Amgen and Arrakis Aim to Destroy Disease with Targeted RNA Degraders

Amgen_Al Seib / Los Angeles Times via Getty

Al Seib / Los Angeles Times via Getty

Amgen will partner with Arrakis Therapeutics for an innovative collaboration to research and develop RNA degrader therapeutics against a range of difficult-to-drug targets in multiple therapeutic areas.

This new class of "targeted RNA degraders" consists of small molecule drugs that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases.

Amgen and Arrakis will work together to design and functionalize these molecules to precisely degrade targeted RNAs, and Amgen will advance further preclinical and clinical development activities.

Arrakis will receive an upfront payment for five initial programs worth $75 million as per the deal terms. Also, the company will have the option to nominate additional programs. Arrakis will be eligible for additional payments for each program of up to low-double digits for preclinical, clinical, regulatory and commercial milestones and royalties. Arrakis' future payment could reach several billion dollars if all milestones are met and future program options are exercised.

Raymond Deshaies, Ph.D., SVP of global research at Amgen, is excited about the partnership for a potential revolution in drug design. "Targeted RNA degradation is an exciting area that is pushing the boundaries of drug discovery and design," Deshaies said.

The collaboration will combine Arrakis' pioneering discovery platform of a new class of medicines that directly target RNA, together with Amgen’s induced proximity expertise in discovering multispecific molecules to target the biologic mechanisms of disease. The two innovative discovery platforms from the two companies show promise in creating an opportunity to design and engineer targeted RNA degraders.

"Combining this approach with Amgen's targeted protein degradation induced proximity research already underway has the potential to significantly expand the druggable genome," added Deshaies.

Michael Gilman, Ph.D., chairman and chief executive officer for Arrakis, shared the same excitement. "We are excited to partner with Amgen's strong research team to pursue a shared goal of creating a new class of medicines that induce degradation of disease-causing RNAs. This collaboration further demonstrates the utility of our proprietary rSM discovery platform for targeting RNA with small molecules and paves the way for creating powerful new treatments for patients."

With Amgen's Induced Proximity Platform (IPP), drugs don't do all the hard work of inhibiting or activating a target. Instead, it brings the target into proximity of powerful cellular mechanisms that then act on the target.

Arrakis pursues the far-reaching therapeutic opportunities presented by RNA biology. With targeted RNA degraders, the effector, such as a ribonuclease or other RNA modulator, is brought closer to the RNA to degrade or otherwise modify the disease-causing RNA of interest.

The collaboration program will complement the two companies' capabilities to create heterobifunctional molecules that trigger degradation of disease-relevant RNA targets.

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