Prosensa Advances Three Exon Skipping Candidates for Duchenne Muscular Dystrophy into the Next Development Stage - Prosensa to receive up to £27M in development and milestone payments from GSK


Leiden, September 12, 2011 – Prosensa, the Dutch biopharmaceutical company focusing on rare diseases with an unmet medical need, announced today that they have agreed with GlaxoSmithKline (GSK) to advance three further exon skipping compounds (PRO044, PRO045 and PRO053) into the next development stage under their ongoing collaboration relationship in Duchenne Muscular Dystrophy (DMD). The two companies have agreed upon the development terms for the exon 44 skipping programme, which sees GSK providing additional support for PRO044, currently in Phase I/II. Prosensa and GSK will also work together on a preclinical and clinical development program for PRO045 and PRO053, which are expected to enter clinical trials in the first half of 2012. The three candidates PRO044, PRO045 and PRO053 are compounds designed to skip exons 44, 45 and 53 respectively, in the dystrophin gene, each targeting different subpopulations of DMD patients. Under the collaboration agreement, Prosensa is eligible for milestone payments and development funding payments which could total up to £27M, depending upon the course and outcome of the clinical development for these programs.

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good to hear this , ambrish

hi raktim ,


does yr son needs any of these?



thats great!. I hope they meet the timeline.

27M looks to be a nice confirmation that things are looking good with the current trials. With some extra juice in the Prosensa machine they should move forward quickly. There is another 400M waiting.


Great post Raktim. From some of these individual exon skipping drugs hope we get data for multiple exon skip too.


Is this a cure for the deletion of exon 45?

Exon skipping will not be a cure for ANY mutation.  Exon skipping, at best, turns Duchenne into Becker. 

Good news! Progress is always great.

For a deletion of exon 45, one would need exon 44 skipped (PRO044) in order to produce an in-frame mutation which will hopefully result in Becker like protein, shorter but functional. The hope is that it will somewhat slow the progression.


PRO044 is in Phase I trials in Europe.


Cheryl Hartwig said:

Is this a cure for the deletion of exon 45?

It is indeed very encouraging to see Prosensa getting out ahead of the curve with respect to trials for other exons, rather than waiting for approval of PRO051.

Hopefully AVI will be encouraged to do the same.

Very much looking forward to a 44exon skip for our sons and nephews (deletion of 45).

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