- ProQR Therapeutics is deepening its ties with Eli Lilly, agreeing on Thursday to expand a deal the two struck in 2021 to use the Dutch biotechnology company’s technology to edit RNA.
- Under the original partnership, Lilly and ProQR planned to collaborate on as many as five programs for liver and neurological diseases. The new arrangement adds more brain disease targets and could triple the total amount ProQR receives if the programs in the alliance hit various milestones.
- Lilly is paying ProQR $75 million in cash and an equity investment to broaden the deal, and could add a $50 million payout if it decides to expand the alliance further. ProQR could receive up to $3.75 billion in future payments, as well as sales royalties, as part of the arrangement.
The expanded partnership comes at the close of what’s been a difficult year for ProQR.
Its most advanced drug prospect, a treatment for genetic blindness, failed a key trial in February. The company has since changed course, twice paring down its workforce and deciding to partner off that eye drug as well as another similar therapy.
That decision was part of a plan by ProQR to focus on an RNA editing technology it’s developed and that’s known as “Axiomer.” The technology enables ProQR to edit single RNA nucleotides, an approach that’s spurred the creation of several new biotechs, like Korro Bio and Shape Therapeutics, in recent years, and was a focus of an alliance between GSK and Wave Life Sciences last week. Proponents of RNA editing believe it offers a way to address genetic mutations with less risk than DNA editing because it involves temporary, not permanent changes.
Lilly has now bet on that technology twice. Since their original deal in 2021, the two claim they’ve made notable research progress. In a statement, Lilly said the partners are now better able to deliver RNA editing medicines, and that those drugs can perform edits more efficiently. That progress has enabled Lilly and ProQR to go after more disease targets than they could previously.
“We have been impressed with the progress to date with our partners at ProQR and have conviction that RNA editing can be an important alternative to other more permanent therapies,” said Andrew Adams, Lilly’s senior vice president of genetic medicine, in the statement.
The relatively quick expansion of the partnership, as well as growing interest in the RNA editing space “have warmed us up to [ProQR] ahead of what we believe could be a big year for the company in 2023,” Cantor Fitzgerald analyst Jennifer Kim wrote in a note to clients Thursday. ProQR also indicated on a conference call that it has more opportunities to forge additional RNA editing partnerships.
ProQR shares climbed 65% in morning trading Friday. But at less than $3 apiece, shares have lost about two thirds of their value in 2022.