Yesterday, GlaxoSmithKline (GSK) entered into an exclusive worldwide collaboration with Prosensa for the development and commercialization of ribonucleic acid (RNA) based therapeutics for Duchenne Muscular Dystrophy (DMD). Prosensa is a Dutch biopharmaceutical company which focuses on RNA modulating therapeutics. 

The agreement covers four RNA-based compounds intended to treat specific, but different, subpopulations of patients suffering from DMD. The lead compound at Prosensa is PRO051, which is being developed for the treatment of DMD. In Sep 2009, the company reported positive results from a phase I/IIa study, which was conducted in patients with DMD. 

Results showed that the systemic delivery of PRO051 in patients with DMD was well tolerated and induced novel expression of dystrophin. The company intends to move the candidate into a double blind placebo controlled phase III study, which is scheduled to commence next year. Glaxo will fund future clinical development costs of PRO051. 

Under the agreement, Prosensa will receive an upfront payment of GBP £16 million (USD $25 million) from Glaxo. Moreover, Prosensa is eligible to receive up to GBP £412 million (USD $655 million) in milestone payments for all four compounds covered by the agreement. The company will also be eligible to receive double-digit royalties on product sales. Prosensa has the option to expand its commercial rights in certain European countries on products arising under the collaboration.
DMD is a severely debilitating childhood neuromuscular disease that affects one in 3,500 newborn boys. Patients suffer from progressive loss of muscle strength due to the absence of the protein dystrophin, often making them wheelchair-bound before the age of 12. Most patients die in early adulthood due to respiratory and cardiac failure.
The alliance is in-line with Glaxo’s strategy of collaborating with biotech companies that have cutting edge technologies and platforms for drug discovery and development. Currently, there is no known cure for DMD. As such, PRO051 could carve a niche for itself on successful development and approval. The candidate could target approximately 13% of all DMD patients.
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