Amicus Reports Preliminary Results From Phase 2 Study Of Plicera

Amicus Therapeutics (Amicus) has reported preliminary results from its phase 2 randomised, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera (afegostat tartrate). The drug is intended for treatment-naive adult patients with type 1 Gaucher disease.

Reportedly, the two dose regimens of Plicera (225mg three days on/four days off and seven days on/seven days off) were studied during the six month trial.

Reportedly, all patients enrolled has experienced an increase in the level of the target enzyme (GCase) as measured in white blood cells. Clinically meaningful improvements in key measures of disease were observed in just one of the eighteen patients who completed the study.

The preliminary results suggested that treatment with Plicera was generally well tolerated, with no serious adverse events (SAEs) reported. Nineteen subjects were enrolled and 18 subjects completed the study.

The company said that once the data is finalalized, Amicus plans to further analyse and evaluate the results in collaboration with its partner, Shire Human Genetic (Shire HGT), and based on the work, will determine the appropriate next steps for the Plicera program.

However, based on these preliminary results, the company does not expect to advance Plicera into phase 3 development at this time.

John Crowley, president and CEO of Amicus, said: “The preliminary results of this phase 2 study certainly do not meet our expectations, but we believe they do provide additional insights into the biological activity of Plicera in Gaucher disease and the pharmacological chaperone technology platform. We plan a detailed analysis to ensure we have a complete understanding of the data.”