ZKN-013 is in development for potential treatment of recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB) and Familial Adenomatous Polyposis (FAP)

ZKN-013 is Eloxx’s first drug candidate from its TURBO-ZM™ platform to gain clearance for clinical testing

WATERTOWN, MA, USA I May 02, 2023 I Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the “Company”), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

“FDA clearance to begin our planned single ascending dose trial is an important milestone towards providing a potential treatment option for patients with RDEB and JEB,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “This update is also a momentous event for Eloxx, as ZKN-013, our lead TURBO-ZM™ based molecule, is the first program developed from hit to lead and is now poised to enter clinical development. Beyond ZKN-013, Eloxx has promising TURBO-ZM based discovery programs in cystic fibrosis (fully funded by the Cystic Fibrosis Foundation) and in certain cMYC-overexpressing cancers.”

Further SAD and multiple ascending dose (MAD) testing is expected to be conducted following the completion of the planned dose cohorts in the SAD study and discussion with the FDA. The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA.

ZKN-013 Preclinical Activity in RDEB, JEB and FAP

ZKN-013 has demonstrated robust functional preclinical activity in RDEB and JEB patient cells and in APCmin (multiple intestinal neoplasia) mice.

Eloxx also plans to develop ZKN-013 for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival.

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. Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit www.eloxxpharma.com.

SOURCE: Eloxx Pharmaceuticals