Almirall is handing over $3 million for Eloxx Pharmaceuticals’ molecule aimed at a pair of rare skin diseases and potentially more just before the drug enters the clinic.
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.
ZKN-013 is designed to enable host cells to produce functional proteins that counteract the root cause of certain dermatological diseases. Specifically, Eloxx has been targeting ZKN-013 at dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). Both of these rare skin conditions are characterized by defects in the Collagen7 gene, which plays a vital role in the correct formation of the skin’s structure and ability to act as a barrier.
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.”
The biotech has also been investigating ZKN-013’s potential as a treatment for familial adenomatous polyposis, an inherited disorder characterized by cancer of the large intestine and rectum.
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.