BioPharma, Pharma, Startups

Arrakis raises $38M for new frontier in drug discovery

Boston-based Arrakis has officially launched with a $38 million Series A and a unique mission to bind RNA with small molecules.

Sunrise in Space new frontier

Some people are really bad at retiring.

CEO and chairman of Arrakis, Michael Gilman, lands on that list. As does his partner-in-crime Russel Petter, founder and CSO of the new Cambridge, Massachusetts startup.

After successful careers, neither could turn down the opportunity to defy conventional scientific thinking and attempt to successfully drug RNA with a targeted small molecule.

“It was like the flash of inspiration for Russ,” Gilman said in a phone interview. “I think it spoke to his contrarian streak — here’s a thing that people said for years couldn’t be done.”

Right or wrong, we may soon find out if its possible. On Monday, Arrakis announced the closing of a $38 million Series A, led by Canaan Partners with support from Advent Life Sciences, Pfizer, Celgene Corporation, Osage University Partners and biotech entrepreneur Henri Termeer. 

Gilman said the money will allow the company to commandeer its own space, bringing the research and development in-house. There will be many skeptics and scientific hurdles to overcome.

There will be many skeptics and scientific hurdles to overcome. Over the years, drug hunters have developed a powerful toolkit for finding new small molecules. However, according to the new Arrakis website, these drugs target approximately 500 proteins in a small number of structural classes. It’s a finite pool for medicine to draw on.

By comparison, humans possess over 200,000 RNA transcripts, which are critical to all the biological pathways involved in disease.

“The question is, can you take that [small molecule] toolkit and point it at RNA instead?” Gilman said.

After extensive troubleshooting, the team identified three buckets; tools that work off-the-shelf, tools that need to be tweaked and tools that would need to be invented from scratch to address some of the many ways RNA differs from proteins. That theoretical structure is now in place, according to Gilman, opening the door for real screening experiments.

“We’ve done several screens using off-the-shelf assay platforms and existing compounds and we’ve found hits,” he said. “So I think that’s very reassuring that there are plenty of RNA-binding small molecules out there, it’s just that they’re hiding in plain sight. No one has gone looking for them before.”

Arrakis is developing two platforms, TRYST and PEARL-seq, to identify new RNA targets and their small molecule drug counterparts. It will also pursue its own therapeutic pipeline. The obvious clinical targets would be conditions that are known to be caused by a genetic mutation, but where the resulting protein hasn’t proven druggable.

That could encompass certain neurologic diseases, cancer, rare genetic diseases and more. Enough to keep the Arrakis team busy for the next few years. Gilman and Petter have truly failed at the whole retirement gig.

Their friendship traces back to the early 2000’s when Gilman was head of drug discovery at Biogen and Petter led the medicinal chemistry division. They parted ways and went on to different startups.

Petter moved to Avila Therapeutics, which was acquired by Celgene in 2012. He transitioned to the new company and worked for three years before leaving for “retirement” in 2015.

Gilman, on the other hand, founded and ran Padlock Therapeutics until it was acquired by Bristol Myers-Squibb in 2016 for up to $600 million.

“I stayed for about a month before I was cut loose into my retirement — my ‘air quotes’ retirement,” Gilman said.

By October 1, he had joined Petter as CEO and chairman of Arrakis.

“I really tried to retire,” Gilman laughed. “But once I start falling for something, I fall pretty hard.”

Photo: Pr3t3nd3r, Getty Images

Shares0
Shares0