The candidate in question is a so-called nonsense mutation readthrough drug called ZKN-013. Nonsense mutations stop DNA synthesis in its tracks, resulting in an incomplete protein that doesn't work.
In preclinical studies ZKN-013 has already demonstrated activity in RDEB/JEB patient cells, Almirall said in a March 13 release, including the production of functional, full-length Collagen7 in RDEB cells.
As a result of these findings, Eloxx has been gearing up to enter ZKN-013 into a phase 1 clinical trial “shortly.”
In return for the global rights to the molecule, Eloxx will be in line for up to $470 million in potential milestone payments as well as tiered royalties on global sales.
"We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” Eloxx CEO Sumit Aggarwal said in the release.
“In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform,” Aggarwal added.