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

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break down the harmful substances that cause symptoms of Pompe and related diseases. “Our drugs promote stability and enable the patients’ own enzyme to fold in the right configuration,” Crowley explains.

Crowley—who left Genzyme in 2002 and went to work with the Princeton, NJ-based venture group Domain Associates—was recruited as CEO, and employee No. 6 of Amicus in January 2005. The company had been founded by Stamford, CT-based venture capital firm CHL Medical Partners, and it was housed in a science incubator in northern New Jersey. Amicus raised $170 million in venture capital and went public in May 2007.

Amicus’ lead compound, AT1001, is in late-stage trials to treat Fabry disease, an enzyme disorder that results in an abnormal build-up of fatty substances in cells. Amicus is testing it alone and in combination with agalsidase (Fabrazyme), another Genzyme treatment. On January 6, the company announced that the combination performed well in a Phase 2 study, with patients showing as much as four times more active enzyme as patients who took enzyme-replacement alone.

Testing the combination is important, Crowley says, because a large subset of patients don’t make any of their own enzyme, and therefore there is nothing for Amicus’ drug to chaperone. In those cases, AT1001 should bind to Genzyme’s enzyme-replacement product, making it more potent, potentially at a lower dose than what patients would normally get, Crowley says. “We know in animals at reduced doses we can significantly enhance enzyme activity,” he says. More details about the clinical trial will be discussed at the Lysosomal Disease Network World Symposium in February.

In October 2010, GlaxoSmithKline licensed AT1001, giving Amicus a $30 million up-front payment and the potential to earn $170 million in milestone payments. GSK also took a nearly 20 percent ownership stake in Amicus for $31 million. Crowley says GSK was one of three pharma companies vying for the molecule, all of whom offered almost identical financial terms. But GSK was the right cultural match, he says. “GSK’s vision is to become one of the leading companies in rare diseases,” he says. “And they did the deepest diligence. They came in here for weeks on end with about 20 people, tearing through our patents and our manufacturing processes. They came back with a lot of ideas about how to make the program better.”

Amicus’ second drug is its Pompe product, AT2220. Initially, Amicus was studying the drug as a monotherapy, but there was a problem: It had to be given in such high doses that patients experienced side effects like fatigue. In March 2009, the FDA put a “clinical hold” on AT2220, essentially killing the program. So Amicus’ scientists began looking at the drug in combination with Genzyme’s Pompe treatment. In early 2011, Amicus presented data to the FDA, along with a plan to develop a combination treatment. The agency removed the clinical hold in March of 2011. Amicus started a Phase 2 study late last year and plans to report preliminary results in the first half of this year.

That’s just one of the milestones Crowley expects to reach this year. “What I hope for 2012 is to be able to deliver data in multiple different programs,” he says. Amicus is also testing a drug for Gaucher disease, another enzyme disorder. At JP Morgan, Crowley plans to discuss that program, as well as Amicus’ early-stage molecule for Parkinson’s disease, which addresses a subset of patients who also carry the Gaucher gene. The link between the two diseases has only recently been discovered, Crowley says, and is still not fully understood. “We think we’re a couple of years ahead of everyone else at developing a toolset to intervene there,” he says.

Leerink Swann analyst Joseph Schwartz believes Amicus’ recent progress bodes well for its technology platform. Schwarz discussed the Fabry data with a doctor specializing in rare diseases, who surmised that Amicus’ chaperone molecule may increase the efficacy of enzyme replacement, lessen the risks, and allow clinicians to give it in lower doses to patients with Fabry. “He believes the benefit may be of even greater value in Pompe,” Schwartz wrote in a Jan. 9 report. Schwartz has a $9 target on the share price, which has risen a bit since the start of the year to $3.72.

Crowley hopes Amicus’ technology can be applied across a range of rare diseases, but he’s not shy about admitting that combating Pompe remains one of his biggest goals. Crowley’s office in Amicus’ suburban New Jersey headquarters is filled with reminders of his family’s moment in the show-biz spotlight. There are photos of Meghan and Patrick with the kids who played them in the movie, and photos of Ford visiting Amicus’ office, so he could observe what real scientists do, Crowley says. Even though Crowley’s children are doing well, it’s conceivable they could benefit from AT2220 if it makes it to market, he says. “There are a lot of things enzyme replacement can do, but there are a lot of limitations,” he says. “I’m hopeful these drugs will make a difference.”

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