Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted.
A phase 2, dose-escalation study of ACE-031 in boys with DMD have been terminated, and the extension study for boys who have completed the original study have been suspended, centered on preliminary safety data.
Both studies have been being carried out in Canada, and the program was to available study web pages in the United States some time in the future.
The adverse events that the trial participants experienced — minor nose and gum bleeding and dilation of blood vessels in the dermis — have been not, in and of themselves, thought to be dangerous. However, the companies and regulatory firms included say they need to fully comprehend these events prior to continuing clinical studies of ACE-031.
ACE-031 is an inhibitor of myostatin and other related, normally occurring proteins that limit muscle growth. The experimental drug acts as a decoy receptor for these proteins, keeping them away from muscle fibers. It’s designed to allow for much better muscle development and fix in DMD.
Development of ACE-031 is fully discussed in Diverting an unwanted Protein, in the April 2011 issue of Quest, MDA’s quarterly research and health magazine.
Companies hope to resume ACE-031 testing soon after safety issues fully explored
Acceleron and Shire are completing studies of ACE-031 in rats and monkeys and will analyze the human and animal data to obtain an knowing with the observed adverse events. The companies say they hope to resume testing ACE-031 in humans soon after these analyses have been obtained and reviewed by the U.S. meal and drug administration (FDA) and health Canada.
Acceleron and Shire issued the pursuing statement on April 21:
“In an effort to keep the Duchenne muscular dystrophy neighborhood informed, Acceleron and Shire wish to provide an update concerning the standing with the ACE-031 clinical program.
“During the course of clinical trials in wholesome adults and in DMD boys, some participants experienced minor nosebleeds, gum bleeding, and/or little dilated blood vessels within the skin. These events all resolved fully upon discontinuation of treatment.
“By themselves, the minor bleeding events and dilated blood vessels have been not thought to be for being a severe safety concern for study subjects.
“However, centered on review of these safety data using the FDA and health Canada, Acceleron has terminated the A031-03 DMD study and has suspended enrollment and dosing in the follow-on extension study A031-06.
“Acceleron and Shire remain committed to the worldwide DMD clinical program and the improvement of ACE-031. To that end, it is our intention to start new studies of ACE-031 in DMD with appropriate safety monitoring pursuing discussions with regulatory agencies.
“In the coming months, we will provide updates to the DMD neighborhood as appropriate.”
Meaning for families with Duchenne MD
Emerging safety concerns associated with an experimental drug are never superior information for families hoping for new therapies. However, clinical trials of experimental drugs are by nature risky, and it’s helpful to patients that potentially severe adverse events be identified prior to they pose a threat to health or life.
“While we are disappointed at this turn of events in the improvement of ACE-031, it underscores the importance of conducting properly designed and monitored studies,” stated Sanjay Bidichandani, vice president–research at MDA.
MDA and ACE-031
In January 2011, MDA announced a $1.5 million award to Acceleron Pharma for ongoing testing of ACE-031 in DMD.
These funds have been for being distributed in increments, depending on research progress and attainment of predetermined milestones.
MDA will provide updated data concerning the ACE-031 trials as it becomes available.