Eloxx Pharmaceuticals Announces Publication Demonstrating the Power of its TURBO-ZM™ Platform to Target the Human Ribosome for Therapeutic Benefit

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.

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SOURCE: Eloxx Pharmaceuticals, Inc.