Arrakis, Roche team up on RNA-targeting drugs in $190M deal

Elizabeth Radcliffe, Vice President of Finance and Strategy; Michael Gilman, Chief Executive Officer; Jennifer Petter, Founder and Chief Scientific Officer; Heather Lounsbury, Vice President of Business and Technical Operations; James Barsoum, Vice President of Research; Jacques Dumas, Senior Vice President of Drug Discovery (Arrakis Therapeutics)

Arrakis Therapeutics has been dodging pharma suitors since its early days, keeping its head down, building its RNA-targeting technology and “politely declining” advances “because we weren’t ready.”

“We started from scratch on this and were trying to develop an understanding of what it was we actually had, and what the magnitude of the opportunity was,” Arrakis CEO Michael Gilman told FierceBiotech. Turns out, that opportunity is far greater than Arrakis could handle on its own, so the company is enlisting Roche to help develop a suite of RNA-targeted small-molecule drugs.

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The duo inked a collaboration and licensing deal that will see Arrakis lead discovery and research for drugs against targets “across all of Roche’s research and development areas,” according to a statement. After a “defined point,” Roche will have the right to pick up preclinical and clinical development for those programs. Arrakis is netting $190 million upfront, but the deal could ultimately reach several billion dollars in worth thanks to clinical, commercial and sales milestone payments.

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Gilman didn’t specify disease areas Roche is particularly interested in, saying only that the targets in the partnership span all three of Roche’s major therapeutic buckets: cancer; immunology and infectious diseases; and neuroscience, ophthalmology and rare diseases.

Some of those targets overlapped with Arrakis’ internal target list, but most didn’t, Gilman said. At the end of the day, there are so many targets to work on, Arrakis is “happy to let them have those,” he added. The biotech is looking into targets in dyslipidemia, or abnormal levels of fats in the blood. And it has three active oncology programs that take aim at RNAs that code for undruggable proteins.

“One is a bit of an oldie but a goodie oncogene—that's MEK—and another is a transcription factor that has long been associated with cancer. The third is a synthetic lethal gene,” Gilman said in a previous interview.

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The Roche deal—and its attendant $190 million in cash—give Arrakis some stability in an uncertain time, Gilman wrote in a blog post. It comes after the biotech banked a $75 million series B round to advance its work in RNA-targeting drugs, a mission that was once considered “nuts” or simply impossible. RNAs are difficult targets because they’re small and aren’t stably folded like proteins. And, unlike proteins, which are made of many different amino acids, RNAs all have the same ingredients—the nucleotide bases A, T, G and C—making them difficult to selectively target with small molecules. 

Despite being told they’d “sail ourselves right off the edge of the earth,” the Arrakis team kept at it and will continue doing so, even through social distancing rules that have forced it to shut its doors.

“Shutting down the lab is obviously not the ideal situation,” Gilman said. “But now that we’ve done it … it’s surprising how much we’ve been able to keep going despite the fact that our own scientists are not at the bench. A lot of our work was already outsourced to CROs, so we’ve been able to ramp that up and move stuff to them that we were doing in our own labs.”

Somewhat ironically, shifting some work across the world to CROs in China has actually sped it up, he wrote in his blog post.

“Now, I’m starting to think about what we would need to see to start sending some people back to the lab,” Gilman said. “I just don’t know what that is yet.”

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