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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I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.

******************************************************************************************************************

Thank you for your message.

We plan a first clinical trial in patients..

Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.
If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.
Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.

With best wishes,




Giulio Cossu

Division of Regenerative Medicine
San Raffaele Scientific Institute
Via Olgettina 58
20132 Milan
Tel: +3902 2643 4954
Fax: +3902 2643 4621
E-mail: cossu.giulio@hsr.it

Department of Biology
University of Milan
Via Celoria 26
20129 Milan
Tel: +3902 503 14798
Fax: +3902 503 14781
E-mail: giulio.cossu@unimi.it
Yes, I spoke with Debra Miller, CureDuchenne, who is helping with funding of Cossu's trial. She mentioned he wanted to start previously but is having trouble with the Italian regulatories. :( more delays but they do plan (perhaps hope) to begin 2011 like Raktim said.

RAKTIM SINGH said:
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
Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!

Melanie Sunny said:
I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.

******************************************************************************************************************

Thank you for your message.

We plan a first clinical trial in patients..

Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.
If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.
Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.

With best wishes,




Giulio Cossu

Division of Regenerative Medicine
San Raffaele Scientific Institute
Via Olgettina 58
20132 Milan
Tel: +3902 2643 4954
Fax: +3902 2643 4621
E-mail: cossu.giulio@hsr.it

Department of Biology
University of Milan
Via Celoria 26
20129 Milan
Tel: +3902 503 14798
Fax: +3902 503 14781
E-mail: giulio.cossu@unimi.it
MAY GOD BLESS.

cheryl cliff said:
Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!

Melanie Sunny said:
I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.

******************************************************************************************************************

Thank you for your message.

We plan a first clinical trial in patients..

Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.
If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.
Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.

With best wishes,




Giulio Cossu

Division of Regenerative Medicine
San Raffaele Scientific Institute
Via Olgettina 58
20132 Milan
Tel: +3902 2643 4954
Fax: +3902 2643 4621
E-mail: cossu.giulio@hsr.it

Department of Biology
University of Milan
Via Celoria 26
20129 Milan
Tel: +3902 503 14798
Fax: +3902 503 14781
E-mail: giulio.cossu@unimi.it
Good news about the stem cell progressing I think! Thanks for sharing!
Thank you for this news. I can't find any information about this trial and method in the internet, could you me the internet address? thank you in advance. This methos suits to all mutations?
And I don't understand what is the main goal of this trial - to prove it safety or to reach therapeutic effect? They write "the number of patients that we will be able to treat will be small because of logistic and economic problems" - why? if this method will work - doesn't it mean that they help all boys, why not?
As such, this will work for DMD kids but one of the important criteria here is 'FULLY HLA-matched donor (a brother or a non carrier sister).' I think that will be tough to find.
Katerina,

Please see the internet addresses below.

Dr. Giulio Cossu's Home Address:
http://www.scienceparkrome.eu/research/stem-cells-and-muscular-dist...

Stem Cell and Muscular Dystrophies Web Page:
http://www.scienceparkrome.eu/research/stem-cells-and-muscular-dist...

On Stem Cell and Musuclar Dystrophies page, it mentions "Based on these discoveries a phase I/II clinical trial is planned for Duchenne Muscular Dystrophy.". I emailed and asked him when a phase I/II clinical trial will be able to start for Duchenne Muscular Dystrophy, and he replied to me with above email.

I do believe this method will suit all DMD boys no matter what mutations they are carrying.

I don't understand the logistic problem for the trial. This trial is extremely costly, and I guess this is the economic problem. As I know CureDuchenne has provided a one year grant for this research.

Best,
Melanie

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