Biotech Dreams of Drugging the ‘Undruggable’ (Not What You Think) | Pharmaguy's Insights Into Drug Industry News |

Pills have served biopharma pretty well over the years. Easy to design and cheap to produce, the so-called small-molecule therapies that fit in a capsule have made billions of dollars for their inventors and treated millions of patients in the process.


Now, however, as yesteryear’s swallowable blockbusters go generic, the industry is running into diseases for which pills can find no quarter.


“This is a classic lament in the drug development business, that all the low-hanging fruit has been plucked and eaten already,” said Michael Gilman, CEO of a newfangled biotech called Arrakis Therapeutics.


But Gilman’s company is among a growing number of startups hoping to challenge the conventional wisdom on small-molecule drugs, relying on advances in science and exuberance from investors in hopes of writing a second act for the humble pill.


Arrakis, borrowing its name from the Dune universe, just raised $38 million in venture funds with a plan to move upstream in the process of disease. While the majority of small-molecule therapies are designed to latch onto problematic proteins, Arrakis’s goal is to attack the RNA molecules that direct protein production in the first place.


Beyond being a novel approach in itself, setting sights on RNA could also allow for the treatment of what scientists call undruggable targets, proteins that are impervious to any kind of therapeutic intervention.