Amicus Counts on “Chaperone” Tech to Enhance Rare Disease Treatments

Xconomy New York — 

On Thursday, Amicus Therapeutics CEO John Crowley will join the long procession of biotech executives making presentations at one of the industry’s most important gatherings, the JP Morgan Healthcare Conference in San Francisco. But Crowley does have one claim to fame that makes him stand out from the crowd: He’s the only biotech CEO whose early career was portrayed in a glitzy Hollywood movie. And that film, Extraordinary Measures starring Harrison Ford, focused on one of the illnesses Amicus is tackling—Pompe disease, an enzyme-related muscle disorder that two of Crowley’s three children have. “That’s what got me into the biotech field,” Crowley (pictured at right) told Xconomy a few weeks before the JP Morgan event.

Like the others speaking at the event, Crowley will have a short 25 minutes to make his pitch to investors, Wall Street analysts, and fellow executives. His goal, in brief, is to build confidence in the company’s pipeline, which includes a drug that Amicus is testing in combination with alglucosidase alfa (Myozyme)—the Genzyme treatment for Pompe that Crowley famously helped develop and that his own children take.

Amicus’ investors will surely be looking for signs of hope. Shares of Amicus (NASDAQ: FOLD), which is based in Cranbury, NJ, dropped 43 percent to $3.44 in the nine months ended December 30. That was a time filled with “some measure of instability,” Crowley says, primarily because it took longer than expected for Amicus to enroll patients in a key clinical trial of a drug it’s developing for a second rare disease, called Fabry.

Before we delve into Amicus, though, let’s briefly review the Hollywood-worthy events leading up to Crowley’s tenure there. In 2000, Crowley, a Harvard-trained MBA, left a management position at New York-based Bristol-Myers Squibb to help start Novazyme, an Oklahoma City-based company founded by a scientist working on a Pompe treatment. They sold Novazyme to Cambridge, MA-based Genzyme in 2001 for $137.5 million and Crowley became chief of Genzyme’s Pompe program. (If you’re not impressed yet, consider that Crowley is also a graduate of Notre Dame law school and a commissioned officer in the U.S. Navy Reserve.)

Genzyme won approval for alglucosidase alfa in 2006. Crowley’s children, Meghan and Patrick, who were put on the drug in 2003 as part of a clinical trial, are still on it and doing well in the 9th and 8th grades, respectively, Crowley says. His family’s experience inspired a Wall Street Journal story, and later, a book called The Cure. Then Harrison Ford optioned the rights to the story, which led to the movie, starring Brendan Fraser as Crowley and Ford as a prickly (and fictional) scientist who worked on an enzyme treatment for Pompe.

What attracted Crowley to Amicus was the opportunity to make Genzyme’s treatment for Pompe—and drugs for related disorders—even more effective. Pompe and Fabry are among about 50 inherited, often fatal disorders that occur when the enzyme-making machinery in the body’s cells malfunction, causing them to make too little of a particular enzyme, or to make a “misfolded,” or unstable, version of it. Genzyme’s treatment is one of a number of enzyme replacement therapies designed to fix those deficiencies.

Amicus was founded in 2002 on a technology developed at Mount Sinai Medical School in New York. The technology yields small molecules that attach themselves to the defective enzymes, stabilize them, then transport them to the part of the cell where they need to go in order to … Next Page »

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