Eloxx Pharmaceuticals, Inc. provided significant pipeline updates. First patient dosed in Phase 1 clinical trial of ZKN-013: The first two subjects have been dosed in the ongoing Phase 1 healthy volunteer single-ascending dose study of ZKN-013. ZKN-013 is designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP).

This Phase 1 study evaluates the safety and pharmacokinetics in healthy volunteers. ZKN-013 has been exclusively licensed to Almirall, S.A., 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.

Positive feedback from the PIND meeting with FDA for ELX-02 clinical development in NMAS patients in the US: Eloxx has received FDA?s final minutes from the hybrid in-person and video conference PIND meeting the company held with the Office of Cardiology, Hematology, Endocrinology, and Nephrology - Division of Cardiology and Nephrology. The company believe that the FDA?s positive feedback and guidance provides a pathway to an IND application submission to initiate a validating Phase 2 clinical trial in the US with ELX-02 in NMAS patients. As part of the meeting, the FDA reviewed the clinical data from the three patients from the Phase 2 trial conducted in the UK and provided feedback on the proposed clinical trial design.

In April 2024, the FDA Office of Orphan Products Development (OOPD) granted Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome.