Ataluren Results Announced

Dear Friends,


Today (March 3, 2010) PTC Thearapeutics and Genzyme sent out a press release regarding the results of the Phase 2b ataluren trial.  We know that there are many questions surrounding the information presented and we are working very hard to get the answers you need and deserve.


PPMD has been in contact with PTC and Genzyme  this morning and tried to ask questions that we know are on your mind. Unfortunately, we were not given very specific answers except that the trial failed to meet primary and secondary endpoints.  It was the suggestion of PTC that all patients currently involved in the trials contact their clinicians for specific information regarding their participation.  While we know this isn’t the update you wanted to hear, it is all the information we have at this moment and so we wanted to make sure to pass it on to you.


In the meantime, PPMD will continue to seek out answers, ask questions, and advocate for the  entire Duchenne community.  We will also make sure to inform you of any updates as we get them.  On Friday, March 5th at 12 noon Eastern, myself and Senior Director of Research and Advocacy Sharon Hesterlee will host a conference call for parents where you are invited  to ask questions you may have and participate in discussions regarding the future of the trials. We have invited leadership from PTC to participate on this call as well. We will also share any additional information we have
received at that time. Details on how you can dial in to this conference call will be posted on our website before the end of the day tomorrow.


As always, PPMD remains committed to aggressively seeking answers for our boys. Please  continue to check our website, community site, and Facebook page for up-to-date information on how you can join these discussions.


Sincerely,

Pat Furlong
Founding President and CEO

Views: 291

Reply to This

Replies to This Discussion

I am anxious for the call on Friday for more answers. I can't believe this is happening...

Perlita & Gordy Hains said:
Joanna- I'm beyond sad - we were banking on PTC124 for Levi... My hubby was home sick from work today & I simply can't bear to tell him now... I'm so overwhlemed.
We were in the Study at Washington University in MO. And were called this evening and told to quit taking the drug as well. I feel just horrible about what I've put my son through for the past two months and to just be told it's over like that. His biopsy wound still hasn't healed all the way. :(
hi
we need to understand! our all son can'thave bear all that for nothing!!

my son is on extensive procedure from september. for him i think he was on placebo on phase B or the drug was not very efficency BUT i have been told that for a very significant population the result was very interesting after 10 months

usually the kids, without ptc, have a decrease of 20% by year and for a lot of kids on the trial they don't see decrease wich was a very goog think. I have never been told of very important side effects
i really don't undestand the position of genzyme. Is it a money problem?

it should be interesting to know for all the kids who are on the trial what was their results at the walking test before the friday call
best
Do the rest of you all feel like the walking test is a reliable measure of success? With my son, I could see the results of the walk test varying significantly by the day just based on his mood alone...
Yes may be , but during along period it is intersting, and above all it is themesure they will take

the endpoint of the trial was an amelioration of the 6 minute walking wich is to my point of view stupid.If after oneyear the boys do the same it is a good point. Without they will have a decrease
so we have to fight as parents to make the trial go on for at least 2 years because the med is efficency only after 10 months
i am very afraid they want to stop for money pb
at least they have to give us ALL the results et the reason of their position, our kids are not mouses

best
My son is in the non-ambulatory trial, or I guess I should say was. I'm sure it's not a reliable measure of success. But with all the blood work and muscle biopsy's they did, that is where the real results are coming from. I'm extremely heartbroken, my son turned 13 last month and I almost felt like if there was one chance for him, this was it. Now I'm just devastated.

Mindy said:
Do the rest of you all feel like the walking test is a reliable measure of success? With my son, I could see the results of the walk test varying significantly by the day just based on his mood alone...
I'm so sorry. My heart hurts.
We need to find out how this trial was designed, what are the endpoints? Obviously 6 MWT is the primary end point, what are the rest? So Ataluren did not produce an improvement, we need to know why. They should make public all the data, including biopsy data.

As per Mindy's question, how was the 6 MWT validated by PTC as a good measure for DMD? How reliable is it? Obviously if the drug produces clear improvement that can easily be seen in the 6 MWT, but is this what we are looking for when only the steroids are approved for DMD, a clear improvement? Wouldn't we be happy with something that offers more than the steroids with less side effects?

I would like to see this data. These were repeated measurements on multiple subjects during the 48-week trial period, which is a good design. If all the strength measures did not produce significant results over the placebo then I buy the fact that the 6 MWK is a reliable measure of improvement, but we need to know why there wasn't one, we need to know how much dystrophin was produced. Shouldn't PTC have a CC presenting the results and their decisin to stop this trial? Other companies are doing this when a trial does not produce good results.

Finally, will the 6 MWT be used as the primary endpoint for all the disease modifying treatments in the pipeline?
BIG Thank you-
to all the families and especially the boys, who participated in the PTC trial. I can't imagine anything more difficult than participating, waiting, watching, then having it called off. You all are extremely brave. Although Ataluren wasn't 100% beneficial it still can prove useful. Plus, there are/were some families reporting improvements, so at least we have that?

From reading the PTC release it appears that they don't plan to give it up...maybe they want to adjust the chemestry somewhat or define what worked and what didn't in order to better understand things. That isn't all bad either.
To say this news is devastating is an understatement. It seemed to take me back to when we first found out our son had the disorder. Being a scientist myself I know the reality is that there would be more data supporting what they have said already, its just frustrating they havent released it yet for us to see if there is anything that can be pulled from the ashes.
I totally agree.

Jonathan said:
To say this news is devastating is an understatement. It seemed to take me back to when we first found out our son had the disorder. Being a scientist myself I know the reality is that there would be more data supporting what they have said already, its just frustrating they havent released it yet for us to see if there is anything that can be pulled from the ashes.
I'm so sad, my son looked so good, he was getting stronger, I just can't believe this, I guess I will learn more today, conference call with the Dr. from CHOP of Philadelphia. So Sad...

Reply to Discussion

RSS

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2019   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service