Dr. Jeffrey Leiden: Transforming Vertex, and rare disease treatments

He bet the company on treating a rare disease, and with success it is now giving back — and pushing forward.

Dr. Jeffrey Leiden, in a lab at Vertex headquarters in the Seaport, no longer needs to worry about the company going out of business. Dina Rudick/Globe Staff

Six years ago, Dr. Jeffrey Leiden walked into the “leaky, bug-ridden, and rodent-infested” Cambridge headquarters of Vertex Pharmaceuticals just as its only source of revenue, a drug for hepatitis C, was about to be displaced by better medicines made by other companies.

Leiden, the company’s newly hired CEO, knew Vertex itself was in a dire spot. As its staff gathered, the discussion of what to do next veered away from the usual quest to find the next Lipitor or Viagra, drugs with vast potential markets. Instead, Vertex decided to dig into the genetic roots of cystic fibrosis, a devastating inherited disease that gradually robs patients of lung function. Rather than a one-drug-fits-all magic pill, Vertex would create a series of medicines to address the multiple genetic variants responsible for the disease.

In effect, the company was betting its future that a science project could overthrow what was a drug industry truism: Drugs for rare diseases can’t make money. A single failed experiment could sink the whole enterprise. There were moments during experiments where “literally, when we turned over some of those data cards, if [the results] had been negative, the company wouldn’t be here,” says Leiden, a former professor at Harvard Medical School. His easy smile gives way to a big laugh.

Leiden can smile now because Vertex pulled off the unlikely. Since then it has doubled down on its effort to treat each of the world’s roughly 75,000 cystic fibrosis patients. Vertex’s first two medicines, Kalydeco and Orkambi, work for roughly 30,000 of them. An improved combination, which a Wall Street analyst dubbed “Morkambi,” should add 12,000 more if, as expected, it wins approval next year.

But the big breakthrough this year, the culmination of that day six years ago, involves what’s known as the triple combination, a group of mix-and-match therapies that help patients who don’t benefit from the company’s other drugs. In July, Vertex announced that a midstage trial of the triple combo showed sterling results. If those effects are replicated in a larger study, the company can treat 90 percent of all cystic fibrosis patients.

With Vertex on pace to book $2 billion in revenue this year, Leiden announced this fall it would establish a foundation and give at least $500 million over the next 10 years for science education, cystic fibrosis outreach, and community building. The gifts will make it one of Boston’s biggest corporate donors, and Leiden intends for the company to be a generous neighbor. Already, its sweeping new headquarters in the Seaport includes a 3,000-square-foot learning lab where Boston Public Schools students conduct science projects of their own on the company’s dime.

Vertex’s drugs are undeniably expensive. Kalydeco has a list price of more than $300,000 per year, Orkambi goes for about $270,000 per year, and analysts expect prices for the company’s next two products to fall in the same ballpark.

But Leiden says the pricing supports the cost of inventing each new incremental medicine, creating a virtuous cycle that expands the number of cystic fibrosis patients who can benefit. And that benefit is hard to quantify: Patients who once struggled to walk a block or a climb a flight of stairs now do so with ease, he says.

“These are medicines that literally transform the lives of these patients,” Leiden says. “Those are the highest-value medicines you can make.”