Bad news!

 

http://www.fiercebiotech.com/story/ptcs-lead-drug-ataluren-flunks-p...

 

SOUTH PLAINFIELD, NJ and CAMBRIDGE, MA – March 03, 2010 – PTC Therapeutics, Inc. and Genzyme Corporation (Nasdaq: GENZ) today announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD). The primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48 week duration of the study. Study results showed that ataluren was well tolerated and no clinical trial patients discontinued treatment due to an adverse event. Additional efficacy analyses are underway in patient subgroups.

"These results further demonstrate the safety profile of ataluren and support continued development," remarked Langdon Miller, M.D., PTC's chief medical officer. "DBMD is a progressive, debilitating, and life-threatening neuromuscular disorder. The variability of symptom onset, disease progression, and 6-minute walk distance creates challenges for clinical development. Importantly, this trial does provide a wealth of valuable data about ataluren and DBMD. Additional analyses will guide the overall clinical and regulatory path forward."

"PTC has a longstanding commitment to discovering and developing new treatments for DBMD and we will continue to collaborate with patients, investigators, and DBMD advocacy groups to advance these efforts," stated Stuart Peltz, Ph.D., president and chief executive officer of PTC Therapeutics. "Data from this Phase 2b study will guide further development of ataluren in nonsense mutation genetic disorders."

"The quality of the data from this well-conducted study and additional analyses will help to inform the clinical development of ataluren in other indications," stated Geoffrey McDonough, M.D., senior vice president and general manager of Genetic Diseases at Genzyme. "We are committed to the development of ataluren and will continue to collaborate with PTC to advance its development for the treatment of genetic disorders."

Ataluren is also currently being investigated for use in patients with nonsense mutation cystic fibrosis in a Phase 3 study and nonsense mutation hemophilia A and B in a Phase 2a study. Ataluren's mechanism of action offers the potential to address multiple genetic disorders with various pathophysiologies and disease manifestations.


ABOUT THE PHASE 2B CLINICAL TRIAL
The randomized, double-blind, placebo-controlled Phase 2b trial was designed to evaluate the safety and efficacy of 48 weeks of ataluren therapy in patients with nmDBMD. The study enrolled 174 participants at 37 sites in North America, Europe, Australia, and Israel. Participants were randomized to receive either a low dose of ataluren (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening), a high dose of ataluren (20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening), or placebo (inactive drug in the morning, at midday, and in the evening). The primary outcome measure was the total distance walked during a 6-minute walk test, a standardized test of ambulation. Other outcome measures in the study evaluated activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and ataluren blood levels were also monitored.

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We are sending out a statement about this shortly and PPMD will also be hosting a conference call on Friday so that people can call in with questions. We might not have all the answers, but we will certainly take those questions back to the company. Details about the call will be posted on the PPMD site by the end of the day.
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
Hi Ofelia--I don't think they have quantified the amount of dystrophin produced yet--that analysis is ongoing.
This will be a very important number to know...

Sharon Hesterlee said:
Hi Ofelia--I don't think they have quantified the amount of dystrophin produced yet--that analysis is ongoing.
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...

Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
Although this wouldn't have helped my grandson, i feel sick now and am absolutely devastated for all the boys this would have helped. I am so sorry for all of you. x

Joanna Johnson said:
My son is in the study. Both of my 2 children have Duchenne. I'm sick to my stomach. I vaguely recall a number of 25% in the mdx mouse, but don't quote me on that.

Ofelia Marin said:
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...
Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
I know. This is very, very sad! My son does not have a stop codon and I am devastated by this news. I remember seeing good numbers like 50% drop in CK levels.

Joanna Johnson said:
My son is in the study. Both of my 2 children have Duchenne. I'm sick to my stomach. I vaguely recall a number of 25% in the mdx mouse, but don't quote me on that.

Ofelia Marin said:
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...
Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
Can the kids not continue the drug even if they have not shown improvement but are stable and have not regressed?????

Ofelia Marin said:
I know. This is very, very sad! My son does not have a stop codon and I am devastated by this news. I remember seeing good numbers like 50% drop in CK levels.

Joanna Johnson said:
My son is in the study. Both of my 2 children have Duchenne. I'm sick to my stomach. I vaguely recall a number of 25% in the mdx mouse, but don't quote me on that.

Ofelia Marin said:
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...
Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
I really tried not to get my hopes up, but it was impossible. :( My son was diagnosed too late to get in the trial, but I WAS very, very hopeful, as I'm sure we all were. I have lots of questions as well.
Not if PTC and Genzyme decide to stop the develoment of Ataluren. I have so many question about this data...My understanding from the press release is that there is NO significant difference b/w the group on placebo and the group on Ataluren, hence the drug is NOT working. I expect them to stop development of Ataluren for DMD. I would like to see the data though, % dustrophin, drop in CK levels etc. The researchers working on other treatments, exon skipping for example, must learn something from this.

Ana Vaish said:
Can the kids not continue the drug even if they have not shown improvement but are stable and have not regressed?????

Ofelia Marin said:
I know. This is very, very sad! My son does not have a stop codon and I am devastated by this news. I remember seeing good numbers like 50% drop in CK levels.

Joanna Johnson said:
My son is in the study. Both of my 2 children have Duchenne. I'm sick to my stomach. I vaguely recall a number of 25% in the mdx mouse, but don't quote me on that.

Ofelia Marin said:
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...
Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.
If some improvement was seen in some boys but it did not meet the endpoints PTC could redesign the study with different end points. Last year in a trial for infantil spasms and the drug sabril they did this as the babys showed some improvement but did not meet the endpoints, the FDA said they needed to do another trial with the different end points or measurements. In addition the nonambulatory trial need not be stopped per my information from the FDA as that just started so the results are not in and it has different endpoints, Likewise it is my understanding the CF trial will continue and a continuation of the nonambulatory trial would provide additional safety data for the FDA. In addition, in the past trials for other diseases have had design issues and have had to be redone. This was the first large scale trial like this in DMD and the design of the trial could also be an issue.

There are many questions, I and pray the best will be done for the boys, Cheri

Ofelia Marin said:
Not if PTC and Genzyme decide to stop the develoment of Ataluren. I have so many question about this data...

Ana Vaish said:
Can the kids not continue the drug even if they have not shown improvement but are stable and have not regressed?????

Ofelia Marin said:
I know. This is very, very sad! My son does not have a stop codon and I am devastated by this news. I remember seeing good numbers like 50% drop in CK levels.

Joanna Johnson said:
My son is in the study. Both of my 2 children have Duchenne. I'm sick to my stomach. I vaguely recall a number of 25% in the mdx mouse, but don't quote me on that.

Ofelia Marin said:
From a friend from UK. They will probably make the % public at some point. Very sad news... Do you remember how much dystrophin was obtained in the previous phase of the trial? I remember reading those numbers but I have too many negative things on my mind now to remember...
Joanna Johnson said:
Where did you get the 10% statistic from?

Ofelia Marin said:
My understanding is that the data shows 10% dystrophin which, as we've heard from scientists, is not enough. :-( I would like to hear what they say about this.

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