Amicus Reports Positive Preliminary Data On Plicera For Gaucher Disease
This article was originally published in The Pink Sheet Daily
Executive Summary
Amicus and partner Shire hope to grab a share of the $1 billion market now dominated by Genzyme’s Cerezyme.
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Amicus Therapeutics released a trickle of data from a Phase II trial of its investigational Gaucher disease therapy Plicera , a potential competitor for Genzyme's enzyme replacement product Cerezyme (imiglucerase), Jan. 7 during the JPMorgan Healthcare Conference in San Francisco. Cranbury, N.J.-based Amicus is developing a suite of novel, small-molecule, oral therapeutics called pharmacological chaperones that "target, bind to and elevate the deficient enzyme" in a lysosomal storage disorder, allowing the body to maintain levels day to day in a more normal process than through current enzyme-replacement therapies that require periodic infusions, CEO John F. Crowley explained during the company's presentation. In December, the firm released Phase II data from its investigational therapy for Fabry disease, Amigal , which would compete with Genzyme's Fabrazyme (agalsidase beta) (1 (Also see "Amicus Reports Positive Phase II Findings For Fabry Disease Candidate Amigal" - Pink Sheet, 20 Dec, 2007.)). Data Crowley presented at the conference were from a four-week trial in which 30 patients with Gaucher disease who were receiving Cerezyme were switched to Plicera. The exec estimated the Gaucher disease therapy market at $1 billion for 5,000 to 10,000 patients. Interim data available for 16 women and four men between the ages of 18 and 63 enrolled in the trial show that Plicera generally was well-tolerated at all doses evaluated, with no serious adverse events reported, Crowley said. Glucocerebrosidase (G-Case) activity - G-Case is the enzyme deficient in Gaucher - was increased in 15 of the 20 patients, he said, noting that the five patients who did not show a clear increase were in either the lowest dose or least frequently dosed group. A trial in treatment-naive patients also is underway, Crowley said. The firm plans to release complete results from the trial at the American College of Medical Genetics meeting March 12-16 in Phoenix. Amicus is partnered with Shire Human Genetic Therapies, a division of Shire, for development of the LSD therapies (2 (Also see "Shire Shores Up Rare Disease Portfolio With Amicus Compounds" - Pink Sheet, 8 Nov, 2007.)). -Shirley Haley ([email protected]) |