Almirall, S.A. and Eloxx Pharmaceuticals, Inc. have announced that the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall obtains global rights to develop and commercialise ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.
The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.
As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future 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,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximising the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform.”
“This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall. “We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations.”
RDEB /JEB are rare skin diseases characterised by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells.
ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterised by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene.
ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx's TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases.