Staff Writer

With three clinical-stage products targeting rare genetic disorders, Amicus Therapeutics Inc. is making plans to go public.

The Cranbury, N.J.-based firm filed for an initial public offering, hoping to raise about $86.25 million, although the number of shares and share price have not yet been determined. Amicus is looking to list its stock on Nasdaq under the ticker "AMTX."

In its prospectus, the company said proceeds from the offering would be used to support clinical development of three programs based on the approach of using pharmacological chaperones to treat rare genetic disorders, such as Fabry's disease, Gaucher's disease and Pompe disease, by correcting proteins that are misfolded.

The standard of treatment for those disorders include enzyme-replacement therapies, such as Cambridge, Mass.-based Genzyme Corp.'s Fabry's disease product, Fabrazyme (agalsidase beta), which recorded sales of $80.5 million for the first quarter. Genzyme also has a Type I Gaucher's disease product, Cerezyme (imiglucerase for injection), which had first-quarter sales of $239 million. Late last month, Genzyme gained FDA approval for Myozyme (alglucosidase alfa), an enzyme-replacement therapy for Pompe disease. (See BioWorld Today, May 1, 2006.)

Unlike enzyme-replacement therapy, Amicus' products are designed as oral small molecules aimed at restoring the function of a patient's own enzymes. The pharmacological chaperone selectively binds to the target protein, helping to fold it into its correct shape to increase protein activity and improve cellular function.

Amicus is in Phase II with its first product, Amigal (migalastat hydrochloride), in Fabry's disease, a condition that typically results in kidney failure, cardiac abnormalities and progressive neurological problems.

Early results from the first four patients after 12 weeks of treatment suggest that Amigal can increase activity in the lysosomal enzyme alpha-galactosidase A, which is deficient in Fabry's disease. The company expects to finish enrolling patients in the trial by the end of 2006, with a Phase III trial anticipated in 2007.

A Gaucher's disease product, AT2101, is set to begin Phase I testing during the second half of the year, with Phase II to start in the first half of 2007. Gaucher's disease, caused by a lack of activity of the enzyme glucocerebrosidase, results in an enlarged liver and spleen, low levels of red blood cells and platelets, and bone pain.

During the second half of this year, Amicus plans to file an investigational new drug application for its third product, AT2220, in Pompe disease, a disorder caused by acid alpha glucosidase deficiency that leads to progressive muscle weakness and can affect respiration and heart function.

Beyond development of those products, funds from Amicus' IPO would be used to support preclinical research and development of pharmacological chaperone molecules to treat cardiovascular or neurological disorders resulting from enzyme deficiency. Money also might go toward the acquisition or in-licensing of complementary technologies, products or businesses.

For the first quarter, the company had a net loss of $7.6 million. As of March 31, cash, cash equivalents and marketable securities totaled $19.6 million.

Since its founding in 2002, Amicus has raised more than $86 million in venture capital, including a $55 million Series C round it closed in September.

Prior to the offering, Amicus' principal stockholders included entities affiliated with three investment funds that each hold 15.2 million shares, or 17 percent of the company: Prospect Venture Partners II LP, of Palo Alto, Calif.; New Enterprise Associates, of Baltimore; and Frazier Healthcare Ventures, of Seattle. Other shareholders are CHL Medical Partners, of Stamford Conn., which holds 14.3 million shares, or 15.9 percent; Canaan Partners, of Rowayton, Conn., which holds 13.9 million shares, or 15.5 percent; and Quaker Bioventures, of Philadelphia, which owns 7.9 million shares, or 8.9 percent.