Prosensa’s Clinical Development Plan Update – March 2012

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline.  Below you can find an update about the current state of development of these compounds.

Prosensa is currently conducting a clinical study to investigate the effect of PRO044 in boys with Duchenne muscular dystrophy, who have a dystrophin gene mutation amenable to an exon 44 skip. This study is a phase I/IIa study that will assess the safety, tolerability, efficacy and pharmacokinetics of PRO044. The inclusion/exclusion criteria, as well as the endpoints and investigator locations are available on (search terms ‘Duchenne and Prosensa’ or ‘PRO044’).


Prosensa has four additional compounds in development that target exons 45, 53, 52 and 55 (PRO045, PRO053, PRO052 and PRO055). These programs currently are in the pre-clinical development phase. It is anticipated that the company will be able to progress PRO045 and PRO053 into the clinic in the second half of 2012. Discussions are on-going with the regulatory authorities to ensure optimum development. Each trial will be posted at the website as soon as the study design is finalized and all the required approvals of authorities and ethics committees are granted.


It is always difficult to predict timelines for drug development. Therefore, please keep in mind that expected start and end dates are based on predictions and could be delayed if unforeseen events occur.


Prosensa is very committed to developing safe and effective treatments and we will do our utmost to succeed herein. Our goal is to develop compounds for as many patients as feasible who may benefit from the exon skipping platform. 

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Comment by Stephanie Matthes on March 29, 2012 at 9:20am

Details regarding the clinical trial(s) are not yet available, but as soon as this information is released we will notify the PPMD community and all registrants on DuchenneConnect. This info will also be posted on Regarding the FDA providing "fast track" for other exon skipping candidates, we do not know if they will agree to this but we are certainly hopeful! Again, we will provide additional details as soon as they are available to us.

Comment by Chris Diemler on March 26, 2012 at 11:47pm

Will the FDA have to make concessions as far as trial requirements for the exon skipping candidates after pro051?  I ask this because i just read where they only have 123 registered for the phase III GSK run trial, they need 180...the patient numbers dont appear to be there for FDA trial requirements...I guess my real question is would all boys who could be possibly treated by pro045 or pro053 be allowed to participate?

Comment by David on March 26, 2012 at 7:31pm

Correction: I meant to type "help the FDA step up and ... consider fast track options". Regulators will get bold if Congress tells provides necessary cover from malicious lawsuits.

WRITE YOUR CONGRESSPERSON AND SENATORS and tell them to support FAST ACT 2012 (HR 4132)

If you aren't in the U.S. contact someone who is. Doesn't take much time, visit and you'll be done in 15 minutes.



Comment by Liisa Underwood on March 26, 2012 at 5:58pm

Sorry, I guess I could have looked at the PRO044 version.  I could have noticed that the GSK study for 51 has study sites in the US.  Sorry, I'm so anxious and sometimes fail to see all the information at hand.

Comment by David on March 26, 2012 at 5:42pm

Each trial will be posted at the website as soon as the study design is finalized and all the required approvals of authorities and ethics committees are granted.

That would include details on dosing regimen, sites, extension study policy, etc. How I read "progress PRO045 and PRO053 into the clinic in the second half of 2012" is to expect the site updated with new trial # no earlier than this summer.

The really good potential news here is that these announcements help the FDA to setup up and announce that they will consider some sort of fast track option for these other skips. With any luck by summer PPMD advocacy will pay off in that regard.


Comment by Liisa Underwood on March 26, 2012 at 5:24pm

Let's say we do have USA study sites.  Here are a better set of questions to ask:  How often and how long would the child need to be present for treatment?  For example, 3 times a week for 6 months.  And if things go well on the treatment, I assume like PRO051 there are additional stages to the treatment study which would then have it's own treatment duration.  Can someone please explain how this or tell me to whom I should talk? I want to have a rough plan in mind when/if the treatment comes to the USA, as to how I'm going to handle the logistics.  In other words, will I have to move temporarily to the location where the treatment may happen and what duration could I be looking at?  Or will I be able to fly there because there is enough time between treatments?  Thanks!!!

Comment by Liisa Underwood on March 26, 2012 at 5:09pm

Here is hoping for USA locations!!!!

Comment by Stephanie Matthes on March 26, 2012 at 4:41pm

Liisa & Traci - Thanks for your questions! I spoke to our DuchenneConnect Coordinator, Ann Martin. She says: 

"We unfortunately do not know the details yet of where the study sites will be located.  When we last asked Prosensa about US trial sites, they stated that it is their 'full intention to include the US in the clinical program.'  However, this is not a guarantee!"

Comment by Liisa Underwood on March 26, 2012 at 12:44pm

Second Traci's question...are the trials for 45 and 53 going to be in the states or do I need to move to Belgium?

Comment by Traci Strafuss on March 26, 2012 at 11:17am
Does this mean 45 & 53 into US clinics in the second half of 2012?

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