• Publication titled “A Novel Class of Ribosome Modulating Agents Exploits Cancer Ribosome Heterogeneity to Selectively Target the CMS2 Subtype of Colorectal Cancer” published in Cancer Research Communications
  
• Results suggest that MYC overexpressing cancers can be targeted by exploiting ribosome heterogeneity in cancer; preclinical data demonstrated activity of ZKN-157 against subtypes of colorectal cancer
• Research potentially opens large opportunities to selectively target MYC-driven cancers with a novel mechanism and possible synergy with existing cancer therapies

WATERTOWN, Mass., July 10, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that Cancer Research Communications has published “A Novel Class of Ribosome Modulating Agents Exploits Cancer Ribosome Heterogeneity to Selectively Target the CMS2 Subtype of Colorectal Cancer”. The publication demonstrates the potential of Eloxx’s 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.

“We are incredibly pleased with this publication, as it highlights the power of our TURBO-ZM platform to target the human ribosome to develop new potential therapeutics. Importantly, for the first time, we also demonstrate that MYC overexpressing cancers can be targeted by exploiting ribosome heterogeneity, as ZKN-157, a novel RMA, demonstrated activity against subtypes of colorectal cancer,” said Vijay Modur MD, PhD, Head of R&D of Eloxx. “As MYC is dysregulated in approximately 70% of human cancers, this research provides opportunities to selectively target MYC-driven cancers with a novel mechanism and has potential for synergy with existing cancer therapies.”

Eloxx uses a unique synthetic chemistry approach to generate novel RMAs that exploit cancer ribosome heterogeneity. ZKN-157 was designed to selectively target the consensus molecular subtype 2 (CMS2) of colorectal cancer, which is distinguished by high MYC activity. In the preclinical data published, ZKN-157 showed efficacy as a single agent and, the potency and efficacy of ZKN-157 synergized with clinically approved DNA-intercalating agents, which have previously been shown to inhibit ribogenesis as well.

The publication is available on Eloxx’s website at link.

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 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 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 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 March 31, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our 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, 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.

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