RARE Daily

Almirall Licenses Eloxx Pharma’s ZKN-013 for Rare Dermatological Diseases

March 13, 2024

Rare Daily Staff

Spanish biopharma Almirall entered an exclusive license agreement with Eloxx Pharmaceuticals to develop and commercialize Eloxx’s ZKN-013 for rare dermatological diseases and other diseases associated with nonsense mutations.

ZKN-013 is a phase 1 ready oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins that counteract the root cause of these rare dermatological diseases such as recessive dystrophic epidermolysis bullosa (RDEB), junctional epidermolysis bullosa (JEB), and familial adenomatous polyposis (FAP), and potentially other diseases.

RDEB /JEB are rare skin diseases characterized 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.

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.

“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.

ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized 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.

“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,” said Sumit Aggarwal, president and CEO of Eloxx.

Photo: Sumit Aggarwal, president and CEO of Eloxx.

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