Prosensa and GlaxoSmithKline initiate development of four additional products under existing alliance in Duchenne Muscular Dystrophy.

http://www.prosensa.eu/press-room/press-releases/2010-06-23-Prosens...

 

 

Leiden, June 23, 2010 – Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the initiation of two further programmes under its existing alliance with GSK covering novel RNA-based treatments for Duchenne Muscular Dystrophy (DMD). The initiation of these additional programs under the terms of the existing alliance agreement is a validation of both the potential of Prosensa’s “exon skipping” platform and the ongoing relationship.

The two new programs are included under the existing alliance agreement between both parties and will address the development of four compounds which target different subpopulations of patients suffering from DMD. To access these new programs, GSK has made two initiation payments to Prosensa and Prosensa becomes eligible for further pre-option milestone payments based on research progress.

Within these new programs, Prosensa and GSK will focus on the skipping of four exons (i.e. exon 45, 52, 53, and 55), in addition to their existing programs which target skipping of exon 51 and 44 (PRO051/GSK2402968 and PRO044). The initiation of these programs confirms the joint commitment of both companies to find treatments for DMD.

Under the terms of the collaboration, GSK has an option to select two of these additional four compounds for later-stage development and commercialization. Prosensa will retain certain limited European commercialization rights alongside GSK for the two compounds selected by GSK. For the two compounds not selected by GSK, Prosensa will retain full commercialization rights.

“We are very pleased with this news. Prosensa and GSK’s commitment to progress further developments of additional products that can provide for a solution in DMD is encouraging and welcomed by all of us” said Elizabeth Vroom, Chair of the United Parent Project Muscular Dystrophy, which unites different parent project organizations set up by parents of children with DMD in many countries all over the world.

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Finally, the news I was hoping to hear. Skipping 44 & 45 will work to fix Alexander. Thank God my son will live longer!! Now, onto back to work to solve the puzzle for all mutations, all patients with DMD.
but WHEN will the trials be able to begin in the US? i am so happy to hear that they are moving to new exons, but when can we expect to actually see our boys benefiting from these. i am tired of watching my sons suffer while deals are made. it is really frustrating
Dr Flanigan @Nationwide Childrens is starting a trial up for skipping 51-non ambulatories, which I think you already know about. More will follow Jenn, but I agree the waiting is very frustrating!!!

jenn said:
but WHEN will the trials be able to begin in the US? i am so happy to hear that they are moving to new exons, but when can we expect to actually see our boys benefiting from these. i am tired of watching my sons suffer while deals are made. it is really frustrating
we did get a promising email an hour ago, saying we will be contacted soon about that trial, austin is on "the list" to at least go for the screaning soon....its a phase 1, if he participates he may not get the drug, if he participates and gets the drug, he may not get to be in phase 2 or 3, depending on protocal....im just devistated lately, i hate that my oldest son may have just missed the treatment that would keep him movng, my youngest son is doing fine now at 8, but i know first hand what is going to happen if he doesnt get treatment! sick of roadblocks, sick of raising money running marathons and hiking mountains and not getting usable results... sorry, just pissed off lately! its rough to watch my kids suffer, even harder now that he is old enough to ask alot of questions

cheryl cliff said:
Dr Flanigan @Nationwide Childrens is starting a trial up for skipping 51-non ambulatories, which I think you already know about. More will follow Jenn, but I agree the waiting is very frustrating!!!

jenn said:
but WHEN will the trials be able to begin in the US? i am so happy to hear that they are moving to new exons, but when can we expect to actually see our boys benefiting from these. i am tired of watching my sons suffer while deals are made. it is really frustrating
Its fantastic that they are doing more trials for other exons. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I have read in the past that the idea is to use these trials as arguments that the concept is the same for other exons and so shouldnt have to go through the same lengthy process. Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped?
Very good news,Thanks all,I'm just woundering if any body knows when and where will take this trials,my son goes to see Dr FINKEL at CHOPhiladelphia,does anybody have any information when the trials will reach this hospital?.
I think that is the ultimate goal of the pharma's involved in exon skipping clinical trials. However, the pharma's can't control the regulatory agencies and since this "boutique" or "personalized" medical/chemical approach hasn't been done before it may be that they have to put through (the old fashioned way) a couple (or several) exon skipping compounds in order to prove they work the same.

Pat will be meeting with FDA this fall in order to begin the long (and frustrating to us) process of communicating about this issue. Meeting with the FDA is going to let us know more about their position regarding boutique medicine. Jenn, I know its hard to wait while our sons deteriorate, but we have no other choice. I'm trying to focus on the positive until we know more this fall. We ARE at an exceptional time in the history of Duchenne!! Our sons WILL live longer, better lives.

