Lilly strikes $1.9bn kidney disease alliance with Ascidian

In its third big licensing deal of the week, Eli Lilly has tapped into Ascidian Therapeutics' RNA-editing capabilities with a $1.9 billion collaboration focused on kidney diseases.

According to the Boston-based startup, it will work with Lilly on the discovery and development of drugs for kidney diseases caused by single gene alterations based on its platform, which can repair mutations at the kilobase scale by replacing entire exons at the RNA level.

Its drugs are DNA constructs that, after being delivered into the body via a viral vector or lipid nanoparticle system, are transcribed into mutation-free, exon-only RNA in the body. That allows healthy protein to be transcribed and avoids the risks associated with direct DNA editing or gene replacement, according to the company.

The alliance gives Lilly exclusive rights to specific but unidentified kidney disease targets, with the prospect of adding to the list if all goes well with the partnership, which includes an upfront payment along with development and commercial milestones, said Ascidian in a statement. The company will lead discovery and some preclinical work, with Lilly taking over once the projects get nearer to the clinic.

More than 60 genetic diseases are known to affect the kidneys, and many of them involve large genes that cannot be targeted with existing technologies, according to Ascidian chief executive, Michael Ehlers, who was formerly head of R&D at Biogen and led neuroscience and rare disease R&D at Pfizer.

"Lilly and Ascidian believe that patients with serious monogenic kidney diseases deserve effective treatment options and that an RNA-based approach is a compelling strategy for those diseases," he said. "Combined with Lilly's genetic medicine expertise, we aim to dramatically reduce the burden of genetic kidney disease."

Haisco pact

News of the Ascidian partnership came right on the heels of an agreement with China's Haisco Pharmaceutical, with little detail other than upfront and near-term payments of $87 million, along with up to $2.97 billion in additional milestones.

There is a commitment for Haisco to work on the discovery and identification of up to five target programmes on behalf of Lilly, with the US group claiming global rights to any resulting medicines for some of them and rights outside China, Hong Kong, Macau, and Taiwan for others. Haisco's core therapeutic categories are pain management, oncology, respiratory diseases, autoimmune disorders, metabolic diseases, and central nervous system disorders.

Earlier this week, Lilly also licensed a GLP-2 analogue for gastrointestinal disease developed by Hanmi Pharma, sonefpeglutide, for $75 million upfront and another $1.185 billion in milestones.

The new deals are the latest in an ever-expanding list of acquisitions and licensing deals signed by Lilly in the last few months, funded by rapidly growing revenues from its GLP-1-based weight-loss and diabetes medicines.

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