The FDA just approved a new drug to treat a form of blood cancer.
The drug, Tibsovo, is used to treat acute myeloid leukemia and is made by Agios Pharmaceuticals. It’s the second approval for the company, whose first drug, Idhifa is also used to treat AML with particular genetic mutations.
The two drugs act on a simple idea: Starve out cancer cells. They target two different mutations related to AML, IDH1 and IDH2. The two mutations make up about 20% of patients with AML.
Getting two drugs from discovery to approval is no small feat, especially considering that development timelines for drugs often go beyond a decade.
Agios CEO David Schenkein, who’s been with the company since 2009, explained in May that there were a few things that helped the company get into this spot.
The first was to focus in on a narrow area of biology, specifically cellular metabolism. For example, cancer cell metabolisms — the process of converting food into energy — work differently than normal cells.
Cancer cells tend to take in more glucose than normal cells, which opens up the opportunity to target just cancer cells, leaving healthy cells untouched. Normal cells go through a whole life cycle (gets born, ages, makes new babies, then dies). Cancer cells, on the other hand, get stuck at the baby stage. While there, they make more and more of themselves. The idea with Agios’s drugs is to help those cancer cells get old and die off instead of sticking around.
The second was hiring people and creating a scientific environment to “challenge the norms” of drug development.
“We’ve set the bar really high,” he said. “As a result, that’s motivated people because they’ve been so excited to challenge the way we do everything out there and go more quickly.”
That’s happening at a time when as Schenkein sees it, the typical process of drug development is starting to shift from going through three distinct phases to becoming a more adaptive process. That helped Agios use the results from earlier-phase trials into studies the company could bring before the FDA.
Agios is also developing treatments for rare genetic diseases connected to cellular metabolism and researching how the metabolism of the immune system could help treat cancer.