Jonathan said:
Its fantastic that they are doing more trials for other exons. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I have read in the past that the idea is to use these trials as arguments that the concept is the same for other exons and so shouldnt have to go through the same lengthy process. Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped?
Is it possible to ask this question to the scientist in Prosensa. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped
It's so hard live without hope, my son has the deletion of 21 exon and he need skipping of 20 or 22 exon....is there any chance...
I suspect the scientists at GSK/Prosensa can't answer that question Katerina. The reason is because the answer you seek isn't up to them, it is up to the regulatory agencies at each country...FDA,EMEA, and the like. This is the first time we are faced with personalized medicine. It might be difficult to get the regulatory agencies to comprehend the need for this. On the other hand, there might be sympathetic people on these regulatory boards who want personalized meds to come through. It's a crap shoot either way. But I do believe in the survival instincts of the human race, and humans are on these regulatory boards. Plus, we've got Pat on our side. She is doing everything possible to make regulatories understand what we need. Gotta give her credit for all the hard work she's done.

As far as having hope...there is some, for your son too, more than EVER in the history of this disease. Exon skipping is moving along but there are other solid approaches too. And they aren't mutation specific. Look into Utrophin Upregulation, there is a phase I trial underway in the UK, Myostatin Inhibitors, with a phase II trial underway in Canada. And of course a phase I using Stem Cells going on in Italy.

Its hard to keep watching this stuff spin in the dmd universe and not have it ready for our boys now. Something will break. We have to remain hopeful.



Katerina said:
Is it possible to ask this question to the scientist in Prosensa. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped
It's so hard live without hope, my son has the deletion of 21 exon and he need skipping of 20 or 22 exon....is there any chance...
Hi Cheryl.

Can you please point me to where I can get more information on the stem cell trials in Italy. I haven't been able to find it.

many thanks!
-Rahul

cheryl cliff said:
I suspect the scientists at GSK/Prosensa can't answer that question Katerina. The reason is because the answer you seek isn't up to them, it is up to the regulatory agencies at each country...FDA,EMEA, and the like. This is the first time we are faced with personalized medicine. It might be difficult to get the regulatory agencies to comprehend the need for this. On the other hand, there might be sympathetic people on these regulatory boards who want personalized meds to come through. It's a crap shoot either way. But I do believe in the survival instincts of the human race, and humans are on these regulatory boards. Plus, we've got Pat on our side. She is doing everything possible to make regulatories understand what we need. Gotta give her credit for all the hard work she's done.

As far as having hope...there is some, for your son too, more than EVER in the history of this disease. Exon skipping is moving along but there are other solid approaches too. And they aren't mutation specific. Look into Utrophin Upregulation, there is a phase I trial underway in the UK, Myostatin Inhibitors, with a phase II trial underway in Canada. And of course a phase I using Stem Cells going on in Italy.

Its hard to keep watching this stuff spin in the dmd universe and not have it ready for our boys now. Something will break. We have to remain hopeful.



Katerina said:
Is it possible to ask this question to the scientist in Prosensa. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped
It's so hard live without hope, my son has the deletion of 21 exon and he need skipping of 20 or 22 exon....is there any chance...
Hi Rahul,
Sorry in the delay with getting back to your question, we were away on holiday for a bit. Look for information related to stem cell work being done at the STEM CELL RESEARCH INSTITUTE, HSR, MILAN, ITALY. Also check into MESOANGIOBLASTS, and DR. G. COSSU. Don't hesitate to share what you find, OK?
best
cheryl

Rahul Grover said:
Hi Cheryl.

Can you please point me to where I can get more information on the stem cell trials in Italy. I haven't been able to find it.

many thanks!
-Rahul

cheryl cliff said:
I suspect the scientists at GSK/Prosensa can't answer that question Katerina. The reason is because the answer you seek isn't up to them, it is up to the regulatory agencies at each country...FDA,EMEA, and the like. This is the first time we are faced with personalized medicine. It might be difficult to get the regulatory agencies to comprehend the need for this. On the other hand, there might be sympathetic people on these regulatory boards who want personalized meds to come through. It's a crap shoot either way. But I do believe in the survival instincts of the human race, and humans are on these regulatory boards. Plus, we've got Pat on our side. She is doing everything possible to make regulatories understand what we need. Gotta give her credit for all the hard work she's done.

As far as having hope...there is some, for your son too, more than EVER in the history of this disease. Exon skipping is moving along but there are other solid approaches too. And they aren't mutation specific. Look into Utrophin Upregulation, there is a phase I trial underway in the UK, Myostatin Inhibitors, with a phase II trial underway in Canada. And of course a phase I using Stem Cells going on in Italy.

Its hard to keep watching this stuff spin in the dmd universe and not have it ready for our boys now. Something will break. We have to remain hopeful.



Katerina said:
Is it possible to ask this question to the scientist in Prosensa. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped
It's so hard live without hope, my son has the deletion of 21 exon and he need skipping of 20 or 22 exon....is there any chance...
Hi Rahul,

Prof Giulio Cossu from 'Division of Regenerative Medicine, San Raffaele Scientific Institute' is working on this. He is planning to use mesoangioblasts stem cells. Also, he had recruited DMD kids where full an HLA-matched donor (a brother or a non carrier sister) were found. I understand that he planning to start the trial in March 2011.

Raktim

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