West Coast Editor

Marching onto the unfriendly competitive field of enzyme-shortage therapy, Amicus Therapeutics Inc. raised $55 million in a Series C financing for what the company describes as an all-new strategy that corrects misfolded proteins.

Amicus now has enough cash to take the firm "into 2008," said John Crowley, CEO of Cranbury, N.J.-based Amicus.

The privately held firm's Amigal (migalastat hydrochloride) has just entered Phase II trials. The drug acts as a pharmacological chaperone that restores natural function to the misfolded target enzyme.

"We've reached into the protein production factory'" - that is, the endoplasmic reticulum - "to correct the problem before it has a chance to misfold," Crowley said.

In a 16-patient Phase I study, Amigal showed a statistically significant and dose-dependent increase in the activity of the enzyme in each of the healthy volunteers who took part, with zero drug-related adverse events and a high degree of availability in the oral formulation.

Amicus detailed the findings this week at the 42nd Annual Symposium of the Society for the Study of Inborn Errors of Metabolism in Paris, and said the Phase II program for Amigal enrolled its first patient at the end of last month.

Preliminary data from the Phase II trial are expected early next year, and Amicus has a small-molecule therapy for Gaucher's disease in the works, as well.

The Gaucher's program is at the "advanced preclinical" stage, Crowley said, and is likely to enter the clinic in the first half of next year as either a Phase I or Phase I/II trial. Money from the Series C "will also allow us to ramp up several other programs," he told BioWorld Today. "Some are enzyme-related and some are non-enzyme protein programs, but we're not going to disclose those quite yet. Patients are so desperate, they literally line up at the door. They show up with sick kids and want them to be treated, and I want to make sure what we've got is the real deal."

Amicus expects its different angle of attack on genetic diseases will keep the company out of the kind of skirmishes that occupied the likes of Cambridge, Mass.-based Genzyme Corp. and Transkaryotic Therapeutics Inc., also of Cambridge. In early 2004, TKT surrendered its bid to win FDA approval of Replagal, an enzyme-replacement therapy for Fabry's disease that would have competed in the U.S. with Fabrazyme, from Genzyme. Genzyme had filed a lawsuit claiming TKT had infringed on its patent, but a federal judge dismissed the case in December 2001. Replagal was approved outside the U.S. (See BioWorld Today, Jan. 14, 2004.)

Another firm with a novel approach to lysosomal storage disorders is Zystor Therapeutics Inc., of Milwaukee, Wis., which deploys Glycosylation Independent Lysosomal Targeting technology licensed from St. Louis-based Symbiontics Inc. (See BioWorld Today, Dec. 16, 2004.)

Crowley knows Zystor well, since he once served on the company's board.

"They're trying to come up with a better way to do enzyme-replacement therapy, just using a different targeting mechanism," he said. Zystor's method enhances the binding ability of replacement enzymes by attaching a peptide tag to the enzyme. The tag targets receptors on cell surfaces, and Zystor is taking aim at Pompe disease first.

Anywhere from two-thirds to three-quarters of patients with diseases such as those targeted by Amicus "make the enzyme," he said. "They just don't make it right, so we're going back and fixing the problem at its source."

Amicus' Series B financing took place in the spring of 2004 and raised $31 million, which the company said at the time would provide enough to operate into early 2006. Crowley said the company does not plan to seek partners. (See BioWorld Today, May 13, 2004.)

"We don't intend to," he said. "We think scientifically, clinically and commercially, we can develop these drugs without the need for partners."

The Series C was led by Quaker BioVentures, of Philadelphia, joined by existing investors Canaan Partners, of Rowayton, Conn.; CHL Medical Partners, of Stamford, Conn.; Frazier Healthcare Ventures, of Seattle; New Enterprise Associates, of Menlo Park, Calif.; Prospect Venture Partners, of Palo Alto, Calif.; and Radius Ventures, of Boston.

Other new investors included Palo Alto Investors, of Palo Alto, Calif., and the Garden State Life Sciences Venture Fund, funded by the New Jersey Economic Development Authority and managed by Quaker. Sherrill Neff, managing partner of Quaker, is joining Amicus' board.

No Comments