Small molecules are the bedrock of the pharmaceutical industry. But they remain frustratingly difficult to develop, as even promising compounds can work differently than envisioned once tested in humans.
Empress Therapeutics, a new biotechnology startup from company creator Flagship Pioneering, wants to make the process for discovering these medicines more predictable. By blending small molecule drug research with genetics and machine learning tools, the company aims to more easily determine, early on, which prospects might succeed in human testing.
Empress is drawing on the role genetics play in the development of biologic medicines, the synthetic antibodies and other treatments made from living cells, said Jason Park, the company’s CEO.
The company, founded in 2020, says it already has 15 potential drug leads and could be in clinical testing as early as next year. Like some other Flagship startups, Cambridge, Massachusetts-based Empress is debuting with $50 million from the firm to fund its work.
“This is an opportunity where you don’t have to choose between platform and product,” said Park, who is also an operating partner at Flagship.
Small molecule drugs make up a majority of the medicines that have won approval, prized for their ability to slip into the body’s cells and be administered in a pill instead of by injection or infusion.
But discovering chemical-based medicines is notoriously difficult, a key reason most of the drugs that reach clinical testing ultimately fail. They often don’t behave in ways researchers expect or prove as safe or effective in patients as initially thought. Some studies indicate that biologics, by comparison, have higher success rates in clinical testing. They are typically designed with a genetic engineering step in which a DNA sequence is put into cells to produce a would-be therapeutic.
Empress is taking a page from that process. It says it’s working to identify DNA sequences that cause the body to make chemicals that could become small molecule drugs. The company claims this approach can help it find more predictable medicines more quickly, though its approach is unproven.
It is starting with potential drug targets for autoimmune and inflammatory conditions, Park said.
“I am very close to people who need medicines faster,” Park said. “And there's nothing more frustrating than looking out at the development landscape at clinical trials, and realizing that there just aren't that many options for a number of really serious diseases.”
Park previously co-founded Sonata Therapeutics, a startup making drugs for autoimmune diseases and cancer. He’s joined by Chief Innovation Officer Sabrina Yang, a principal at Flagship, and Chief Scientific Officer Murray McKinnon, an executive who oversaw immunology research at Johnson & Johnson.