Roche and Arrakis Link on RNA-targeted Drugs

15.04.2020 -

Swiss drugmaker Roche has entered into a strategic collaboration and license agreement with US biopharma Arrakis Therapeutics for the discovery of RNA-targeted small molecule (rSM) drugs.

The Waltham, Massachusetts-based firm will lead discovery and research activities to a defined point on a broad set of targets across all of Roche’s R&D areas. Roche will then have the right to exclusively pursue further preclinical and clinical development.

Under the terms of the deal, Roche will make an upfront payment of $190 million cash to Arrakis, which is also eligible to receive further payments, which could exceed several billion dollars, on reaching various milestones as well as royalties on any resulting products.

“Together, we share a common vision of accessing new drug targets at the RNA level and thereby discovering novel medicines to treat diseases with high unmet medical need. The collaboration will increase the number of new treatments for patients arising from our proprietary rSM discovery platform,” said Michael Gilman, CEO of Arrakis.

“In addition to the Roche collaboration, we are further building our capabilities and  advancing our wholly-owned rSM programs for diseases unaddressed by today’s medicines,” Gilman said.

Arrakis’s founder and chief scientific officer, Jennifer Petter, added that the company’s platform allows it to “predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits and conduct medicinal chemistry programs to discover a new class of RNA-targeted medicines optimized for potency, selectivity, and safety.”

As well as partnering other companies, Arrakis is also developing an internal pipeline of rSMs to treat a range of serious illnesses, including cancers and other diseases where it said strongly validated targets and drivers of disease have been identified but have proven challenging with other drug approaches and modalities.

In December 2013, Arrakis entered into an exclusive license agreement with the University of Pennsylvania to access intellectual property and technology related to small molecule drug candidates that directly target RNA.

The biopharma said the ability to target RNA with small molecules opens up the potential to access the biological and potentially disease-relevant activity of more than 200,000 RNA transcripts, representing a vast number of therapeutic targets that are largely out of reach with today’s drug development tools.