CAMBRIDGE — Ariad Pharmaceuticals Inc. has spent two decades in classic biotechnology start-up mode. Company scientists have labored at designing drugs and identifying diseases to target, while its investors have waited for Ariad’s first product to hit the market.
Some time soon, the perseverance may finally pay off.
Federal regulators have granted priority review to the biotech’s experimental treatment for chronic myeloid leukemia, promising to decide by March 27 whether the company, founded in 1991, can start selling the drug in the United States. The drug is now called ponatinib, but Ariad plans to give it a more consumer-friendly brand name once approval is granted by the Food and Drug Administration.
As it anticipates that ruling, Ariad is moving quickly to transition from a research operation to a fully commercial company. The workforce has been more than doubled to nearly 300 employees since the start of the year. Ariad also has opened a European headquarters in Lausanne, Switzerland, and hired a sales force, including many refugees from Big Pharma companies, as preparations are made to market the new leukemia drug to oncology and hematology specialists on both sides of the Atlantic.
“We are launch-ready for ponatinib,” said Ariad founder and chief executive Harvey J. Berger. “If we had approval today, we’d have the drug in the hands of patients in the United States in two weeks. We are on the verge of commercialization of our first product.”
Industry analysts believe the closely watched leukemia drug — which is also awaiting a mid-2013 decision by European regulators — is destined to become a blockbuster within the next five years, meaning it will generate more than $1 billion in annual sales.
But even more important, they say, is the science behind Ariad’s impending commercial debut. The company has pioneered a “structure-based” design that can produce multiple cancer drugs, each binding to tumors and blocking the mutations that have undermined existing medicines by allowing diseases to outsmart treatments. Next up in Ariad’s pipeline: a lung cancer drug.
While initial FDA approval would allow use of ponatinib for leukemia patients who haven’t responded to other treatments or whose cancer has mutated while on other drugs, analysts believe it has the potential to soon become a “first-line” therapy — one given to leukemia patients from the start of their treatment regimens. That would give Ariad access to a larger share of the estimated $4.5 billion annual chronic myeloid leukemia market now dominated by Novartis AG’s Gleevec and Tasigna, and Sprycel from Bristol-Myers Squibb Co. Each year, about 5,000 Americans are diagnosed with the often fatal disease, known as CML, a cancer of the white blood cells.
“Ponatinib is a best-in-class drug and a potential first-line therapy with very compelling efficacy,” said biopharmaceutical analyst Michael J. Yee, managing director for RBC Capital Markets in San Francisco, who cited the drug’s global market potential. “What’s very interesting about Ariad is that they are the rare biotech that has decided to retain worldwide commercial rights to their drugs. That could be particularly valuable in the future.”
Hematologists and CML patients also are eagerly awaiting the treatment’s approval.
“This represents a huge advance for patients whose drugs have failed them and patients who have developed resistance to those drugs,” said Dr. Daniel J. DeAngelo, associate professor of medicine at Harvard Medical School and an oncologist who treats CML patients at Dana-Farber Cancer Institute in Boston. “Ponatinib offers a wonderful option for those patients.”
One such patient, Hans Loland, a 44-year-old systems analyst for Microsoft Corp. who lives in Woodinville, Wash., was diagnosed with CML at the end of 2008 when his wife was pregnant with their second child. After failing to respond to two other drugs, he learned of ponatinib through an online forum and enrolled in a clinical trial in Portland, Ore. He started on the experimental drug in November 2009 and within six weeks doctors saw a 10-fold reduction in the disease.
“It’s just more powerful and effective than the previous drugs,” said Loland, whose wife last year gave birth to their third son. “I’m able to do everything. I work a full schedule. I play soccer. It’s really fantastic, it’s given me my life back.” Loland said he is sometimes fatigued, but chalks that up more to playing with his three young sons than to side effects of ponatinib.
Clare Karten, senior director for mission education at the Leukemia & Lymphoma Society in White Plains, N.Y., a patient advocacy group that helps fund blood cancer research, said patients have high expectations for ponatinib. Their enthusiasm is driven partly by a Nov. 29 article in the New England Journal of Medicine, which reported Ariad’s treatment was “rationally developed to address the limitations of currently available” CML drugs.
“This drug is effective against a number of mutations that there are no other therapeutic options for today,” Karten said.
Doctors and analysts caution that it is too soon to say whether resistance could eventually develop in patients taking ponatinib, as it has for those taking other drugs. For now, however, many are anticipating the drug could join recently approved therapies from other Cambridge biotechnology companies with the potential to achieve blockbuster status, notably a Vertex Pharmaceuticals Inc. hepatitis C treatment and an Ironwood Pharmaceuticals Inc. drug for irritable bowel disease with constipation. Other local companies, including Aveo Pharmaceuticals Inc., also have drug applications pending before the FDA.
Marty J. Duvall, a biopharma veteran Berger recruited 15 months ago as senior vice president for commercial operations, has been assembling a field force — quizzing salespeople and job candidates on the details of ponatinib.
Duvall said they can hardly wait to start selling the product.
“We’re looking under the tree every morning,” he said.
The company has experienced some growing pains as it shifts into high commercial gear, including an overflow of employees at its Landsdowne Street headquarters that forced Ariad to lease space for about 50 employees on nearby Sidney Street. For now, Ariad workers are trudging back and forth between the two locations for meetings and consultations, but Berger said he expects to unveil a plan next month to consolidate the expanding company in new space in Cambridge.
Berger said he is also being asked at meetings and employee breakfasts he hosts whether Ariad can retain its culture of integrity, teamwork, and scientific excellence as it grows.
“People ask how we can be sure we don’t mess up the great thing we created,” Berger said. “We’re going to keep talking. It’s going to come down to the leadership. It’s all about conveying clarity of message, clarity of vision. . . . Patience and perseverance are the two watch words.”