CureDuchenne - Together we Can CureDuchenne
August 2013
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Prosensa Provides Updates on Duchenne Development Programs

Prosensa

On August 13, 2013, Prosensa’s Chief Executive Officer Hans Schikan and Chief Medical Officer Dr. Giles Campion provided updates on the Company’s development programs for the treatment of Duchenne. CureDuchenne is pleased to see the progress that Prosensa has made, now with six exon skipping programs at different stages of development.

CureDuchenne was the lead organization that provided funding to Prosensa in 2004 and we have been so happy to see the further funding that followed from the venture capital firms, GlaxoSmithKline, and other Duchenne organizations. It’s been a very long 10 years and until these drugs are actually approved and treating our sons, we still need to be cautious with our expectations, but this is the dream we had as we entered into this partnership with Prosensa shortly after CureDuchenne was founded. Although it will take a few years to see all six of these drugs make it into clinical use, if they are all eventually approved, they will treat approximately 41% of Duchenne patients. CureDuchenne will continue to lead exon skipping research so that all boys with a skip-able mutation will have a treatment.

Product Candidate % Duchenne Patients Phase Status Timeline
Drisapersen (PRO051) 13% Phase III >300 patients fully enrolled worldwide Results expected Q4 2013
PRO044 6% European Phase I/II completed Clinical report at World Muscle meeting Oct 2013 European extension study anticipated to start Q3 2013

Pivotal trial (includes US) Q4 2014
PRO045 8% Phase I/II Europe European Phase I/II study started in Q1-2013 US patients in trial in 2014
PRO053 8% Preclinical European Phase I/II study anticipated to start in Q3-2013 US trial anticipated to start in 2014
PRO052 4% Preclinical Pre_IND meeting in 2014 European and US based Phase I/II 2014-2015
PRO055 2% Preclinical Pre-IND meeting in 2014 European and US based Phase I/II 2014-2015
PROSPECT Skip 10-18 = 5%
Skip 10-30 = 13%
Discovery Investigating multiple exon skipping for rare and duplication mutations No timeline. Early research: currently in preclinical development

As the table above shows, drisapersen, the exon skipping drug designed to treat mutations amenable to exon 51 (and the result of the Prosensa/GSK partnership), is expected to complete its pivotal phase III trial this year. We expect that this could be the first drug ever approved for the treatment of Duchenne, and CureDuchenne is proud to have had a role at such a crucial time in the development of this drug.

CureDuchenne has also been dedicated to addressing rare and duplication mutations. We are funding Dr. Kevin Flanigan (Nationwide Children’s Hospital Columbus) to study exon skipping for duplication mutations http://cureduchenne.com/blog/cure-duchenne-supports-duchenne-duplication-mutation-research-with-dr-kevin-flanigan/, and are very pleased to see Prosensa address as many patients as possible with their research.

In order to help parents, patients and researchers understand mutations, especially Becker mutations, which is the goal of exon skipping, we have partnered with eDystrophin: http://edystrophin.genouest.org/index.php?page=home (open in Chrome). You will find this site to offer extensive information on mutations.

Prosensa plans to include non-ambulatory patients in all of its upcoming trials. Prosensa is also enrolling 250 patients in a large, international natural history trial that will include patients that are amenable to their current development candidates, 44, 45, 52, 53, 55. For more information: http://clinicaltrials.gov/ct2/show/NCT01753804?term=prosensa+natural+history&rank=1

The chart is a very simple summary of two very comprehensive presentations. We hope it makes it easier for the Duchenne community to understand the programs and timelines that Prosensa and GSK are anticipating.

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