Now comes the hard part for Vertex Pharmaceuticals Inc.
After launching two drugs aimed at nearly 40 percent of the 75,000 patients worldwide who suffer from cystic fibrosis, the biotech is mounting an assault on a range of mutations causing the obstructive lung disease in the remaining patients. Until the Vertex medicines hit the market, there was no treatment for the life-threatening condition, which causes serious lung infections that can result in respiratory failure.
Boston-based Vertex also is working to apply the scientific model it crafted for CF — targeting a family of mutations driving a single disease — to other rare genetic conditions. They range from the blood disease sickle cell anemia to a brain disorder known as adrenoleukodystrophy to another lung illness called antitrypsin deficiency.
The company’s goal is “to create transformative drugs for serious diseases,” chief executive Jeffrey Leiden said in an interview at Vertex’s headquarters and research site in the Seaport District. “We believe scientific innovation is where you create the value. . . . We don’t have a big marketing budget. You’ll never see us make a me-too drug. We’ll only make drugs that totally transform or cure the disease. That’s all we’re interested in.”
But the company, which has about 1,400 employees in Massachusetts and 2,100 globally, faces some daunting expectations — and potentially weighty decisions — as it makes the transition to a new phase of growth. A stumble could sour investors, while success could turn the state’s second largest biotech company into a takeover target for larger drug makers.
“If their strategy works, there’s a lot of big companies that would love to have a franchise like Vertex that’s going to grow over the next four or five years,” said biotechnology analyst Phil Nadeau, managing director at the investment bank Cowen & Co. in New York. “They have demonstrated their ability to develop drugs that help patients.”
Vertex shares soared in March after the company released positive data from a clinical trial of an experimental combination drug for CF. The data suggested Vertex had, in Leiden’s words, “cracked the code of cystic fibrosis” in its bid to correct genetic defects not susceptible to its drugs now on the market — Orkambi and Kalydeco, which help patients breathe more easily and lead normal lives.
Investors will be watching closely later this year when the company reports findings from much anticipated studies of a new class of three-drug combination medicines for the largest remaining segment of untreated patients. Like the current Vertex drugs, the so-called triple combos will be pills that can bind to misfolded mutant proteins and fold them correctly so they can move to the surface of the patients’ cells, allowing the cells to function correctly.
It’s as complicated as it sounds — and the kind of challenge Vertex embraces, said David Altshuler, the company’s executive vice president for global research and chief scientific officer. “We are willing to take on hard problems and find new ways of doing things,” he said. Among the company’s top research projects: finding a nonaddictive, genetic-based pain medication.
The market value of Vertex has climbed over 70 percent since the start of the year to more than $30 billion, making it the best performing stock on the Standard & Poor’s 500 index. Some analysts suggest the high expectations for the combination drugs mean a setback could send the company’s shares plunging. “We believe the current valuation already prices in considerable pipeline success,” a team of analysts led by Laura Chico at research firm Raymond James & Associates in St. Petersburg, Fla., wrote in a recent note to investors.
To continue its expansion, Vertex will also try to maintain high prices for its drugs, which treat relatively small numbers of patients. Like other specialty genetic drugs, its approved CF medicines carry premium list prices: $259,000 a year for Orkambi and $311,000 for Kalydeco.
Critics, including lawmakers in both major parties, say the cost of such drugs is unsustainable. Vertex executives consider the price tags crucial for the continuation of innovation that creates life-extending treatments — at least until lower-priced generics are allowed on the market.
The pricing model “is not only sustainable, it’s necessary,” Leiden said. He said new drugs will help contain health care costs by keeping patients healthy and out of the hospital. “This is a system that actually works. It rewards innovation and stimulates it. And then after the period of [market] exclusivity is over, it actually makes these innovations free” for future patients.
Vertex is pursuing a three-pronged approach in its final push against CF. It plans to apply to the Food and Drug Administration in the third quarter of this year for approval of a two-drug combination that could treat another 1,500 patients. An even bigger catalyst would be the data from the company’s triple-combination therapies, scheduled for release in the second half of 2017, which have the potential to treat another 24,000 patients around the world.
Lastly, the company is exploring different approaches, such as gene therapies and messenger RNA that can carry protein replacement drugs inside cell membranes, in partnership with collaborators such as Crispr Therapeutics Inc. and Moderna Therapeutics Inc. These would be used to treat the roughly 7,000 patients who don’t respond to its current approach.
The experimentation underscores Vertex’s scientific philosophy — it starts with a disease and finds a treatment, rather than starting with a technology.
“We will only work on programs where the human biology of the disease is understood,” Altshuler said. “We’ll invent the technology we need or acquire it through partnerships.”
Whether the company will eventually buy Crispr or Moderna if the new approaches bear fruit is the subject of much industry speculation. For now, Leiden said of the Vertex partners, “we want to let them run with the technology” and see how it evolves.
Conversely, the progress of Vertex’s research pipeline could set it up as a buyout candidate. Among its attractions is a research-heavy focus enabled by a small but effective sales force. Because its products don’t treat large numbers of patients, only about 20 company sales representatives are needed to sell Vertex drugs. That frees up more money to invest in the science behind the company’s medicines.
Leiden said Vertex is focused intensely on its business, not on positioning the company as takeover bait for Big Pharma. But he acknowledged the company would have to entertain serious offers. In the end, Leiden said, “shareholders make the decision.”Robert Weisman can be reached at email@example.com. Follow him on Twitter @GlobeRobW.