February 27, 2017
By Alex Keown, BioSpace.com Breaking News Staff
WALTHAM, Mass. – Michael Gilman, who founded and sold Padlock Therapeutics, along other industry vets with backgrounds at Biogen , teamed together to launch startup Arrakis Therapeutics, a company that intends to use chemicals to identify new RNA targets and create new small-molecule drugs.
The new company launched with $38 million in Series A financing led by Canaan Partners with participation by Advent Life Sciences, Pfizer, Inc. , Celgene Corporation , Osage University Partners, and biotech industry leader Henri Termeer. Gilman, who has launched and sold other startups, was named chairman and chief executive officer of the new company.
Arrakis said the funds will be used to finance the company’s proprietary discovery platforms, Tryst and Pearl-Seq. Using those two platforms, the company is planning to develop a pipeline of RNA-targeted small molecules focused on neurology, oncology, and rare genetic disorders. The company said its technology “interrogates structures of folded RNAs in their native state, within cells, to enable discovery and rational design of potent, selective small-molecule inhibitors of RNA function.”
“RNA is the locus of most human biology, yet our current pharmacopeia is largely limited to protein targets,” said Gilman, who also held previous leadership roles at Biogen and Ariad Pharmaceuticals . “Arrakis intends to re-architect small molecule drug discovery by redirecting, modifying, and creating tools that enable medicinal chemistry to directly address therapeutically-important RNA molecules. Our substantial intellectual property, financial resources, and the extensive experience of our team in discovering and developing novel drugs will enable us to build a pipeline of new small-molecule therapeutics for patients not helped by today’s medicines.”
In addition to Gilman, Arrakis has also tapped a number of other industry vets to guide the fledgling company. Other members of the Arrakis leadership team include former Celgene, Biogen and Novartis executive Russell Petter, who is serving as the chief scientific officer of Arrakis. Daniel Koerwer, Arrakis’ chief business officer, held former executive roles at BIND and Biogen. James Barsoum, senior vice president of biology of Arrakis, formerly served in executive roles at Synta , and Biogen.
Arrakis said there are more than 200,000 RNA transcripts in the human transcriptome, but small-molecule medicines address approximately 500 protein targets. The company said it intended to use “attractive and predictable pharmaceutical properties of small-molecule drugs, including oral bioavailability, broad bio-distribution, and efficient cell penetration” to open RNA biology to medicines.
Petter said the importance of targeting RNA in disease is well-recognized, but attempting to do so with small molecules has been thought to be too challenging. In a statement, Petter said there are a number of precedents for small molecules to “bind and modulate RNA,” but only after initial development.
“Our goal is to intentionally discover RNA-modulating small-molecule medicines. We believe that new developments in informatics, structural biology, and biophysical tools now make that possible,” Petter said.
