Eli Lilly Expands RNA Editing Collaboration with ProQR Therapeutics

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Thursday, shares of ProQR Therapeutics surged in premarket trading in response to an expanded RNA editing licensing agreement with Eli Lilly. The new agreement will reportedly center around the discovery and development of additional assets using ProQR's Axiomer technology.

Lilly and Netherlands-based ProQR initially entered into a five-target collaboration in 2021. The partnership harnesses ProQR’s proprietary Axiomer RNA editing platform to target disorders of the liver and nervous systems.

The Axiomer platform enables the editing of single nucleotides within RNA in a highly-specific manner. This expanded partnership builds on the prior agreement by exploring further potential applications of Axiomer’s novel RNA editing platform.  

Daniel de Boer, co-founder and chief executive officer of ProQR, said Lilly has an additional option to further expand the collaboration for another five targets, for a total of 15. In a call with investors Thursday, de Boer said he anticipates Lilly will opt-in on those additional targets within the next year or so.

If Lilly further expands the collaboration, the value of the partnership is estimated at $3.9 billion, not including potential royalties on commercialization, de Boer said.

“Lilly is a leader in RNA therapeutics and the expansion of our partnership is a validation of our Axiomer platform,” de Boer said.

Andrew C. Adams, senior vice president of genetic medicine at Lilly and co-director of the Institute for Genetic Medicine, touted ProQR’s technology. In a brief statement, Adams said Lilly is convinced that RNA editing can become an important alternative to “other more permanent therapies.”

Beyond ProQR, Eli Lilly also has an RNA-editing partnership with MiNA Therapeutics. The 2021 collaboration with London-based MiNA focuses on small activating RNAs, which can upregulate intracellular or secreted proteins for therapeutic benefit. Eli Lilly also has RNA-based deals with Avidity, Dicerna and CureVac.

Under the terms of the expanded agreement, Eli Lilly will pay ProQR $75 million in a combination of cash and equity investment. Lilly has an option to further expand the collaboration for an additional $50 million.

Following this cash infusion, ProQR’s cash runway ought to extend through 2026, de Boer said.

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