Dive Brief:
- Amgen will team with Arrakis Therapeutics to develop oral drugs that target and destroy RNA, announcing a deal Tuesday that will pay the Waltham, Massachusetts-based biotech $75 million in cash.
- Arrakis, which is privately held, could receive “several billion dollars” more should research and development advance as planned across the five initial programs selected by the companies, as well as any further ones Amgen decides to nominate.
- The companies did not disclose which diseases they aim to treat, saying only that the partnership will focus on “difficult-to-drug targets in multiple therapeutic areas.” Arrakis will identify small molecules that can lock onto RNA targets chosen by Amgen, after which the two companies will collaborate to refine those drug candidates further.
Dive Insight:
While companies like Alnylam Pharmaceuticals and Ionis Pharmaceuticals have found success targeting RNA with infused medicines made up of nucleic acid themselves, developing small molecule drugs that can bind and interact with RNA is considered a much tougher challenge.
Recently, however, a number of companies, Arrakis included, have emerged with plans to do just that, taking advantage of research advances that make targeting RNA with pills more feasible.
Large pharmaceutical companies like Roche, AstraZeneca and Vertex have taken notice, inking partnership deals to further explore what might be possible. Roche, for example, has already joined forces with Arrakis, reaching an agreement last April that paid the biotech $190 million.
Amgen, which announced the deal the same day it presented to investors at the J.P. Morgan Healthcare Conference, sees Arrakis as partner to expand research it already has underway on protein-degrading and RNA-targeting drugs.
“This is a collaboration where we expect to try to develop medicines that are designed to degrade RNA and, in doing that, to prevent harmful proteins from being assembled in the body,” Amgen CEO Robert Bradway said in a presentation at the conference, which is being held virtually due to COVID-19.
The science behind Amgen and Arrakis’ collaboration is complex and cutting-edge. Amgen has developed a way to design molecules that can bind to a target as well as what’s known as an “effector,” which blocks, activates or destroys the target. (Several Amgen drugs in development for cancer work similarly by bringing together immune cells with cancerous ones.)
With Arrakis, the companies plan to add RNA to the mix, creating molecules that can bind to the nucleic acid and bring it near enzymes in the cell that can separate and degrade it.
