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

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I'll email you.

Mindy said:
I hope that the call will be summarized by someone on the boards afterwards for those of us who can't get in because the call is full...
I will be appreciated that if u email me a summarized conference call..

Ofelia Marin said:
I'll email you.

Mindy said:
I hope that the call will be summarized by someone on the boards afterwards for those of us who can't get in because the call is full...
Post Conference Call Thoughts

Dear Friends,

I wanted to take an opportunity to reach out to the community after today’s conference call with PTC Therapeutics.

I want to echo Pat’s comments on the call – it is incredible to have a pharmaceutical company participate in a call like we conducted today and respond so quickly to our questions and concerns. The ataluren trials in Duchenne are not just important for the patients who participated or for the percentage of young men who would have been affected by this treatment. It is important for continued clinical trials in Duchenne and in rare disease. This is an historic trial and that fact cannot be denied.

But while we appreciate PTC and their answers, and while we understand that the analysis of the data collected from this trial will take time - that’s the one thing as the father of a child with Duchenne I don’t have. I’m sure you feel the same way, especially if your child was in this trial and you believe he was benefitting.

Please know that PPMD will continue to represent the patients and the families of this community to assure that the patients’ voices are heard, questions are answered, and that progress is made. PTC has been a great champion and collaborator in our fight to end Duchenne. And they have promised that they will continue to fight with us. We will hold them to that promise.

For those of you who were not able to join the call, we want to reiterate that we will provide a detailed summary of what was said, as well as some additional clarification. If you were on the call and did not have your specific questions answered, please continue to forward us your questions (info@parentprojectmd.org) and we will pass them along to PTC. We have been told by PTC that they will address each of your individual concerns in a timely manner.

The journey none of us wanted to take, continues. But we continue together, as a community, as a family. Know that PPMD is here for you and your family and please continue to check back with our website and community site for updated information as it becomes available.

Best,
John Killian
PPMD Board Chairman
My kid was not to benefit from PTC124, however this sudden and unexplained decision has shaken me up. Due to a lack of logical explanantion for stopping this trial, I am back to square one. There are too many negetive thoughts which include a suspicion on any other claim to help DMD.

Probably with time I will latch on to the next guy selling hope, but till then I am lost and confused. Lets see if PTC tries to come out clean and offers a good explanation on what happened.
My son Kyle was in the study; in fact I found out today that he was also on the high dose from day 1 of the study (ie. during the blind study period). I am too confused on how they could stop it based on the 6 minute walk test alone. I infact emailed during the conference call that while there was no "improvement", what about just status quo - ie. if they could walk the same distance from day 1 as to their last clinical - why wouldn't that have been enough to let us keep going. they said they had to finish compiling the data - well, then let us stay on the drug!!!!! This was also scary to me because Kyle started the clincial study within 6 months of diagnosis (he had to be on steriods for 6 months first); so now I am doubting where he is at, or wondering: is he really in as good of shape as he is because that's just him, or is it because of the steriods, OR was it because of the drug and are we going to start seeing him progress??? I am not angry at PTC - their hands are tied but I just wish/hope that they will get through all the data quickly and hopefully there'll still be something good to come out of this. I"m not sure who mentioned it in this blog, but I agree - it feels alittle like diagnosis day all over again.....
I don't feel the 6MWR was reliable. My grandson would go into the testing often very tired from the travel to the hospital and not always trying his best. He was doing better strenght wise and was holding his own. He was not declining and we were pleased with that. We are devastated that they told us to stop taking the Altaluren. We pray that they will continue again. It was very tempting to go ahead and keep him on what we have left and not return it. This is so heartbreaking, but we are keeping our faith that God has all this in control. Alex will get better.

Vicki

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...
Nick Catlin from Action Duchenne in the UK has put somes notes of his own from the meeting on the Action Duchenne website:

http://www.actionduchenne.org/viewarticle?news=53

best wishes
Mark
After reading this, I'm a bit upset at PTC. I feel they have an obligation to the DMD community and to those families that have helped them during this trial. The question of continuing the trial would be hard because they would have to ask regulators for permission? Wow thats very arrogant when you are dealing with our children's lives. If they have any sort of proof that a number or even 1 boy has been helped in any way they need to fight to continue the trial. If CPK levels have dropped, dystrophin made, or even just slowed down the progression it's more then we have now. My son was only allowed on the medicine for 4 weeks, he now has a big scar on his leg and has been through numerous blood draws, and essentially for nothing because the pulled the plug on all of this. I want some real answers and some real data put out on the table for our sons. They owe that to the DMD community.

Marks33 said:
Nick Catlin from Action Duchenne in the UK has put somes notes of his own from the meeting on the Action Duchenne website:

http://www.actionduchenne.org/viewarticle?news=53

best wishes
Mark
Matt i do agree with you, we can't accept it like that
our sons have had scares , we have to obtain the result of biopsy before stopping the trial.
they owe it

best

Matt O'Harver said:
After reading this, I'm a bit upset at PTC. I feel they have an obligation to the DMD community and to those families that have helped them during this trial. The question of continuing the trial would be hard because they would have to ask regulators for permission? Wow thats very arrogant when you are dealing with our children's lives. If they have any sort of proof that a number or even 1 boy has been helped in any way they need to fight to continue the trial. If CPK levels have dropped, dystrophin made, or even just slowed down the progression it's more then we have now. My son was only allowed on the medicine for 4 weeks, he now has a big scar on his leg and has been through numerous blood draws, and essentially for nothing because the pulled the plug on all of this. I want some real answers and some real data put out on the table for our sons. They owe that to the DMD community.

Marks33 said:
Nick Catlin from Action Duchenne in the UK has put somes notes of his own from the meeting on the Action Duchenne website:

http://www.actionduchenne.org/viewarticle?news=53

best wishes
Mark
Notes from the call will go up on Monday. NO ONE (not this community and not PTC) is giving up on Ataluren. The sub analysis is underway. We are learning and will learn a great deal more in the upcoming weeks and months. This is a roadblock and we have to figure out how to get around it and we will. We want the best way forward, the 'way' that has the greatest chance of success for every boy/young man who has the potential to benefit from this approach.
Thank you for reassuring us Pat. :) We all want something good to come of Ataluren. Something in this fight has to break for our guys. Hopefully this set-back won't take too long to straighten out.

Pat Furlong said:
Notes from the call will go up on Monday. NO ONE (not this community and not PTC) is giving up on Ataluren. The sub analysis is underway. We are learning and will learn a great deal more in the upcoming weeks and months. This is a roadblock and we have to figure out how to get around it and we will. We want the best way forward, the 'way' that has the greatest chance of success for every boy/young man who has the potential to benefit from this approach.
Pat while I hope this is the case. IMHO pulling children from the medicine who are seeing possible benefits from the drug they are already on is irresponsible. You as well as anyone know that TIME is something we as parents don't have. If they need to tweak things, I'm sure that can be investigated and then changes made. But unless there are safety issues they are hiding pulling the plug on all studies, especially the 30 Non Amublatory ones is just senseless. I'm furious with them for what I put my son through only to see 4 weeks of medicine and no usable data due to them pulling the plug. It was essentially all for nothing.

Pat Furlong said:
Notes from the call will go up on Monday. NO ONE (not this community and not PTC) is giving up on Ataluren. The sub analysis is underway. We are learning and will learn a great deal more in the upcoming weeks and months. This is a roadblock and we have to figure out how to get around it and we will. We want the best way forward, the 'way' that has the greatest chance of success for every boy/young man who has the potential to benefit from this approach.

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