Much drug research has focused on small molecules that target disease-causing proteins. However, the easy protein targets have already been drugged so some biotech companies are going upstream by addressing RNA, the molecules that carry the genetic instructions for making proteins. Johnson & Johnson sees promise in Remix Therapeutics’ approach and the pharmaceutical giant is paying $45 million to begin a multi-drug research alliance.
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The sum is an upfront payment and research funding, according to terms announced Thursday. In exchange, J&J subsidiary Janssen Pharmaceutica gets exclusive rights to three targets that have applications in immunology and oncology.
Various types of RNA carries out different functions. Remix’s focus is on the type that carries the genetic information needed to make proteins. The startup aims to modulate these RNA so that the proteins that are made don’t cause disease. But the small molecules Remix is developing don’t directly target RNA. Instead, Remix aims to target the cellular components that process it.
Remix says its technology, called REMaster, identifies patterns in RNA processing and finds ways to use them to modulate RNA expression. On its website, the startup compares its approach to the way musical instruments are tuned to adjust the sound. Remix’s drugs can work in a number of ways, selectively degrading RNA to prevent protein expression or alternatively by boosting RNA expression to increase levels of a particular protein. Another approach is rescuing or skipping over damaged portions of DNA. In each case, the goal is to address the cause of disease at its origin.
RNA has been an elusive target for drug hunters because it’s hard to get small molecules to bind to it. Ribometrix and Arrakis Therapeutics both use technologies that study the structures of RNA, finding the places where small molecule drugs can bind. Durham, North Carolina-based Ribometrix has R&D alliances with Vertex Pharmaceuticals and Roche subsidiary Genentech. Waltham, Massachusetts-based Arrakis has an R&D partnership with Roche. Skyhawk Therapeutics is developing RNA-targeting small molecules in partnerships with companies that include Bristol Myers Squibb, Merck, and Takeda Pharmaceutical.
Remix has not disclosed details about its internal pipeline. The biotech is a relatively newer player, having launched in late 2020 backed by an $84 million Series A round of financing led by Foresite Capital. Under the alliance with Janssen, Remix is eligible for milestone payments that could top $1 billion if any drugs reach the market. The biotech would also earn royalties from sales of any drugs commercialized from the alliance. The agreement gives Remix the option to share in the clinical development of one of the programs in exchange for higher royalties.
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