Post by icemandios on Nov 14, 2023 3:02:03 GMT
Eloxx Pharmaceuticals Reports Third Quarter 2023 Financial and Operating Results and Provides Business Update
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 NIPOKA GmbH
Clinical data from Phase 2 study of ELX-02 for Alport Syndrome included in presentations at the American Society of Nephrology (ASN) Kidney Week 2023
Significant progress toward completion of a strategic partnership for ZKN-013
WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.
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.
Raised additional cash in ongoing efforts to strengthen balance sheet.
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.
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.
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.
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.
For the three months ended September 30, 2023, we incurred a net loss of $3.6 million, or $1.31 per share, which included $0.4 million in stock-based compensation. For the same period in the prior year, we incurred a net loss of $7.5 million, or $3.47 per share, which included $0.7 million in stock-based compensation.
R&D expenses were $1.3 million for the three months ended September 30, 2023, which included $0.1 million in stock-based compensation. For the same period in the prior year, R&D expenses were $4.9 million, which included $0.3 million of stock-based compensation. The decrease was primarily related to a decrease in expenses related to subcontractors, advisors, and laboratory supplies in connection with preclinical research and development activities related to inhaled delivery of ELX-02 in CF, a decrease in CFF funded activities, a decrease in salaries and other personnel costs, a decrease related to stock-based compensation, and a decrease in facility and overhead expenses.
General and administrative (G&A) expenses were $2.4 million for the three months ended September 30, 2023, which included $0.4 million in stock-based compensation. For the same period in the prior year, G&A expenses were $2.3 million, which included $0.4 million of stock-based compensation. The increase was primarily related to a $0.2 million increase in expenses attributable to professional and consulting fees, offset by a decrease of $0.1 million related to facility and overhead expenses.
As of September 30, 2023, we had unrestricted cash and cash equivalents of $4.8 million. During the quarter, we raised gross proceeds of $2.0 million from the sale of shares of common stock, pre-funded warrants and warrants. Additionally, we sold shares of common stock through our ATM program during the quarter for gross proceeds of $1.8 million. Eloxx remains focused on its liquidity position and raising additional capital in the near term in order to fund its operating plan through the end of 2023 and beyond. Assuming that we initiate Phase 3 clinical trial activities when sufficient funding allows, and that we maintain compliance with our debt covenants, we believe that our current cash position will be sufficient to fund our operations through the end of the fourth quarter of 2023.
As previously reported, we received a delisting determination from the Listing Qualifications Department of the Nasdaq Stock Market LLC as the company did not regain compliance with the Nasdaq Listing Rule 5550(b)(2), which requires a listed company to have at least $35 million in market value of listed securities. Effective October 16, 2023, trading of our common stock was suspended on the Nasdaq Capital Market and began trading on the OTC Pink Marketplace under the symbol “ELOX.” On October 26, 2023, we requested a review of the Nasdaq delisting determination by the Nasdaq Listing Council. Trading of or common stock on the Nasdaq Capital Market will remain suspended pending a decision by the Nasdaq Listing Council.
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, Europe, Japan and China with Nonsense Mutation Alport Syndrome. These patients have no approved treatment options.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com .