Almirall and Eloxx Pharmaceuticals Forge Exclusive Global License Agreement for Rare Disease Treatment ZKN-013

14 March 2024 | Thursday | News

New Collaboration Aims to Advance Treatment for Dermatological Conditions Linked to Nonsense Mutations
Image Source : Public Domain

Image Source : Public Domain

Almirall, S.A. (BME:ALM), a global biopharmaceutical company, and Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a clinical-stage biopharmaceutical innovator, have announced a groundbreaking exclusive license agreement for ZKN-013. This partnership grants Almirall the global rights to develop and commercialize ZKN-013 for the treatment of rare dermatological diseases and other conditions associated with nonsense mutations.

ZKN-013, a leading oral therapy poised for Phase I development, is designed to enable the production of functional proteins, addressing the underlying cause of diseases such as recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB), and familial adenomatous polyposis (FAP). These conditions, characterized by premature stop codons leading to the production of nonfunctional proteins, have significant unmet medical needs.

Under the terms of the agreement, Eloxx will receive an upfront payment of $3 million from Almirall, with the potential for additional development, regulatory, and sales milestone payments that could total up to $470 million. Furthermore, Eloxx is entitled to receive tiered royalties on any future global sales.

Sumit Aggarwal, President and CEO of Eloxx, expressed enthusiasm about the partnership, emphasizing the potential of ZKN-013 to significantly impact the treatment of these debilitating diseases. This agreement also allows Eloxx to concentrate on advancing ELX-02 for rare kidney diseases and to continue its discovery efforts on the TURBO-ZM™ platform.

Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer at Almirall, highlighted the alignment of this license agreement with Almirall’s R&D strategy. The collaboration aims to develop novel treatments for people with dermatological conditions, including those with rare diseases, reinforcing Almirall's commitment to addressing challenging medical conditions.

ZKN-013 has shown promising preclinical activity in patient cells and animal models, suggesting its potential to induce the production of functional proteins critical for skin structure and barrier function in RDEB/JEB, as well as to address FAP, a gastrointestinal disease leading to colon cancer due to APC gene mutations.

This partnership marks a significant step towards developing new therapies for rare diseases caused by nonsense mutations, leveraging Eloxx's TURBO-ZM™ technology platform aimed at modulating the human ribosome to create innovative therapeutics.

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