Prosensa Named a Fierce 15 Biotech Company - 'One of the most promising private biotechnology companies in the industry'

Prosensa Named a Fierce 15 Biotech Company - 'One of the most promising private biotechnology companies in the industry'

Leiden, the Netherlands, September 19, 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has been named by FierceBiotech as one of 2012's Fierce 15, designating it as one of the most promising private biotechnology companies in the industry.

Prosensa's main focus is the treatment of rare neuromuscular diseases. Drisapersen (PRO051/GSK2402968), its leading therapy for the treatment of Duchenne muscular dystrophy (DMD), is currently in Phase III trials in partnership with GlaxoSmithKline (GSK).

"When New Enterprise Associates went looking for its first European biotech investment, they found an ideal candidate in Prosensa," says FierceBiotech Editor John Carroll.  "Its Big Pharma partner GlaxoSmithKline is executing a pivotal study on its lead drug for Duchenne muscular dystrophy, making Prosensa − which has five other DMD programs in the pipeline − a leader in rare diseases worth watching closely. A strong investment syndicate gives Prosensa some real options to ponder about its future. And we'll be watching every step of the way."

Hans Schikan, Prosensa CEO said: "Prosensa is very pleased to have been recognised as a 'fierce' biopharmaceutical company. Thanks to our partnerships with patient organisations, academia and pharmaceutical companies, along with the support of our investors, we will passionately continue on our path of growth to become a leading rare disease player with an initial focus on Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease. Prosensa's platform of RNA modulation can be applied to a large number of unmet medical needs."

Prosensa has the most advanced portfolio of drug candidates for the treatment of DMD, with two compounds in clinical trials in partnership with GSK (drisapersen and PRO044) and four additional compounds in preclinical development, as well as 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.

This is FierceBiotech's tenth annual Fierce 15 selection. The Fierce 15 celebrates the spirit of being "free" – championing innovation and creativity, even in the face of intense competition. A complete list of the "Fierce 15" companies can be found at

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About DMD

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to a defective exon and thereby correct the reading frame, enabling the production of a novel, functional dystrophin protein.

About Prosensa

Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa's current focus is on developing treatments for Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington's disease. In 2009, Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa's lead compound, drisapersen (GSK2402968/ PRO051), being developed by GSK, is in Phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit

Press contacts for this press release:

Luc Dochez Chief Business Officer Prosensa

Phone: +31 (0) 71 33 22 085