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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OK, this is yet another blip since we've been aware of this drugs development in 2002. At 16 and a half our son is likely one of the oldest ambulatory Duchenne kids in the trial. Our son has had 4 biopsies within these earlier and later trials for PTC-124 and now we are pretty close to real success; As we would define it: making dystrophin and halting or significantly slowing thee disease. We are not giving up on this yet, but my family is disappointed at being so very close and then calling for a STOP; Our son cannot make new "good" muscle, but he has been doing so well on this stuff;
Hi Michael. I strongly agree with you and hope that these guys will not just close shop and leave everybody empty handed. We first heard of PTC124 in 2003. My son is now 19 years old. We live in Argentina so there was no chance he could enroll in the trials, but he sure has a stop codon mutation, which we had confirmed by two US labs (City of Hope and the University of Utah), just in case the trials were expanded to this part of the world. My son stopped walkiing at age 16 (most likely due to the steroids he's been taking since he was 8) and I wasn't really looking for a miracle cure, but certainly I think that our sons and we as families are entitled to see that a beneficial drug is produced and marketed. By beneficial I understand, as you say, a drug that (for what I gather from thousands of miles away) has succeeded in producing dystrophin where there was no dystrophin, and by doing that has also succeeded in at lest stopping or greatly reducing the relentless destruction of muscle fibers. I cannot believe that they are quitting on this because the kids did not show greater walking speed.

By the way: What happened to the summary of Friday's call, which was supposed to be posted during the weekend?

Best regards

Bernardo
Michael said:
OK, this is yet another blip since we've been aware of this drugs development in 2002. At 16 and a half our son is likely one of the oldest ambulatory Duchenne kids in the trial. Our son has had 4 biopsies within these earlier and later trials for PTC-124 and now we are pretty close to real success; As we would define it: making dystrophin and halting or significantly slowing thee disease. We are not giving up on this yet, but my family is disappointed at being so very close and then calling for a STOP; Our son cannot make new "good" muscle, but he has been doing so well on this stuff;
In response to the press release that went out on March 3, 2010 from PTC Therapeutics regarding the ataluren trials, an open conference call was held on 3/5 for parents to have their questions answered by PTC. Click here to read notes compiled from this important open conference call.
HI

I just read the summarize of the conference call

did i dream or i read that may be we will not have the result of the biopsy

i would like to know if it is legal, ethic

according to the parent's consent form that we sign at the beginning of the trial i think they HAVE TO give us this information
can someone of PPMD answer this question

thank you very much

françoise
Françoise--we will pass along this question to the company. I'm interested in knowing the answer as well.
Sharon
I was on the call and feel very good about PTC. They seemed committed to continue this process. Keep in mind they have a decade's worth of time and resources into this and don't want to see it fail either.

And please don't feel it was for nothing ! (My son has two scars and 18 months of blood draws to show for it !) We have to believe that something can be learned from this. This type of research is so new, that it will take some trial and error!

Try not to be discouraged ! The fight continues !

Tamara Walters, St. Louis
Tamara, my comment that is was for nothing was a personal one. The boys who just started on the trial, some just had their biopsies and never made it to the three week visit for one blood draw. No matter the outcome we will not know what it would have, could have done for our boys.IMHO the decision to stop this trial was not done overnight, they had to have seen this coming, the results didn't appear overnight, just as they said it takes months to compile this data. So if they knew months ago things weren't going to come close to what they needed, why even start the new Non ambulatory and put our boys through what they did? Our boys go through enough already. Had I known we were going to be on the medication 4 weeks and essentially get no good data, there is no way I would have put my son through this. That is what upsets me the most. They had to have an idea things weren't going where they needed to be, yet they added the new trial of 30 boys.

Tamara Walters said:
I was on the call and feel very good about PTC. They seemed committed to continue this process. Keep in mind they have a decade's worth of time and resources into this and don't want to see it fail either.

And please don't feel it was for nothing ! (My son has two scars and 18 months of blood draws to show for it !) We have to believe that something can be learned from this. This type of research is so new, that it will take some trial and error!

Try not to be discouraged ! The fight continues !

Tamara Walters, St. Louis
Matt, unfortunately they really didn't know--this caught the PTC staff totally off guard. The trial data was blinded--only the Data Safety Monitoring Board (DSMB) knew who was on what drug and they weren't allowed to tell anyone. When the results were unblinded last week it was probably a relatively quick analysis to see that primary and secondary endpoints weren't met...what will take weeks is all the subgroup analyses and the biopsy results.

PTC did have access to "interim" data at the the half way point of the study that looked pretty good. I think everybody thought that as the boys stayed on the drug longer, the results would look even better. As we all know now, that wasn't the case. The statistics that drive all this decision making are so counter-intuitive that it's really hard to convince yourself to believe the numbers and not what you want to see in the data. As humans we've been programmed to look for patterns in things--it's probably a skill that served our caveman ancestors well as they tried to make sense of the world. The problem is that we see patterns all the time when they aren't really there because we haven't looked long enough or looked at enough numbers.

It's absolutely horrible to not even get anyresults back, negative or positive--I can see that there's no closure for all of the families in that position who were just starting the trial. I think we are all hoping that the subgroup analyses might identify a dose that is more promising or a group of boys who might be more likely to benefit based on the type of stop, or their age or whatever.

Sharon
Sharon - I, too, was just bummed beyond words when I heard this. I do understand. But, there is utrophin and exon skipping both that are in clinical trial stages...There is also mini dystrophin with micro dystrophin, small moleculars like calpain, myostatin,etc. There are alot of trials for viagra and ibederone. So, your son has a stop condon....isn't that lighter on the spectrum of DMD? I hope you can find some hope again. One of the DMD moms - Char Burke with a 7/12 yr old with a duplication of 54-57

Sharyn Thompson said:
My son would have benefitted from the drug but was too young to take part in the trial. Apparently they have been told in AUstralia to cease the trial immediately as well. My heart is broken. Again. I had a horrible feeling this was going to happen, but kept hoping it was simply me being a pessimist. I have been hoping for this drug ever since I heard about it 3 years ago. Now I am back to no hope as he is almost 6 and realistic treatments are still years away as they have to go through so many years of trials. I am so sad.
Hey Char,

Can you provide any more information on mini/micro dystrophin? I know about Idenebone but what trials are running involving viagra?

thanks
Mark

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