Data from Eloxx’s proof-of-concept trial in patients with nonsense mutation Alport Syndrome (NMAS) presented in late-breaking presentation at American Society of Nephrology (ASN) Kidney Week New protein analyses confirm production of both collagen alpha 4 and collagen alpha 5 in patients post treatment with ELX-02 Results justify the need to conduct a larger clinical trial of ELX-02 in NMAS to confirm clinical benefit U.S. Food and Drug Administration (FDA) allows for continued dosing of subjects in Phase 1 trial of ZKN-013 WATERTOWN, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases with nonsense mutations, today provided significant key corporate updates. “The demonstration of both collagen alpha 4 and collagen alpha 5 in patients in our proof-of-concept study of ELX-02 in NMAS patients is an important milestone in the development of this crucial potential therapy. These new results, presented in a late-breaking presentation at ASN Kidney Week, strongly justify the need to conduct a larger trial to confirm these encouraging results in this underserved patient population,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “Also, the FDA allowing for continued dosing of subjects in the Phase 1 trial for ZKN-013 is a substantial achievement for the program and makes Eloxx eligible for the next milestone payment in the company’s agreement with Almirall, once confirmed by our partner, Almirall.” Additional Alport Syndrome data presented at the ASN Kidney Week In late October, Daniel Gale, Professor of Nephrology at University College London Department of Renal Medicine, presented additional results from Eloxx’s proof-of-concept study in three NMAS patients treated with ELX-02 in a late-breaking presentation at ASN Kidney Week. The presentation was titled: “Small molecule premature termination codon readthrough therapy: a Phase 2 pediatric and adult trial in Nonsense Mutation Alport syndrome” at the ASN Kidney Week conference. “Although this was a small trial of limited duration, the appearance of new Type IV Collagen in the glomerular basement membranes is the first time a missing podocyte protein has been reconstituted in a human and provides evidence that ELX-02 is acting as intended in the kidneys of patients. A longer, controlled study will be needed to quantify the clinical effectiveness of this therapy,” said Professor Gale. New protein immunostaining and mass spectrometry analyses that were presented, confirmed the production of both collagen a4 (IV) and collagen a5 (IV) proteins in NMAS patients treated with ELX-02. Prior to treatment, collagen a4 (IV) and a5 (IV) were absent in the glomerulus in all three patients. Post treatment biopsy of the patient with the largest improvement in Filtration Slit Density showed a 34% increase in collagen a4 (IV) immunostaining intensity and presence of collagen a3/a4/a5 (IV) peptide with a greater than 6-fold increase in collagen a4 and detectable collagen a5 consistent with formation of a functional protein induction. No collagen alpha 5 was detected pre-treatment in this patient. These additional data support ELX-02’s mechanism of protein induction, and the observed morphology change and reduced foot process effacement in all the patients that was previously reported. The results also justify the conduct of a larger clinical trial with longer ELX-02 treatment duration to validate the observed clinical benefit in NMAS patients. The demonstrated readthrough of premature stop codons also suggests ELX-02 has potential in other rare kidney diseases, including autosomal dominant polycystic kidney disease and cystinosis. FDA allows for continued dosing of subjects in Phase 1 trial of ZKN-013 Following successful dosing of the initial subjects, FDA has allowed continued dosing of healthy volunteers for ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa and junctional epidermolysis bullosa. Eloxx is awaiting confirmation from Almirall to proceed with the study based on their review of the results and FDA feedback. ZKN-013 has been exclusively licensed to Almirall, S.A. (BME:ALM), who has global rights to develop and commercialize ZKN-013. Under the terms of the license agreement, Eloxx is eligible for additional development, regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales. 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. Forward-looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement, the expected timeline for clinical development, the efficacy of the Company’s product candidates ZKN-013 and ELX-02, the Company’s expected and planned communications with and submissions to the FDA and anticipated funding under the bridge loan 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: the Company’s incurrence of significant losses; the Company has identified conditions and events that raise substantial doubt about its ability to continue as a going concern; its ability, or its licensees’ 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 the Company’s, and its licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the Company’s ability to obtain the capital necessary to fund its operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; the Company’s ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for the Company’s 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 the Company’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in the Company’s other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of the Company’s website at https://investors.eloxxpharma.com/financials-filings. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, the Company has 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. ContactJohn Woolfordjohn.woolford@westwicke.com 443.213.0506 Source: Eloxx Pharmaceuticals
