ELOX - Eloxx Pharmaceuticals Provides Pipeline and Financing Updates | Benzinga
First two subjects dosed in Phase 1 clinical trial of ZKN-013; ZKN-013 is being developed for the potential treatment of rare dermatological and other diseases associated with nonsense mutations
Positive written FDA feedback and guidance from a pre-Investigational New Drug Application (PIND) meeting provides pathway to IND application submission to initiate a Phase 2 clinical trial in the US with ELX-02 in patients with nonsense mutation alport syndrome (NMAS)
Secured binding commitment for additional $3.2 million financing to advance our clinical programs
WATERTOWN, Mass., July 11, 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 pipeline and financing updates.
"Dosing of the first healthy volunteers in the Phase 1 trial for ZKN-013, our lead TURBO-ZM™ is a meaningful milestone for Eloxx, as it further demonstrates our ability to advance novel candidates targeting rare diseases into clinical trials," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. "We were also very pleased with the outcome of our pre-IND meeting with the FDA, which has increased our confidence in the potential of ELX-02 to have a meaningful impact on the treatment of Alport syndrome."
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. (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.
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 - ...