CAMBRIDGE — Local labs are working to develop treatments for neurological disorders, multiple forms of cancer, and dozens of rare genetic diseases. But the next potential blockbuster drug to emerge from the Boston area may target a less fearsome condition that nonetheless afflicts 30 million to 40 million Americans: irritable bowel syndrome.
Within the next few weeks, the Food and Drug Administration will decide whether an experimental drug called linaclotide can be approved for sale in the United States. If the FDA signs off on it, the drug would bring a new treatment option to people suffering from abdominal pain, bloating, and eating limitations as a result of irritable bowel syndrome — known as IBS — as well as chronic constipation. It would also catapult linaclotide’s maker, 14-year-old Ironwood Pharmaceuticals Inc., into the elite club of the area’s commercial drug companies.
“We’re working feverishly to launch this drug,” said Ironwood chief executive Peter Hecht. “We’ve got to be able to stock 40,000 pharmacies within a few weeks of launch.”
The treatment’s long journey to the marketplace has been watched with anticipation by patients afflicted with a condition many are reluctant to discuss, even though it causes them to miss work or school, cancel flights, or turn down promotions because they require lunching with clients or making presentations when they have symptoms.
“It’s an embarrassing condition,” said Bill Norton, vice president of the International Foundation for Functional Gastrointestinal Disorders, a patient advocacy group in Milwaukee. “People don’t like to talk about bowel disorders. You think about the symptoms — diarrhea, constipation, gas goes along with it — nobody wants to talk about that. And if someone’s uncomfortable talking about it, that could be an impediment to this market.”
Still, stock analysts project the Ironwood drug could attain blockbuster status — ringing up sales of more than $1 billion a year worldwide — within a decade.
“Linaclotide should provide a new standard of care,” said Irina Rivkind, specialty pharmaceuticals analyst for bond trading firm Cantor Fitzgerald in New York. Rivkind estimates the drug could generate as much as $3.8 billion globally by 2027, the year it would lose patent protection in the United States and face competition from low-cost generics.
“It has the potential to be a blockbuster drug based on very solid efficacy and a good safety profile” in clinical trials, said Graig Suvannavejh, specialty pharmaceuticals analyst for New York investment firm Jefferies & Co. “A lot of the patients for this condition don’t really have a prescription or branded product available to them. They’re using laxatives, diets, home remedies, or whatever they find on the Internet. This will address a very large market.”
To reach an important chunk of that market, Ironwood has struck a partnership with Forest Laboratories Inc., a New York-based drug maker that has a strong network of sales people calling on primary care physicians who can prescribe the new treatment. But the Cambridge company also is hiring its own sales force to focus on gastroenterologists and other medical specialists who often see patients with more advanced cases of IBS or chronic constipation.
The company is rapidly expanding its 325-person workforce in preparation for linaclotide’s hoped-for launch, and has begun hunting for potential sites for a larger headquarters and research campus in Cambridge, Boston, or the western suburbs. Among the areas being considered is the South Boston Waterfront, where another Cambridge company with newly approved drugs, Vertex Pharmaceuticals Inc., is building its headquarters.
But even as it grows, Ironwood is working to preserve a culture that its leaders and rank-and-file employees describe as unique among area biotechnology companies.
The company, formerly called Microbia Inc., is wedded more to the principles of adaptability and “patient-centric” drug development than to any specific therapeutic area. Among other things, its scientists seek to make quick no-go decisions on drug candidates that don’t look promising and “celebrate failure” with cake when research projects are discontinued.
“If the data says this is not the path to go down anymore, you want to get on to the next thing,” said Mark Currie, the Ironwood senior vice president and chief scientific officer.
Ironwood employee meetings, dubbed “pharmapaloozas,” are collaborative affairs that partners and suppliers are sometimes invited to attend. Job candidates at the company interview with more than a dozen prospective colleagues. Researchers sit beside sales managers during interviews for sales hires.
“It demystifies what the sales people are all about,” said Thomas McCourt, senior vice president and chief commercial officer. “We’re all here to help each other and help our partners.”
Most striking, perhaps, is Ironwood’s commitment to remaining independent in a rapidly consolidating industry in which mergers and acquisitions are often the ultimate goal.
Toward that end, Ironwood has created a dual-class stock share structure that gives super-voting rights to investors who owned stock before the company went public in 2010. The rights can be exercised only on change of control decisions, such as a hostile takeover bid by a corporate raider.
“We’re excited about building a company for the long term,” said Michael Higgins, chief operating officer and chief financial officer. “It’s an important part of the overall story.”
The company's immediate concern is getting ready to market and sell its drug, an oral peptide that mimics naturally occurring hormones that regulate gut function. The track record of treatments for IBS with constipation isn’t good. At least two previous prescription drugs — from other companies — were pulled from the market because of serious side effects. The only FDA-approved medication currently on the market — Amitiza, sold by Japan’s Takeda Pharmaceutical Co. — has had only limited success because it can cause nausea at high doses.
“Over the past decade, our understanding of IBS has improved,” said Dr. Anthony Lembo, associate professor at Harvard Medical School and a gastroenterologist at Beth Israel Deaconess Medical Center in Boston, who consults for Ironwood. “But there haven’t been a lot of new therapies that have come out for IBS. Sometimes the science lags behind our understanding of the condition.”