Shire Finds Big Value in Rare Diseases, and a Strong Growth Path for its Boston Unit

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55 percent to $105 million. Analysts expect the company will report total sales of about $4 billion this year, and Russell has told them the company hopes to achieve “mid-teens growth” through 2015.

Rare diseases have been so good to Shire that the company is now pursuing a niche within a niche: a new “intrathecal” pump that delivers drugs directly to the brain via the spinal fluid. “In these rare genetic diseases, a proportion of patients have the more severe form, which has crossed the blood-brain barrier,” Russell explains. “The intravenous dosing of these proteins doesn’t do anything for those patients.” So Shire is making a bet that delivering its drugs directly into the brain will be the answer for those gravely ill patients. Shire has begun human trials of the pump in Hunter syndrome, where 20 percent of patients suffer from the most severe form, Russell says. The company is planning to test the pump in two other rare diseases, as well.

The pool of patients that might use such a pump is tiny: About 300 people with Hunter would be eligible globally, Russell says. But the CEO is convinced the insurance community will support what is likely to turn out to be a high-ticket treatment. “It all depends on our ability to prove value,” he says. “If you’re the only company producing these products, you can show the value of your drugs over the cost of someone being institutionalized for a decade,” because he is too ill to function independently, he says. “If you look at the opportunity for cost savings, it’s big. That argument has a lot of traction with payers.”

In addition to looking for new opportunities in gene therapy and stem cells, Shire is diversifying its pipeline, which includes finding new uses for existing drugs. The company is currently testing its hit drug for attention deficit disorder, lisdexamfetamine (Vyvanse), in schizophrenia. Russell says the drug may be useful in combination with currently used anti-psychotics, because it reduces what are known as “negative” symptoms—such as lack of emotion and diminished social skills—but doesn’t exacerbate other symptoms such as hallucinations and delusions.

And Shire has been busy boosting its presence in Boston. On May 11, the company was among the investors in a $7 million funding round for Boston-based Prexa Pharmaceuticals, which is working on drugs to treat neurological disorders. And last September, it teamed up with Cambridge-based Acceleron to develop a treatment for Duchenne Muscular Dystrophy. The deal could bring Acceleron as much as $498 million.

During our lunch, it was easy for me to see that Shire’s devotion to rare diseases has become personal for the CEO. Russell told me that he recently met a 17-year-old with Hunter syndrome who has participated in Shire’s clinical trials for 11 years. The teen interned in the marketing department of Shire’s Boston unit, and asked to meet the CEO. During their discussion, the boy asked Russell if he could work at Shire after he graduated from high school. “That was my wake-up moment,” Russell says. “I thought ‘Wow, without the treatment, this kid would have been dead by now.’ Now he’s holding out the prospect of being in a job, paying taxes. His contribution to society will be very valuable.”

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