No collagen 4 alpha 5 was expressed in the glomerular basement membrane (GBM) at baseline.
- Collagen 4 Alpha 5 expression in the GBM post-treatment was reported post-treatment based on immunofluorescence staining of patient biopsies
- ELX-02 increased the filtration slit density (FSD) by an average of 60% as compared to baseline levels, consistent with reduction of podocyte foot process effacement.
- Biopsies were analyzed on a blinded basis by
NIPOKA GmbH . FSD was estimated for 15 to 20 glomeruli per sample. - Data supporting FSD as a precise histopathological estimator of podocyte health and its inverse correlation with proteinuria was presented at the ASN meeting on
November 2, 2023 .
- Biopsies were analyzed on a blinded basis by
- Visual assessment of improvement in foot process effacement in Transmission Electron Micrography (TEM) images was independently confirmed by highly regarded renal pathologist and TEM expert.
- All three treated patients had reduced proteinuria variability and two patients had a reduction in proteinuria compared to baseline during or in 2 months post treatment consistent with improvement in kidney morphology.
- ELX-02 was well-tolerated in the study with no discontinuations.
- Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the ASN Kidney Week 2023.
ZKN-013 in nonsense mutation Recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and Familial Adenomatous Polyposis
- Eloxx made significant progress in completing a strategic transaction for ZKN-013.
- Following FDA clearance to begin a single ascending dose trial in healthy volunteers for ZKN-013 for the treatment of recessive RDEB and JEB, Eloxx received significant strategic interest in ZKN-013.
- Should these discussions lead to a transaction, it will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform.
Third Quarter 2023 Financial Results
For the three months ended
R&D expenses were
General and administrative (G&A) expenses were
As of
As previously reported, we received a delisting determination from the
About Nonsense Mutation Alport Syndrome
Nonsense Mutation Alport syndrome is a rare Type IV Collagenopathy characterized by mutations in the genes (COL4A3, COL4A4, and COL4A5) that result in a less than full length (truncated) Type 4 Collagen. This disorder mostly affects children with a median age at diagnosis of 9 to 20 years. It is characterized by rapid and progressive damage to the kidneys, ear and eyes, starting with worsening of kidney morphology to proteinuria and finally kidney failure, hearing loss and eye abnormalities. It is estimated that there are approximately 7,500 patients in the US and 20,000 patients in US,
About
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 cash runway to fund our operating plan, our plans to raise additional capital, our ability to comply with the covenants in our debt agreement, the expected timing of and results from trials of our product candidates, the potential of our product candidate to treat nonsense mutations, the outcome of the Nasdaq Listing Council’s review of the delisting determination; the strategic partnership for the clinical development of ZKN-013 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 obtain the capital necessary to fund our operations; our ability to regain and maintain compliance with the continued listing requirements of the Nasdaq Capital Market; 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;; 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.
Contact
Investors
john.woolford@westwicke.com
443.213.0506
Media
laureen@outcomescg.com
Source:
| UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
| (Amounts in thousands, except share and per share data) | ||||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 4,775 | $ | 19,207 | ||||
| Restricted cash | 219 | 261 | ||||||
| Prepaid expenses and other current assets | 635 | 661 | ||||||
| Total current assets | 5,629 | 20,129 | ||||||
| Property and equipment, net | 107 | 169 | ||||||
| Operating lease right-of-use asset | 306 | 825 | ||||||
| Total assets | $ | 6,042 | $ | 21,123 | ||||
| LIABILITIES AND STOCKHOLDERS’ DEFICIT | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 3,134 | $ | 3,020 | ||||
| Accrued expenses | 2,442 | 2,799 | ||||||
| Current portion of long-term debt | 2,819 | 3,980 | ||||||
| Advances from collaboration partners | 12,966 | 12,535 | ||||||
| Warrant liabilities | 1,695 | — | ||||||
| Current portion of operating lease liability | 315 | 712 | ||||||
| Derivative liabilities | 95 | 45 | ||||||
| Total current liabilities | 23,466 | 23,091 | ||||||
| Long-term debt, net of current portion | 2,621 | 8,557 | ||||||
| Operating lease liability | 3 | 135 | ||||||
| Total liabilities | 26,090 | 31,783 | ||||||
| Total stockholders’ deficit: | (20,048 | ) | (10,660 | ) | ||||
| Total liabilities and stockholders’ deficit | $ | 6,042 | $ | 21,123 | ||||
| UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
| (Amounts in thousands, except share and per share data) | ||||||||||||||||
| Three Months Ended |
Nine Months Ended |
|||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 1,250 | $ | 4,880 | $ | 7,076 | $ | 20,430 | ||||||||
| General and administrative | 2,387 | 2,262 | 6,184 | 7,961 | ||||||||||||
| Total operating expenses | 3,637 | 7,142 | 13,260 | 28,391 | ||||||||||||
| Loss from operations | (3,637 | ) | (7,142 | ) | (13,260 | ) | (28,391 | ) | ||||||||
| Other (income) expense, net | (45 | ) | 366 | 903 | 1,355 | |||||||||||
| Net loss | $ | (3,592 | ) | $ | (7,508 | ) | $ | (14,163 | ) | $ | (29,746 | ) | ||||
| Net loss per share, basic and diluted | $ | (1.31 | ) | $ | (3.47 | ) | $ | (5.96 | ) | $ | (13.73 | ) | ||||
| Weighted average number of shares of common stock used in computing net loss per share, basic and diluted | 2,747,687 | 2,166,404 | 2,377,599 | 2,166,344 | ||||||||||||
Source: Eloxx Pharmaceuticals
