He was saved by a hot biotech trend: Medicines that are targeted to a protein made by a particular gene, often discovered by sequencing the cancer’s DNA. Sometimes they work in only a small number of people, requiring diagnostic testing. In Brandt’s case, Agios Pharmaceuticals, just a 15-minute ride from his Harvard office, had a drug that targeted a genetic mutation he was lucky enough to have. Only a few thousand of the 21,000 people diagnosed with AML each year do.
What drives drug companies to invest in markets of just a few thousand people? Speed is one reason—big benefits for very sick people mean smaller, shorter studies. One Agios drug, Idhifa, was approved by the Food & Drug Administration just four years after clinical trials started—a process that typically takes 12 years. Another reason to invest is price. Agios licenced Idhifa to Celgene, which charges $25,000 a month for it. (Brandt gets his medicine for free, because he’s in a clinical trial.)
The strategy paid off in June, when Loxo’s first drug presented stunning results: Of 50 patients who took it, 38 had their tumours shrink. (Updated results show tumours shrinking in 44 out of 55 patients.) The drug, larotrectinib, was tested against a rare mutation called TRK, which can produce tumours in the lungs, skin and brain. And Loxo took another unprecedented step. It had already started developing a second drug for patients whose tumours had become resistant to the first, so when patients are failed by the company’s first medicine, another option will be at the ready. It’s been tested in two patients.
Loxo shares are up 175 percent so far this year, thanks to the excitement about larotrectinib and two other rare-cancer medicines it is developing. Other companies taking the rare-disease approach are doing equally well. Ignyta, which is testing a drug called entrectinib that targets TRK and a rare mutation called ROS1 that is a cause of lung cancers, is up 190 percent. Blueprint Medicines, focussed on drugs targeting other rare mutations, including a type of digestive-tract cancer, is up 150 percent. But Agios’s story should make investors cautious. That company’s shares are up 100 percent since its 2013 initial public offering, but down 50 percent from their high in January 2015. The results can be so great, investors forget how small the target markets are.
Small companies aren’t the only ones playing. The first big targeted drugs came from giants like Novartis, Pfizer and AstraZeneca, all of which are in the mix. “There is this Talmudic concept that the way to save humanity is one life at a time,” says José Baselga, the physician-in-chief at Memorial Sloan Kettering Cancer Center. “Our obligation is to question every dogma in drug development.”
First Published: Dec 19, 2017, 06:45
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