Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

New release from Prosensa:

£10m in milestone payments received from GlaxoSmithKline


Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted orphan drug designation for two additional DMD compounds in its pipeline.


Prosensa identified suitable oligonucleotide candidates for PRO052 and PRO055, designed for the skipping of exons 52 and 55 of the dystrophin gene. PRO052 and PRO055 are currently in pre-clinical development and will be moved to clinical trials as soon as possible.


Prosensa has also received orphan drug designation by the European Commission for PRO045 and PRO053. These compounds are designed to skip exons 45 and 53 of the dystrophin gene and are expected to enter clinical development within the next 6 months. In parallel, Prosensa and GSK have initiated a large global natural history study in order to generate important data on the progress of DMD that will help to facilitate the development pathways for these compounds.


Together with drisapersen (PRO051/GSK2402968) and PRO044, already in clinical development, the Prosensa portfolio of six DMD compounds has the potential to treat more than 40% of all DMD patients.


Hans Schikan, CEO of Prosensa, commented: “Achieving orphan drug status for PRO045 and PRO053 is an important milestone that we believe will allow us to accelerate development of these compounds and initiate clinical trials within the next half year. While GSK has an option to license one compound, PRO045 or PRO053, the other compound will be developed and eventually commercialized by Prosensa in our ambition to become a fully integrated biopharmaceutical player in rare diseases. Together with PRO052 and PRO055, where the same principle applies, Prosensa will be in a position to target rare mutations in specific DMD subpopulations and may provide treatment to more patients with this debilitating disease.”


Prosensa has the most advanced portfolio of drug candidates for the treatment of DMD, and has also preclinical compounds for Myotonic Dystrophy and Huntington’s Disease. Prosensa’s DMD compounds are based on its proprietary exon-skipping technology that uses antisense oligonucleotides to restore expression of a functional dystrophin protein and to provide potential treatment for patients affected by this progressively debilitating neuromuscular disease.

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Comment by Sharon Hesterlee on October 24, 2012 at 8:31pm

No, it sounds like Prosensa would take the other compound forward internally--if the 51 compound is successful then I'm sure Prosensa will get some kind of milestone payment for GSK that would give it the cash to do some of the clinical trials for other compounds in house.  I think the plan is to move both forward.

Comment by Denise Bretsik on October 24, 2012 at 7:53pm

I am so confused about GSK being able to license one, PRO45 or PRO53. What does this mean? Would only only move forward at this time? 

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