Amicus Therapeutics has suspended enrolment for the phase-2 clinical trial of its investigational drug AT2220 (1-deoxynojirimycin HCI) for the treatment of Pompe disease and that it has received verbal notice from the US Food and Drug Administration (FDA) that the trial is on clinical hold. After evaluation of available data the company plans to work closely with the FDA and if appropriate will amend the phase-2 protocol with the objective of restarting the trial as expeditiously as possible.
Amicus initiated the phase-2 clinical trial of AT2220 in adults with Pompe disease in June, 2008. Based on encouraging safety data from both preclinical and phase-1 studies, the approved phase-2 trial protocol involved initial treatment with a high dose of AT2220. Two patients enrolled in the trial experienced self-reported adverse events and subsequently withdrew from the trial. The events were categorized by the site investigator as serious and probably related to treatment with AT2220.
After initial evaluation of the events, Amicus suspended patient enrolment for the phase-2 trial. In compliance with regulatory requirements and internal standard operating procedures, the two events were reported to the relevant health authorities, including FDA, and in a subsequent discussion with the FDA the trial was placed on clinical hold status pending further evaluation of the events.
John F Crowley, president and CEO of Amicus commented, "We will work closely with the investigators and the FDA to address these issues, modify the protocol as appropriate, and get the trial back on track as quickly as possible. We are committed to exploring this new therapy for people living with Pompe disease in as safe a manner as possible."
The company previously announced that the phase-2 results with AT2220 will be available in the second half of 2009. The company will provide updated guidance for reporting results in the upcoming months.
These events have no impact on Amicus' ongoing studies with its investigational drugs Amigal (migalastat hydrochloride) for Fabry disease and Plicera (afegostat tartrate) for Gaucher disease.
Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases.