“All in for CF.”
That pledge adorns the lobby of Vertex Pharmaceuticals, the Boston biotech best known for its groundbreaking but costly treatments for the deadly, rare disease cystic fibrosis.
CEO Jeffrey Leiden, however, says Vertex is making progress in developing a new class of drugs for an all-too-common malady: pain.
With three cystic fibrosis drugs on the market and a fourth expected to win approval early next year, he said, Vertex is diversifying to develop treatments for other disorders that desperately need effective medicines, from Duchenne muscular dystrophy to sickle cell diseases.
Pain might seem an unlikely fit, he said, but it’s not.
“Pain is one of the most common and actually severe ailments out there,” Leiden, 63, said at the company’s sleek headquarters in the Seaport District. “We haven’t had a new class of pain medicine in a long, long time.”
The opioid crisis, he said, makes the need for a non-addictive pain reliever even more urgent.
Late last month, Vertex announced Leiden will step down as CEO and president in April, after seven years in those roles, and become executive chairman. His handpicked successor is Reshma Kewalramani, the chief medical officer, who will become the first female CEO at a top-tier US biotech company.
Although Leiden plans to serve as executive chairman for three years, he stressed that Kewalramani will be running the business. “I’m a big believer that there’s one CEO at a time, and starting in April, that will be her,” he said.
Nonetheless, he said, long-term strategy won’t change.
Vertex, he said, will continue to focus on serious medical conditions that have few, if any, effective treatments, and use any valid approach. In June, for example, it the company announced it was expanding its efforts to use gene-editing to treat two forms of muscular dystrophy.
Although its work on a new painkiller has garnered less attention, Vertex sees a multibillion-dollar opportunity.
The pain relievers people rely on — aspirin, acetaminophen, ibuprofen, and naproxen — were invented generations ago and aren’t always effective for severe pain. Opioids date back thousands of years. While often highly effective, they can cause serious side effects, including, of course, addiction.
In recent years, Vertex has developed an experimental non-opioid painkiller — dubbed VX-150 — that performed well in three mid-stage clinical trials. Eight similar compounds are in the pipeline.
The biotech is basing its work on scientific insights more than a decade old, including the strange case of a 10-year-old Pakistani who was able to walk on hot co als without discomfort.
Geneticists investigating the case identified a gene that they concluded was central to the perception of pain. A mutation had wiped out all perception of injury, raising hopes of creating news drugs to eliminate pain by blocking the gene’s function.
Vertex was one of several companies that pursued that ambitious goal. After years of research and millions invested, Leiden said, the drug maker created a series of molecules that target a related gene.
The lead drug candidate was tested in mid-stage trials in three categories of pain, from the short-lived pain that patients feel after surgery to the neuropathic pain caused by diabetes.
“It worked in every one of those trials,” Leiden said, including one whose results were reported in December.
The company has begun clinical trials of a few of the other molecules it has developed. Vertex hopes to enter late-stage clinical trials of the most promising ones in 2021.
Leiden, who started his career as a cardiologist and has been among the state’s highest paid executives, said he used an opioid, one that had been prescribed, only once in his life and immediately understood its power. He was 40 years old and took the medicine for a couple of days after surgery on an Achilles tendon he had ruptured while playing paddle tennis.
“It’s a feeling like nothing you’ve ever had — incredible euphoria,” he said of the drug. “And so I really understood, this is it, this is the reason people get addicted. I’ve stayed away from them for that reason.”