Computer models are being used to discover new RNA targets.

Rather than target proteins that cause disease, which is an established approach to drug development, Arrakis Therapeutics is looking to identify new compounds that can target the RNA molecules that encode the instructions for constructing proteins.

The company has been developing computer models and modifying various drug-screening techniques to enable the identification of molecules that inhibit RNA activity. Recently, Arrakis announced (https://www.statnews.com/2019/04/18/arrakis-cancer-target-rna-myc/) that through these early screening efforts it has found several hits, identifying compounds that can be formulated into oral drugs that block RNA messaging involved in the production of well-known cancer-causing proteins previously thought to be undruggable.

One target is myc, an oncogene first discovered in the early 1980s that is thought to be important in nearly three-quarters of human tumors. Even with this result, CEO Michael Gilman acknowledges there is a lot of work yet to be done. The lead compounds need to be further refined and formulated into oral drugs that can be investigated for safety and efficacy in preclinical trials. Arrakis will use $75 million recently raised in a Series B round of private financing to fund this work.

The first RNA interfering (RNAi) drug — developed by Alnylam Pharmaceuticals — received approval from the U.S. Food and Drug Administration in 2018. The drug prevents the production of harmful proteins inside nerve cells. Few RNAi drugs have been successful, however, because it is a challenge to deliver them to the right targets.

Arrakis hopes to get around the delivery problem by using oral formulations of small molecule APIs –– an approach once thought to be impossible, though Gilman believes there are huge opportunities.