A drugmaker developing medicines to directly target RNA has received an investment to scale up its development system and pipeline of new drugs.
Waltham, Massachusetts-based Arrakis Therapeutics said Thursday that it had closed a $75 million Series B financing to advance what its RNA-targeted small molecules, or rSMs. Nextech Invest and venBio Partners led the round, while new investors Omega Funds, HBM Healthcare Investments, GV, WuXi AppTec Venture Fund and Alexandria Venture Investments participated. Existing investors participating included Canaan Partners, Advent Life Sciences, Pfizer Ventures, Celgene, Osage University Partners and the estate of Henri Termeer.
Michael Gilman, a partner in Atlas Venture, will become full-time CEO of the company, which is named for the fictional planet of Arrakis in Frank Herbert’s “Dune” science fiction series, the planet being the source of the fictional substance spice melange. The company completed a $38 million Series A round in February 2017, led by Canaan and with participation from Advent Life Sciences, Pfizer, Celgene, Osage University Partners and Termeer. The former CEO of Genzyme, Termeer died in May 2017.
The company plans to use the new funding to build its pipeline of RNA-targeted drugs, with the goal of bringing one or more into clinical development. In particular, it plans to focus on oncology and genetically validated targets in other disease areas. It will also put money toward development of its discovery platform, which includes computer systems, biophysical and cellular assays and chemical libraries.
“We have built an end-to-end platform for the discovery of rSMs by creating or adapting tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits and conduct medicinal chemistry programs to improve potency, selectivity and safety,” Gilman said in a statement.
According to the company, while targeting RNA has required use of oligonucleotide drugs, those drugs have challenging pharmaceutical properties that limit their utility for patients. But there are precedents for targeting it with small-molecule drugs, which unlike oligonucleotides were discovered through luck, when it was found after their discovery that they acted on RNA.
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