ACE-031 Program Update From Acceleron and Shire

 

In an effort to keep the Duchenne muscular dystrophy community informed, Acceleron and Shire wish to provide an update about the status of the ACE-031 clinical program. During the course of clinical trials in healthy adults and in DMD boys, some participants experienced minor nosebleeds, gum bleeding, and/or small 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 were not considered to be a serious safety concern for study subjects. However, based on review of these safety data with 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 global DMD clinical program and the development of ACE-031. To that end, it is our intention to start new studies of ACE-031 in DMD with appropriate safety monitoring following discussions with regulatory agencies. In the coming months, we will provide updates to the DMD community as appropriate.

 

 

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Permalink Reply by amit gupta on April 21, 2011 at 12:50pm
it is the worst thing i have read today.
Permalink Reply by Ofelia Marin on April 21, 2011 at 12:53pm
TREAT-NMD newsletter no. 99
Permalink Reply by Ofelia Marin on April 21, 2011 at 3:47pm

This is very positive and shows that not everything is lost:

"Acceleron and Shire remain committed to the global DMD clinical program and the development of ACE-031. To that end, it is our intention to start new studies of ACE-031 in DMD with appropriate safety monitoring following discussions with regulatory agencies. In the coming months, we will provide updates to the DMD community as appropriate."

Permalink Reply by amit gupta on April 21, 2011 at 4:03pm
ofelia, that sounds like the standard 'forward looking statement'
Permalink Reply by Ofelia Marin on April 21, 2011 at 4:07pm
They would have canceled the development of ACE-031 as BioMarin did with the utrophin upregulator... these big companies do not want to lose money. I think we need to wait for more info about their plans.
Permalink Reply by Moein on April 21, 2011 at 4:16pm

I phoned Acceleron Pharma today,and they WILL NOT DISCONTINUE THE DRUG ,BUT CHANGE THE PROTOCOL TO GIVE THE DOSE EVERY 4 WEEKS NOT BIWEEKLY

 

Permalink Reply by Ofelia Marin on April 21, 2011 at 4:18pm
Thanks Moein! That's what I thought, from a regulatory standpoint it's easier to start a new trial with a new protocol.
Permalink Reply by Moein on April 21, 2011 at 4:19pm

On the contrary,I felt that they insist on developing this drug.I am thinking that if they saw no improvement+serious side effects then they would discontinue the drug,but I think they saw improvement and they DON'T WANT SACRIFICE this for side effects that can be managed.Do you think that?

Permalink Reply by Moein on April 21, 2011 at 4:19pm


Moein said:

I phoned Acceleron Pharma today,and they WILL NOT DISCONTINUE THE DRUG ,BUT CHANGE THE PROTOCOL TO GIVE THE DOSE EVERY 4 WEEKS NOT BIWEEKLY

 

Permalink Reply by Ofelia Marin on April 21, 2011 at 4:21pm
That's exactly what I meant...they would not continue development if the data weren't promising.

Moein said:

On the contrary,I felt that they insist on developing this drug.I am thinking that if they saw no improvement+serious side effects then they would discontinue the drug,but I think they saw improvement and they DON'T WANT SACRIFICE this for side effects that can be managed.Do you think that?

Permalink Reply by Moein on April 21, 2011 at 4:22pm

I knew something,that they now know what is the reason for nosebleeds

Permalink Reply by Moein on April 21, 2011 at 4:28pm

Ofelia,My sonis on the waiting list,and his neurologist who directs the trial (first site)called me to update me.

My son is supposed to enter the trial maximum in fall.

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